A Phase 3 interventional study of sutimlimab (BIVV009) and placebo in Cold Agglutinin Disease, sponsored by Bioverativ, a Sanofi company. Completed at 53 sites in 14 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2022-12-23.
Sponsored by Bioverativ, a Sanofi company (part of Sanofi) · Phase 3, Interventional, and Treatment
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Among 31 studies in Anemia, Hemolytic, Autoimmune with a site in United States — 4 open to participants now, 9 under way, 4 of them phase 3. Anemia, Hemolytic, Autoimmune · United States.
Among the 31 studies in Anemia, Hemolytic, Autoimmune with a site in United States, this one is the 7th largest by enrollment of the 30 interventional ones that give a count, is one of 4 that started in 2018, is one of 8 in phase 3, and was the 8th registered.
103 studies on the registry are indexed under Anemia, Hemolytic, Autoimmune; 39 are open to participants now.
Browse Anemia, Hemolytic, Autoimmune studies →4 of the 31 studies in Anemia, Hemolytic, Autoimmune with a site in United States are open now. Recruiting first, the newest first:
Sanofi · Sites in United States, Argentina, Austria and 15 more countries
Ages 18+
Lakefront Biotherapeutics West LLC · Sites in United States, Australia
Ages 18–75
Rigel Pharmaceuticals · Sites in United States, Australia, Austria and 15 more countries
Ages 18–100
Juno Therapeutics, Inc., a Bristol-Myers Squibb Company · Sites in United States, Denmark, Germany and 1 more country
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 24 Anemia, Hemolytic, Autoimmune studies recruiting now, 12 (50%) set no upper age limit and 10 (42%) stop at 75 or younger; 5 take children under 18; none take healthy volunteers.
This study: 18 Years and older · 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.
31 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 6 · Phase 2 18 · Phase 3 9
First in 1997; this study’s year is marked. 4 started in 2018.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 42 is above the median of 23 across 78 interventional studies indexed under Anemia, Hemolytic, Autoimmune.
Median enrollment 24 across the 30 interventional studies, 429 across the 1 observational. Of the 18 that completed or were terminated, 13 (72%) have results posted.
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26 of 31 have an industry lead sponsor. The 10 most frequent:
This study is registered under Bioverativ, a Sanofi company, one of the names counted as Sanofi. Under that name alone:
Bioverativ, a Sanofi company is the lead sponsor of 16 studies on the registry; none are open to participants now.
Of its 12 completed or terminated interventional studies of FDA-regulated products, 7 (58%) have results posted.
Sanofi has 9 studies filed under Anemia, Hemolytic, Autoimmune — fewer than ten, so there is no comparison to draw.
1 of the studies in Anemia, Hemolytic, Autoimmune with a site in United States that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT07083960Lakefront Biotherapeutics West LLCOM336 in Autoimmune Cytopenias | 2026-12 |
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Among the 31 studies in Anemia, Hemolytic, Autoimmune with a site in United States, from their registry updates:
Nothing recorded among them since 2026-09-25.
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The purpose of Part A was to determine whether sutimlimab administration resulted in a greater than or equal to (>=)1.5 grams per deciliter (g/dL) increase in hemoglobin (Hgb) level and avoidance of transfusion in participants with primary cold agglutinin disease (CAD) without a recent history of blood transfusion. The purpose of Part B was to evaluate the long-term safety and tolerability of sutimlimab in participants with primary CAD.
The planned total study duration per participant was approximately 1.5 to 2.5 years.
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.
Exclusion criteria:
The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
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.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
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.
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.
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.
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.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
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.
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.
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.
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.
Participants with primary CAD and without a recent history of blood transfusion during the last 6 months prior to enrollment in this study, received an intravenous (IV) infusion of BIVV009 6.5 g (for participants less than \[\<\]75 kilograms \[kg\]) or 7.5 g dose (for participants greater than or equal to \[\>=\]75 kg) on Day 0 and Day 7 and every 14 days thereafter in Part A up to Week 25. Participants who completed Part A per protocol through the end of treatment visit (Week 26), received placebo on Week 26 and continued to receive BIVV009 6.5 or 7.5 g in Part B, every 2 weeks starting at Week 27 for up to an additional 149 weeks (for 6.5 g) or 121 weeks (for 7.5 g). All participants who completed Part A elected to continue in Part B.
Drug: sutimlimab (BIVV009)
Participants with primary CAD and without a recent history of blood transfusion during the last 6 months prior to enrollment in this study, received an IV infusion of placebo matched to BIVV009 on Day 0 and Day 7 and every 14 days thereafter in Part A up to Week 25. Participants who completed Part A per protocol through the end of treatment visit (Week 26) received BIVV009 6.5 (if \<75 kg) or 7.5 g (if \>=75 kg) in Part B, on Week 26 and Week 27 and every 2 weeks thereafter for up to an additional 123 weeks (for 6.5 g) or 137 weeks (for 7.5 g). All participants who completed Part A elected to continue in Part B.
Drug: sutimlimab (BIVV009) · Drug: placebo
Pharmaceutical form: solution for injection Route of administration: intravenous (i.v.)
Pharmaceutical form: solution for injection Route of administration: intravenous (i.v.)
Part A: Percentage of Participants With Response to Treatment
A participant was considered a responder: if he or she did not receive blood transfusion from Week 5 through Week 26 (end of treatment) and did not receive treatment for CAD beyond what was permitted per protocol. Additionally, participant's hemoglobin (Hgb) level must have increased to \>=1.5 grams per deciliter (g/dL) from baseline (defined as last Hgb value before administration of first dose of study drug) at treatment assessment timepoint (defined as average of values from the Week 23, 25, and 26 visits). Percentage of responders was calculated together with 95% exact Clopper-Pearson confidence interval (CI).
Time frame: From Week 5 through Week 26
Part B: Number of Participants With Treatment-emergent Adverse Events (AEs) and Serious AEs (SAEs)
Adverse Event (AE): any untoward medical occurrence in a participant who received study drug and did not necessarily have to have a causal relationship with the treatment. Treatment emergent serious adverse events (TESAEs) was defined as any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was congenital anomaly/birth defect, was medically important event. Treatment emergent adverse events (TEAEs): AEs that developed, worsened or became serious during the treatment-emergent (TE) period (from first investigational medicinal product \[IMP\] administration in Part B to last IMP administration + 9 weeks follow-up period).
Time frame: Part B, 6.5 g cohort: From first dose (Week 26) up to 149 weeks of treatment + 9 weeks of follow-up (i.e., up to Week 184); Part B, 7.5 g cohort: From first dose (Week 26) up to 137 weeks of treatment + 9 weeks of follow-up (i.e., up to Week 172)
Part A: Mean Change From Baseline in Hemoglobin (Hgb) Level at the Treatment Assessment Timepoint
Mean change from baseline (Week 0) in Hemoglobin (Hgb) at the treatment assessment timepoint is reported in this outcome measure. Treatment assessment timepoint was defined as the average of the values from the Week 23, 25, and 26 visits. Least squares (LS) mean and 95 % confidence interval (CI) was assessed by Mixed Model for Repeated Measures (MMRM) approach using heterogeneous Toeplitz (TOEPH) covariance matrix with change from baseline as the dependent variable and baseline value and visits as independent variables. Baseline was defined as the last non-missing value prior to the first administration of study drug.
Time frame: Baseline (Week 0), treatment assessment timepoint (i.e., average of Week 23, 25 and 26)
Part A: Mean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score at the Treatment Assessment Timepoint
FACIT-Fatigue scale consists of 13 questions assessed using a 5-point scale (0=not at all; 1 = a little bit, 2 = somewhat, 3 = quite a bit and 4 = very much). Responses to each question were added to obtain a total score. Total score ranged from 0 to 52, with higher score indicating more fatigue. Treatment assessment timepoint was defined as the average of the values from the Week 23, 25, and 26 visits. LS mean and 95% CI was assessed by MMRM approach using TOEPH covariance matrix with change from baseline (Week 0) as the dependent variable and baseline value and visits as independent variables. Baseline was defined as the last non-missing value prior to the first administration of study drug.
Time frame: Baseline (Week 0), treatment assessment timepoint (i.e., average of Week 23, 25 and 26)
Part A: Mean Change From Baseline in Total Bilirubin Levels at the Treatment Assessment Timepoint
Mean change from baseline (Week 0) in total bilirubin at the treatment assessment timepoint is reported in this outcome measure. Treatment assessment timepoint was defined as the average of the values from the Week 23, 25, and 26 visits. Baseline was defined as the last non-missing value prior to the first administration of study drug.
Time frame: Baseline (Week 0), treatment assessment timepoint (i.e., average of Week 23, 25 and 26)
Part A: Mean Change From Baseline in Lactate Dehydrogenase (LDH) at the Treatment Assessment Timepoint
Mean change from baseline (Week 0) in LDH at the treatment assessment timepoint is reported in this outcome measure. Treatment assessment timepoint was defined as the average of the values from the Week 23, 25, and 26 visits. Baseline was defined as the last non-missing value prior to the first administration of study drug.
Time frame: Baseline (Week 0), treatment assessment timepoint (i.e., average of Week 23, 25 and 26)
Part A: Percentage of Participants With Solicited Symptomatic Anemia at Week 26
Symptomatic anemia was defined as having following symptoms: i. Fatigue; ii. Weakness; iii. Shortness of breath; iv. Palpitations, fast heartbeat; v. Light headedness and/or vi. Chest pain. Percentage of participants with solicited symptomatic anemia symptoms was reported in this outcome measure.
