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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)

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ClinicalTrials.gov Identifier: NCT03347396
Recruitment Status : Completed
First Posted : November 20, 2017
Last Update Posted : October 5, 2021
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Bioverativ, a Sanofi company )

Tracking Information
First Submitted Date  ICMJE November 16, 2017
First Posted Date  ICMJE November 20, 2017
Last Update Posted Date October 5, 2021
Actual Study Start Date  ICMJE March 5, 2018
Actual Primary Completion Date September 15, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 29, 2018)
  • Part A: Percentage of Participants With Response (R) [ Time Frame: Up to Week 26 ]
    A participant who meets all of the following criteria will be considered a responder: who did not receive a blood transfusion from Week 5 through Week 26 (end of treatment) and did not receive treatment for cold agglutinin disease (CAD) beyond what is permitted per protocol. Additionally the participant's hemoglobin (Hgb) level must meet either of the following criteria: Hgb level greater than or equal to (>=) 12 gram per deciliter (g/dL) at the treatment assessment endpoint, or Hgb increased >= 2 g/dL from baseline (defined as the last Hgb value before administration of the first dose of study drug) at treatment assessment endpoint.
  • Part B: Number of Participants With Treatment-emergent Adverse Events (AEs) and Serious AEs (SAEs) [ Time Frame: Approximately 1 year ]
    An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship.
Original Primary Outcome Measures  ICMJE
 (submitted: November 16, 2017)
  • Part A: Percentage of Participants With Response (R) [ Time Frame: Up to Week 26 ]
    A participant who meets all of the following criteria will be considered a responder: who did not receive a blood transfusion from Week 5 through Week 26 (end of treatment) and did not receive treatment for cold agglutinin disease (CAgD) beyond what is permitted per protocol. Additionally the participant's hemoglobin (Hgb) level must meet either of the following criteria: Hgb level greater than or equal to (>=) 12 gram per deciliter (g/dL) at the treatment assessment endpoint, or Hgb increased >= 2 g/dL from baseline (defined as the last Hgb value before administration of the first dose of study drug) at treatment assessment endpoint.
  • Part B: Number of Participants With Treatment-emergent Adverse Events (AEs) and Serious AEs (SAEs) [ Time Frame: Approximately 1 year ]
    An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: November 16, 2017)
  • Part A: Mean Change From Baseline in Bilirubin up to Week 26 [ Time Frame: Baseline up to Week 26 ]
    Mean change from baseline in bilirubin up to Week 26 will be assessed.
  • 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 ]
    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.
  • Part A: Mean Change From Baseline in Lactate Dehydrogenase (LDH) up to Week 26 [ Time Frame: Baseline up to Week 26 ]
    Mean change from baseline in LDH up to Week 26 will be assessed.
  • Part A: Number of Blood Transfusions After the First 5 Weeks of Study Drug Administration [ Time Frame: 5 Weeks ]
    Number of transfusions after the first 5 weeks of study drug administration will be assessed.
  • Part A: Number of Blood Units Transfused After the First 5 Weeks of Study Drug Administration [ Time Frame: 5 Weeks ]
    Number of blood units transfused after the first 5 weeks of study drug administration will be assessed.
  • Part A: Mean Change From Baseline in Hemoglobin (Hgb) Level up to Week 26 [ Time Frame: Baseline up to Week 26 ]
    Mean change from baseline in Hgb level up to Week 26 will be assessed.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE 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)
Official Title  ICMJE 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
Brief Summary The purpose of Part A is to determine whether sutimlimab administration results in a greater than or equal to (>=) 2 gram per deciliter (g/dL) increase in hemoglobin (Hgb) levels or increases Hgb to >= 12 g/dL and obviates the need for blood transfusion during treatment in participants with primary cold agglutinin disease (CAD) who have 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 CAD.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Agglutinin Disease, Cold
Intervention  ICMJE Drug: Sutimlimab
Sutimlimab will be administered as IV infusion.
Study Arms  ICMJE Experimental: Sutimlimab
Participants will receive an intravenous (IV) infusion of sutimlimab. Participants who complete Part A per protocol through the end of treatment visit (Day 182) will participate in Part B, and continue to receive sutimlimab up to 1 year after last patient out (LPO) in Part A.
Intervention: Drug: Sutimlimab
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: November 16, 2017)
20
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE September 15, 2021
Actual Primary Completion Date September 15, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   Austria,   Belgium,   Canada,   France,   Germany,   Israel,   Italy,   Japan,   Netherlands,   Norway,   Spain,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03347396
Other Study ID Numbers  ICMJE EFC16215
BIVV009-03 ( Other Identifier: Bioverativ Therapeutics Inc. )
2017-003538-10 ( EudraCT Number )
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Yes
Plan Description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://www.clinicalstudydatarequest.com/
Responsible Party Sanofi ( Bioverativ, a Sanofi company )
Study Sponsor  ICMJE Bioverativ, a Sanofi company
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Clinical Sciences & Operations Sanofi
PRS Account Sanofi
Verification Date October 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP