A Phase 3 interventional study of BIVV009 in Agglutinin Disease, Cold, sponsored by Bioverativ, a Sanofi company. Completed at 49 sites in 14 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2022-10-31.
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 15th 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 24 is close to 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.
This tab as a report to print or save as PDF → With an account.
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 |
Add it to your calendar (.ics) → With an account.
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 (>=) 2 grams per deciliter (g/dL) increase in hemoglobin (Hgb) levels or increased Hgb to >= 12 g/dL and obviated the need for blood transfusion during treatment in participants with primary cold agglutinin disease (CAD) who had 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 CAD.
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:
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 who had a recent history of transfusion (defined as at least 1 transfusion during the last 6 months prior to screening) received an intravenous (IV) infusion of BIVV009 6.5 grams (g) (if body weight was less than \[\<\] 75 kilograms \[kg\]) or BIVV009 7.5 g (if body weight was 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) could continue to receive BIVV009 in Part B, every 2 weeks starting at Week 27 for up to an additional 149 weeks. All participants who completed Part A elected to continue in Part B.
Drug: BIVV009
Sutimlimab was administered as intravenous (IV) infusion.
Also known as: Sutimlimab
Part A: Percentage of Participants With Response to Treatment
A participant was considered a responder: if he or she did not receive a blood transfusion from Week 5 through Week 26 (end of treatment in Part A) and did not receive treatment for CAD beyond what was permitted per protocol. Additionally, the participant's hemoglobin (Hgb) level must meet either of the following criteria: Hgb level \>= 12 grams per deciliter (g/dL) at the treatment assessment endpoint (defined as the average of the values from the Week 23, 25, and 26 visits), or Hgb increased \>= 2 g/dL from baseline (defined as the last Hgb value before administration of the first dose of study drug) at treatment assessment endpoint. Percentage of responders was calculated together with a 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 (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
An Adverse Event (AE) was defined as any untoward medical occurrence in a participant who received study drug and did not necessarily have to have a causal relationship with the treatment. 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 a congenital anomaly/birth defect, was a medically important event. TEAEs were defined as AEs that developed, worsened or became serious during the treatment-emergent (TE) period (from the first investigational medicinal product \[IMP\] administration in Part B to the last IMP administration + 9 weeks follow up period).
Time frame: Part B, 6.5 g cohort: From first dose (Week 27) up to 143 weeks of treatment + 9 weeks of follow-up (i.e., up to Week 179); Part B, 7.5 g cohort: From first dose (Week 27) up to 149 weeks of treatment + 9 weeks of follow-up (i.e., up to Week 185)
Part A: Mean Change From Baseline in Bilirubin Levels at the Treatment Assessment Timepoint
Mean change from baseline in bilirubin levels at the treatment assessment timepoint was 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, 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 (Quality of Life) at 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 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, 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 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. LS mean and 95% CI was assessed by MMRM approach using 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, treatment assessment timepoint (i.e., average of Week 23, 25 and 26)
Part A: 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. Number of transfusions after the first 5 weeks of study drug administration and up to Week 26 are reported in this outcome measure.
Time frame: From Week 5 up to Week 26
Part A: Number of Blood Units Transfused 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. Number of blood units transfused after the first 5 weeks of study drug administration and up to Week 26 are reported in this outcome measure.
Time frame: From Week 5 up to Week 26
Part A: Mean Change From Baseline in Hemoglobin (Hgb) Level at the Treatment Assessment Timepoint
Mean change from baseline (Week 0) in Hgb at 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. LS mean and 95% CI was assessed by MMRM approach using 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, treatment assessment timepoint (i.e., average of Week 23, 25 and 26)
Part B: Change From Baseline in Hemoglobin (Hgb) Level at Each Specified Time Points
Change From Hgb level from baseline (Week 0) at each specified time points (i.e., Weeks 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, 83, 87, 91, 95, 99, 103, 107, 111, 115, 119, 123, 127, 131, 135, 139, 143, 147, 151, 155, 159, 163, 167, 171, 175 and early termination/safety follow up \[ET/SFU\] 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 185).
Time frame: Baseline, Weeks 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, 83, 87, 91, 95, 99, 103, 107, 111, 115, 119, 123, 127, 131, 135, 139, 143, 147, 151, 155, 159, 163, 167, 171, 175, ET/SFU (up to Week 185)
Part B: Change From Baseline in Bilirubin Levels at Each Specified Time Points
Change from baseline (Week 0) in bilirubin levels at each specified time point (i.e., Weeks 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, 83, 87, 91, 95, 99, 103, 107, 111, 115, 119, 123, 127, 131, 135, 139, 143, 147, 151, 155, 159, 163, 167, 171, 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 185).
Time frame: Baseline, Weeks 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, 83, 87, 91, 95, 99, 103, 107, 111, 115, 119, 123, 127, 131, 135, 139, 143, 147, 151, 155, 159, 163, 167, 171, 175, ET/SFU (up to Week 185)
Part B: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score (Quality of Life) at Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135, 147,159, 171 and Early Termination (ET) Visit/Safety Follow-up Visit
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 185).
Time frame: Baseline, Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135, 147,159, 171 and ET Visit/SFU visit (i.e., up to Week 185)
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 for which, score ranged 0 (poor health) to 100 (better health). Higher scores = better HRQOL. Baseline (Week 0) was defined as the last non-missing value prior to 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 185). Change from baseline in SF-12 PCS and MCS scores is reported in this outcome measure.
Time frame: Baseline, Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135 and ET Visit/SFU visit (i.e., up to Week 185)
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 Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135, 147,159, 171 and ET Visit/SFU Visit
EQ-5D-5L:standardized, participant-rated questionnaire to assess health-related quality of life. EQ-5D-5L includes 2 components: EQ-5D-5L 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 problems, slight problems, moderate problems, severe problems, and extreme problems measured with Likert scale. EQ-5D-5L responses relating to 5 dimensions are converted into a single index utility score between 0 to 1, where higher score=better health state. The EQ-5D-5L VAS rated participant's current health state on a scale from 0=worst imaginable health state to 100 =best imaginable health state. Baseline (Week 0): last non-missing value prior to 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 185).
Time frame: Baseline, Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135, 147,159, 171 and ET Visit/SFU visit (i.e., up to Week 185)
Part B: Number of Participants With Response to Participant's Global Impression of (Fatigue) Severity (PGIS) Questionnaire at Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135, 147,159, 171 and at ET Visit/SFU Visit
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 185).
Time frame: At Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135, 147,159, 171 and at ET Visit/SFU visit (i.e., up to Week 185)
Part B: Number of Participants With Response to Participant's Global Impression of Change (PGIC) Questionnaire at Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135, 147,159, 171 and at ET Visit/SFU Visit
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 185).
Time frame: At Weeks 39, 51, 63, 75, 87, 99, 111, 123, 135, 147,159, 171 and at ET Visit/SFU visit (i.e., up to Week 185)
Part B: Mean Change From Baseline in Lactate Dehydrogenase (LDH) Level at Each Specified Time Points
Mean change from baseline in LDH levels at each specified time points (i.e., Weeks 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, 83, 87, 91, 95, 99, 103, 107, 111, 115, 119, 123, 127, 131, 135, 139, 143, 147, 151, 155, 159, 163, 167, 171, 175 and ET/SFU Visit) is reported in this outcome measure. 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 185).
Time frame: Baseline, Weeks 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, 83, 87, 91, 95, 99, 103, 107, 111, 115, 119, 123, 127, 131, 135, 139, 143, 147, 151, 155, 159, 163, 167, 171, 175, ET/SFU (up to Week 185)
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: Number of Blood Units Transfused 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: Change From Baseline in Haptoglobin Values at Each Specified Time Points
Change in Haptoglobin values from baseline at each specified time points (i.e., Weeks 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, 83, 87, 91, 95, 99, 103, 107, 111, 115, 119, 123, 127, 131, 135, 139, 143, 147, 151, 155, 159, 163, 167, 171, 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 185). Haptoglobin values \<0.2 were imputed as 0.2.
Time frame: Baseline, Weeks 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, 83, 87, 91, 95, 99, 103, 107, 111, 115, 119, 123, 127, 131, 135, 139, 143, 147, 151, 155, 159, 163, 167, 171, 175, ET/SFU (up to Week 185)
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, or visit to a generalist doctor, or visit to a specialist doctor) and 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 16 active sites in 8 countries. Out of 42 screened participants, a total of 24 participants were enrolled from 05 March 2018 to 10 Jan 2019. This was a single arm study that consisted of 2 Parts: Part A and Part B.
| Milestone | BIVV009 6.5 g | BIVV009 7.5 g |
|---|---|---|
| Started | 17 | 7 |
| Completed | 16 | 6 |
| Not completed | 1 | 1 |
| Withdrew: Adverse event | 1 | 0 |
| Withdrew: Death | 0 | 1 |
| Milestone | BIVV009 6.5 g | BIVV009 7.5 g |
|---|---|---|
| Started | 16 | 6 |
| Completed | 14 | 5 |
| Not completed | 2 | 1 |
| Withdrew: Adverse event | 1 | 1 |
| Withdrew: Death | 1 | 0 |
A participant was considered a responder: if he or she did not receive a blood transfusion from Week 5 through Week 26 (end of treatment in Part A) and did not receive treatment for CAD beyond what was permitted per protocol. Additionally, the participant's hemoglobin (Hgb) level must meet either of the following criteria: Hgb level \>= 12 grams per deciliter (g/dL) at the treatment assessment endpoint (defined as the average of the values from the Week 23, 25, and 26 visits), or Hgb increased \>= 2 g/dL from baseline (defined as the last Hgb value before administration of the first dose of study drug) at treatment assessment endpoint. Percentage of responders was calculated together with a 95% exact Clopper-Pearson confidence interval (CI).
| percentage of participants | BIVV009 |
|---|---|
| Part A: Percentage of Participants With Response to Treatment | 54.2 (32.8 to 74.4) |
An Adverse Event (AE) was defined as any untoward medical occurrence in a participant who received study drug and did not necessarily have to have a causal relationship with the treatment. 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 a congenital anomaly/birth defect, was a medically important event. TEAEs were defined as AEs that developed, worsened or became serious during the treatment-emergent (TE) period (from the first investigational medicinal product \[IMP\] administration in Part B to the last IMP administration + 9 weeks follow up period).
| Participants | BIVV009 6.5 g | BIVV009 7.5 g |
|---|---|---|
| TEAEs | 16 | 6 |
| TESAEs | 10 | 2 |
Mean change from baseline in bilirubin levels at the treatment assessment timepoint was 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.
| micromoles per Liter (mcmol/L) | BIVV009 |
|---|---|
| Part A: Mean Change From Baseline in Bilirubin Levels at the Treatment Assessment Timepoint | -38.18 (-42.52 to -33.84) |
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 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 |
|---|---|
| Part A: Mean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score (Quality of Life) at Treatment Assessment Timepoint | 10.85 (8.00 to 13.70) |
Mean change from baseline 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. LS mean and 95% CI was assessed by MMRM approach using 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.
| units per liter | BIVV009 |
|---|---|
| Part A: Mean Change From Baseline in Lactate Dehydrogenase (LDH) at the Treatment Assessment Timepoint | -126.95 (-218.47 to -35.42) |
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. Number of transfusions after the first 5 weeks of study drug administration and up to Week 26 are reported in this outcome measure.
| blood transfusions per participant | BIVV009 |
|---|---|
| Part A: Number of Blood Transfusions Per Participant | 0.9 ± 2.75 |
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. Number of blood units transfused after the first 5 weeks of study drug administration and up to Week 26 are reported in this outcome measure.
| blood units transfused per participant | BIVV009 |
|---|---|
| Part A: Number of Blood Units Transfused Per Participant | 5.8 ± 8.47 |
Mean change from baseline (Week 0) in Hgb at 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. LS mean and 95% CI was assessed by MMRM approach using 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.
| g/dL | BIVV009 |
|---|---|
| Part A: Mean Change From Baseline in Hemoglobin (Hgb) Level at the Treatment Assessment Timepoint | 2.60 (0.74 to 4.46) |
Change From Hgb level from baseline (Week 0) at each specified time points (i.e., Weeks 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, 83, 87, 91, 95, 99, 103, 107, 111, 115, 119, 123, 127, 131, 135, 139, 143, 147, 151, 155, 159, 163, 167, 171, 175 and early termination/safety follow up \[ET/SFU\] 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 185).
| g/dL | BIVV009 |
|---|---|
| Week 27 | 2.76 ± 2.20 |
| Week 29 | 2.77 ± 2.20 |
| Week 31 | 2.74 ± 1.96 |
| Week 33 | 2.87 ± 1.97 |
| Week 35 | 2.82 ± 2.10 |
| Week 37 | 2.71 ± 2.08 |
| Week 39 | 2.72 ± 2.26 |
| Week 41 | 2.76 ± 2.24 |
| Week 43 | 2.73 ± 2.06 |
| Week 45 | 2.84 ± 2.21 |
| Week 47 | 2.74 ± 1.95 |
| Week 49 | 2.73 ± 2.09 |
| Week 51 | 2.71 ± 1.97 |
| Week 53 | 2.66 ± 1.97 |
| Week 55 | 2.73 ± 1.96 |
| Week 57 | 3.01 ± 2.36 |
| Week 59 | 2.81 ± 2.10 |
| Week 61 | 2.88 ± 2.37 |
| Week 63 | 2.99 ± 2.33 |
| Week 65 | 2.53 ± 2.18 |
| Week 67 | 2.52 ± 2.20 |
| Week 69 | 2.96 ± 2.08 |
| Week 71 | 2.86 ± 2.31 |
| Week 73 | 2.79 ± 2.25 |
| Week 75 | 2.70 ± 2.18 |
| Week 77 | 2.93 ± 2.59 |
| Week 79 | 3.13 ± 2.23 |
| Week 83 | 2.89 ± 2.47 |
| Week 87 | 2.63 ± 2.36 |
| Week 91 | 3.23 ± 2.17 |
| Week 95 | 3.12 ± 2.06 |
| Week 99 | 2.98 ± 2.01 |
| Week 103 | 2.92 ± 2.09 |
| Week 107 | 2.60 ± 2.10 |
| Week 111 | 2.82 ± 2.17 |
| Week 115 | 2.96 ± 2.25 |
| Week 119 | 2.79 ± 2.57 |
| Week 123 | 2.82 ± 2.35 |
| Week 127 | 2.89 ± 1.99 |
| Week 131 | 3.03 ± 2.19 |
| Week 135 | 3.35 ± 1.99 |
| Week 139 | 3.42 ± 2.14 |
| Week 143 | 3.43 ± 2.31 |
| Week 147 | 3.75 ± 2.38 |
| Week 151 | 3.43 ± 1.91 |
| Week 155 | 3.74 ± 1.94 |
| Week 159 | 5.20 ± 1.65 |
| Week 163 | 4.18 ± 1.14 |
| Week 167 | 4.80 ± 0.42 |
| Week 171 | 4.80 |
| Week 175 | 4.40 |
| ET/SFU Visit | 1.21 ± 1.56 |
Change from baseline (Week 0) in bilirubin levels at each specified time point (i.e., Weeks 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, 83, 87, 91, 95, 99, 103, 107, 111, 115, 119, 123, 127, 131, 135, 139, 143, 147, 151, 155, 159, 163, 167, 171, 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 185).
