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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 Study)

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ClinicalTrials.gov Identifier: NCT03347422
Recruitment Status : Recruiting
First Posted : November 20, 2017
Last Update Posted : November 7, 2018
Sponsor:
Information provided by (Responsible Party):
Bioverativ Therapeutics Inc.

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Agglutinin Disease, Cold Drug: Sutimlimab Drug: Placebo Phase 3

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : November 20, 2017
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2020

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Part A: sutimlimab or Placebo
In Part A, participants will be randomized 1:1 to receive an intravenous (IV) infusion of sutimlimab or placebo.
Drug: Sutimlimab
Sutimlimab will be administered by IV.

Drug: Placebo
Placebo will be administered by IV.

Experimental: Part B: Response Extension Phase (sutimlimab)
In Part B, all participants will undergo blinded cross-over loading doses to allow all participants to receive sutimlimab while maintaining Part A blinding.
Drug: Sutimlimab
Sutimlimab will be administered by IV.




Primary Outcome Measures :
  1. Part A: Percentage of Participants With Response (R) [ Time Frame: Up to Week 26 ]
    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) at treatment assessment endpoint.

  2. 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.


Secondary Outcome Measures :
  1. 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 hemoglobin (Hgb) level up to Week 26 will be assessed.

  2. 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.

  3. 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.

  4. 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.

  5. Part A: Percentage of Participants With Solicited Symptomatic Anemia at End of Treatment (EOT) [ Time Frame: At EOT (Day 182) ]
    Symptomatic anemia is defined as fatigue, weakness, shortness of breath, palpitations, fast heart beat, light headedness, and/or chest pain.



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03347422


Contacts
Contact: Bioverativ Therapeutics Inc, Waltham, MA, USA 1-844-308-0808(US only) clinicaltrials@bioverativ.com

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Sponsors and Collaborators
Bioverativ Therapeutics Inc.

Responsible Party: Bioverativ Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT03347422     History of Changes
Other Study ID Numbers: BIVV009-04
2017-003539-12 ( EudraCT Number )
BIVV009-04 ( Other Identifier: Bioverativ Therapeutics Inc. )
First Posted: November 20, 2017    Key Record Dates
Last Update Posted: November 7, 2018
Last Verified: November 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Anemia, Hemolytic, Autoimmune
Anemia, Hemolytic
Anemia
Hematologic Diseases
Autoimmune Diseases
Immune System Diseases
Agglutinins
Cold agglutinins
Immunologic Factors
Physiological Effects of Drugs
Hemagglutinins