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)

• Status: Completed
• Trial end date: 2021-09-15
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of Part A is to determine whether sutimlimab administration results in a greater than or equal to (>=) 2 gram per deciliter (g/dL) increase in hemoglobin (Hgb) levels or increases Hgb to >= 12 g/dL and obviates the need for blood transfusion during treatment in participants with primary cold agglutinin disease (CAD) who have a recent history of blood transfusion.The purpose of Part B is to evaluate the long-term safety and tolerability of sutimlimab in participants with CAD.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Bioverativ Therapeutics Inc.; Bioverativ, a Sanofi company
• Treatments: Agglutinins; Cold agglutinins

Criteria

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