A Phase 2 interventional study of Pomalidomide (CC-4047) and Placebo in Scleroderma, Systemic, Sclerosis, Systemic and Systemic Scleroderma, sponsored by Celgene. Terminated at 56 sites in 10 countries. Open to participants aged 18 Years to 80 Years. Per ClinicalTrials.gov, last updated 2023-12-01.
Sponsored by Celgene (part of Bristol-Myers Squibb) · Phase 2, Interventional, and Treatment
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Among 165 studies in Scleroderma, Systemic with a site in United States — 40 open to participants now, 52 under way, 6 of them phase 3. Scleroderma, Systemic · United States.
Among the 165 studies in Scleroderma, Systemic with a site in United States, this one is the 93rd largest by enrollment of the 142 interventional ones that give a count, is one of 3 that started in 2012, is one of 54 in phase 2, and was the 53rd registered.
613 studies on the registry are indexed under Scleroderma, Systemic; 221 are open to participants now.
Browse Scleroderma, Systemic studies →40 of the 165 studies in Scleroderma, Systemic with a site in United States are open now. Recruiting first, the newest first:
Fate Therapeutics · Sites in United States
Ages 18–70
AstraZeneca · Sites in United States, Argentina, Australia and 32 more countries
Ages 5–80
Boehringer Ingelheim · Sites in United States, Argentina, Australia and 42 more countries
Ages 18+
Miltenyi Biomedicine GmbH · Sites in United States
Ages 18+
University of Michigan · Sites in United States
Ages 18+
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 145 Scleroderma, Systemic studies recruiting now, 83 (57%) set no upper age limit and 49 (34%) stop at 75 or younger; 19 take children under 18; 16 take healthy volunteers.
This study: 18 Years to 80 Years · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
165 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 46 · Phase 2 74 · Phase 3 17 · Phase 4 3
First in 1992; this study’s year is marked. 3 started in 2012.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 23 is below the median of 37 across 449 interventional studies indexed under Scleroderma, Systemic.
Median enrollment 34 across the 142 interventional studies, 67 across the 22 observational. Of the 104 that completed or were terminated, 56 (54%) have results posted.
This tab as a report to print or save as PDF → With an account.
69 of 165 have an industry lead sponsor. The 10 most frequent:
This study is registered under Celgene, one of the names counted as Bristol-Myers Squibb. Under that name alone:
Celgene is the lead sponsor of 428 studies on the registry; 12 are open to participants now.
Of its 104 completed or terminated interventional studies of FDA-regulated products, 30 (29%) have results posted.
Bristol-Myers Squibb has 5 studies filed under Scleroderma, Systemic — fewer than ten, so there is no comparison to draw.
15 of the studies in Scleroderma, Systemic with a site in United States that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05925803Phase 3AstraZenecaDetermine Effectiveness of Anifrolumab In SYstemic Sclerosis (DAISY) | 2027-01-06 |
| NCT06195072Scleroderma Research Foundation, Inc.Platform Clinical Study for Conquering Scleroderma | 2026-11 |
| NCT06375993Adicet TherapeuticsA Phase 1 Study of Prulacabtagene Leucel (Prula-cel, Formerly ADI-001) in Autoimmune Disease | 2026-12 |
| NCT04380831City of Hope Medical CenterTBI Using IMRT and Cyclophosphamide Prior to Stem Cell Transplant for the Treatment of Severe Systemic Sclerosis | 2026-12-22 |
| NCT06655155argenxA Study to Assess the Efficacy and Safety of Efgartigimod PH20 SC in Adults With Systemic Sclerosis | 2027-03 |
Add all 15 to your calendar (.ics) → With an account.
Among the 165 studies in Scleroderma, Systemic with a site in United States, from their registry updates:
This tab as a report to print or save as PDF → With an account.
The primary objective of this study is to evaluate the safety, tolerability, and efficacy of pomalidomide in the treatment of patients with systemic sclerosis with interstitial lung disease.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Subjects are required to meet at least one of the following 2 pulmonary-related criteria to be eligible for the study:.
i) FVC readings ≥ 70% and ≤ 80% at Screening and Baseline (Visit 2) with a documented history of either or both of:.
A. A ≥ 5% decrease (expressed as percent predicted or in liters) in FVC in the 24-month period prior to Baseline (Visit 2) based on 3 or more assessments. Two assessments may be done during the Screening phase provided the assessments are completed at least 2 weeks apart.
B. A high resolution computed tomography (HRCT) fibrosis score > 20%.
ii) Forced vital capacity (FVC) ≥ 45% and \<70% at Screening and Baseline (Visit 2) [with or without a documented pre-specified FVC decline or fibrosis score].
