A Phase 2/3 interventional study of ULSC (1.5 x 10^8 cells/dose) and Placebo (no cells) in Idiopathic Inflammatory Myositis (IIM) and DERMATOMYOSITIS OR POLYMYOSITIS, sponsored by Restem, LLC.. Recruiting at 2 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-04-27.
Sponsored by Restem, LLC. · Phase 2/3, Interventional, and Treatment
The goal of this clinical trial is to learn about how an umbilical cord lining-derived stem cell (ULSC) product performs when treating Dermatomyositis/Polymyositis (DM/PM), also known as idiopathic inflammatory myopathy (IIM) in adults. It will assess safety and efficacy in relieving symptoms of DM/PM with ULSC administered in three intravenous (IV) doses of 150 million cells per dose.
The main questions that this study plans to answer are:
Participants will have been diagnosed with either DM or PM:
Participants in this study will:
184 studies on the registry are indexed under Dermatomyositis; 63 are open to participants now.
This study's planned enrollment of 40 is above the median of 35 across 130 interventional studies indexed under Dermatomyositis.
Browse Dermatomyositis studies →Restem, LLC. is the lead sponsor of 3 studies on the registry; 2 are open to participants now.
Compared within Dermatomyositis, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Myositis. The counts are of registry records indexed under each term, refreshed daily. How we classify →
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.
Active disease as defined by any one of the following test results:
Muscle weakness or active cutaneous manifestations of dermatomyositis assessed at Screening and documented with either of the following scores:
Participants must be receiving standard of care treatment with one or more or at least 5 mg prednisone (or equivalent).
Participants will either be:
Note: The minimum workup to be performed on all prospective participants to exclude mimics is as standardly followed, which may include:
Exclusion Criteria:
The presence of any of the following criteria excludes a participant from study enrollment:
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.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
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.
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.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
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.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
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.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
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.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
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.
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.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
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.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
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.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
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.
Cohort 1 will receive 1.5 x 10\^8 ULSC per dose through IV infusion on Day 0, Month 3, and Month 6 (total of three doses). One month after the third dose, Cohort 1 will cross-over to receive Placebo IV infusion on Month 7, Month 10, and Month 13.
Biological: ULSC (1.5 x 10^8 cells/dose) · Biological: Placebo (no cells)
Cohort 2 will receive Placebo IV infusion on Day 0, Month 3, and Month 6 (total of three Placebo infusions). One month after the third dose, Cohort 2 will cross-over to receive 1.5 x 10\^8 ULSC per dose through IV infusion on Month 7, Month 10, and Month 13.
Biological: ULSC (1.5 x 10^8 cells/dose) · Biological: Placebo (no cells)
Allogeneic umbilical-cord lining stem cells (ULSC) are cryopreserved and supplied in vials to be thawed and prepared for infusion at point of use. Each dose of 1.5 x 10\^8 ULSC will be added into 250 sterile saline IV bag for infusion (total volume of 260 mL volume).
The Placebo will be 250 ml sterile saline with vehicle (total volume of 260 mL) IV bag for infusion.
Safety based on Adverse Events (AEs) and Serious Adverse Events (SAEs) that begin during or following treatment infusion.
Cumulative listing of all AEs/SAEs per cohort with descriptive statistics for categorical variables and count variables to compare between ULSC and Placebo, with emphasis on All SAEs and AEs/SAEs suspected to be treatment infusion-related.
Time frame: Each visit from Day 0, 7 days, and 30 days after each infusion, and all follow-up visits up to 12 months after the final treatment infusion.
Efficacy based on Total Improvement Score (TIS, expressed as continuous variable) in the 2016 ACR/EULAR Myositis Response Criteria
Total Improvement Score (TIS) as continuous variable (scale of 0 to 100 points) will be assessed; TIS is a weighted average of the sub-scores in the 6 core set measures that comprise the validated outcome measure in the 2016 ACR/EULAR Myositis Response Criteria.
Time frame: From baseline (i.e., before first dose per treatment) to 7 months (i.e., one month after the third/final dose per treatment) for each treatment group and each study period.
