A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy and Safety of Sutimlimab in Patients With Primary Cold Agglutinin Disease Without a Recent History of Blood Transfusion

EFC16216

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.

2018-03-17

2021-12-03

2021-12-03

Completed

Early phase I

42

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.

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2022-12-23