Exclusion Criteria
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
From a breathing test (spirometry): how much air you can blow out in the first second. Often given as a percentage of what is expected for your age, height and sex.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
FEV1
From a breathing test (spirometry): how much air you can blow out in the first second. Often given as a percentage of what is expected for your age, height and sex.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: Placebo
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Drug: Pomalidomide (CC-4047)
1 mg orally every day for 52 weeks
Matching placebo capsules taken orally once a day
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
An adverse event (AE) is any noxious, unintended, or untoward medical occurrence that may appear or worsen during the course of a study. A TEAE is any AE that began or worsened on or after the start of study drug through 28 days after the last dose. A treatment-related TEAE is a TEAE which was considered by the investigator to be related to study drug. The severity/intensity of AEs was assessed by the investigator as Mild (asymptomatic or mild symptoms; intervention not indicated), Moderate (symptoms cause moderate discomfort, intervention may be required), or Severe (symptoms cause severe discomfort/pain, requiring medical intervention, inability to perform daily activities). A serious AE is any AE that: - Resulted in death; - Was life-threatening; - Required inpatient hospitalization or prolongation of existing hospitalization - Resulted in persistent or significant disability/incapacity; - Was a congenital anomaly/birth defect; - Constituted an important medical event.
Time frame: From the start of study drug to 28 days after last dose; Treatment Phase median duration of treatment was 358 and 320 days for Placebo and Pomalidomide; Extension phase median duration of treatment was 161 days and 194 days for Placebo and pomalidomide.
Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 52
Forced vital capacity (FVC) is a pulmonary function test and is the volume of air in the lungs that can forcibly be blown out after a full inhalation. Percent predicted values are based comparison between the participant's measured value with expected FVC for someone of the same sex, age and height (reference value). For the analysis of FVC, the baseline value was defined as the average of all values between Screening and Baseline (inclusive), and the average of Weeks 48 and 52 was treated as the Week 52 value, to reduce the total data variability at the key time points.
Time frame: Baseline (defined as the average of all values between Screening and Baseline) and Weeks 48 and 52
Change From Baseline in the Modified Rodnan Skin Score (mRSS) at Week 52/Early Termination
Improvement in skin thickening is associated with improved survival and may be useful as a surrogate measurement in clinical studies. The mRSS is an assessment tool which is used to evaluate the extent and severity of the skin thickening associated with systemic sclerosis (SSc). Seventeen body areas were evaluated on a 4-point scale (0 \[normal\], 1 \[mild\], 2 \[moderate\]), or 3 \[severe\]). The total score, which is the sum of the 17 individual body assessments, can range from 0 to 51.
Time frame: Baseline and Week 52 (or the Treatment Phase Early Termination visit)
Change From Baseline in University of California, Los Angeles, Scleroderma Clinical Trial Consortium Gastrointestinal Tract (UCLA SCTC GIT 2.0) Total Score at Week 52/Early Termination
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets gastrointestinal (GI) activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health (except for Questions 15 and 31 which are scored as 0 (better health) or 1 (worse health). The total score is calculated as the average of the first 6 scale scores (excluding constipation) which captures overall burden (severity) of SSc-associated GIT. The overall score ranges from 0 to 3, where higher scores indicate more severe symptoms.
Time frame: Baseline and Week 52 (or Treatment Phase Early Termination visit)
Change From Baseline in Percent Predicted Forced Vital Capacity Over Time
Forced vital capacity (FVC) is a pulmonary function test and is the volume of air in the lungs that can forcibly be blown out after a full inhalation. Percent predicted values are based comparison between the participant's measured value with expected FVC for someone of the same sex, age and height (reference value).
Time frame: Baseline (defined as the average of all values between Screening and Baseline) and Weeks 12, 24, 36, 64, 76, and 156
Change From Baseline in Modified Rodnan Skin Score Over Time
Improvement in skin thickening is associated with improved survival and may be useful as a surrogate measurement in clinical studies. The mRSS is an assessment tool which is used to evaluate the extent and severity of the skin thickening associated with systemic sclerosis (SSc). Seventeen body areas were evaluated on a 4-point scale (0 \[normal\], 1 \[mild\], 2 \[moderate\], or 3 \[severe\]). The total score, which is the sum of the 17 individual body assessments, can range from 0 to 51.
Time frame: Baseline and Weeks 12, 24, 64, 76, and 156 (or the Extension Phase Early Termination visit).
Change From Baseline in UCLA SCTC GIT 2.0 Total Score Over Time
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health (except for Questions 15 and 31 which are scored as 0 (better health) or 1 (worse health). The total score is calculated as the average of the first 6 scale scores (excluding constipation) which captures overall burden (severity) of SSc-associated GIT. The overall score ranges from 0 to 3, where higher scores indicate more severe symptoms.
Time frame: Baseline and Weeks 12, 24, 64, 76, and 156 (or the Extension Phase Early Termination visit).
Change From Baseline in UCLA SCTC GIT 2.0 Reflux Subscale Score Over Time
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The reflux subscale score is calculated as the average of eight reflux-related questions; the score ranges from 0 to 3, where higher scores indicate more frequent symptoms.
Time frame: Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).
Change From Baseline in UCLA SCTC GIT 2.0 Distension/Bloating Subscale Score Over Time
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The distension/bloating subscale score is calculated as the average of four distension/bloating-related questions; the score ranges from 0 to 3, where higher scores indicate more frequent symptoms.
Time frame: Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).
Change From Baseline in UCLA SCTC GIT 2.0 Fecal Soilage Subscale Score Over Time
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The fecal soilage subscale score is calculated from one soilage question; the score ranges from 0 to 3, where higher scores indicate more frequent symptoms.
Time frame: Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).
Change From Baseline in UCLA SCTC GIT 2.0 Diarrhea Subscale Score Over Time
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The diarrhea subscale score is calculated as the average of one diarrhea question about the frequency of loose stools (on a scale from 0 \[none\] to 3 \[5-7 days/week\] and one question about the presence of watery stools (scored as 0 \[No\] or 1 \[Yes\]); the score ranges from 0 to 2, where a higher score indicates more frequent symptoms.
Time frame: Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).
Change From Baseline in UCLA SCTC GIT 2.0 Social Functioning Subscale Score Over Time
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The social functioning subscale score is calculated as the average of six questions about how often symptoms interfered with social activities; the score ranges from 0 to 3, where higher scores indicate more frequent symptoms.
Time frame: Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).
Change From Baseline in UCLA SCTC GIT 2.0 Emotional Well Being Subscale Score Over Time
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The emotional well-being subscale score is calculated as the average of nine questions regarding the impact of bowel problems on emotional status; the score ranges from 0 to 3, where higher scores indicate more frequent problems.
Time frame: Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).
Change From Baseline in UCLA SCTC GIT 2.0 Constipation Subscale Score Over Time
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The constipation subscale score is calculated as the average of three questions regarding the frequency of constipation (scored from 0 \[no days\] to 3 \[5-7 days/week\] and one question about the presence of stools becoming harder (scored as 0 \[No\] or 1 \[Yes\]); the score ranges from 0 to 2.5, where higher scores indicate more frequent symptoms.
Time frame: Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).
Change From Baseline in Dyspnea Functional Impairment at Week 12
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath.
Time frame: Week 12
Change From Baseline in Dyspnea Functional Impairment at Week 24
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath.
Time frame: Week 24
Change From Baseline in Dyspnea Functional Impairment at Week 52/Early Termination
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath.
Time frame: Week 52 or at the Treatment Phase Early Termination visit
Change From Baseline in Dyspnea Functional Impairment at Week 64
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath.
Time frame: Week 64
Change From Baseline in Dyspnea Functional Impairment at Week 76
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath.
Time frame: Week 76
Change From Baseline in Dyspnea Functional Impairment at Week 156/Early Termination
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath.
Time frame: Week 156 or the Extension Phase Early Termination visit
Change From Baseline in Dyspnea Magnitude of Task at Week 12
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain.
Time frame: Week 12
Change From Baseline in Dyspnea Magnitude of Task at Week 24
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain.
Time frame: Week 24
Change From Baseline in Dyspnea Magnitude of Task at Week 52/Early Termination
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain.
Time frame: Week 52 or at the Treatment Phase Early Termination visit
Change From Baseline in Dyspnea Magnitude of Task at Week 64
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain.
Time frame: Week 64
Change From Baseline in Dyspnea Magnitude of Task at Week 76
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain.
Time frame: Week 76
Change From Baseline in Dyspnea Magnitude of Task at Week 156/Early Termination
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carry very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain.
Time frame: Week 156 or the Extension Phase Early Termination visit
Change From Baseline in Dyspnea Magnitude of Effort at Week 12
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath.
Time frame: Week 12
Change From Baseline in Dyspnea Magnitude of Effort at Week 24
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath.
Time frame: Week 24
Change From Baseline in Dyspnea Magnitude of Effort at Week 52/Early Termination
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath.
Time frame: Week 52 or at the Treatment Phase Early Termination visit
Change From Baseline in Dyspnea Magnitude of Effort at Week 64
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath.
Time frame: Week 64
Change From Baseline in Dyspnea Magnitude of Effort at Week 76
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath.
Time frame: Week 76
Change From Baseline in Dyspnea Magnitude of Effort at Week 156/Early Termination
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath.
Time frame: Week 156 or at the Extension Phase Early Termination visit
Oxygen Saturation Over Time
Oxygen saturation was measured by pulse oximetry.
Time frame: Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).
Pharmacokinetic Parameters of Pomalidomide in Plasma
Pharmacokinetic (PK) analyses were not conducted as there were too few participants with available data.
