Primary Cold Agglutinin Disease MedDRA version: 20.0 Level: PT Classification code 10073785 Term: Autoimmune haemolytic anaemia System Organ Class: 10005329 - Blood and lymphatic system disorders
Conditions
Sponsors
Bioverativ USA Inc.
Eligibility
Sex/Gender
All
Inclusion 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 for C3d, d) Cold agglutinin titer >= 64 at 4 degree Celsius, and e) Immunoglobulin G (IgG) DAT less than or equal to (=65 years) yes F.1.3.1 Number of subjects for this age range 36
Exclusion criteria
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The purpose of Part A is to determine whether sutimlimab administration results in a greater than or equal to (>=)1.5 gram 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 is to evaluate the long-term safety and tolerability of sutimlimab in participants with primary CAD. ;Secondary Objective: Part A: To assess the effect of BIVV009 on clinical events and laboratory parameters related to hemolysis and anemia in patients with primary CAgD. To assess the effect of BIVV009 on specific complications of CAgD (acrocyanosis, Raynaud's syndrome, hemoglobinuria, and thromboembolism) To assess the effect of BIVV009 on quality of life (QOL) in patients with primary CAgD. Part B: The secondary objective of Part B is to investigate the durability of response during long-term treatment with BIVV009 in patients with primary CAgD.;Primary end point(s): Part A: Percentage of Participants With Response (R). A participant will be considered a responder if he or she did not receive a blood transfusion from Week 5 through Week 26 (EOT) and did not receive treatment for primary cold agglutinin disease (CAD) beyond what is permitted per protocol. Additionally, the participant's hemoglobin (Hgb) level must meet the following criterion: Hgb increase greater than or equal to (>=) 1.5 gram per deciliter (g/dL) from baseline (defined as the last Hgb value before administration of the first dose of study drug. Part B: Number of Participants With Treatment-emergent Adverse Events (AEs) and Serious AEs (SAEs). An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship.;Timepoint(s) of evaluation of this end point: Part A: Up to Week 26 Part B: Approximately 1 year from end of Week 26 of Part A | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Part A: 1. Mean Change From Baseline in Hemoglobin (Hgb) Level up to Week 26. 2. Mean Change From Baseline in Bilirubin up to Week 26. 3. Mean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score (Quality of Life). FACITFatigue scale consists of 13 questions assessed using a 5 point scale (0=not at all; 1 = a little bit, 2 = somewhat, 3 = quite a bit and 4 = very much). Responses to each question are added to obtain a total score. The range of possible scores is 0-52, with higher score indicating more fatigue. 4. Mean Change From Baseline in Lactate Dehydrogenase (LDH) up to Week 26. 5. Percentage of Participants With Solicited Symptomatic Anemia at End of Treatment (EOT). Symptomatic anemia is defined as fatigue, weakness, shortness of breath, palpitations, fast heart beat, light headedness, and/or chest pain. Part B: 6. Mean Change From Week 27 in Hemoglobin (Hgb) Level. 7. Mean Change From Week 27 in Bilirubin (total). 8. Mean Change From Week 27 in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score (Quality of Life). FACIT Fatigue scale consists of 13 questions assessed using a 5 point scale (0=not at all; 1 = a little bit, 2 = somewhat, 3 = quite a bit and 4 = very much). Responses to each question are added to obtain a total score. The range of possible scores is 0-52, with higher score indicating more fatigue. 9. Mean Change From Week 27 in five level EuroQol five dimensions questionnaire (EQ-5D-5L). The EQ-5D descriptive system comprises 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has a 5-level response: no problems, slight problems, moderate problems, severe problems, and extreme problems. A scale with score 0-100 is used to collect response on current health status. Higher the score better the health. 10. Mean Change From Week 27 in 12-item short form survey (SF-12). SF-12 health sur | — |
Countries
Australia, Austria, Belgium, Canada, France, Germany, Israel, Italy, Japan, Netherlands, New Zealand, Norway, Spain, United Kingdom, United States
Contacts
Public ContactClinical Trial Dept
Pharm-Olam, LLC
Outcome results
None listed