Time frame: Week 26
Part B: Change From Baseline in Hemoglobin (Hgb) Level at Each Specified Time Points
Change from baseline (Week 0) in Hgb levels at each specified time points (i.e., Week 27, 29, 31, 33, 35, 37, 39, 41, 43,45, 47, 49, 51, 53, 55, 57, 59, 61, 63, 65, 67, 69, 71, 73, 75, 77, 79, 81, 83,85,87, 89,91, 93, 95, 97, 99,101,103, 105, 107,109, 111,113,115,117,119, 121,123, 125, 127,129,131,133,135,137,139,141,143,145,147,149,151,153, 155,157,159,161,163,165,167,169,171,173,175 and ET/SFU Visit) is reported in this outcome measure. Baseline was defined as the last non-missing value prior to the first administration of study drug in Part A. Early Termination (ET) visit/safety follow up (SFU) visit was 9 weeks after administration of last dose (i.e., up to Week 184). Here, "0" in the number analyzed field signifies that none of participants were available for assessment at the specified timepoints.
Time frame: Baseline (Week 0), every 2 weeks starting from Week 27 till Week 175 and at ET/SFU visit (i.e., up to Week 184)
Part B: Change From Baseline in Total Bilirubin Levels at Each Specified Time Points
Change from baseline (Week 0) in total bilirubin levels at each specified time points (i.e., Week 27, 29, 31, 33, 35, 37, 39, 41, 43, 45, 47, 49, 51, 53, 55, 57, 59, 61, 63, 65,67,69,71,73,75, 77, 79, 81,83, 85, 87, 89, 91, 93, 95, 97, 99,101,103, 105, 107,109, 111,113,115,117,119, 121, 123, 125, 127,129,131,133,135,137,139,141,143, 145,147,149,151,153,155,157,159, 161,163,165,167,169,171,173,175 and ET/SFU Visit) is reported in this outcome measure. Baseline was defined as the last non-missing value prior to the first administration of study drug in Part A. ET visit/SFU visit was 9 weeks after administration of last dose (i.e., up to Week 184). Here, "0" in the number analyzed field signifies that none of participants were available for assessment at the specified timepoints.
Time frame: Baseline (Week 0), every 2 weeks starting from Week 27 till Week 175 and at ET/SFU visit (i.e., up to Week 184)
Part B: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score (Quality of Life) at Each Specified Time Points
FACIT-Fatigue scale consists of 13 questions assessed using a 5-point scale (0=not at all; 1 = a little bit, 2 = somewhat, 3 = quite a bit and 4 = very much). Responses to each question were added to obtain a total score. The Total score ranged from 0 to 52, with higher score indicating more fatigue. Baseline (Week 0) was defined as the last non-missing value prior to the first administration of study drug in Part A. ET visit/SFU visit was 9 weeks after administration of last dose (i.e., up to Week 184).
Time frame: Baseline (Week 0), Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135, 147, 159, 171 and ET Visit/SFU visit (i.e., up to Week 184)
Part B: Change From Baseline in 12-Item Short-Form Survey (SF-12) Physical Component Summary (PCS) and Mental Component Summary (MCS) Scores at Each Specified Time Points
SF-12: 12 item-questionnaire assessed health-related quality of life (HRQOL), contained 12 items, categorized into 8 domains (subscales) of functioning and well-being: physical functioning, role-physical, role emotional, mental health, bodily pain, general health, vitality and social functioning, with each domain score ranged from 0 (poor health) to 100 (better health). Higher scores = good health condition. These 8 domains were further summarized into 2 summary scores, PCS and MCS that ranged from 0 (poor health) to 100 (better health). Higher scores = better HRQOL. Baseline (Week 0): last non-missing value prior to first administration of study drug in Part A. ET visit/SFU visit: 9 weeks after administration of last dose (i.e., up to Week 184).
Time frame: Baseline (Week 0), Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135, 147, 159, 171 and ET Visit/SFU visit (i.e., up to Week 184)
Part B: Change From Baseline in 5-level European Quality of Life 5- Dimensions 5-Level Questionnaire (EQ-5D-5L) Health State Utility Index and VAS Scores at Each Specified Time Points
EQ-5D-5L included 2 components: health state utility index (descriptive system) and Visual Analog Scale (VAS). EQ-5D descriptive system comprises 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 5 response option: no problem, slight problem, moderate problem, severe problem and extreme problems measured with Likert scale. EQ-5D-5L responses converted into single index utility score between 0 to 1. Higher score=better health. EQ-5D-5L VAS rated participant's current health state on scale from 0 (worst imaginable health) to 100 (best imaginable health). Baseline (Week 0): last non-missing value prior to first administration of study drug in Part A. ET visit/SFU visit: 9 weeks after administration of last dose (i.e., up to Week 184).
Time frame: Baseline (Week 0), Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135, 147, 159, 171 and ET Visit/SFU visit (i.e., up to Week 184)
Part B: Number of Participants With Response to Participant's Global Impression of (Fatigue) Severity (PGIS) Questionnaire at Each Specified Time Points
The PGIS is a self-reported scale. The PGIS is a 1-item questionnaire designed to assess participant's impression of disease severity using a 5-point scale ranging from 1 to 5, where 1=none, 2=mild, 3=moderate, 4=severe, 5=very severe. Higher scores indicated greater severity. ET visit/SFU visit was 9 weeks after administration of last dose (i.e., up to Week 184).
Time frame: Baseline (Week 0), Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135, 147, 159, 171 and ET Visit/SFU visit (i.e., up to Week 184)
Part B: Number of Participants With Response to Participant's Global Impression of Change (PGIC) Questionnaire at Each Specified Time Points
PGIC is a self-administered questionnaire to evaluate the improvement or worsening compared to the start of the study. PGIC was assessed on a 7-point Likert scale ranged from 1 (greatly improved) to 7 (greatly worsened). Categories were defined based on the PGIC scores as follows: 1=very much improved, 2=much improved, 3=minimally improved, 4=no change, 5=minimally worse, 6=much worse and 7=very much worsen. Higher scores indicated greater severity. ET visit/SFU visit was 9 weeks after administration of last dose (i.e., up to Week 184).
Time frame: Baseline (Week 0), Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135, 147, 159, 171 and ET Visit/SFU visit (i.e., up to Week 184)
Part B: Mean Change From Baseline in Lactate Dehydrogenase (LDH) Level at Each Specified Time Points
Change from baseline (Week 0) in LDH levels at each specified time points (i.e., Week 27, 29, 31, 33, 35, 37, 39, 41, 43, 45, 47, 49, 51, 53, 55, 57, 59, 61, 63, 65, 67, 69, 71, 73, 75, 77, 79, 81, 83, 85, 87, 89, 91, 93, 95, 97, 99, 101, 103, 105, 107,109, 111, 113, 115, 117, 119, 121,123, 125, 127,129,131,133,135,137,139,141,143,145,147, 149, 151,153,155,157,159,161,163,165,167,169, 171, 173, 175 and ET/SFU Visit) is reported in this outcome measure. Baseline was defined as the last non-missing value prior to the first administration of study drug in Part A. ET/SFU visit was 9 weeks after administration of last dose (i.e., up to Week 184).
Time frame: Baseline (Week 0), every 2 weeks starting from Week 27 till Week 175 and at ET/SFU visit (i.e., up to Week 184)
Part B: Number of Blood Transfusions Per Participant
A participant was to receive a transfusion if his or her Hgb level met either of the following criteria: Hgb was \<9 g/dL and the participant had symptoms of anemia or Hgb was \<7 g/dL and the participant was asymptomatic.
Time frame: From Week 27 up to 149 weeks of treatment (i.e., up to Week 176)
Part B: Mean Change From Baseline in Haptoglobin Values at Each Specified Time Points
Change from baseline (Week 0) in haptoglobin values at each specified time points (i.e., Week 27, 29, 31, 33, 35, 37, 39, 41, 43, 45, 47, 49, 51, 53, 55, 57, 59, 61, 63, 65, 67, 69, 71, 73, 75, 77, 79, 81, 83, 85, 87, 89, 91, 93, 95, 97,99,101,103, 105, 107,109, 111,113,115,117,119, 121,123, 125, 127, 129,131,133,135,137,139,141,143, 145,147,149,151,153,155,157,159, 161,163,165,167,169,171,173,175 and ET/SFU Visit) is reported in this outcome measure. Baseline was defined as the last non-missing value prior to the first administration of study drug in Part A. ET/SFU visit was 9 weeks after administration of last dose (i.e., up to Week 184). Haptoglobin values \<0.2 were imputed as 0.2.
Time frame: Baseline (Week 0), every 2 weeks starting from Week 27 till Week 175 and at ET/SFU visit (i.e., up to Week 184)
Part B: Number of Healthcare Visits by Type
In this outcome measure, number of healthcare visits which included non-study healthcare resource utilization visit (consisted mainly of extra visits to the office of the study doctor, visit to a generalist doctor or visit to a specialist doctor), hospitalization visit and visit to hospital emergency is reported.
Time frame: From Week 27 up to 149 weeks of treatment (i.e., up to Week 176)
The study was conducted at 27 sites in 13 countries. Out of 66 screened participants, a total of 42 participants were enrolled and randomized from 17 March 2018 to 30 March 2020. This study consisted of 2 Parts: Part A and Part B.
| Milestone | BIVV009/BIVV009 | Placebo/BIVV009 |
|---|---|---|
| Started | 22 | 20 |
| Completed | 19 | 20 |
| Not completed | 3 | 0 |
| Withdrew: Adverse event | 3 | 0 |
| Milestone | BIVV009/BIVV009 | Placebo/BIVV009 |
|---|---|---|
| Started | 19 | 20 |
| Completed | 16 | 16 |
| Not completed | 3 | 4 |
| Withdrew: Lack of efficacy | 1 | 2 |
| Withdrew: Adverse event | 0 | 1 |
| Withdrew: Withdrawal by subject | 1 | 1 |
| Withdrew: Other | 1 | 0 |
A participant was considered a responder: if he or she did not receive blood transfusion from Week 5 through Week 26 (end of treatment) and did not receive treatment for CAD beyond what was permitted per protocol. Additionally, participant's hemoglobin (Hgb) level must have increased to \>=1.5 grams per deciliter (g/dL) from baseline (defined as last Hgb value before administration of first dose of study drug) at treatment assessment timepoint (defined as average of values from the Week 23, 25, and 26 visits). Percentage of responders was calculated together with 95% exact Clopper-Pearson confidence interval (CI).
| percentage of participants | BIVV009 | Placebo |
|---|---|---|
| Part A: Percentage of Participants With Response to Treatment | 72.7 (49.8 to 89.3) | 15.0 (3.2 to 37.9) |
Adverse Event (AE): any untoward medical occurrence in a participant who received study drug and did not necessarily have to have a causal relationship with the treatment. Treatment emergent serious adverse events (TESAEs) was defined as any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was congenital anomaly/birth defect, was medically important event. Treatment emergent adverse events (TEAEs): AEs that developed, worsened or became serious during the treatment-emergent (TE) period (from first investigational medicinal product \[IMP\] administration in Part B to last IMP administration + 9 weeks follow-up period).
| Participants | Part B: BIVV009 6.5 g | Part B: BIVV009 7.5 g |
|---|---|---|
| TEAEs | 29 | 7 |
| TESAEs | 6 | 1 |
Mean change from baseline (Week 0) in Hemoglobin (Hgb) at the treatment assessment timepoint is reported in this outcome measure. Treatment assessment timepoint was defined as the average of the values from the Week 23, 25, and 26 visits. Least squares (LS) mean and 95 % confidence interval (CI) was assessed by Mixed Model for Repeated Measures (MMRM) approach using heterogeneous Toeplitz (TOEPH) covariance matrix with change from baseline as the dependent variable and baseline value and visits as independent variables. Baseline was defined as the last non-missing value prior to the first administration of study drug.
| grams per deciliter | BIVV009 | Placebo |
|---|---|---|
| Part A: Mean Change From Baseline in Hemoglobin (Hgb) Level at the Treatment Assessment Timepoint | 2.66 (2.09 to 3.22) | 0.09 (-0.50 to 0.68) |
FACIT-Fatigue scale consists of 13 questions assessed using a 5-point scale (0=not at all; 1 = a little bit, 2 = somewhat, 3 = quite a bit and 4 = very much). Responses to each question were added to obtain a total score. Total score ranged from 0 to 52, with higher score indicating more fatigue. Treatment assessment timepoint was defined as the average of the values from the Week 23, 25, and 26 visits. LS mean and 95% CI was assessed by MMRM approach using TOEPH covariance matrix with change from baseline (Week 0) as the dependent variable and baseline value and visits as independent variables. Baseline was defined as the last non-missing value prior to the first administration of study drug.
| score on a scale | BIVV009 | Placebo |
|---|---|---|
| Part A: Mean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score at the Treatment Assessment Timepoint | 10.83 (7.45 to 14.22) | 1.91 (-1.65 to 5.46) |
Mean change from baseline (Week 0) in total bilirubin at the treatment assessment timepoint is reported in this outcome measure. Treatment assessment timepoint was defined as the average of the values from the Week 23, 25, and 26 visits. Baseline was defined as the last non-missing value prior to the first administration of study drug.
| micromoles per liter | BIVV009 | Placebo |
|---|---|---|
| Part A: Mean Change From Baseline in Total Bilirubin Levels at the Treatment Assessment Timepoint | -22.881 ± 10.401 | -1.388 ± 13.901 |
Mean change from baseline (Week 0) in LDH at the treatment assessment timepoint is reported in this outcome measure. Treatment assessment timepoint was defined as the average of the values from the Week 23, 25, and 26 visits. Baseline was defined as the last non-missing value prior to the first administration of study drug.
| units per liter | BIVV009 | Placebo |
|---|---|---|
| Part A: Mean Change From Baseline in Lactate Dehydrogenase (LDH) at the Treatment Assessment Timepoint | -150.833 ± 160.824 | 7.600 ± 212.690 |
Symptomatic anemia was defined as having following symptoms: i. Fatigue; ii. Weakness; iii. Shortness of breath; iv. Palpitations, fast heartbeat; v. Light headedness and/or vi. Chest pain. Percentage of participants with solicited symptomatic anemia symptoms was reported in this outcome measure.
| percentage of participants | BIVV009 | Placebo |
|---|---|---|
| Fatigue | 31.6 | 68.4 |
| Weakness | 5.3 | 31.6 |
| Shortness of breath | 5.3 | 36.8 |
| Palpitations | 0 | 15.8 |
| Light headedness | 5.3 | 15.8 |
| Chest pain | 0 | 5.3 |
Change from baseline (Week 0) in Hgb levels at each specified time points (i.e., Week 27, 29, 31, 33, 35, 37, 39, 41, 43,45, 47, 49, 51, 53, 55, 57, 59, 61, 63, 65, 67, 69, 71, 73, 75, 77, 79, 81, 83,85,87, 89,91, 93, 95, 97, 99,101,103, 105, 107,109, 111,113,115,117,119, 121,123, 125, 127,129,131,133,135,137,139,141,143,145,147,149,151,153, 155,157,159,161,163,165,167,169,171,173,175 and ET/SFU Visit) is reported in this outcome measure. Baseline was defined as the last non-missing value prior to the first administration of study drug in Part A. Early Termination (ET) visit/safety follow up (SFU) visit was 9 weeks after administration of last dose (i.e., up to Week 184). Here, "0" in the number analyzed field signifies that none of participants were available for assessment at the specified timepoints.
