A Study to Assess the Efficacy and Safety of BIVV009 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 : March 22, 2018
|Condition or disease||Intervention/treatment||Phase|
|Agglutinin Disease, Cold||Drug: BIVV009 Drug: Placebo||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||40 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Double (Participant, Investigator)|
|Official Title:||A Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy and Safety of BIVV009 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|
Experimental: Part A: BIVV009 or Placebo
In Part A, participants will be randomized 1:1 to receive an intravenous (IV) infusion of BIVV009 or placebo.
BIVV009 will be administered by IV.Drug: Placebo
Placebo will be administered by IV.
Experimental: Part B: Response Extension Phase (BIVV009)
In Part B, all participants will undergo blinded cross-over loading doses to allow all participants to receive BIVV009 while maintaining Part A blinding.
BIVV009 will be administered by IV.
- 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 (CAgD) 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.
- 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.
- 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.
- 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: 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.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03347422
|Contact: Bioverativ Therapeutics Inc, Waltham, MA, USA||1-844-308-0808(US only)||firstname.lastname@example.org|
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