A Study to Assess the Efficacy and Safety of BIVV009 (Sutimlimab) in Participants With Primary Cold Agglutinin Disease Who Have a Recent History of Blood Transfusion (Cardinal Study)
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ClinicalTrials.gov Identifier: NCT03347396 |
Recruitment Status :
Completed
First Posted : November 20, 2017
Last Update Posted : April 25, 2022
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Condition or disease | Intervention/treatment | Phase |
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Agglutinin Disease, Cold | Drug: Sutimlimab | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 20 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Phase 3, Pivotal, Open-label, Multicenter Study to Assess the Efficacy and Safety of Sutimlimab in Patients With Primary Cold Agglutinin Disease Who Have a Recent History of Blood Transfusion |
Actual Study Start Date : | March 5, 2018 |
Actual Primary Completion Date : | September 15, 2021 |
Actual Study Completion Date : | September 15, 2021 |

Arm | Intervention/treatment |
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Experimental: Sutimlimab
Participants will receive an intravenous (IV) infusion of sutimlimab. Participants who complete Part A per protocol through the end of treatment visit (Day 182) will participate in Part B, and continue to receive sutimlimab up to 1 year after last patient out (LPO) in Part A.
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Drug: Sutimlimab
Sutimlimab will be administered as IV infusion. |
- Part A: Percentage of Participants With Response (R) [ Time Frame: Up to Week 26 ]A participant who meets all of the following criteria will be considered a responder: who did not receive a blood transfusion from Week 5 through Week 26 (end of treatment) and did not receive treatment for cold agglutinin disease (CAD) beyond what is permitted per protocol. Additionally the participant's hemoglobin (Hgb) level must meet either of the following criteria: Hgb level greater than or equal to (>=) 12 gram per deciliter (g/dL) at the treatment assessment endpoint, 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.
- Part B: Number of Participants With Treatment-emergent Adverse Events (AEs) and Serious AEs (SAEs) [ Time Frame: Approximately 1 year ]An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship.
- Part A: Mean Change From Baseline in Bilirubin up to Week 26 [ Time Frame: Baseline up to Week 26 ]Mean change from baseline in bilirubin up to Week 26 will be assessed.
- Part A: Mean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score (Quality of Life) [ Time Frame: Baseline up to Week 26 ]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 are added to obtain a total score. The range of possible scores is 0-52, with higher score indicating more fatigue.
- Part A: Mean Change From Baseline in Lactate Dehydrogenase (LDH) up to Week 26 [ Time Frame: Baseline up to Week 26 ]Mean change from baseline in LDH up to Week 26 will be assessed.
- Part A: Number of Blood Transfusions After the First 5 Weeks of Study Drug Administration [ Time Frame: 5 Weeks ]Number of transfusions after the first 5 weeks of study drug administration will be assessed.
- Part A: Number of Blood Units Transfused After the First 5 Weeks of Study Drug Administration [ Time Frame: 5 Weeks ]Number of blood units transfused after the first 5 weeks of study drug administration will be assessed.
- Part A: Mean Change From Baseline in Hemoglobin (Hgb) Level up to Week 26 [ Time Frame: Baseline up to Week 26 ]Mean change from baseline in Hgb level up to Week 26 will be assessed.

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Body weight of greater than or equal to (>=) 39 kilogram (kg) at Screening
- Confirmed diagnosis of primary cold agglutinin disease (CAD) based on the following criteria: a) Chronic hemolysis; b) Polyspecific direct antiglobulin test (DAT) positive; c) Monospecific DAT strongly positive for C3d; d) Cold agglutinin titer >= 64 at 4 degree celsius; e) Immunoglobulin G (IgG) DAT less than or equal to (<=) 1+, and f) No overt malignant disease
- History of at least one documented blood transfusion within 6 months of enrollment
- Hemoglobin level <= 10.0 gram per deciliter (g/dL)
- Bilirubin level above the normal reference range, including patients with Gilbert's Syndrome
Exclusion Criteria:
- Cold agglutinin syndrome secondary to infection, rheumatologic disease, or active hematologic malignancy
- Clinically relevant infection of any kind within the month preceding enrollment (eg, active hepatitis C, pneumonia)
- Clinical diagnosis of systemic lupus erythematosus (SLE); or other autoimmune disorders with anti-nuclear antibodies at Screening. Anti-nuclear antibodies of long-standing duration without associated clinical symptoms will be adjudicated on a case-by-case basis during the Confirmatory Review of Patient Eligibility
- Positive hepatitis panel (including hepatitis B surface antigen and/or hepatitis C virus antibody) prior to or at Screening
- Positive human immunodeficiency virus (HIV) antibody at Screening
- Treatment with rituximab monotherapy within 3 months or rituximab combination therapies (eg, with bendamustine, fludarabine, ibrutinib, or cytotoxic drugs) within 6 months prior to enrollment

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03347396

Study Director: | Clinical Sciences & Operations | Sanofi |
Publications of Results:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | Bioverativ, a Sanofi company |
ClinicalTrials.gov Identifier: | NCT03347396 |
Other Study ID Numbers: |
EFC16215 BIVV009-03 ( Other Identifier: Bioverativ Therapeutics Inc. ) 2017-003538-10 ( EudraCT Number ) |
First Posted: | November 20, 2017 Key Record Dates |
Last Update Posted: | April 25, 2022 |
Last Verified: | April 2022 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Yes |
Plan Description: | 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 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Anemia, Hemolytic, Autoimmune Anemia, Hemolytic Anemia |
Hematologic Diseases Autoimmune Diseases Immune System Diseases |