Time frame: Day 1 and week 4 pre-dose and up to 24 hours post-dose.
| Milestone | Placebo | Pomalidomide |
|---|---|---|
| Started | 12 | 11 |
| Received treatment | 12 | 10 |
| Completed | 7 | 4 |
| Not completed | 5 | 7 |
| Withdrew: Adverse event | 0 | 2 |
| Withdrew: Progressive disease | 1 | 1 |
| Withdrew: Lack of efficacy | 0 | 1 |
| Withdrew: Study terminated by sponsor | 3 | 2 |
| Withdrew: Protocol exclusion criteria | 1 | 0 |
| Withdrew: Did not receive study drug | 0 | 1 |
| Milestone | Placebo | Pomalidomide |
|---|---|---|
| Started | 6 | 2 |
| Completed | 0 | 0 |
| Not completed | 6 | 2 |
| Withdrew: Adverse event | 1 | 0 |
| Withdrew: Study terminated by sponsor | 5 | 2 |
An adverse event (AE) is any noxious, unintended, or untoward medical occurrence that may appear or worsen during the course of a study. A TEAE is any AE that began or worsened on or after the start of study drug through 28 days after the last dose. A treatment-related TEAE is a TEAE which was considered by the investigator to be related to study drug. The severity/intensity of AEs was assessed by the investigator as Mild (asymptomatic or mild symptoms; intervention not indicated), Moderate (symptoms cause moderate discomfort, intervention may be required), or Severe (symptoms cause severe discomfort/pain, requiring medical intervention, inability to perform daily activities). A serious AE is any AE that: - Resulted in death; - Was life-threatening; - Required inpatient hospitalization or prolongation of existing hospitalization - Resulted in persistent or significant disability/incapacity; - Was a congenital anomaly/birth defect; - Constituted an important medical event.
| participants | Treatment Phase: Placebo | Treatment Phase: Pomalidomide | Extension Phase: Placebo/Pomalidomide | Extension Phase: Pomalidomide/Pomalidomide |
|---|---|---|---|---|
| Any TEAE | 12 | 9 | 5 | 2 |
| Drug-related TEAE | 8 | 6 | 3 | 1 |
| Severe TEAE | 1 | 4 | 0 | 0 |
| Serious TEAE | 1 | 4 | 0 | 1 |
| Serious Drug-related TEAE | 0 | 1 | 0 | 0 |
| TEAE Leading to Drug Interruption | 2 | 1 | 0 | 0 |
| TEAE Leading to Drug Withdrawal | 0 | 4 | 0 | 0 |
| TEAE Leading to Death | 0 | 0 | 0 | 0 |
Forced vital capacity (FVC) is a pulmonary function test and is the volume of air in the lungs that can forcibly be blown out after a full inhalation. Percent predicted values are based comparison between the participant's measured value with expected FVC for someone of the same sex, age and height (reference value). For the analysis of FVC, the baseline value was defined as the average of all values between Screening and Baseline (inclusive), and the average of Weeks 48 and 52 was treated as the Week 52 value, to reduce the total data variability at the key time points.
| percent predicted | Placebo | Pomalidomide |
|---|---|---|
| Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 52 | -2.8 ± 4.02 | -5.2 ± 5.29 |
Improvement in skin thickening is associated with improved survival and may be useful as a surrogate measurement in clinical studies. The mRSS is an assessment tool which is used to evaluate the extent and severity of the skin thickening associated with systemic sclerosis (SSc). Seventeen body areas were evaluated on a 4-point scale (0 \[normal\], 1 \[mild\], 2 \[moderate\]), or 3 \[severe\]). The total score, which is the sum of the 17 individual body assessments, can range from 0 to 51.
| units on a scale | Placebo | Pomalidomide |
|---|---|---|
| Change From Baseline in the Modified Rodnan Skin Score (mRSS) at Week 52/Early Termination | -3.7 ± 6.99 | -2.7 ± 5.66 |
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets gastrointestinal (GI) activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health (except for Questions 15 and 31 which are scored as 0 (better health) or 1 (worse health). The total score is calculated as the average of the first 6 scale scores (excluding constipation) which captures overall burden (severity) of SSc-associated GIT. The overall score ranges from 0 to 3, where higher scores indicate more severe symptoms.
| units on a scale | Placebo | Pomalidomide |
|---|---|---|
| Change From Baseline in University of California, Los Angeles, Scleroderma Clinical Trial Consortium Gastrointestinal Tract (UCLA SCTC GIT 2.0) Total Score at Week 52/Early Termination | 0.00 ± 0.18 | 0.1 ± 0.29 |
Forced vital capacity (FVC) is a pulmonary function test and is the volume of air in the lungs that can forcibly be blown out after a full inhalation. Percent predicted values are based comparison between the participant's measured value with expected FVC for someone of the same sex, age and height (reference value).
| percent predicted | Placebo | Pomalidomide |
|---|---|---|
| Week 12 | -2.4 ± 3.07 | -1.8 ± 3.39 |
| Week 24 | -1.3 ± 3.33 | 2.3 ± 12.58 |
| Week 36 | -2.6 ± 2.48 | -4.4 ± 3.97 |
| Week 64 | -7.0 ± 4.29 | -6.4 ± 4.49 |
| Week 76 | -8.7 ± 10.34 | -5.2 ± NA |
| Week 156 | -6.7 ± 6.19 | -5.9 ± 2.31 |
Improvement in skin thickening is associated with improved survival and may be useful as a surrogate measurement in clinical studies. The mRSS is an assessment tool which is used to evaluate the extent and severity of the skin thickening associated with systemic sclerosis (SSc). Seventeen body areas were evaluated on a 4-point scale (0 \[normal\], 1 \[mild\], 2 \[moderate\], or 3 \[severe\]). The total score, which is the sum of the 17 individual body assessments, can range from 0 to 51.
| units on a scale | Placebo | Pomalidomide |
|---|---|---|
| Week 12 | -1.8 ± 3.94 | -0.3 ± 1.83 |
| Week 24 | -3.6 ± 5.68 | -2.0 ± 3.38 |
| Week 64 | -4.3 ± 8.36 | -4.0 ± 2.83 |
| Week 76 | -8.5 ± 7.78 | -7.0 ± NA |
| Week 156/Early Termination | -3.7 ± 8.52 | -4.5 ± 3.54 |
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health (except for Questions 15 and 31 which are scored as 0 (better health) or 1 (worse health). The total score is calculated as the average of the first 6 scale scores (excluding constipation) which captures overall burden (severity) of SSc-associated GIT. The overall score ranges from 0 to 3, where higher scores indicate more severe symptoms.
| units on a scale | Placebo | Pomalidomide |
|---|---|---|
| Week 12 | 0.2 ± 0.36 | -0.1 ± 0.30 |
| Week 24 | 0.1 ± 0.23 | -0.1 ± 0.20 |
| Week 64 | 0.0 ± 0.20 | 0.4 ± 0.01 |
| Week 76 | -0.1 ± 0.01 | 0.5 ± NA |
| Week 156/Early Termination | 0.0 ± 0.14 | 0.0 ± 0.14 |
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The reflux subscale score is calculated as the average of eight reflux-related questions; the score ranges from 0 to 3, where higher scores indicate more frequent symptoms.
| units on a scale | Placebo | Pomalidomide |
|---|---|---|
| Week 12 | 0.1 ± 0.36 | -0.1 ± 0.51 |
| Week 24 | -0.1 ± 0.27 | 0.0 ± 0.47 |
| Week 52/Early Termination | -0.1 ± 0.54 | 0.1 ± 0.38 |
| Week 64 | -0.2 ± 0.14 | 0.2 ± 0.46 |
| Week 76 | -0.5 ± 0.18 | -0.2 ± NA |
| Week 156/Early Termination | 0.0 ± 0.44 | 0.1 ± 0.28 |
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The distension/bloating subscale score is calculated as the average of four distension/bloating-related questions; the score ranges from 0 to 3, where higher scores indicate more frequent symptoms.
| units on a scale | Placebo | Pomalidomide |
|---|---|---|
| Week 12 | 0.4 ± 0.56 | 0.2 ± 1.21 |
| Week 24 | 0.0 ± 0.53 | 0.3 ± 0.80 |
| Week 52/Early Termination | 0.0 ± 0.55 | 0.2 ± 1.07 |
| Week 64 | -0.2 ± 0.70 | 1.0 ± 1.41 |
| Week 76 | 0.0 ± 0.00 | 3.0 ± NA |
| Week 156/Early Termination | -0.3 ± 0.30 | 0.9 ± 1.24 |
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The fecal soilage subscale score is calculated from one soilage question; the score ranges from 0 to 3, where higher scores indicate more frequent symptoms.
| units on a scale | Placebo | Pomalidomide |
|---|---|---|
| Week 12 | 0.0 ± 0.00 | 0.0 ± 0.00 |
| Week 24 | 0.2 ± 0.42 | 0.0 ± 0.00 |
| Week 52/Early Termination | 0.0 ± 0.00 | 0.1 ± 0.33 |
| Week 64 | 0.2 ± 0.41 | 0.0 ± 0.00 |
| Week 76 | 0.0 ± 0.00 | 0.0 ± NA |
| Week 156/Early Termination | 0.2 ± 0.41 | 0.0 ± 0.00 |
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The diarrhea subscale score is calculated as the average of one diarrhea question about the frequency of loose stools (on a scale from 0 \[none\] to 3 \[5-7 days/week\] and one question about the presence of watery stools (scored as 0 \[No\] or 1 \[Yes\]); the score ranges from 0 to 2, where a higher score indicates more frequent symptoms.
| units on a scale | Placebo | Pomalidomide |
|---|---|---|
| Week 12 | 0.4 ± 0.57 | -0.2 ± 0.65 |
| Week 24 | 0.1 ± 0.21 | -0.3 ± 0.46 |
| Week 52/Early Termination | 0.3 ± 0.34 | 0.0 ± 0.75 |
| Week 64 | 0.1 ± 0.38 | 0.5 ± 0.71 |
| Week 76 | 0.0 ± 0.00 | -0.5 ± NA |
| Week 156/Early Termination | 0.3 ± 0.42 | -0.8 ± 0.35 |
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The social functioning subscale score is calculated as the average of six questions about how often symptoms interfered with social activities; the score ranges from 0 to 3, where higher scores indicate more frequent symptoms.
| units on a scale | Placebo | Pomalidomide |
|---|---|---|
| Week 12 | 0.3 ± 0.49 | 0.0 ± 0.28 |
| Week 24 | 0.1 ± 0.30 | -0.1 ± 0.20 |
| Week 52/Early termination | 0.0 ± 0.25 | 0.2 ± 0.45 |
| Week 64 | 0.0 ± 0.18 | 0.3 ± 0.35 |
| Week 76 | 0.2 ± NA | 0.0 ± NA |
| Week 156/Early Termination | 0.0 ± 0.25 | -0.2 ± 0.23 |
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The emotional well-being subscale score is calculated as the average of nine questions regarding the impact of bowel problems on emotional status; the score ranges from 0 to 3, where higher scores indicate more frequent problems.
| units on a scale | Placebo | Pomalidomide |
|---|---|---|
| Week 12 | 0.2 ± 0.40 | -0.4 ± 0.80 |
| Week 24 | 0.1 ± 0.16 | -0.3 ± 0.56 |
| Week 52/Early Termination | 0.1 ± 0.25 | -0.1 ± 0.42 |
| Week 64 | 0.0 ± 0.36 | 0.2 ± 0.00 |
| Week 76 | 0.0 ± 0.00 | 0.3 ± NA |
| Week 156/Early Termination | -0.1 ± 0.15 | 0.0 ± 0.00 |
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The constipation subscale score is calculated as the average of three questions regarding the frequency of constipation (scored from 0 \[no days\] to 3 \[5-7 days/week\] and one question about the presence of stools becoming harder (scored as 0 \[No\] or 1 \[Yes\]); the score ranges from 0 to 2.5, where higher scores indicate more frequent symptoms.
| units on a scale | Placebo | Pomalidomide |
|---|---|---|
| Week 12 | -0.1 ± 0.36 | 0.2 ± 0.98 |
| Week 24 | -0.2 ± 0.31 | 0.3 ± 0.81 |
| Week 52/Early Termination | 0.0 ± 0.25 | 0.3 ± 0.51 |
| Week 64 | 0.0 ± 0.60 | 0.1 ± 0.18 |
| Week 76 | 0.0 ± 0.00 | 0.0 ± NA |
| Week 156/Early Termination | -0.2 ± 0.20 | 0.1 ± 0.18 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath.
| Participants | Placebo | Pomalidomide |
|---|---|---|
| Major deterioration | 0 | 0 |
| Moderate deterioration | 1 | 1 |
| Minor deterioration | 0 | 1 |
| No change | 7 | 3 |
| Minor improvement | 2 | 1 |
| Moderate improvement | 0 | 0 |
| Major improvement | 0 | 0 |
| Further impairment for other reasons | 0 | 1 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath.
| Participants | Placebo | Pomalidomide |
|---|---|---|
| Major deterioration | 1 | 0 |
| Moderate deterioration | 0 | 2 |
| Minor deterioration | 1 | 1 |
| No change | 4 | 2 |
| Minor improvement | 3 | 1 |
| Moderate improvement | 1 | 0 |
| Major improvement | 0 | 0 |
| Further impairment for other reasons | 0 | 1 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath.
| Participants | Placebo | Pomalidomide |
|---|---|---|
| Major deterioration | 0 | 0 |
| Moderate deterioration | 0 | 4 |
| Minor deterioration | 1 | 1 |
| No change | 8 | 4 |
| Minor improvement | 3 | 1 |
| Moderate improvement | 0 | 0 |
| Major improvement | 0 | 0 |
| Further impairment for other reasons | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath.
| Participants | Placebo | Pomalidomide |
|---|---|---|
| Major deterioration | 0 | 1 |
| Moderate deterioration | 0 | 1 |
| Minor deterioration | 1 | 0 |
| No change | 4 | 0 |
| Minor improvement | 1 | 0 |
| Moderate improvement | 0 | 0 |
| Major improvement | 0 | 0 |
| Further impairment for other reasons | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath.
| Participants | Placebo | Pomalidomide |
|---|---|---|
| Major deterioration | 0 | 0 |
| Moderate deterioration | 0 | 1 |
| Minor deterioration | 1 | 0 |
| No change | 0 | 0 |
| Minor improvement | 1 | 0 |
| Moderate improvement | 0 | 0 |
| Major improvement | 0 | 0 |
| Further impairment for other reasons | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath.
| Participants | Placebo | Pomalidomide |
|---|---|---|
| Major deterioration | 0 | 0 |
| Moderate deterioration | 0 | 1 |
| Minor deterioration | 2 | 1 |
| No change | 2 | 0 |
| Minor improvement | 1 | 0 |
| Moderate improvement | 1 | 0 |
| Major improvement | 0 | 0 |
| Further impairment for other reasons | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain.
| Participants | Placebo | Pomalidomide |
|---|---|---|
| Major deterioration | 0 | 0 |
| Moderate deterioration | 0 | 0 |
| Minor deterioration | 2 | 1 |
| No change | 5 | 5 |
| Minor improvement | 3 | 1 |
| Moderate improvement | 0 | 0 |
| Major improvement | 0 | 0 |
| Further impairment for other reasons | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain.
| Participants | Placebo | Pomalidomide |
|---|---|---|
| Major deterioration | 0 | 0 |
| Moderate deterioration | 1 | 1 |
| Minor deterioration | 2 | 1 |
| No change | 5 | 2 |
| Minor improvement | 2 | 3 |
| Moderate improvement | 0 | 0 |
| Major improvement | 0 | 0 |
| Further impairment for other reasons | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain.
| Participants | Placebo | Pomalidomide |
|---|---|---|
| Major deterioration | 0 | 0 |
| Moderate deterioration | 0 | 3 |
| Minor deterioration | 1 | 1 |
| No change | 8 | 6 |
| Minor improvement | 2 | 0 |
| Moderate improvement | 0 | 0 |
| Major improvement | 1 | 0 |
| Further impairment for other reasons | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain.
| Participants | Placebo | Pomalidomide |
|---|---|---|
| Major deterioration | 0 | 1 |
| Moderate deterioration | 0 | 0 |
| Minor deterioration | 2 | 0 |
| No change | 3 | 0 |
| Minor improvement | 1 | 1 |
| Moderate improvement | 0 | 0 |
| Major improvement | 0 | 0 |
| Further impairment for other reasons | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain.
| Participants | Placebo | Pomalidomide |
|---|---|---|
| Major deterioration | 0 | 0 |
| Moderate deterioration | 0 | 0 |
| Minor deterioration | 1 | 0 |
| No change | 1 | 0 |
| Minor improvement | 0 | 1 |
| Moderate improvement | 0 | 0 |
| Major improvement | 0 | 0 |
| Further impairment for other reasons | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carry very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain.
| Participants | Placebo | Pomalidomide |
|---|---|---|
| Major deterioration | 0 | 0 |
| Moderate deterioration | 1 | 0 |
| Minor deterioration | 1 | 1 |
| No change | 2 | 0 |
| Minor improvement | 2 | 1 |
| Moderate improvement | 0 | 0 |
| Major improvement | 0 | 0 |
| Further impairment for other reasons | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath.
| Participants | Placebo | Pomalidomide |
|---|---|---|
| Major deterioration | 0 | 0 |
| Moderate deterioration | 1 | 0 |
| Minor deterioration | 2 | 2 |
| No change | 4 | 4 |
| Minor improvement | 2 | 1 |
| Moderate improvement | 1 | 0 |
| Major improvement | 0 | 0 |
| Further impairment for other reasons | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath.
| Participants | Placebo | Pomalidomide |
|---|---|---|
| Major deterioration | 1 | 1 |
| Moderate deterioration | 0 | 1 |
| Minor deterioration | 1 | 0 |
| No change | 4 | 3 |
| Minor improvement | 3 | 2 |
| Moderate improvement | 1 | 0 |
| Major improvement | 0 | 0 |
| Further impairment for other reasons | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath.
| Participants | Placebo | Pomalidomide |
|---|---|---|
| Major deterioration | 0 | 0 |
| Moderate deterioration | 0 | 3 |
| Minor deterioration | 1 | 1 |
| No change | 7 | 6 |
| Minor improvement | 3 | 0 |
| Moderate improvement | 1 | 0 |
| Major improvement | 0 | 0 |
| Further impairment for other reasons | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath.
| Participants | Placebo | Pomalidomide |
|---|---|---|
| Major deterioration | 0 | 1 |
| Moderate deterioration | 0 | 0 |
| Minor deterioration | 3 | 0 |
| No change | 2 | 1 |
| Minor improvement | 1 | 0 |
| Moderate improvement | 0 | 0 |
| Major improvement | 0 | 0 |
| Further impairment for other reasons | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath.
| Participants | Placebo | Pomalidomide |
|---|---|---|
| Major deterioration | 0 | 0 |
| Moderate deterioration | 0 | 0 |
| Minor deterioration | 1 | 0 |
| No change | 0 | 1 |
| Minor improvement | 1 | 0 |
| Moderate improvement | 0 | 0 |
| Major improvement | 0 | 0 |
| Further impairment for other reasons | 0 | 0 |
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath.
| Participants | Placebo | Pomalidomide |
|---|---|---|
| Major deterioration | 0 | 0 |
| Moderate deterioration | 1 | 0 |
| Minor deterioration | 2 | 1 |
| No change | 2 | 1 |
| Minor improvement | 1 | 0 |
| Moderate improvement | 0 | 0 |
| Major improvement | 0 | 0 |
| Further impairment for other reasons | 0 | 0 |
Oxygen saturation was measured by pulse oximetry.
| percent saturation | Placebo | Pomalidomide |
|---|---|---|
| Baseline | 97.5 ± 2.07 | 96.8 ± 1.92 |
| Week 12 | 97.1 ± 2.18 | 97.4 ± 1.51 |
| Week 24 | 97.6 ± 1.65 | 96.5 ± 4.31 |
| Week 52/Early Termination | 96.8 ± 2.18 | 96.8 ± 1.86 |
| Week 64 | 96.3 ± 2.94 | 94.0 ± 2.83 |
| Week 76 | 98.5 ± 0.71 | 97.0 ± NA |
| Week 156/Early Termination | 95.8 ± 5.12 | 97.5 ± 0.71 |
Pharmacokinetic (PK) analyses were not conducted as there were too few participants with available data.
