A study to determine the safety and effectiveness of study drug BIVV009 in patients with Primary Agglutinin Disease without recent history of blood transfusions

Phase 1

Active, not recruiting

• Conditions: Primary Cold Agglutinin Disease; Therapeutic area: Diseases [C] - Immune System Diseases [C20]; MedDRA version: 20.0Level: PTClassification code 10073785Term: Autoimmune haemolytic anaemiaSystem Organ Class: 10005329 - Blood and lymphatic system disorders

• Registration Number: EUCTR2017-003539-12-NL
• Lead Sponsor: Bioverativ USA Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2017-12-19
• Last Update Posted: 2 March 2022

Recruitment & Eligibility

• Status: Not Recruiting
• Sex: All
• Target Recruitment: 40

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, and e) Immunoglobulin G (IgG) DAT less than or equal to (<=) 1+, and, f) No overt malignant disease
• Hemoglobin level <= 10.0 gram per deciliter (g/dL)
• Bilirubin level above the normal reference range, including patients with Gilbert's Syndrome
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 4
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 36

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 (example, active hepatitis C, pneumonia)
• Clinical diagnosis of systemic lupus erythematosus (SLE); or other autoimmune disorders with anti-nuclear antibodies at Screening. Antinuclear
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 (example, with bendamustine, fludarabine,
ibrutinib, or cytotoxic drugs) within 6 months prior to enrollment

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified