A Study to Assess the Efficacy and Safety of BIVV009 (Sutimlimab) in Participants With Primary Cold Agglutinin Disease Without A Recent History of Blood Transfusion

Purpose

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.

Condition

  • Cold Agglutinin Disease

Eligibility

Eligible Ages
Over 18 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Body weight of >=39 kg at screening. - Confirmed diagnosis of primary 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, and e) Immunoglobulin G DAT less than or equal to (<=) 1+, and, f) No overt malignant disease. - Hemoglobin level <= 10.0 g/dL. - Bilirubin level above the normal reference range, including participants with Gilbert's Syndrome.

Exclusion Criteria

  • Cold agglutinin syndrome secondary to infection, rheumatologic disease, or active hematologic malignancy. - History of blood transfusion within 6 months of screening, or history of more than one blood transfusion within 12 months of screening. - Clinically relevant infection of any kind within the month preceding enrollment (example, active hepatitis C, pneumonia). - Clinical diagnosis of systemic lupus erythematosus; or other autoimmune disorders with anti-nuclear antibodies at screening. Anti-nuclear antibodies of long-standing duration without associated clinical symptoms would be adjudicated on a case-by-case basis during the confirmatory review of participant eligibility. - Positive hepatitis panel (including hepatitis B surface antigen and/or hepatitis C virus antibody) prior to or at screening. - Positive human immunodeficiency virus antibody at screening. - Treatment with rituximab monotherapy within 3 months or rituximab combination therapies (example, with bendamustine, fludarabine, ibrutinib, or cytotoxic drugs) within 6 months prior to enrollment. The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Double (Participant, Investigator)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
BIVV009/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 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)
    Pharmaceutical form: solution for injection Route of administration: intravenous (i.v.)
Experimental
Placebo/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)
    Pharmaceutical form: solution for injection Route of administration: intravenous (i.v.)
  • Drug: placebo
    Pharmaceutical form: solution for injection Route of administration: intravenous (i.v.)

More Details

Status
Completed
Sponsor
Bioverativ, a Sanofi company

Study Contact

Detailed Description

The planned total study duration per participant was approximately 1.5 to 2.5 years.