| grams per deciliter | BIVV009/BIVV009 | Placebo/BIVV009 |
|---|---|---|
| Week 27 | 2.647 ± 1.348 | 1.125 ± 1.545 |
| Week 29 | 2.507 ± 1.669 | 1.947 ± 1.467 |
| Week 31 | 2.490 ± 1.550 | 2.358 ± 1.471 |
| Week 33 | 2.519 ± 1.520 | 1.987 ± 2.054 |
| Week 35 | 2.611 ± 1.522 | 1.977 ± 2.010 |
| Week 37 | 2.197 ± 1.461 | 2.469 ± 1.550 |
| Week 39 | 2.528 ± 1.607 | 2.368 ± 1.835 |
| Week 41 | 2.162 ± 1.636 | 2.074 ± 1.825 |
| Week 43 | 2.436 ± 1.103 | 2.182 ± 1.643 |
| Week 45 | 2.644 ± 1.688 | 2.249 ± 1.858 |
| Week 47 | 2.335 ± 1.108 | 2.315 ± 2.129 |
| Week 49 | 2.531 ± 1.280 | 2.121 ± 1.883 |
| Week 51 | 2.349 ± 1.260 | 2.394 ± 1.870 |
| Week 53 | 2.346 ± 1.182 | 2.411 ± 1.786 |
| Week 55 | 2.438 ± 1.396 | 2.539 ± 1.403 |
| Week 57 | 2.531 ± 1.446 | 2.250 ± 1.774 |
| Week 59 | 2.590 ± 1.397 | 2.008 ± 2.615 |
| Week 61 | 4.073 ± 6.761 | 2.059 ± 1.911 |
| Week 63 | 2.382 ± 1.546 | 2.102 ± 1.780 |
| Week 65 | 2.925 ± 1.447 | 2.311 ± 1.986 |
| Week 67 | 4.170 ± 6.509 | 2.534 ± 2.036 |
| Week 69 | 2.777 ± 1.411 | 2.505 ± 2.093 |
| Week 71 | 2.654 ± 1.495 | 2.357 ± 1.758 |
| Week 73 | 2.800 ± 2.132 | 2.197 ± 1.989 |
| Week 75 | 2.391 ± 1.700 | 2.175 ± 2.151 |
| Week 77 | 3.074 ± 2.088 | 2.451 ± 1.878 |
| Week 79 | 2.614 ± 1.874 | 2.419 ± 2.247 |
| Week 81 | 2.598 ± 1.801 | 2.458 ± 1.579 |
| Week 83 | 2.469 ± 1.777 | 2.762 ± 1.539 |
| Week 85 | 2.755 ± 1.942 | 2.489 ± 1.375 |
| Week 87 | 2.477 ± 1.779 | 2.765 ± 1.605 |
| Week 89 | 2.551 ± 1.835 | 2.482 ± 2.004 |
| Week 91 | 2.380 ± 2.084 | 2.556 ± 1.538 |
| Week 93 | 2.931 ± 2.386 | 2.655 ± 2.033 |
| Week 95 | 2.262 ± 2.014 | 2.578 ± 1.970 |
| Week 97 | 2.413 ± 2.262 | 2.551 ± 1.544 |
| Week 99 | 2.019 ± 1.799 | 2.412 ± 1.822 |
| Week 101 | 2.276 ± 1.986 | 2.171 ± 1.852 |
| Week 103 | 2.378 ± 2.342 | 2.527 ± 1.807 |
| Week 105 | 2.291 ± 1.719 | 2.321 ± 1.805 |
| Week 107 | 2.319 ± 1.511 | 2.560 ± 1.858 |
| Week 109 | 2.509 ± 1.772 | 2.645 ± 1.608 |
| Week 111 | 2.416 ± 1.908 | 2.750 ± 1.707 |
| Week 113 | 2.860 ± 1.832 | 2.268 ± 1.497 |
| Week 115 | 2.622 ± 1.986 | 2.326 ± 1.809 |
| Week 117 | 3.135 ± 1.598 | 2.474 ± 1.808 |
| Week 119 | 3.106 ± 1.493 | 2.608 ± 2.045 |
| Week 121 | 3.388 ± 1.616 | 2.325 ± 2.223 |
| Week 123 | 3.255 ± 1.346 | 2.878 ± 2.667 |
| Week 125 | 3.197 ± 1.366 | 1.700 ± 2.684 |
| Week 127 | 3.371 ± 1.551 | 2.102 ± 3.211 |
| Week 129 | 3.171 ± 1.566 | 2.129 ± 2.739 |
| Week 131 | 3.453 ± 1.823 | 1.423 ± 3.184 |
| Week 133 | 3.520 ± 1.707 | 2.025 ± 1.167 |
| Week 135 | 3.611 ± 1.458 | 3.048 ± 1.620 |
| Week 137 | 4.074 ± 2.392 | 2.400 ± 1.808 |
| Week 139 | 3.929 ± 1.889 | 3.334 ± 2.669 |
| Week 141 | 3.500 ± 0.656 | 2.733 ± 1.674 |
| Week 143 | 3.220 ± 1.064 | 2.985 ± 1.904 |
| Week 145 | 3.350 ± 0.636 | 3.467 ± 2.108 |
| Week 147 | 3.202 ± 0.591 | 2.300 ± 0.566 |
| Week 149 | 2.900 | 2.550 ± 0.778 |
| Week 151 | 2.961 ± 0.370 | 3.000 |
| Week 153 | 2.800 | 2.800 |
| Week 155 | 2.700 | 3.600 |
| Week 157 | 2.700 | 3.000 |
| Week 159 | 3.300 | 2.300 |
| Week 161 | 4.100 | 2.400 |
| Week 163 | 4.100 | 2.600 |
| Week 165 | 3.800 | — |
| Week 167 | 4.700 | — |
| Week 169 | 4.300 | — |
| Week 171 | 4.200 | — |
| Week 173 | 4.000 | — |
| Week 175 | 2.500 | — |
| ET/SFU Visit | 0.149 ± 2.073 | 0.359 ± 1.872 |
Change from baseline (Week 0) in total bilirubin levels at each specified time points (i.e., Week 27, 29, 31, 33, 35, 37, 39, 41, 43, 45, 47, 49, 51, 53, 55, 57, 59, 61, 63, 65,67,69,71,73,75, 77, 79, 81,83, 85, 87, 89, 91, 93, 95, 97, 99,101,103, 105, 107,109, 111,113,115,117,119, 121, 123, 125, 127,129,131,133,135,137,139,141,143, 145,147,149,151,153,155,157,159, 161,163,165,167,169,171,173,175 and ET/SFU Visit) is reported in this outcome measure. Baseline was defined as the last non-missing value prior to the first administration of study drug in Part A. ET visit/SFU visit was 9 weeks after administration of last dose (i.e., up to Week 184). Here, "0" in the number analyzed field signifies that none of participants were available for assessment at the specified timepoints.
| micromoles per liter | BIVV009/BIVV009 | Placebo/BIVV009 |
|---|---|---|
| Week 27 | -22.965 ± 9.899 | -18.761 ± 13.353 |
| Week 29 | -20.119 ± 12.170 | -19.889 ± 14.500 |
| Week 31 | -21.675 ± 11.025 | -22.153 ± 14.962 |
| Week 33 | -19.860 ± 11.527 | -24.263 ± 15.169 |
| Week 35 | -20.540 ± 10.557 | -21.888 ± 13.930 |
| Week 37 | -21.350 ± 13.234 | -22.527 ± 16.315 |
| Week 39 | -23.493 ± 11.425 | -21.173 ± 15.901 |
| Week 41 | -18.694 ± 12.189 | -21.138 ± 16.150 |
| Week 43 | -20.593 ± 11.449 | -21.544 ± 16.603 |
| Week 45 | -18.556 ± 11.213 | -20.156 ± 16.757 |
| Week 47 | -20.400 ± 9.803 | -20.250 ± 15.536 |
| Week 49 | -21.450 ± 10.271 | -21.024 ± 13.630 |
| Week 51 | -19.686 ± 11.072 | -19.444 ± 15.942 |
| Week 53 | -19.107 ± 11.267 | -20.335 ± 17.126 |
| Week 55 | -19.354 ± 12.209 | -23.481 ± 12.300 |
| Week 57 | -21.708 ± 10.467 | -18.953 ± 15.825 |
| Week 59 | -19.979 ± 9.706 | -19.820 ± 16.209 |
| Week 61 | -18.254 ± 10.541 | -21.073 ± 17.457 |
| Week 63 | -21.587 ± 9.680 | -21.753 ± 16.663 |
| Week 65 | -20.400 ± 9.866 | -20.950 ± 19.833 |
| Week 67 | -22.680 ± 10.317 | -20.150 ± 20.250 |
| Week 69 | -21.279 ± 9.423 | -17.827 ± 22.194 |
| Week 71 | -19.008 ± 8.255 | -20.585 ± 21.125 |
| Week 73 | -23.058 ± 7.766 | -20.080 ± 17.803 |
| Week 75 | -21.492 ± 7.453 | -20.771 ± 20.261 |
| Week 77 | -23.267 ± 8.972 | -19.814 ± 19.337 |
| Week 79 | -21.125 ± 12.701 | -21.027 ± 19.830 |
| Week 81 | -19.255 ± 9.478 | -22.331 ± 20.117 |
| Week 83 | -16.567 ± 12.565 | -25.091 ± 13.838 |
| Week 85 | -18.045 ± 12.183 | -23.292 ± 12.976 |
| Week 87 | -19.990 ± 12.043 | -21.473 ± 14.444 |
| Week 89 | -17.790 ± 11.036 | -24.782 ± 13.390 |
| Week 91 | -16.944 ± 13.476 | -23.091 ± 14.949 |
| Week 93 | -18.300 ± 11.132 | -24.970 ± 13.852 |
| Week 95 | -18.613 ± 12.265 | -23.564 ± 11.122 |
| Week 97 | -18.656 ± 11.462 | -26.122 ± 11.870 |
| Week 99 | -17.389 ± 10.932 | -23.700 ± 12.919 |
| Week 101 | -18.411 ± 10.175 | -25.940 ± 12.309 |
| Week 103 | -18.178 ± 11.156 | -22.655 ± 11.308 |
| Week 105 | -19.422 ± 8.444 | -26.920 ± 10.996 |
| Week 107 | -18.100 ± 9.955 | -23.018 ± 12.755 |
| Week 109 | -18.456 ± 10.606 | -25.422 ± 14.100 |
| Week 111 | -19.767 ± 9.992 | -24.380 ± 12.298 |
| Week 113 | -19.389 ± 9.192 | -23.940 ± 15.514 |
| Week 115 | -19.622 ± 8.194 | -21.533 ± 14.215 |
| Week 117 | -22.088 ± 10.247 | -22.225 ± 15.256 |
| Week 119 | -20.038 ± 8.529 | -22.713 ± 14.859 |
| Week 121 | -22.063 ± 7.215 | -21.183 ± 10.750 |
| Week 123 | -21.438 ± 8.058 | -22.029 ± 12.421 |
| Week 125 | -20.657 ± 8.889 | -19.560 ± 12.997 |
| Week 127 | -20.550 ± 9.782 | -21.433 ± 15.850 |
| Week 129 | -22.883 ± 11.291 | -19.020 ± 11.749 |
| Week 131 | -19.420 ± 12.936 | -23.100 ± 15.797 |
| Week 133 | -27.600 ± 7.477 | -17.450 ± 16.917 |
| Week 135 | -29.500 ± 7.816 | -21.900 ± 19.552 |
| Week 137 | -28.833 ± 8.615 | -11.650 ± 18.173 |
| Week 139 | -25.800 ± 9.081 | -24.733 ± 17.470 |
| Week 141 | -34.400 ± 2.404 | -13.833 ± 16.669 |
| Week 143 | -26.967 ± 9.235 | -20.033 ± 22.861 |
| Week 145 | -32.900 ± 3.677 | -18.100 ± 27.217 |
| Week 147 | -29.300 ± 7.418 | -3.300 ± 34.083 |
| Week 149 | -31.700 | -7.300 ± 28.284 |
| Week 151 | -21.500 ± 0.566 | 0.300 |
| Week 153 | -30.800 | -11.400 |
| Week 155 | -35.300 | 0.700 |
| Week 157 | -32.400 | -0.100 |
| Week 159 | -32.500 | 4.700 |
| Week 161 | -36.800 | 5.500 |
| Week 163 | -37.100 | 13.000 |
| Week 165 | -36.300 | — |
| Week 167 | -34.300 | — |
| Week 169 | -32.700 | — |
| Week 171 | -33.100 | — |
| Week 173 | -32.100 | — |
| Week 175 | -28.400 | — |
| ET/SFU Visit | 1.976 ± 9.444 | 1.165 ± 18.773 |
FACIT-Fatigue scale consists of 13 questions assessed using a 5-point scale (0=not at all; 1 = a little bit, 2 = somewhat, 3 = quite a bit and 4 = very much). Responses to each question were added to obtain a total score. The Total score ranged from 0 to 52, with higher score indicating more fatigue. Baseline (Week 0) was defined as the last non-missing value prior to the first administration of study drug in Part A. ET visit/SFU visit was 9 weeks after administration of last dose (i.e., up to Week 184).