| mcmol/L | BIVV009 |
|---|---|
| Week 27 | -34.96 ± 18.31 |
| Week 29 | -34.92 ± 15.78 |
| Week 31 | -32.58 ± 17.07 |
| Week 33 | -32.67 ± 17.52 |
| Week 35 | -32.25 ± 22.39 |
| Week 37 | -33.24 ± 15.53 |
| Week 39 | -35.92 ± 13.17 |
| Week 41 | -35.03 ± 13.71 |
| Week 43 | -36.46 ± 17.25 |
| Week 45 | -34.81 ± 15.76 |
| Week 47 | -34.57 ± 16.53 |
| Week 49 | -32.36 ± 19.50 |
| Week 51 | -34.88 ± 15.90 |
| Week 53 | -35.25 ± 18.32 |
| Week 55 | -34.79 ± 16.70 |
| Week 57 | -36.32 ± 16.54 |
| Week 59 | -35.77 ± 17.73 |
| Week 61 | -36.39 ± 16.14 |
| Week 63 | -38.25 ± 15.43 |
| Week 65 | -35.46 ± 16.84 |
| Week 67 | -35.74 ± 18.37 |
| Week 69 | -33.88 ± 16.83 |
| Week 71 | -32.54 ± 23.01 |
| Week 73 | -33.03 ± 21.82 |
| Week 75 | -30.55 ± 21.68 |
| Week 77 | -30.95 ± 22.83 |
| Week 79 | -35.51 ± 14.79 |
| Week 83 | -38.60 ± 13.90 |
| Week 87 | -37.45 ± 16.29 |
| Week 91 | -35.19 ± 16.07 |
| Week 95 | -33.57 ± 15.93 |
| Week 99 | -28.71 ± 21.98 |
| Week 103 | -32.04 ± 22.45 |
| Week 107 | -34.46 ± 15.27 |
| Week 111 | -37.67 ± 14.28 |
| Week 115 | -36.41 ± 16.76 |
| Week 119 | -34.65 ± 13.71 |
| Week 123 | -29.99 ± 24.96 |
| Week 127 | -35.57 ± 17.83 |
| Week 131 | -35.03 ± 18.72 |
| Week 135 | -36.88 ± 14.71 |
| Week 139 | -37.60 ± 14.59 |
| Week 143 | -44.50 ± 15.96 |
| Week 147 | -44.82 ± 15.12 |
| Week 151 | -46.53 ± 10.80 |
| Week 155 | -50.32 ± 12.63 |
| Week 159 | -46.30 ± 13.90 |
| Week 163 | -46.13 ± 11.86 |
| Week 167 | -36.30 ± 12.59 |
| Week 171 | -32.50 |
| Week 175 | -29.10 |
| ET/SFU Visit | -9.98 ± 18.11 |
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 185).
| score on a scale | BIVV009 |
|---|---|
| Week 39 | 9.37 ± 16.00 |
| Week 51 | 10.50 ± 12.05 |
| Week 63 | 10.21 ± 11.88 |
| Week 75 | 11.00 ± 11.51 |
| Week 87 | 10.39 ± 13.41 |
| Week 99 | 9.94 ± 8.19 |
| Week 111 | 9.11 ± 12.35 |
| Week 123 | 6.79 ± 11.28 |
| Week 135 | 11.71 ± 13.85 |
| Week 147 | 13.29 ± 13.39 |
| Week 159 | 24.75 ± 10.24 |
| Week 171 | 32.00 |
| ET/SFU Visit | 1.05 ± 8.15 |
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 for which, score ranged 0 (poor health) to 100 (better health). Higher scores = better HRQOL. Baseline (Week 0) was defined as the last non-missing value prior to 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 185). Change from baseline in SF-12 PCS and MCS scores is reported in this outcome measure.
| score on a scale | BIVV009 |
|---|---|
| Week 39-PCS | 7.813 ± 10.486 |
| Week 39-MCS | 5.859 ± 10.249 |
| Week 51-PCS | 6.677 ± 9.925 |
| Week 51-MCS | 3.912 ± 9.493 |
| Week 63-PCS | 8.318 ± 7.148 |
| Week 63-MCS | 2.385 ± 9.777 |
| Week 75-PCS | 6.580 ± 9.214 |
| Week 75-MCS | 3.102 ± 9.881 |
| Week 87-PCS | 6.398 ± 9.021 |
| Week 87-MCS | 1.611 ± 10.336 |
| Week 99-PCS | 9.054 ± 5.803 |
| Week 99-MCS | 2.581 ± 9.169 |
| Week 111-PCS | 11.958 ± 3.832 |
| Week 111-MCS | 2.523 ± 12.585 |
| Week 123-PCS | 4.743 ± 6.900 |
| Week 123-MCS | 3.810 ± 14.071 |
| Week 135-PCS | 10.450 |
| Week 135-MCS | 27.900 |
| ET/SFU Visit-PCS | -8.920 |
| ET/SFU Visit-MCS | -1.180 |
EQ-5D-5L:standardized, participant-rated questionnaire to assess health-related quality of life. EQ-5D-5L includes 2 components: EQ-5D-5L 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 problems, slight problems, moderate problems, severe problems, and extreme problems measured with Likert scale. EQ-5D-5L responses relating to 5 dimensions are converted into a single index utility score between 0 to 1, where higher score=better health state. The EQ-5D-5L VAS rated participant's current health state on a scale from 0=worst imaginable health state to 100 =best imaginable health state. Baseline (Week 0): last non-missing value prior to 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 185).
| score on a scale | BIVV009 |
|---|---|
| Week 39 - Index score | 0.067 ± 0.264 |
| Week 39 - VAS score | 18.111 ± 20.642 |
| Week 51 - Index score | 0.078 ± 0.194 |
| Week 51 - VAS score | 14.500 ± 19.050 |
| Week 63 - Index score | 0.092 ± 0.166 |
| Week 63 - VAS score | 18.053 ± 16.844 |
| Week 75 - Index score | 0.069 ± 0.211 |
| Week 75 - VAS score | 17.842 ± 16.604 |
| Week 87 - Index score | 0.022 ± 0.226 |
| Week 87 - VAS score | 14.421 ± 20.815 |
| Week 99 - Index Score | 0.060 ± 0.136 |
| Week 99 - VAS Score | 14.059 ± 13.818 |
| Week 111 - Index score | 0.099 ± 0.174 |
| Week 111 - VAS score | 18.889 ± 15.408 |
| Week 123 - Index Score | 0.009 ± 0.190 |
| Week 123 - VAS score | 8.842 ± 18.765 |
| Week 135 - Index score | 0.085 ± 0.233 |
| Week 135 - VAS score | 17.067 ± 21.608 |
| Week 147 - Index score | 0.143 ± 0.131 |
| Week 147 - VAS score | 17.857 ± 20.178 |
| Week 159 - Index Score | 0.250 ± 0.145 |
| Week 159 - VAS score | 36.250 ± 14.930 |
| Week 171 - Index score | 0.535 |
| Week 171 - VAS score | 35.000 |
| ET/SFU Visit - Index score | -0.025 ± 0.173 |
| ET/SFU Visit - VAS score | 1.263 ± 19.287 |
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 185).
| Participants | BIVV009 |
|---|---|
| Week 39 - None | 5 |
| Week 39 - Mild | 8 |
| Week 39 - Moderate | 4 |
| Week 39 - Severe | 1 |
| Week 39 - Very severe | 0 |
| Week 51 - None | 5 |
| Week 51 - Mild | 13 |
| Week 51 - Moderate | 3 |
| Week 51 - Severe | 0 |
| Week 51 - Very severe | 0 |
| Week 63 - None | 4 |
| Week 63 - Mild | 12 |
| Week 63 - Moderate | 3 |
| Week 63 - Severe | 1 |
| Week 63 - Very severe | 0 |
| Week 75 - None | 3 |
| Week 75 - Mild | 12 |
| Week 75 - Moderate | 5 |
| Week 75 - Severe | 0 |
| Week 75 - Very severe | 0 |
| Week 87 - None | 5 |
| Week 87 - Mild | 9 |
| Week 87 - Moderate | 3 |
| Week 87 - Severe | 2 |
| Week 87 - Very severe | 0 |
| Week 99 - None | 4 |
| Week 99 - Mild | 10 |
| Week 99 - Moderate | 4 |
| Week 99 - Severe | 0 |
| Week 99 - Very severe | 0 |
| Week 111 - None | 7 |
| Week 111 - Mild | 6 |
| Week 111 - Moderate | 6 |
| Week 111 - Severe | 0 |
| Week 111 - Very severe | 0 |
| Week 123 - None | 4 |
| Week 123 - Mild | 7 |
| Week 123 - Moderate | 7 |
| Week 123 - Severe | 2 |
| Week 123 - Very severe | 0 |
| Week 135 - None | 4 |
| Week 135 - Mild | 8 |
| Week 135 - Moderate | 3 |
| Week 135 - Severe | 0 |
| Week 135 - Very severe | 0 |
| Week 147 - None | 3 |
| Week 147 - Mild | 3 |
| Week 147 - Moderate | 1 |
| Week 147 - Severe | 0 |
| Week 147 - Very severe | 0 |
| Week 159 - None | 3 |
| Week 159 - Mild | 1 |
| Week 159 - Moderate | 0 |
| Week 159 - Severe | 0 |
| Week 159 - Very severe | 0 |
| Week 171 - None | 0 |
| Week 171 - Mild | 1 |
| Week 171 - Moderate | 0 |
| Week 171 - Severe | 0 |
| Week 171 - Very severe | 0 |
| ET/SFU Visit - None | 4 |
| ET/SFU Visit - Mild | 6 |
| ET/SFU Visit - Moderate | 4 |
| ET/SFU Visit - Severe | 5 |
| ET/SFU Visit - Very severe | 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 185).
