Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
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ClinicalTrials.gov |
Last refreshed on:
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6 January 2025 |
Main ID: |
NCT05116787 |
Date of registration:
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02/11/2021 |
Prospective Registration:
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No |
Primary sponsor: |
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Public title:
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BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants Not Receiving Other Complement Inhibitor Therapy
REDEEM-2 |
Scientific title:
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A Randomized, Double-Blind, Multicenter, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria |
Date of first enrolment:
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October 26, 2021 |
Target sample size:
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12 |
Recruitment status: |
Terminated |
URL:
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https://clinicaltrials.gov/ct2/show/NCT05116787 |
Study type:
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Interventional |
Study design:
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Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
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Phase:
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Phase 2
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Countries of recruitment
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Korea, Republic of
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Malaysia
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South Africa
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Spain
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Taiwan
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United States
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Contacts
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Name:
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David J Kuter, MD, DPhil |
Address:
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Telephone:
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Email:
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Affiliation:
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Massachusetts General Hospital |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Male or female, aged = 18 years old
- Body weight = 40 kg
- Documented diagnosis of PNH
- No complement inhibitor therapy for = 12 months prior to screening
- Contraindication to or no access to approved (C3 or C5) complement inhibitor
therapies
- Documentation of current vaccinations against Neisseria meningitidis and
Streptococcus pneumoniae or willingness to start vaccination series
- At screening: PNH clone of = 10%, hemoglobin = 10.5 g/dL and lactate dehydrogenase =
2 × upper limit of normal
Exclusion Criteria:
- Known history of or existing diagnosis of hereditary complement deficiency
- History of hematopoietic cell transplant or solid organ transplant or anticipated
candidate for transplantation
- Myocardial infarction or cerebrovascular accident within 30 days prior to screening,
or current and uncontrolled clinically significant cardiovascular or cerebrovascular
condition
- History of malignancy within 5 years prior to the screening visit
- Active bacterial, viral, or fungal infection or any other serious infection within
14 days prior to screening
- Treatment with anti-thymocyte globulin within 180 days prior to screening
- Initiation of treatment with an erythrocyte or platelet growth factor, or danazol
within 28 days prior to screening
- Receiving iron supplementation with an unstable dose in the 28 days prior to
screening
Age minimum:
18 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Paroxysmal Nocturnal Hemoglobinuria (PNH)
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Intervention(s)
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Drug: BCX9930 monotherapy
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Drug: Placebo
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Primary Outcome(s)
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Part 1: Change From Baseline in Hemoglobin at Week 12
[Time Frame: Baseline, Week 12]
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Secondary Outcome(s)
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Part 1: Change From Baseline in Aspartate Aminotransferase (AST)
[Time Frame: Baseline, Week 12]
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Part 1: Change From Baseline in Absolute Reticulocyte Count (ARC)
[Time Frame: Baseline, Week 12]
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Part 1: Change From Baseline in Haptoglobin
[Time Frame: Baseline, Week 12]
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Part 1: Number of Participants Who Were Transfusion-free
[Time Frame: From Week 4 to Week 12]
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Part 1: Number of Participants With ARC in the Normal Range
[Time Frame: Week 12]
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Part 1: Percentage (%) Reduction in the Rate of pRBC Units Transfused
[Time Frame: Prestudy (12 months prior to screening) and from Week 4 to Week 12]
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Part 1: Change From Baseline in Ratio of Total PNH RBC Clone Size to PNH White Blood Cell (WBC) Clone Size
[Time Frame: Baseline, Week 12]
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Part 1: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score
[Time Frame: Baseline, Week 12]
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Part 1: Change From Baseline in Total Bilirubin
[Time Frame: Baseline, Week 12]
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Part 1: Number of Participants With Haptoglobin = Lower Limit of Normal (LLN) Reference Range
[Time Frame: Week 12]
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Part 1: Number of Units of pRBCs Transfused
[Time Frame: From Week 4 to Week 12]
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Part 1: Change From Baseline in Total Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Clone Size
[Time Frame: Baseline, Week 12]
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Part 1: Number of Participants Achieving Hemoglobin Stabilization
[Time Frame: From Week 4 to Week 12]
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Part 1: Percent Change From Baseline in Lactate Dehydrogenase
[Time Frame: Baseline, Week 12]
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Part 1: Number of Participants With Hemoglobin = 12 g/dL
[Time Frame: At Week 12]
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Secondary ID(s)
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2020-004403-14
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BCX9930-203
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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