Need for Rescue Therapy (Incidence)
The incidence of need for rescue therapy will be assessed by the number of participants that require administration of rescue therapy.
Time frame: From baseline to 7 months for both study periods.
Need for Rescue Therapy (Time)
Need for rescue therapy will be assessed in terms of the time to rescue therapy, i.e. time from the first dose of study treatment to the first administration of rescue therapy required.
Time frame: From baseline to 7 months for both study periods.
Glucocorticoid Dose (prednisone equivalents per day) Tapering
Changes from baseline in glucocorticoid dose expressed in prednisone equivalents (mg/day) will be assessed as a measure of steroid tapering over time.
Time frame: From baseline to 4 months and 7 months for both study periods.
Total Improvement Score (TIS) improvement category (minimal, moderate, or major improvement) in the 2016 ACR/EULAR Myositis Response Criteria
Response relative to baseline will be assessed in terms of Total improvement category (minimal TIS ≥20, moderate TIS ≥40, or major TIS ≥60). TIS is a weighted average of the sub-scores in the 6 core set measures that comprise the validated outcome measure in the 2016 ACR/EULAR Myositis Response Criteria.
Time frame: From baseline to 7 months for both study periods.
Total Improvement Score (TIS, expressed as continuous variable) in the 2016 ACR/EULAR Myositis Response Criteria after each repeat dose
Response to repeat dosing will be assessed by TIS (scale of 0 to 100 points) at 1 month after each repeat dose: TIS at 1 month (after 1 dose), 4 months (after 2 doses), and 7 months (after 3 doses).
Time frame: From baseline to 1 month, 4 months, and 7 months for both study periods.
Modified Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) Activity Score
The mean changes in the modified CDASI activity score (scale of 0-100) will be assessed. The CDASI is a partially validated, clinician-scored, 1-page outcome measure for assessing skin disease in DM patients. It evaluates the skin in 15 anatomic locations and provides two separate scores based on activity and damage. The CDASI activity score reflects the presence and severity of active skin inflammation, and it is the sum of scores for erythema, scaling, ulceration/erosion, and activity due to Gottron's papules on the hands, recent hair loss, and periungual changes. Higher scores indicate greater disease severity.
Time frame: From baseline to 7 months for both study periods.
Modified Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) Damage Score
The mean changes in the modified CDASI damage score (scale of 0-32) will be assessed. The CDASI is a partially validated, clinician-scored, 1-page outcome measure for assessing skin disease in DM patients. It evaluates the skin in 15 anatomic locations and is comprised of two separate scores based on activity and damage. The CDASI damage score reflects the chronic manifestations of skin disease, and it is the sum of scores for poikiloderma and calcinosis and damage due to Gottron's papules on the hands, recent hair loss, and periungual changes. Higher scores indicate greater disease severity.
Time frame: From baseline to 7 months for both study periods.
Myositis Disease Activity Assessment Tool (MDAAT)
The mean changes in scores on the MDAAT will be assessed. The MDAAT utilizes Visual Analogue Scale (VAS). The VAS is a 10-point scale (left end of line = no evidence of disease activity, midpoint of line = moderate disease activity, and right end of line = extreme or maximum disease activity) which the physician assesses, for each of several pre-designated extra-muscular organ systems. The common format is a horizontal bar or line. Physicians assess each designated organ system based on how active the patient's disease has been within the prior four weeks. These VAS ratings are determined with the help of a listed set of clinical and laboratory findings.
Time frame: From baseline to 7 months for both study periods.
Myositis Damage Index (MDI) Extent Score
The mean changes in scores on the myositis damage index (MDI) extent score will be assessed. Each organ system is scored 0 or 1 (0 means never present and 1 means present for at least 6 months). The total extent score is the sum of all items scored across all systems divided by the total possible maximum score (scale of 0 to 38 in adults).
Time frame: From baseline to 7 months for both study periods.
Myositis Damage Index (MDI) Severity Score
The mean changes in scores on the myositis damage index (MDI) Severity Score will be assessed. Clinician rates the severity of the damage of each organ system using a 10 cm visual analogue scale (VAS) from 0 to 10 (0 means no damage and 10 means extreme damage). Final score is the sum of all systems (scale of 0 to 110 for all 11 systems).
Time frame: From baseline to 7 months for both study periods.
Cutaneous Dermatomyositis Investigator Global Assessment (CDM-IGA)
The mean changes in scores on the CDM-IGA will be assessed. The CDM-IGA observes the skin on 11 areas of the body, which are scored on a scale of 0 to 4 based on color and extent of the active cutaneous disease. The final CDM-IGA score is equal to the highest scoring characteristic.
Time frame: From baseline to 7 months for both study periods.
Self-reported physical function capability measured by PROMIS PF-20a
The mean change in self-reported physical function capability in each treatment group as measured by PROMIS PF-20 will be assessed. The PROMIS PF-20 measures asks 20 questions about the functioning of the upper extremities (dexterity), lower extremities (walking or mobility), and central regions (neck, back), as well as instrumental activities of daily living, such as running errands. The PROMIS PF-20a provides a single Physical Function capability score and assesses current function.
Time frame: From baseline to 7 months for both study periods.
Glucocorticoid Dose (prednisone equivalents per day) Tapering after repeat doses of study treatment
The mean change from baseline in glucocorticoid dose expressed in prednisone equivalents (mg/day) will be assessed as a measure of steroid tapering after two and three doses of study treatment.
Time frame: Baseline, from 3 months to 4 months, and from 6 months to 7 months, for each study period.
Time to achieve maximum TIS improvement category
The time (months) from study treatment initiation to when the maximum improvement category is achieved in TIS will be assessed. The improvement in each subject will be categorized as minimal (≤ 20 points change), moderate (21 - 40 points), or major (\> 40 points).
Time frame: From baseline to within 7 months for each study period.
Time to confirmed deterioration in the TIS expressed as continuous variable
The time (months) from study treatment initiation to confirmed deterioration in TIS (continuous variable, scale of 0 to100) will be assessed.
Time frame: From baseline to each visit within 7 months for each study period.
Pulmonary function test - Forced Expiratory Volume in one second (FEV1)
Pulmonary function will be evaluated for normal or abnormal based on the Forced Expiratory Volume in one second (FEV1, Liters) measured by spirometry. FEV1 measures how quickly air can be exhaled in the first second of a forced exhalation. A low FEV1 indicates difficulty with quick air flow out of the lungs, such as in obstructive lung disease. Changes will be assessed.
Time frame: From baseline to 7 months for both study periods.
Pulmonary function test - Forced Vital Capacity (FVC)
Pulmonary function will be evaluated for normal or abnormal based on the Forced Vital Capacity (FVC, Liters) measured by spirometry. FVC measures the maximum amount of air a person can forcefully blow out after taking the deepest possible breath. A low FVC can result from poor effort or conditions that restrict the ability of the lungs to fill, such as in restrictive lung diseases. Changes will be assessed.
Time frame: From baseline to 7 months for both study periods.
Pulmonary function test - FEV1/FVC ratio
Pulmonary function will be evaluated for normal or abnormal based on the ratio of spirometry measurements of FEV1/FVC (%). The FEV1/FVC ratio helps differentiate between obstructive lung diseases (like asthma or COPD, which lead to disproportionately low FEV1) that result in low FEV1/FVC ratio, and restrictive lung diseases (like pulmonary fibrosis, which lead to low FVC) that can have FEV1/FVC ratio appear normal or high. Changes will be assessed.
Time frame: From baseline to 7 months for both study periods.
Pulmonary function test - Peak Expiratory Flow (PEF)
Pulmonary function will be evaluated for normal or abnormal based on the Peak Expiratory Flow (PEF, Liters per second) measured by spirometry. PEF measures the maximum speed (flow rate) at which air can be exhaled from the lungs after a deep breath. A high PEF indicates strong lung airflow, and a low PEF suggests potential issues like airway obstruction. Changes will be assessed.
Time frame: From baseline to 7 months for both study periods.
Pulmonary function test - Diffusing Capacity of the Lungs for carbon monoxide (DLCO)
Pulmonary function will be evaluated for normal or abnormal based on the Diffusing Capacity of the Lungs for Carbon monoxide (DLCO, %). DLCO measures the efficiency of gas exchange in the lungs. A normal DLCO indicates healthy gas exchange in the lungs. A low DLCO may suggest possible reduction in the surface area for gas exchange or possible issue with blood flow or blood vessels in the lungs. DLCO is typically measured along with other pulmonary function tests, such as spirometry. Changes will be assessed.
Time frame: From baseline to 7 months for both study periods.
Left ventricular function evaluated by transthoracic echocardiography
Left ventricular function will be evaluated for normal or abnormal based on transthoracic echocardiography (TTE) parameters measuring left ventricular ejection fraction (LVEF), left ventricular end diastolic volume (LVEDV), and left ventricular end systolic volume (LVESV).
Time frame: From baseline to 7 months for both study periods.
Functional Index-3 (FI-3) test
Changes in endurance of repetitive muscle activity will be assessed by the Functional Index-3 (FI-3) test. The FI-3 is a validated method for assessing functional disability (i.e., muscle endurance and stamina) in patients with DM or PM. The assessment consists of 3 tasks, performed either unilaterally or bilaterally, to test shoulder, neck, and hip flexion, with a total score (0-100) computed from the task results. For this study, neck (30), arm (60), and leg lifts (60) on dominant side will be tested using metronome.
Time frame: From baseline to 7 months for both study periods.
Myositis Antibody Panel
Changes in disease activity will be assessed using the extended myositis specific antibody panel IgG titers as biomarkers of myositis disease activity.
Time frame: From baseline to 7 months for both study periods.
Erythrocyte Sedimentation Rate (ESR) - systemic inflammatory/immune marker
Changes in erythrocyte sedimentation rate (ESR, mm/hour) will be measured as a systemic inflammatory/immune marker. The ESR lab test measures the rate at which red blood cells (erythrocytes) settle in a vertical blood tube over one hour. During inflammation, blood proteins increase that result in ESR increase.
Time frame: From baseline to 7 months for both study periods and post-study follow-up at 25 months.
C-Reactive Protein (CRP) - systemic inflammatory/immune marker
Changes in C-Reactive Protein (CRP, mg/L) will be measured as a systemic inflammatory/immune marker in serum samples using a high-sensitivity CRP (HS-CRP) imunoturbidimetric test.
Time frame: From baseline to 7 months for both study periods and post-study follow-up at 25 months.
TGF-β1 - systemic inflammatory/immune marker
Changes in cytokine levels of transforming growth factor-beta 1 (TGF-β1, pg/mL) will be measured as a systemic inflammatory/immune marker in plasma samples using a Luminex-based assay panel.
Time frame: From baseline to 7 months for both study periods and post-study follow-up at 25 months.
TNF-α - systemic inflammatory/immune marker
Changes in cytokine levels of tumor necrosis factor-alpha (TNF-α, pg/mL) will be measured as a systemic inflammatory/immune marker in plasma samples using a Luminex-based assay panel.
Time frame: From baseline to 7 months for both study periods and post-study follow-up at 25 months.
IL-1β - systemic inflammatory/immune marker
Changes in cytokine levels of interleukin-1 beta (IL-1β, pg/mL) will be measured as a systemic inflammatory/immune marker in plasma samples using a Luminex-based assay panel.
Time frame: From baseline to 7 months for both study periods and post-study follow-up at 25 months.
IL-1ra - systemic inflammatory/immune marker
Changes in cytokine levels of interleukin-1 receptor antagonist (IL-1ra, pg/mL) will be measured as a systemic inflammatory/immune marker in plasma samples using a Luminex-based assay panel.
Time frame: From baseline to 7 months for both study periods and post-study follow-up at 25 months.
IL-2 - systemic inflammatory/immune marker
Changes in cytokine levels of interleukin-2 (IL-2, pg/mL) will be measured as a systemic inflammatory/immune marker in plasma samples using a Luminex-based assay panel.