| Participants | BIVV009 |
|---|---|
| Week 39 - Very much improved | 9 |
| Week 39 - Much improved | 5 |
| Week 39 - Minimally improved | 3 |
| Week 39 - No change | 1 |
| Week 39 - Minimally worse | 0 |
| Week 39 - Much worse | 1 |
| Week 39 - Very much worse | 0 |
| Week 51 - Very much improved | 6 |
| Week 51 - Much improved | 13 |
| Week 51 - Minimally improved | 2 |
| Week 51 - No change | 0 |
| Week 51 - Minimally worse | 0 |
| Week 51 - Much worse | 0 |
| Week 51- Very much worse | 0 |
| Week 63 - Very much improved | 8 |
| Week 63 - Much improved | 9 |
| Week 63 - Minimally improved | 1 |
| Week 63 - No change | 1 |
| Week 63 - Minimally worse | 1 |
| Week 63 - Much worse | 0 |
| Week 63 - Very much worse | 0 |
| Week 75 - Very much improved | 5 |
| Week 75 - Much improved | 11 |
| Week 75 - Minimally improved | 1 |
| Week 75 - No change | 2 |
| Week 75 - Minimally worse | 1 |
| Week 75 - Much worse | 0 |
| Week 75 - Very much worse | 0 |
| Week 87 - Very much improved | 5 |
| Week 87 - Much improved | 11 |
| Week 87 - Minimally improved | 2 |
| Week 87 - No change | 1 |
| Week 87 - Minimally worse | 0 |
| Week 87 - Much worse | 0 |
| Week 87 - Very much worse | 0 |
| Week 99 - Very much improved | 4 |
| Week 99 - Much improved | 10 |
| Week 99 - Minimally improved | 3 |
| Week 99 - No Change | 1 |
| Week 99 - Minimally worse | 0 |
| Week 99 - Much worse | 0 |
| Week 99 - Very much worse | 0 |
| Week 111 - Very much improved | 7 |
| Week 111 - Much improved | 7 |
| Week 111 - Minimally improved | 5 |
| Week 111 - No change | 0 |
| Week 111 - Minimally worse | 0 |
| Week 111 - Much worse | 0 |
| Week 111 - Very much worse | 0 |
| Week 123 - Very much improved | 8 |
| Week 123 - Much improved | 6 |
| Week 123 - Minimally improved | 6 |
| Week 123 - No change | 0 |
| Week 123 - Minimally worse | 0 |
| Week 123 - Much worse | 0 |
| Week 123 - Very much worse | 0 |
| Week 135 - Very much improved | 5 |
| Week 135 - Much improved | 6 |
| Week 135 - Minimally improved | 2 |
| Week 135 - No change | 1 |
| Week 135 - Minimally worse | 1 |
| Week 135 - Much worse | 0 |
| Week 135 - Very much worse | 0 |
| Week 147 - Very much improved | 4 |
| Week 147 - Much improved | 2 |
| Week 147 - Minimally improved | 1 |
| Week 147 - No change | 0 |
| Week 147 - Minimally worse | 0 |
| Week 147 - Much worse | 0 |
| Week 147 - Very much worse | 0 |
| Week 159 - Very much improved | 4 |
| Week 159 - Much improved | 0 |
| Week 159 - Minimally improved | 0 |
| Week 159 - No change | 0 |
| Week 159 - Minimally worse | 0 |
| Week 159 - Much worse | 0 |
| Week 159 - Very much worse | 0 |
| Week 171 - Very much improved | 1 |
| Week 171 - Much improved | 0 |
| Week 171 - Minimally improved | 0 |
| Week 171 - No Change | 0 |
| Week 171 - Minimally worse | 0 |
| Week 171 - Much worse | 0 |
| Week 171 - Very much worse | 0 |
| ET/SFU Visit - Very much improved | 3 |
| ET/SFU Visit - Much improved | 8 |
| ET/SFU Visit - Minimally improved | 2 |
| ET/SFU Visit - No Change | 4 |
| ET/SFU Visit - Minimally worse | 2 |
| ET/SFU Visit - Much worse | 0 |
| ET/SFU Visit - Very much worse | 1 |
Mean change from baseline in LDH levels at each specified time points (i.e., Weeks 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, 83, 87, 91, 95, 99, 103, 107, 111, 115, 119, 123, 127, 131, 135, 139, 143, 147, 151, 155, 159, 163, 167, 171, 175 and ET/SFU Visit) is reported in this outcome measure. 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 185).