| score on a scale | BIVV009/BIVV009 | Placebo/BIVV009 |
|---|---|---|
| Week 39 | 11.048 ± 12.333 | 7.958 ± 12.003 |
| Week 51 | 10.080 ± 11.506 | 6.354 ± 9.680 |
| Week 63 | 10.672 ± 13.225 | 8.499 ± 11.146 |
| Week 75 | 9.861 ± 12.953 | 10.304 ± 9.722 |
| Week 87 | 11.015 ± 13.972 | 8.483 ± 11.323 |
| Week 99 | 12.109 ± 14.786 | 8.788 ± 10.985 |
| Week 111 | 11.563 ± 13.411 | 10.688 ± 11.768 |
| Week 123 | 10.806 ± 13.736 | 6.583 ± 16.613 |
| Week 135 | 16.683 ± 18.953 | 12.650 ± 15.243 |
| Week 147 | 9.208 ± 1.120 | 0.500 ± 2.121 |
| Week 159 | 20.000 | 10.000 |
| Week 171 | 15.000 | — |
| ET/SFU Visit | -1.257 ± 10.399 | -1.551 ± 12.840 |
SF-12: 12 item-questionnaire assessed health-related quality of life (HRQOL), contained 12 items, categorized into 8 domains (subscales) of functioning and well-being: physical functioning, role-physical, role emotional, mental health, bodily pain, general health, vitality and social functioning, with each domain score ranged from 0 (poor health) to 100 (better health). Higher scores = good health condition. These 8 domains were further summarized into 2 summary scores, PCS and MCS that ranged from 0 (poor health) to 100 (better health). Higher scores = better HRQOL. Baseline (Week 0): last non-missing value prior to first administration of study drug in Part A. ET visit/SFU visit: 9 weeks after administration of last dose (i.e., up to Week 184).
| score on a scale | BIVV009/BIVV009 | Placebo/BIVV009 |
|---|---|---|
| Week 39-PCS | 4.814 ± 8.830 | 6.745 ± 11.255 |
| Week 39-MCS | 8.543 ± 9.155 | 1.151 ± 11.591 |
| Week 51-PCS | 5.320 ± 6.913 | 5.654 ± 8.527 |
| Week 51-MCS | 8.461 ± 6.754 | 1.704 ± 8.813 |
| Week 63-PCS | 5.840 ± 6.916 | 6.216 ± 9.514 |
| Week 63-MCS | 8.117 ± 9.955 | 1.722 ± 10.175 |
| Week 75-PCS | 5.723 ± 7.563 | 7.661 ± 10.418 |
| Week 75-MCS | 7.932 ± 9.279 | 3.519 ± 7.891 |
| Week 87-PCS | 5.226 ± 8.196 | 8.778 ± 12.405 |
| Week 87-MCS | 6.058 ± 10.981 | 2.219 ± 9.111 |
| Week 99-PCS | 3.945 ± 7.463 | 8.262 ± 12.534 |
| Week 99-MCS | 7.136 ± 11.850 | 4.682 ± 7.698 |
| Week 111-PCS | 4.800 ± 7.985 | 8.080 ± 12.302 |
| Week 111-MCS | 8.705 ± 7.777 | 4.411 ± 10.249 |
| Week 123-PCS | 4.147 ± 7.110 | 7.973 ± 13.200 |
| Week 123-MCS | 8.650 ± 8.323 | -2.538 ± 13.136 |
| Week 135-PCS | 6.660 ± 9.096 | 6.070 ± 13.819 |
| Week 135-MCS | 8.860 ± 9.817 | 2.790 ± 6.170 |
| Week 147-PCS | -1.655 ± 3.769 | -3.740 ± 1.824 |
| Week 147-MCS | 11.220 ± 6.095 | -1.110 ± 0.679 |
| Week 159-PCS | 5.620 | -10.410 |
| Week 159-MCS | 16.400 | 9.590 |
| Week 171-PCS | 11.980 | — |
| Week 171-MCS | 0.250 | — |
| ET/SFU Visit-PCS | -3.478 ± 10.875 | -0.429 ± 10.922 |
| ET/SFU Visit-MCS | 1.835 ± 11.882 | -2.137 ± 10.148 |
EQ-5D-5L included 2 components: health state utility index (descriptive system) and Visual Analog Scale (VAS). EQ-5D descriptive system comprises 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 5 response option: no problem, slight problem, moderate problem, severe problem and extreme problems measured with Likert scale. EQ-5D-5L responses converted into single index utility score between 0 to 1. Higher score=better health. EQ-5D-5L VAS rated participant's current health state on scale from 0 (worst imaginable health) to 100 (best imaginable health). Baseline (Week 0): last non-missing value prior to first administration of study drug in Part A. ET visit/SFU visit: 9 weeks after administration of last dose (i.e., up to Week 184).
| score on a scale | BIVV009/BIVV009 | Placebo/BIVV009 |
|---|---|---|
| Week 39-Index score | 0.020 ± 0.165 | 0.054 ± 0.135 |
| Week 51-Index score | 0.075 ± 0.173 | -0.008 ± 0.197 |
| Week 63-Index score | 0.060 ± 0.214 | 0.015 ± 0.157 |
| Week 75-Index score | -0.004 ± 0.144 | 0.063 ± 0.152 |
| Week 87-Index score | -0.034 ± 0.240 | 0.059 ± 0.158 |
| Week 99-Index score | -0.058 ± 0.216 | 0.058 ± 0.146 |
| Week 111-Index score | -0.033 ± 0.205 | 0.050 ± 0.194 |
| Week 123-Index score | -0.020 ± 0.235 | 0.031 ± 0.221 |
| Week 135-Index score | 0.008 ± 0.351 | 0.094 ± 0.245 |
| Week 147-Index score | 0.054 ± 0.037 | -0.087 ± 0.222 |
| Week 159-Index score | 0.174 | 0.087 |
| Week 171-Index score | 0.053 | — |
| ET/SFU Visit-Index score | -0.108 ± 0.238 | -0.077 ± 0.169 |
| Week 39-VAS | 20.647 ± 16.871 | 12.000 ± 17.146 |
| Week 51-VAS | 14.800 ± 27.589 | 9.125 ± 21.112 |
| Week 63-VAS | 19.867 ± 21.603 | 14.375 ± 15.573 |
| Week 75-VAS | 16.846 ± 22.120 | 18.933 ± 20.243 |
| Week 87-VAS | 14.077 ± 25.221 | 16.867 ± 17.912 |
| Week 99-VAS | 19.364 ± 20.796 | 18.385 ± 20.706 |
| Week 111-VAS | 18.273 ± 19.463 | 22.833 ± 20.621 |
| Week 123-VAS | 21.667 ± 18.371 | 18.000 ± 30.389 |
| Week 135-VAS | 26.000 ± 20.433 | 23.400 ± 27.574 |
| Week 147-VAS | 17.500 ± 17.678 | 3.500 ± 26.163 |
| Week 159-VAS | 40.000 | 27.000 |
| Week 171-VAS | 35.000 | — |
| ET/SFU Visit-VAS | 1.526 ± 17.976 | -2.056 ± 14.957 |
The PGIS is a self-reported scale. The PGIS is a 1-item questionnaire designed to assess participant's impression of disease severity using a 5-point scale ranging from 1 to 5, where 1=none, 2=mild, 3=moderate, 4=severe, 5=very severe. Higher scores indicated greater severity. ET visit/SFU visit was 9 weeks after administration of last dose (i.e., up to Week 184).
