A PHASE 3, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO ASSESS THE EFFICACY AND SAFETY OF BIVV009 IN PATIENTS WITH PRIMARY COLD AGGLUTININ DISEASE WITHOUT A RECENT HISTORY OF BLOOD TRANSFUSIO
- Conditions
- PRIMARY COLD AGGLUTININ DISEASE1001891110003816
- Registration Number
- NL-OMON55483
- Lead Sponsor
- Bioverativ USA Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 6
* 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
* 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
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Part A: Percentage of Participants With Response (R). A participant will be<br /><br>considered a responder if he or she did not receive a blood transfusion from<br /><br>Week 5 through Week 26 (EOT) and did not receive treatment for primary cold<br /><br>agglutinin disease (CAD) beyond what ispermitted per protocol. Additionally,<br /><br>the participant's hemoglobin (Hgb) level must meet the following criterion: Hgb<br /><br>increase greater than or equal to (>=) 1.5 gram per deciliter (g/dL) from<br /><br>baseline (defined as the last Hgb value before administration of the first dose<br /><br>of study drug).<br /><br><br /><br>Part B: Number of Participants With Treatment-emergent Adverse Events (AEs) and<br /><br>Serious AEs (SAEs). An adverse event (AE) was any untoward medical occurrence<br /><br>in a participant who received study drug without regard to possibility of<br /><br>causal relationship.</p><br>
- Secondary Outcome Measures
Name Time Method