Time frame: From baseline to 7 months for both study periods and post-study follow-up at 25 months.
IL-4 - systemic inflammatory/immune marker
Changes in cytokine levels of interleukin-4 (IL-4, pg/mL) will be measured as a systemic inflammatory/immune marker in plasma samples using a Luminex-based assay panel.
Time frame: From baseline to 7 months for both study periods and post-study follow-up at 25 months.
IL-6 - systemic inflammatory/immune marker
Change in cytokine levels of interleukin-6 (IL-6, pg/mL) will be measured as a systemic inflammatory/immune marker in plasma samples using a Luminex-based assay panel.
Time frame: From baseline to 7 months for both study periods and post-study follow-up at 25 months.
IL-8 - systemic inflammatory/immune marker
Changes in cytokine levels of interleukin-8 (IL-8, pg/mL) will be measured as a systemic inflammatory/immune marker in plasma samples using a Luminex-based assay panel.
Time frame: From baseline to 7 months for both study periods and post-study follow-up at 25 months.
IL-10 - systemic inflammatory/immune marker
Changes in cytokine levels of interleukin-10 (IL-10, pg/mL) will be measured as a systemic inflammatory/immune marker in plasma samples using a Luminex-based assay panel.
Time frame: From baseline to 7 months for both study periods and post-study follow-up at 25 months.
IL-17A - systemic inflammatory/immune marker
Changes in cytokine levels of interleukin-17A (IL-17A, pg/mL) will be measured as a systemic inflammatory/immune marker in plasma samples using a Luminex-based assay panel.
Time frame: From baseline to 7 months for both study periods and post-study follow-up at 25 months.
IFN-γ - systemic inflammatory/immune marker
Changes in cytokine levels of interferon gamma (IFN-γ, pg/mL) will be measured as a systemic inflammatory/immune marker in plasma samples using a Luminex-based assay panel.
Time frame: From baseline to 7 months for both study periods and post-study follow-up at 25 months.
IP-10/ CXCL10 - systemic inflammatory/immune marker
Changes in chemokine levels of interferon gamma-induced protein 10 (IP-10/ CXCL10, pg/mL) will be measured as a systemic inflammatory/immune marker in plasma samples using a Luminex-based assay panel.
Time frame: From baseline to 7 months for both study periods and post-study follow-up at 25 months.
Leukocyte phenotyping (if feasible)
Changes in leukocyte, lymphocyte effector, and regulatory phenotype profiles will be assessed by flow cytometry analysis of CD3+, CD4+, and CD8+; CD25+/Foxp3+ cells.
Time frame: From baseline to 7 months for both study periods and post-study follow-up at 25 months.
Monocyte M1 and M2 phenotyping (if feasible)
Changes in Monocyte M1 and M2 phenotyping (CD14+, CD16+, CD200+) will be assessed by flow cytometry analysis.
Time frame: From baseline to 7 months for both study periods and post-study follow-up at 25 months.
PROMIS Fatigue 13a (FACIT Fatigue Scale)
Changes in patient responses on the PROMIS Fatigue 13a (FACIT Fatigue Scale) questionnaire will be evaluated. This is a 13-item measure that assesses self-reported fatigue and its impact upon daily activities and function during the past week. The level of fatigue is measured on a four point Likert scale (4 = not at all fatigued to 0 = very much fatigued), and the Score is the sum of the scores for all 13 items. The Score range is from 0 to 52; a score of less than 30 indicates severe fatigue, and higher scores indicate better quality of life.
Time frame: From baseline to 7 months for both study periods and post-study follow-up at 25 months.
Patient Global Assessment of Pain (PtGA-Pain)
Changes in patient responses on the Patient Global Assessment of Pain (PtGA-Pain) will be evaluated. The PtGA-Pain is a tool for recording patient reported pain levels without use of Visual Analog Scale (VAS).
Time frame: From baseline to 7 months for both study periods and post-study follow-up at 25 months.
Physician Global Disease Activity Visual Analog Scale (VAS) - TIS Core Set Measure 1 of 6
Changes in the Physician Global Disease Activity Visual Analog Scale (VAS) TIS sub-score will be assessed. The VAS is a 10-point scale, across which a respondent selects a point that best reflects the intensity of disease activity. The common format is a horizontal bar or line. The VAS is anchored at each end by terms that describe disease severity extremes: 0 for no disease activity, and 10 for the most severe disease activity. The IIM six core set measures include three VAS 10 scales, consisting of physician-reported, patient-reported, and extramuscular disease activity.
Time frame: From baseline to 7 months for both study periods and post-study follow-up at 25 months.
Patient Global Disease Activity (VAS) - TIS Core Set Measure 2 of 6
Changes in the Patient Global Disease Activity (VAS) TIS sub-score will be assessed. The VAS is a 10-point scale, across which a respondent selects a point that best reflects the intensity of disease activity. The common format is a horizontal bar or line. The VAS is anchored at each end by terms that describe disease severity extremes: 0 for no disease activity, and 10 for the most severe disease activity. The IIM six core set measures include three VAS 10 scales, consisting of physician-reported, patient-reported, and extramuscular disease activity.
Time frame: From baseline to 7 months for both study periods and post-study follow-up at 25 months.
Manual Muscle Strength Test (MMT-8) - TIS Core Set Measure 3 of 6
Changes in the MMT-8 TIS sub-score will be assessed. The MMT-8 testing is performed as part of the 2016 ACR/EULAR Myositis Response Criteria for Minimal, Moderate, and Major Clinical Response in Adult Dermatomyositis and Polymyositis and is a series of physical tasks done by the patient in tandem with the physician to assess resistance and endurance on target muscles (some of which overlap with the FI-3 below) for an overall score between 0 and 80 (when assessed unilaterally).
Time frame: From baseline to 7 months for both study periods and post-study follow-up at 25 months.
Health Assessment Questionnaire Disability Index (HAQ-DI) - TIS Core Set Measure 4 of 6
Changes in the HAQ-DI TIS sub-score will be assessed. The HAQ was developed as a comprehensive measure of outcome in patients with a wide variety of rheumatic diseases. There are 8 sections: dressing, arising, eating, walking, hygiene, reach, grip, and activities. There are 2 or 3 questions for each section. Scoring within each section is from 0 (without any difficulty) to 3 (unable to do). For each section the score given to that section is the worst score within the section, i.e. if one question is scored 1 and another 2, then the score for the section is 2. In addition, if an aid or device is used or if help is required from another individual, then the minimum score for that section is 2. If the section score is already 2 or more then no modification is made.
Time frame: From baseline to 7 months for both study periods and post-study follow-up at 25 months.
Extramuscular Disease Activity (VAS) - TIS Core Set Measure 5 of 6
Changes in the Extramuscular Disease Activity (VAS) TIS sub-score will be assessed. The VAS is a 10-point scale, across which a respondent selects a point that best reflects the extramuscular disease activity. The common format is a horizontal bar or line. The VAS is anchored at each end by terms that describe disease severity extremes: 0 for no disease activity, and 10 for the most severe disease activity. The IIM six core set measures include three VAS 10 scales, consisting of physician-reported, patient-reported, and extramuscular disease activity.
Time frame: From baseline to 7 months for both study periods and post-study follow-up at 25 months.
Muscle Enzymes - TIS Core Set Measure 6 of 6
Changes in the TIS sub-score for serum level of muscle enzymes will be assessed.
Time frame: From baseline to 7 months for both study periods and post-study follow-up at 25 months.
The percentage of patients by treatment group who met the criteria for clinical deterioration
The percentage of participants in each cohort who met the criteria for clinical deterioration up to 7 months will be assessed.
Time frame: From baseline to within 7 months for each study period.
The percentage of participants in each cohort who require rescue therapy other than steroids
The percentage of participants in each cohort who require rescue therapy other than steroids will be assessed.
Time frame: From baseline to within 7 months for each study period.
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