| Participants | BIVV009/BIVV009 | Placebo/BIVV009 |
|---|---|---|
| Week 39 - None | 7 | 5 |
| Week 39 - Mild | 8 | 7 |
| Week 39 - Moderate | 1 | 5 |
| Week 39 - Severe | 1 | 0 |
| Week 39 - Very Severe | 0 | 0 |
| Week 51 - None | 5 | 4 |
| Week 51 - Mild | 8 | 7 |
| Week 51 - Moderate | 2 | 5 |
| Week 51 - Severe | 0 | 0 |
| Week 51 - Very Severe | 0 | 0 |
| Week 63 - None | 6 | 4 |
| Week 63 - Mild | 8 | 7 |
| Week 63 - Moderate | 3 | 5 |
| Week 63 - Severe | 0 | 0 |
| Week 63 - Very Severe | 0 | 0 |
| Week 75 - None | 5 | 6 |
| Week 75 - Mild | 6 | 6 |
| Week 75 - Moderate | 2 | 3 |
| Week 75 - Severe | 0 | 0 |
| Week 75 - Very Severe | 0 | 0 |
| Week 87 - None | 6 | 7 |
| Week 87 - Mild | 5 | 4 |
| Week 87 - Moderate | 1 | 3 |
| Week 87 - Severe | 1 | 1 |
| Week 87 - Very Severe | 0 | 0 |
| Week 99 - None | 5 | 8 |
| Week 99 - Mild | 5 | 3 |
| Week 99 - Moderate | 1 | 2 |
| Week 99 - Severe | 0 | 0 |
| Week 99 - Very Severe | 0 | 0 |
| Week 111 - None | 5 | 7 |
| Week 111 - Mild | 4 | 3 |
| Week 111 - Moderate | 2 | 2 |
| Week 111 - Severe | 0 | 0 |
| Week 111 - Very Severe | 0 | 0 |
| Week 123 - None | 2 | 5 |
| Week 123 - Mild | 6 | 2 |
| Week 123 - Moderate | 1 | 2 |
| Week 123 - Severe | 0 | 0 |
| Week 123 - Very Severe | 0 | 0 |
| Week 135 - None | 0 | 2 |
| Week 135 - Mild | 3 | 1 |
| Week 135 - Moderate | 2 | 2 |
| Week 135 - Severe | 0 | 0 |
| Week 135 - Very Severe | 0 | 0 |
| Week 147 - None | 0 | 1 |
| Week 147 - Mild | 1 | 0 |
| Week 147 - Moderate | 1 | 1 |
| Week 147 - Severe | 0 | 0 |
| Week 147 - Very Severe | 0 | 0 |
| Week 159 - None | 0 | 0 |
| Week 159 - Mild | 0 | 0 |
| Week 159 - Moderate | 1 | 1 |
| Week 159 - Severe | 0 | 0 |
| Week 159 - Very Severe | 0 | 0 |
| Week 171 - None | 0 | 0 |
| Week 171 - Mild | 1 | 0 |
| Week 171 - Moderate | 0 | 0 |
| Week 171 - Severe | 0 | 0 |
| Week 171 - Very Severe | 0 | 0 |
| ET/SFU - None | 2 | 4 |
| ET/SFU - Mild | 5 | 3 |
| ET/SFU - Moderate | 7 | 5 |
| ET/SFU - Severe | 3 | 5 |
| ET/SFU - Very Severe | 2 | 1 |
PGIC is a self-administered questionnaire to evaluate the improvement or worsening compared to the start of the study. PGIC was assessed on a 7-point Likert scale ranged from 1 (greatly improved) to 7 (greatly worsened). Categories were defined based on the PGIC scores as follows: 1=very much improved, 2=much improved, 3=minimally improved, 4=no change, 5=minimally worse, 6=much worse and 7=very much worsen. Higher scores indicated greater severity. ET visit/SFU visit was 9 weeks after administration of last dose (i.e., up to Week 184).
| Participants | BIVV009/BIVV009 | Placebo/BIVV009 |
|---|---|---|
| Week 39 - Very much improved | 4 | 3 |
| Week 39 - Much improved | 8 | 6 |
| Week 39 - Minimally improved | 2 | 6 |
| Week 39 - No Change | 2 | 1 |
| Week 39 - Minimally worse | 1 | 0 |
| Week 39 - Much worse | 0 | 0 |
| Week 39 - Very much worse | 0 | 0 |
| Week 51 - Very much improved | 5 | 3 |
| Week 51 - Much improved | 3 | 6 |
| Week 51 - Minimally improved | 2 | 4 |
| Week 51 - No Change | 5 | 2 |
| Week 51 - Minimally worse | 0 | 0 |
| Week 51 - Much worse | 0 | 1 |
| Week 51 - Very much worse | 0 | 0 |
| Week 63 - Very much improved | 5 | 3 |
| Week 63 - Much improved | 8 | 4 |
| Week 63 - Minimally improved | 0 | 5 |
| Week 63 - No change | 4 | 3 |
| Week 63 - Minimally worse | 0 | 0 |
| Week 63 - Much worse | 0 | 1 |
| Week 63 - Very much worse | 0 | 0 |
| Week 75 - Very much improved | 6 | 6 |
| Week 75 - Much improved | 2 | 3 |
| Week 75 - Minimally improved | 1 | 5 |
| Week 75 - No Change | 4 | 1 |
| Week 75 - Minimally worse | 0 | 0 |
| Week 75 - Much worse | 0 | 0 |
| Week 75 - Very much worse | 0 | 0 |
| Week 87 - Very much improved | 7 | 7 |
| Week 87 - Much improved | 0 | 3 |
| Week 87 - Minimally improved | 2 | 1 |
| Week 87 - No Change | 2 | 3 |
| Week 87 - Minimally worse | 1 | 1 |
| Week 87 - Much worse | 1 | 0 |
| Week 87 - Very much worse | 0 | 0 |
| Week 99 - Very much improved | 3 | 8 |
| Week 99 - Much improved | 3 | 2 |
| Week 99 - Minimally improved | 1 | 3 |
| Week 99 - No change | 3 | 0 |
| Week 99 - Minimally worse | 1 | 0 |
| Week 99 - Much worse | 0 | 0 |
| Week 99 - Very much worse | 0 | 0 |
| Week 111 - Very much improved | 3 | 6 |
| Week 111 - Much improved | 5 | 4 |
| Week 111 - Minimally improved | 1 | 1 |
| Week 111 - No Change | 2 | 1 |
| Week 111 - Minimally worse | 0 | 0 |
| Week 111 - Much worse | 0 | 0 |
| Week 111 - Very much worse | 0 | 0 |
| Week 123 - Very much improved | 3 | 4 |
| Week 123 - Much improved | 3 | 3 |
| Week 123 - Minimally improved | 2 | 1 |
| Week 123 - No Change | 1 | 1 |
| Week 123 - Minimally worse | 0 | 0 |
| Week 123 - Much worse | 0 | 0 |
| Week 123 - Very much worse | 0 | 0 |
| Week 135 - Very much improved | 2 | 2 |
| Week 135 - Much improved | 2 | 1 |
| Week 135 - Minimally improved | 0 | 1 |
| Week 135 - No Change | 0 | 1 |
| Week 135 - Minimally worse | 1 | 0 |
| Week 135 - Much worse | 0 | 0 |
| Week 135 - Very much worse | 0 | 0 |
| Week 147 - Very much improved | 1 | 0 |
| Week 147 - Much improved | 0 | 1 |
| Week 147 - Minimally improved | 0 | 1 |
| Week 147 - No Change | 1 | 0 |
| Week 147 - Minimally worse | 0 | 0 |
| Week 147 - Much worse | 0 | 0 |
| Week 147 - Very much worse | 0 | 0 |
| Week 159 - Very much improved | 1 | 0 |
| Week 159 - Much improved | 0 | 0 |
| Week 159 - Minimally improved | 0 | 1 |
| Week 159 - No Change | 0 | 0 |
| Week 159 - Minimally worse | 0 | 0 |
| Week 159 - Much worse | 0 | 0 |
| Week 159 - Very much worse | 0 | 0 |
| Week 171- Very much improved | 1 | 0 |
| Week 171- Much improved | 0 | 0 |
| Week 171- Minimally improved | 0 | 0 |
| Week 171- No Change | 0 | 0 |
| Week 171- Minimally worse | 0 | 0 |
| Week 171- Much worse | 0 | 0 |
| Week 171- Very much worse | 0 | 0 |
| ET/SFU Visit- Very much improved | 3 | 3 |
| ET/SFU Visit- Much improved | 5 | 6 |
| ET/SFU Visit- Minimally improved | 4 | 1 |
| ET/SFU Visit- No Change | 3 | 3 |
| ET/SFU Visit- Minimally worse | 1 | 2 |
| ET/SFU Visit- Much worse | 3 | 3 |
| ET/SFU Visit- Very much worse | 0 | 0 |
Change from baseline (Week 0) in LDH levels at each specified time points (i.e., Week 27, 29, 31, 33, 35, 37, 39, 41, 43, 45, 47, 49, 51, 53, 55, 57, 59, 61, 63, 65, 67, 69, 71, 73, 75, 77, 79, 81, 83, 85, 87, 89, 91, 93, 95, 97, 99, 101, 103, 105, 107,109, 111, 113, 115, 117, 119, 121,123, 125, 127,129,131,133,135,137,139,141,143,145,147, 149, 151,153,155,157,159,161,163,165,167,169, 171, 173, 175 and ET/SFU Visit) is reported in this outcome measure. Baseline was defined as the last non-missing value prior to the first administration of study drug in Part A. ET/SFU visit was 9 weeks after administration of last dose (i.e., up to Week 184).
