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)

• Register: ClinicalTrials.gov
• Last refreshed on: 27 June 2022
• Main ID: NCT03347396
• Date of registration: 16/11/2017
• Prospective Registration: Yes
• Primary sponsor: Bioverativ, a Sanofi company
• Public title: 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)
• Scientific title: A Phase 3, Pivotal, Open-label, Multicenter Study to Assess the Efficacy and Safety of Sutimlimab in Patients With Primary Cold Agglutinin Disease Who Have a Recent History of Blood Transfusion
• Date of first enrolment: March 5, 2018
• Target sample size: 20
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03347396
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Australia, Austria, Belgium, Canada, France, Germany, Israel, Italy, Japan, Netherlands, Norway, Spain, United Kingdom, United States

Contacts

• Name: Clinical Sciences & Operations
• Affiliation: Sanofi

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Agglutinin Disease, Cold

Intervention(s)

Drug: Sutimlimab

Primary Outcome(s)

Part A: Percentage of Participants With Response (R) [Time Frame: Up to Week 26]

Part B: Number of Participants With Treatment-emergent Adverse Events (AEs) and Serious AEs (SAEs) [Time Frame: Approximately 1 year]

Secondary Outcome(s)

Part A: Mean Change From Baseline in Bilirubin up to Week 26 [Time Frame: Baseline up to Week 26]

Part A: Mean Change From Baseline in Hemoglobin (Hgb) Level up to Week 26 [Time Frame: Baseline up to Week 26]

Part A: Number of Blood Units Transfused After the First 5 Weeks of Study Drug Administration [Time Frame: 5 Weeks]

Part A: Mean Change From Baseline in Lactate Dehydrogenase (LDH) up to Week 26 [Time Frame: Baseline up to Week 26]

Part A: Number of Blood Transfusions After the First 5 Weeks of Study Drug Administration [Time Frame: 5 Weeks]

Part A: Mean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score (Quality of Life) [Time Frame: Baseline up to Week 26]

Secondary ID(s)

BIVV009-03

EFC16215

2017-003538-10

Source(s) of Monetary Support

Please refer to primary and secondary sponsors