No measurements were reported for this outcome.
Collected over From the start of study drug to 28 days after last dose; Treatment Phase median duration of treatment was 358 and 320 days for Placebo and Pomalidomide; Extension phase median duration of treatment was 161 days and 194 days for Placebo and Pomalidomide.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Treatment Phase: Placebo | — | 1/12 (8.3%) | 12/12 (100%) |
| Treatment Phase: Pomalidomide | — | 4/10 (40%) | 9/10 (90%) |
| Extension Phase: Placebo/Pomalidomide | — | 0/6 (0%) | 5/6 (83.3%) |
| Extension Phase: Pomalidomide/Pomalidomide | — | 1/2 (50%) | 2/2 (100%) |
| Event | Treatment Phase: Placebo | Treatment Phase: Pomalidomide | Extension Phase: Placebo/Pomalidomide | Extension Phase: Pomalidomide/Pomalidomide |
|---|---|---|---|---|
| Acute respiratory failureRespiratory, thoracic and mediastinal disorders | 0/12 | 0/10 | 0/6 | 1/2 |
| Chronic respiratory failureRespiratory, thoracic and mediastinal disorders | 0/12 | 0/10 | 0/6 | 1/2 |
| Pulmonary hypertensionRespiratory, thoracic and mediastinal disorders | 0/12 | 0/10 | 0/6 | 1/2 |
| PneumoniaInfections and infestations | 1/12 | 1/10 | 0/6 | 0/2 |
| Upper respiratory tract infectionInfections and infestations | 0/12 | 1/10 | 0/6 | 0/2 |
| Renal failureRenal and urinary disorders | 0/12 | 1/10 | 0/6 | 0/2 |
| Pulmonary embolismRespiratory, thoracic and mediastinal disorders | 0/12 | 1/10 | 0/6 | 0/2 |
| SepsisInfections and infestations | 1/12 | 0/10 | 0/6 | 0/2 |
| Event | Treatment Phase: Placebo | Treatment Phase: Pomalidomide | Extension Phase: Placebo/Pomalidomide | Extension Phase: Pomalidomide/Pomalidomide |
|---|---|---|---|---|
| Gastrooesophageal reflux diseaseGastrointestinal disorders | 0/12 | 1/10 | 0/6 | 1/2 |
| OedemaGeneral disorders | 1/12 | 0/10 | 0/6 | 1/2 |
| PainGeneral disorders | 1/12 | 1/10 | 0/6 | 1/2 |
| HyperglycaemiaMetabolism and nutrition disorders | 0/12 | 0/10 | 0/6 | 1/2 |
| HypophosphataemiaMetabolism and nutrition disorders | 0/12 | 0/10 | 0/6 | 1/2 |
| Pain in jawMusculoskeletal and connective tissue disorders | 0/12 | 0/10 | 0/6 | 1/2 |
| DizzinessNervous system disorders | 1/12 | 0/10 | 0/6 | 1/2 |
| DyspnoeaRespiratory, thoracic and mediastinal disorders | 2/12 | 0/10 | 0/6 | 1/2 |
| Pulmonary hypertensionRespiratory, thoracic and mediastinal disorders | 1/12 | 0/10 | 1/6 | 1/2 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 4/12 | 4/10 | 0/6 | 0/2 |
| Age, Continuous(years) | Placebo | Pomalidomide | Total |
|---|---|---|---|
| Mean | 44.8 ± 13.77 | 48.9 ± 9.84 | 46.7 ± 11.97 |
| Age, Customized(Participants) | Placebo | Pomalidomide | Total |
|---|---|---|---|
| < 65 years | 10 | 10 | 20 |
| ≥ 65 years | 2 | 1 | 3 |
| Sex: Female, Male(Participants) | Placebo | Pomalidomide | Total |
|---|---|---|---|
| Female | 10 | 10 | 20 |
| Male | 2 | 1 | 3 |
| Race/Ethnicity, Customized(Participants) | Placebo | Pomalidomide | Total |
|---|---|---|---|
| Asian | 0 | 2 | 2 |
| Black or African American | 0 | 1 | 1 |
| Native Hawaiian or Other Pacific Islanders | 1 | 0 | 1 |
| White | 10 | 8 | 18 |
| Other | 1 | 0 | 1 |
| Body Mass Index (BMI)(kg/m^2) | Placebo | Pomalidomide | Total |
|---|---|---|---|
| Mean | 30.64 ± 5.392 | 27.61 ± 9.4 | 29.19 ± 7.556 |
Ran from Aug 2012 to primary completion in Nov 2016, 50 months.
Results were first posted in Oct 2019, 2 years 11 months after primary completion.
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Studies of the drug tested here: Pomalidomide