| units per liter | BIVV009/BIVV009 | Placebo/BIVV009 |
|---|---|---|
| Week 27 | -151.000 ± 184.640 | 0.650 ± 208.051 |
| Week 29 | -115.944 ± 226.913 | 11.000 ± 222.383 |
| Week 31 | -85.111 ± 247.506 | 38.882 ± 211.386 |
| Week 33 | -66.176 ± 324.145 | 4.813 ± 203.939 |
| Week 35 | -64.667 ± 261.431 | -19.313 ± 142.989 |
| Week 37 | 4.438 ± 398.843 | 25.765 ± 219.679 |
| Week 39 | -88.500 ± 271.941 | 27.313 ± 210.705 |
| Week 41 | 23.778 ± 342.195 | -6.000 ± 173.810 |
| Week 43 | -22.118 ± 310.381 | -21.706 ± 181.668 |
| Week 45 | 26.529 ± 329.717 | -13.500 ± 190.460 |
| Week 47 | -22.600 ± 302.511 | -24.944 ± 153.580 |
| Week 49 | -29.250 ± 289.392 | 15.722 ± 193.630 |
| Week 51 | -12.563 ± 290.489 | -9.556 ± 155.646 |
| Week 53 | -16.938 ± 299.788 | 12.529 ± 206.642 |
| Week 55 | -89.500 ± 219.634 | -11.250 ± 177.647 |
| Week 57 | -78.200 ± 285.140 | 64.188 ± 266.395 |
| Week 59 | -84.438 ± 220.376 | 31.647 ± 226.128 |
| Week 61 | -74.353 ± 215.414 | 20.588 ± 263.984 |
| Week 63 | -98.235 ± 185.259 | 38.059 ± 325.329 |
| Week 65 | -97.438 ± 212.335 | -16.467 ± 246.765 |
| Week 67 | -127.824 ± 192.148 | 29.667 ± 269.918 |
| Week 69 | -113.625 ± 197.270 | 36.563 ± 303.550 |
| Week 71 | -101.692 ± 320.348 | 4.067 ± 307.924 |
| Week 73 | -174.500 ± 177.153 | 7.063 ± 245.723 |
| Week 75 | -157.429 ± 184.525 | 7.067 ± 285.183 |
| Week 77 | -142.857 ± 199.169 | -24.867 ± 291.754 |
| Week 79 | -98.077 ± 271.632 | -20.063 ± 265.761 |
| Week 81 | -127.846 ± 233.663 | 7.200 ± 219.952 |
| Week 83 | -59.000 ± 346.917 | 8.818 ± 250.068 |
| Week 85 | -72.000 ± 350.021 | 28.400 ± 282.626 |
| Week 87 | -101.385 ± 285.590 | 83.385 ± 288.731 |
| Week 89 | -78.667 ± 351.059 | 10.833 ± 323.647 |
| Week 91 | -143.000 ± 273.540 | 0.846 ± 288.451 |
| Week 93 | -133.500 ± 254.332 | 10.818 ± 268.308 |
| Week 95 | -59.364 ± 368.708 | 18.231 ± 241.354 |
| Week 97 | -53.364 ± 343.820 | -38.500 ± 188.403 |
| Week 99 | -80.909 ± 312.405 | -12.917 ± 183.844 |
| Week 101 | -90.273 ± 293.680 | -17.182 ± 249.134 |
| Week 103 | -50.273 ± 331.072 | 5.692 ± 211.233 |
| Week 105 | -96.455 ± 276.907 | -34.250 ± 147.738 |
| Week 107 | -28.091 ± 354.244 | 20.769 ± 222.772 |
| Week 109 | -50.727 ± 335.078 | -2.000 ± 243.243 |
| Week 111 | -83.000 ± 303.258 | -5.583 ± 219.850 |
| Week 113 | -80.700 ± 319.283 | 0.667 ± 294.904 |
| Week 115 | -109.500 ± 256.395 | 44.700 ± 295.946 |
| Week 117 | -140.444 ± 280.944 | 12.700 ± 254.786 |
| Week 119 | -149.556 ± 237.771 | -26.500 ± 274.168 |
| Week 121 | -195.778 ± 225.978 | -42.375 ± 211.840 |
| Week 123 | -185.000 ± 231.489 | 22.222 ± 278.037 |
| Week 125 | -99.429 ± 285.513 | -85.143 ± 265.374 |
| Week 127 | -154.714 ± 231.922 | -28.875 ± 312.483 |
| Week 129 | -136.429 ± 234.954 | -18.571 ± 354.584 |
| Week 131 | -120.833 ± 300.454 | -2.000 ± 270.245 |
| Week 133 | -179.000 ± 298.811 | -47.500 ± 367.639 |
| Week 135 | -211.800 ± 265.438 | 58.000 ± 449.233 |
| Week 137 | -252.500 ± 264.591 | 46.500 ± 768.625 |
| Week 139 | -181.800 ± 284.966 | -48.667 ± 373.339 |
| Week 141 | -259.667 ± 362.136 | 30.667 ± 372.889 |
| Week 143 | -160.250 ± 357.021 | -7.500 ± 21.920 |
| Week 145 | -340.500 ± 458.912 | 290.000 ± 562.857 |
| Week 147 | -248.667 ± 355.136 | 311.000 ± 547.301 |
| Week 149 | -78.000 | 116.000 ± 175.362 |
| Week 151 | 58.000 ± 32.527 | 295.000 |
| Week 153 | 7.000 | 75.000 |
| Week 155 | -50.000 | — |
| Week 157 | -50.000 | 369.000 |
| Week 159 | -155.000 | 365.000 |
| Week 161 | -175.000 | — |
| Week 163 | -149.000 | 782.000 |
| Week 165 | -155.000 | — |
| Week 167 | -162.000 | — |
| Week 169 | -180.000 | — |
| Week 171 | -167.000 | — |
| Week 173 | -171.000 | — |
| Week 175 | -121.000 | — |
| ET/SFU Visit | -2.500 ± 195.967 | -11.706 ± 206.321 |
A participant was to receive a transfusion if his or her Hgb level met either of the following criteria: Hgb was \<9 g/dL and the participant had symptoms of anemia or Hgb was \<7 g/dL and the participant was asymptomatic.
| blood transfusions per participant | BIVV009/BIVV009 | Placebo/BIVV009 |
|---|---|---|
| Part B: Number of Blood Transfusions Per Participant | 0.4 ± 0.8 | 0.3 ± 0.4 |
Change from baseline (Week 0) in haptoglobin values at each specified time points (i.e., Week 27, 29, 31, 33, 35, 37, 39, 41, 43, 45, 47, 49, 51, 53, 55, 57, 59, 61, 63, 65, 67, 69, 71, 73, 75, 77, 79, 81, 83, 85, 87, 89, 91, 93, 95, 97,99,101,103, 105, 107,109, 111,113,115,117,119, 121,123, 125, 127, 129,131,133,135,137,139,141,143, 145,147,149,151,153,155,157,159, 161,163,165,167,169,171,173,175 and ET/SFU Visit) is reported in this outcome measure. Baseline was defined as the last non-missing value prior to the first administration of study drug in Part A. ET/SFU visit was 9 weeks after administration of last dose (i.e., up to Week 184). Haptoglobin values \<0.2 were imputed as 0.2.
| grams per liter | BIVV009/BIVV009 | Placebo/BIVV009 |
|---|---|---|
| Week 27 | 0.219 ± 0.380 | 0.082 ± 0.197 |
| Week 29 | 0.240 ± 0.317 | 0.182 ± 0.482 |
| Week 31 | 0.183 ± 0.311 | 0.256 ± 0.516 |
| Week 33 | 0.212 ± 0.423 | 0.229 ± 0.379 |
| Week 35 | 0.207 ± 0.355 | 0.151 ± 0.280 |
| Week 37 | 0.232 ± 0.419 | 0.187 ± 0.291 |
| Week 39 | 0.213 ± 0.330 | 0.165 ± 0.295 |
| Week 41 | 0.119 ± 0.238 | 0.082 ± 0.168 |
| Week 43 | 0.100 ± 0.224 | 0.161 ± 0.234 |
| Week 45 | 0.114 ± 0.205 | 0.189 ± 0.275 |
| Week 47 | 0.135 ± 0.243 | 0.255 ± 0.416 |
| Week 49 | 0.139 ± 0.205 | 0.172 ± 0.345 |
| Week 51 | 0.083 ± 0.197 | 0.207 ± 0.369 |
| Week 53 | 0.045 ± 0.137 | 0.122 ± 0.296 |
| Week 55 | 0.149 ± 0.251 | 0.179 ± 0.343 |
| Week 57 | 0.158 ± 0.234 | 0.158 ± 0.362 |
| Week 59 | 0.132 ± 0.229 | 0.131 ± 0.306 |
| Week 61 | 0.153 ± 0.286 | 0.184 ± 0.350 |
| Week 63 | 0.202 ± 0.265 | 0.177 ± 0.357 |
| Week 65 | 0.101 ± 0.232 | 0.198 ± 0.384 |
| Week 67 | 0.178 ± 0.241 | 0.162 ± 0.363 |
| Week 69 | 0.168 ± 0.255 | 0.169 ± 0.348 |
| Week 71 | 0.220 ± 0.288 | 0.236 ± 0.388 |
| Week 73 | 0.261 ± 0.335 | 0.174 ± 0.348 |
| Week 75 | 0.275 ± 0.321 | 0.197 ± 0.362 |
| Week 77 | 0.249 ± 0.374 | 0.232 ± 0.398 |
| Week 79 | 0.299 ± 0.419 | 0.290 ± 0.464 |
| Week 81 | 0.438 ± 0.463 | 0.207 ± 0.421 |
| Week 83 | 0.297 ± 0.379 | 0.233 ± 0.394 |
| Week 85 | 0.374 ± 0.374 | 0.266 ± 0.426 |
| Week 87 | 0.334 ± 0.409 | 0.344 ± 0.471 |
| Week 89 | 0.348 ± 0.482 | 0.198 ± 0.351 |
| Week 91 | 0.313 ± 0.449 | 0.279 ± 0.449 |
| Week 93 | 0.262 ± 0.383 | 0.178 ± 0.373 |
| Week 95 | 0.263 ± 0.495 | 0.198 ± 0.414 |
| Week 97 | 0.245 ± 0.306 | 0.210 ± 0.407 |
| Week 99 | 0.266 ± 0.351 | 0.213 ± 0.370 |
| Week 101 | 0.289 ± 0.289 | 0.207 ± 0.417 |
| Week 103 | 0.219 ± 0.291 | 0.212 ± 0.389 |
| Week 105 | 0.216 ± 0.291 | 0.174 ± 0.399 |
| Week 107 | 0.182 ± 0.273 | 0.245 ± 0.459 |
| Week 109 | 0.207 ± 0.311 | 0.254 ± 0.507 |
| Week 111 | 0.282 ± 0.556 | 0.263 ± 0.459 |
| Week 113 | 0.248 ± 0.366 | 0.335 ± 0.614 |
| Week 115 | 0.324 ± 0.413 | 0.165 ± 0.432 |
| Week 117 | 0.450 ± 0.492 | 0.181 ± 0.489 |
| Week 119 | 0.348 ± 0.400 | 0.187 ± 0.404 |
| Week 121 | 0.459 ± 0.414 | 0.174 ± 0.479 |
| Week 123 | 0.350 ± 0.375 | 0.192 ± 0.512 |
| Week 125 | 0.303 ± 0.425 | 0.114 ± 0.298 |
| Week 127 | 0.309 ± 0.401 | 0.220 ± 0.420 |
| Week 129 | 0.393 ± 0.506 | 0.191 ± 0.506 |
| Week 131 | 0.480 ± 0.529 | 0.222 ± 0.430 |
| Week 133 | 0.342 ± 0.469 | 0.125 ± 0.155 |
| Week 135 | 0.454 ± 0.643 | 0.280 ± 0.425 |
| Week 137 | 0.545 ± 0.631 | 0.070 ± 0.121 |
| Week 139 | 0.494 ± 0.676 | 0.293 ± 0.508 |
| Week 141 | 0.667 ± 0.583 | 0.025 ± 0.035 |
| Week 143 | 0.513 ± 0.604 | 0.270 ± 0.292 |
| Week 145 | 0.580 ± 0.820 | 0.130 ± 0.141 |
| Week 147 | 0.400 ± 0.592 | 0.195 ± 0.276 |
| Week 149 | 0.000 | 0.000 ± 0.000 |
| Week 151 | 0.000 ± 0.000 | 0.000 |
| Week 153 | 0.000 | 0.000 |
| Week 155 | 0.000 | 0.000 |
| Week 157 | 0.000 | 0.000 |
| Week 159 | 0.000 | 0.000 |
| Week 161 | 0.180 | 0.000 |
| Week 163 | 0.000 | 0.000 |
| Week 165 | 0.000 | — |
| Week 167 | 0.000 | — |
| Week 169 | 0.000 | — |
| Week 171 | 0.000 | — |
| Week 173 | 0.000 | — |
| Week 175 | 0.000 | — |
| ET/SFU Visit | 0.055 ± 0.200 | 0.181 ± 0.426 |
In this outcome measure, number of healthcare visits which included non-study healthcare resource utilization visit (consisted mainly of extra visits to the office of the study doctor, visit to a generalist doctor or visit to a specialist doctor), hospitalization visit and visit to hospital emergency is reported.
| visits | BIVV009/BIVV009 | Placebo/BIVV009 |
|---|---|---|
| Non-study healthcare resource utilization visits | 13 | 12 |
| Hospitalization | 3 | 1 |
| Visit to a hospital emergency room | 1 | 3 |
Collected over Part A (both 6.5 g and 7.5 g cohorts): first dose (Day 0) up to Week 25; Part B, 6.5 g cohort: From first dose (Week 26) up to 149 weeks of treatment + 9 weeks of follow-up (i.e., up to Week 184); Part B, 7.5 g cohort: From first dose (Week 26) up to 137 weeks of treatment + 9 weeks of follow-up (i.e., up to Week 172). Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Part A: BIVV009 6.5 g | 0/17 (0%) | 2/17 (11.8%) | 16/17 (94.1%) |
| Part A: BIVV009 7.5 g | 0/5 (0%) | 1/5 (20%) | 5/5 (100%) |
| Part A: Placebo | 0/20 (0%) | 0/20 (0%) | 3/20 (15%) |
| Part B: BIVV009 6.5 g | 1/32 (3.1%) | 6/32 (18.8%) | 28/32 (87.5%) |
| Part B: BIVV009 7.5 g | 0/7 (0%) | 1/7 (14.3%) | 7/7 (100%) |
| Event | Part A: BIVV009 6.5 g | Part A: BIVV009 7.5 g | Part A: Placebo | Part B: BIVV009 6.5 g | Part B: BIVV009 7.5 g |
|---|---|---|---|---|---|
| Febrile InfectionInfections and infestations | 0/17 | 1/5 | 0/20 | 0/32 | 0/7 |
| Blood Immunoglobulin M IncreasedInvestigations | 0/17 | 1/5 | 0/20 | 0/32 | 0/7 |
| Urinary Tract InfectionInfections and infestations | 0/17 | 0/5 | 0/20 | 0/32 | 1/7 |
| Lumbar Spinal StenosisMusculoskeletal and connective tissue disorders | 0/17 | 0/5 | 0/20 | 0/32 | 1/7 |
| OsteoarthritisMusculoskeletal and connective tissue disorders | 0/17 | 0/5 | 0/20 | 1/32 | 1/7 |
| Cerebral Venous Sinus ThrombosisNervous system disorders | 1/17 | 0/5 | 0/20 | 0/32 | 0/7 |
| Raynaud's PhenomenonVascular disorders | 1/17 | 0/5 | 0/20 | 0/32 | 0/7 |
| AnaemiaBlood and lymphatic system disorders | 0/17 | 0/5 | 0/20 | 1/32 | 0/7 |
| Polycystic Liver DiseaseCongenital, familial and genetic disorders | 0/17 | 0/5 | 0/20 | 1/32 | 0/7 |
| CholelithiasisHepatobiliary disorders | 0/17 | 0/5 | 0/20 | 1/32 | 0/7 |
| Event | Part A: BIVV009 6.5 g | Part A: BIVV009 7.5 g | Part A: Placebo | Part B: BIVV009 6.5 g | Part B: BIVV009 7.5 g |
|---|---|---|---|---|---|
| FatigueGeneral disorders | 1/17 | 1/5 | 0/20 | 7/32 | 5/7 |
| DyspnoeaRespiratory, thoracic and mediastinal disorders | 0/17 | 2/5 | 0/20 | 2/32 | 3/7 |
| AnaemiaBlood and lymphatic system disorders | 0/17 | 2/5 | 0/20 | 9/32 | 2/7 |
| NasopharyngitisInfections and infestations | 0/17 | 2/5 | 0/20 | 5/32 | 2/7 |
| PalpitationsCardiac disorders | 0/17 | 0/5 | 0/20 | 1/32 | 2/7 |
| NauseaGastrointestinal disorders | 1/17 | 0/5 | 0/20 | 3/32 | 2/7 |
| AstheniaGeneral disorders | 0/17 | 1/5 | 0/20 | 4/32 | 2/7 |
| CystitisInfections and infestations | 0/17 | 0/5 | 0/20 | 2/32 | 2/7 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 2/17 | 1/5 | 1/20 | 6/32 | 2/7 |
| DizzinessNervous system disorders | 2/17 | 0/5 | 0/20 | 3/32 | 2/7 |
Analysis was performed on full analysis set (FAS) which included all randomized participants who received at least 1 dose (including partial dose) of study drug (BIVV009 or placebo).
| Age, Continuous(years) | BIVV009/BIVV009 | Placebo/BIVV009 | Total |
|---|---|---|---|
| Mean | 65.3 ± 10.9 | 68.2 ± 10.1 | 66.7 ± 10.5 |
| Sex: Female, Male(Participants) | BIVV009/BIVV009 | Placebo/BIVV009 | Total |
|---|---|---|---|
| Female | 17 | 16 | 33 |
| Male | 5 | 4 | 9 |
| Race (NIH/OMB)(Participants) | BIVV009/BIVV009 | Placebo/BIVV009 | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 5 | 2 | 7 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 |
| White | 0 | 4 | 4 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 17 | 14 | 31 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Yes — Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Ran from Mar 2018 to primary completion in Dec 2021, 44 months.
Results were first posted in Dec 2022, 1 year after primary completion.
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Studies of the drug tested here: Sutimlimab
Anemia, Hemolytic, Autoimmune →
Bioverativ, a Sanofi company →