A Study to Assess the Efficacy and Safety of BIVV009 (Sutimlimab) in Participants With Primary Cold Agglutinin Disease Without A Recent History of Blood Transfusion

• Register: ClinicalTrials.gov
• Last refreshed on: 2 May 2022
• Main ID: NCT03347422
• 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 Without A Recent History of Blood Transfusion Cadenza
• Scientific title: 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
• Date of first enrolment: March 17, 2018
• Target sample size: 42
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03347422
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
• 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, 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

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. 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 (example, with bendamustine, fludarabine, ibrutinib, or cytotoxic drugs)
within 6 months prior to enrollment

The above information is not intended to contain all considerations relevant to a patient's
potential participation in a clinical trial.



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

Health Condition(s) or Problem(s) studied

Cold Agglutinin Disease

Intervention(s)

Drug: placebo

Drug: sutimlimab (BIVV009)

Primary Outcome(s)

Part B: Number of Participants With Treatment-emergent Adverse Events (AEs) and Serious AEs (SAEs) [Time Frame: Approximately 1 year from end of Week 26 of Part A]

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

Secondary Outcome(s)

Part B: Mean Change From Week 27 in Lactate Dehydrogenase (LDH) level [Time Frame: From Week 27 (Part B baseline) up to Week 77]

Part B: Number of transfusions [Time Frame: From Week 27 (Part B baseline) up to Week 77]

Part B: Patient's Global Impression of [Fatigue] Severity (PGIS) total score in different timepoints [Time Frame: From Week 27 (Part B baseline) up to Week 77]

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

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 Lactate Dehydrogenase (LDH) up to Week 26 [Time Frame: Baseline up to Week 26]

Part A: Percentage of Participants With Solicited Symptomatic Anemia at End of Treatment (EOT) [Time Frame: At Part A EOT (Day 182)]

Part B: Mean Change From Week 27 in 12-item short form survey (SF-12) [Time Frame: From Week 27 (Part B baseline) up to Week 77]

Part B: Mean Change From Week 27 in five level EuroQol five dimensions questionnaire (EQ-5D-5L) [Time Frame: From Week 27 (Part B baseline) up to Week 77]

Part B: Patient's Global Impression of Change (PGIC) total score in different timepoints [Time Frame: From Week 27 (Part B baseline) up to Week 77]

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]

Part B: Mean Change From Week 27 in Haptoglobin [Time Frame: From Week 27 (Part B baseline) up to Week 77]

Part B: Mean Change From Week 27 in Hemoglobin (Hgb) Level [Time Frame: From Week 27 (Part B baseline) up to Week 77]

Part B: Mean Change From Week 27 in Bilirubin (total) [Time Frame: From Week 27 (Part B baseline) up to Week 77]

Part B: Mean Change From Week 27 in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score (Quality of Life) [Time Frame: From Week 27 (Part B baseline) up to Week 77]

Part B: Number of healthcare visits by type [Time Frame: Approximately 1 year from end of Week 26 of Part A]

Secondary ID(s)

2017-003539-12

BIVV009-04

EFC16216

Source(s) of Monetary Support

Please refer to primary and secondary sponsors