| units per liter | BIVV009 |
|---|---|
| Week 27 | -111.59 ± 327.80 |
| Week 29 | -129.76 ± 331.91 |
| Week 31 | -95.27 ± 319.25 |
| Week 33 | -82.19 ± 330.04 |
| Week 35 | -126.64 ± 346.75 |
| Week 37 | -151.57 ± 307.15 |
| Week 39 | -49.27 ± 172.53 |
| Week 41 | -116.10 ± 305.11 |
| Week 43 | -150.26 ± 271.72 |
| Week 45 | -85.48 ± 308.01 |
| Week 47 | -79.81 ± 286.13 |
| Week 49 | -104.38 ± 313.66 |
| Week 51 | -76.84 ± 344.60 |
| Week 53 | -87.00 ± 299.09 |
| Week 55 | -68.29 ± 305.09 |
| Week 57 | -101.71 ± 273.44 |
| Week 59 | -97.71 ± 296.74 |
| Week 61 | -89.48 ± 298.05 |
| Week 63 | -94.20 ± 288.34 |
| Week 65 | -91.05 ± 305.75 |
| Week 67 | -106.32 ± 327.48 |
| Week 69 | -75.00 ± 333.47 |
| Week 71 | -52.39 ± 378.82 |
| Week 73 | -70.35 ± 348.56 |
| Week 75 | -18.68 ± 323.02 |
| Week 77 | -64.67 ± 335.73 |
| Week 79 | -54.10 ± 338.58 |
| Week 83 | -87.21 ± 344.88 |
| Week 87 | -89.17 ± 392.80 |
| Week 91 | -78.47 ± 349.15 |
| Week 95 | -132.75 ± 301.36 |
| Week 99 | -13.83 ± 155.04 |
| Week 103 | -106.40 ± 309.44 |
| Week 107 | -85.45 ± 312.50 |
| Week 111 | -107.58 ± 286.18 |
| Week 115 | -107.84 ± 293.61 |
| Week 119 | -63.68 ± 310.56 |
| Week 123 | -58.90 ± 339.49 |
| Week 127 | -57.72 ± 400.26 |
| Week 131 | -113.63 ± 344.53 |
| Week 135 | -97.06 ± 339.41 |
| Week 139 | -1.00 ± 256.65 |
| Week 143 | 180.70 ± 318.62 |
| Week 147 | -10.29 ± 196.90 |
| Week 151 | -24.13 ± 146.79 |
| Week 155 | -17.38 ± 146.80 |
| Week 159 | -126.00 ± 150.38 |
| Week 163 | -31.50 ± 35.49 |
| Week 167 | -48.50 ± 68.59 |
| Week 171 | -23.00 |
| Week 175 | 6.00 |
| ET/SFU visit | -107.50 ± 249.28 |
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 |
|---|---|
| Part B: Number of Blood Transfusions Per Participant | 2.86 ± 6.58 |
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 units transfused per participants | BIVV009 |
|---|---|
| Part B: Number of Blood Units Transfused Per Participant | 16.57 ± 16.85 |
Change in Haptoglobin values from baseline at each specified time points (i.e., Weeks 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, 83, 87, 91, 95, 99, 103, 107, 111, 115, 119, 123, 127, 131, 135, 139, 143, 147, 151, 155, 159, 163, 167, 171, 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 185). Haptoglobin values \<0.2 were imputed as 0.2.
| grams per liter | BIVV009 |
|---|---|
| Week 27 | 0.21 ± 0.38 |
| Week 29 | 0.21 ± 0.45 |
| Week 31 | 0.21 ± 0.43 |
| Week 33 | 0.28 ± 0.53 |
| Week 35 | 0.25 ± 0.41 |
| Week 37 | 0.14 ± 0.28 |
| Week 39 | 0.27 ± 0.49 |
| Week 41 | 0.39 ± 0.59 |
| Week 43 | 0.29 ± 0.47 |
| Week 45 | 0.16 ± 0.26 |
| Week 47 | 0.15 ± 0.35 |
| Week 49 | 0.21 ± 0.41 |
| Week 51 | 0.20 ± 0.37 |
| Week 53 | 0.23 ± 0.46 |
| Week 55 | 0.26 ± 0.45 |
| Week 57 | 0.26 ± 0.43 |
| Week 59 | 0.29 ± 0.44 |
| Week 61 | 0.23 ± 0.43 |
| Week 63 | 0.38 ± 0.50 |
| Week 65 | 0.25 ± 0.41 |
| Week 67 | 0.25 ± 0.42 |
| Week 69 | 0.24 ± 0.47 |
| Week 71 | 0.19 ± 0.33 |
| Week 73 | 0.13 ± 0.29 |
| Week 75 | 0.13 ± 0.24 |
| Week 77 | 0.17 ± 0.29 |
| Week 79 | 0.09 ± 0.22 |
| Week 83 | 0.14 ± 0.25 |
| Week 87 | 0.35 ± 0.53 |
| Week 91 | 0.17 ± 0.28 |
| Week 95 | 0.19 ± 0.33 |
| Week 99 | 0.21 ± 0.37 |
| Week 103 | 0.15 ± 0.25 |
| Week 107 | 0.26 ± 0.40 |
| Week 111 | 0.22 ± 0.33 |
| Week 115 | 0.21 ± 0.35 |
| Week 119 | 0.16 ± 0.31 |
| Week 123 | 0.19 ± 0.45 |
| Week 127 | 0.14 ± 0.23 |
| Week 131 | 0.18 ± 0.38 |
| Week 135 | 0.18 ± 0.35 |
| Week 139 | 0.10 ± 0.17 |
| Week 143 | 0.07 ± 0.16 |
| Week 147 | 0.06 ± 0.16 |
| Week 151 | 0.12 ± 0.24 |
| Week 155 | 0.09 ± 0.22 |
| Week 159 | 0.15 ± 0.34 |
| Week 163 | 0.30 ± 0.35 |
| Week 167 | 0.00 ± 0.00 |
| Week 171 | 0.00 |
| Week 175 | 0.00 |
| ET/SFU visit | 0.02 ± 0.13 |
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, or visit to a generalist doctor, or visit to a specialist doctor) and hospitalization visit and visit to hospital emergency is reported.
| visits | BIVV009 |
|---|---|
| Non-study healthcare resource utilization visits | 16 |
| Hospitalization | 8 |
| Visit to a hospital emergency room | 3 |
Collected over Part A (both 6.5 g and 7.5 g cohorts): From first dose (Day 0) up to Week 26; Part B, 6.5 g cohort: From first dose (Week 27) up to 143 weeks of treatment + 9 weeks of follow-up (i.e., up to Week 179). Part B, 7.5 g cohort: From first dose (Week 27) up to 149 weeks of treatment + 9 weeks of follow-up (i.e., up to Week 185). Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Part A: BIVV009 6.5 g | 0/17 (0%) | 4/17 (23.5%) | 15/17 (88.2%) |
| Part A: BIVV009 7.5 g | 1/7 (14.3%) | 3/7 (42.9%) | 6/7 (85.7%) |
| Part B: BIVV009 6.5 g | 2/16 (12.5%) | 10/16 (62.5%) | 16/16 (100%) |
| Part B: BIVV009 7.5 g | 0/6 (0%) | 2/6 (33.3%) | 6/6 (100%) |
| Event | Part A: BIVV009 6.5 g | Part A: BIVV009 7.5 g | Part B: BIVV009 6.5 g | Part B: BIVV009 7.5 g |
|---|---|---|---|---|
| IridocyclitisEye disorders | 0/17 | 0/7 | 0/16 | 1/6 |
| Retinal DetachmentEye disorders | 0/17 | 0/7 | 0/16 | 1/6 |
| UveitisEye disorders | 0/17 | 0/7 | 0/16 | 1/6 |
| Vitreous HaemorrhageEye disorders | 0/17 | 1/7 | 0/16 | 1/6 |
| Abdominal Pain UpperGastrointestinal disorders | 0/17 | 0/7 | 0/16 | 1/6 |
| Asymptomatic Covid-19Infections and infestations | 0/17 | 0/7 | 0/16 | 1/6 |
| Covid-19 PneumoniaInfections and infestations | 0/17 | 0/7 | 0/16 | 1/6 |
| ErysipelasInfections and infestations | 0/17 | 0/7 | 0/16 | 1/6 |
| Pneumococcal SepsisInfections and infestations | 0/17 | 0/7 | 0/16 | 1/6 |
| Gastrointestinal HaemorrhageGastrointestinal disorders | 0/17 | 1/7 | 0/16 | 0/6 |
| Event | Part A: BIVV009 6.5 g | Part A: BIVV009 7.5 g | Part B: BIVV009 6.5 g | Part B: BIVV009 7.5 g |
|---|---|---|---|---|
| DiarrhoeaGastrointestinal disorders | 1/17 | 1/7 | 2/16 | 3/6 |
| FatigueGeneral disorders | 1/17 | 0/7 | 3/16 | 3/6 |
| AnaemiaBlood and lymphatic system disorders | 0/17 | 1/7 | 5/16 | 2/6 |
| NauseaGastrointestinal disorders | 1/17 | 0/7 | 3/16 | 2/6 |
| PyrexiaGeneral disorders | 0/17 | 1/7 | 3/16 | 2/6 |
| Urinary Tract InfectionInfections and infestations | 1/17 | 0/7 | 3/16 | 2/6 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 1/17 | 0/7 | 2/16 | 2/6 |
| HeadacheNervous system disorders | 1/17 | 1/7 | 2/16 | 2/6 |
| DyspnoeaRespiratory, thoracic and mediastinal disorders | 0/17 | 0/7 | 1/16 | 2/6 |
| Skin LesionSkin and subcutaneous tissue disorders | 0/17 | 0/7 | 0/16 | 2/6 |
Analysis was performed on all enrolled participants.
| Age, Continuous(years) | BIVV009 6.5 g | BIVV009 7.5 g | Total |
|---|---|---|---|
| Mean | 71.8 ± 9.05 | 70.1 ± 6.01 | 71.3 ± 8.18 |
| Sex: Female, Male(Participants) | BIVV009 6.5 g | BIVV009 7.5 g | Total |
|---|---|---|---|
| Female | 11 | 4 | 15 |
| Male | 6 | 3 | 9 |
| Race (NIH/OMB)(Participants) | BIVV009 6.5 g | BIVV009 7.5 g | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 3 | 0 | 3 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 |
| White | 2 | 1 | 3 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 12 | 6 | 18 |
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 Oct 2021, 43 months.
Results were first posted in Oct 2022, 1 year after primary completion.
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Studies of the drug tested here: Sutimlimab
Anemia, Hemolytic, Autoimmune →
Bioverativ, a Sanofi company →