A Phase 1/2, Open-Label Study to Evaluate the Safety and Efficacy of Dibotatug in Adults With Bone Marrow Failure Syndromes
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Summary
This is a multicenter, open-label, Phase 1/2 basket study to evaluate the safety and efficacy of Dibotatug (DR-01) in adults with Bone Marrow Failure syndromes.
Description
Study DR-01-HEM-001 is an open-label Phase 1/2 study evaluating the safety and efficacy of dibotatug in adult patients with BMF syndromes. Participants will be enrolled across three cohorts. Cohort A includes relapsed Severe Aplastic Anemia participants, Cohort B includes refractory Severe Aplastic Anemia participants, and Cohort C includes relapsed or refractory transfusion dependent Nonsevere Aplastic Anemia participants. Stage 1 will evaluate the safety and preliminary efficacy in up to 12 evaluable participants, and if deemed appropriate by a Safety Review Committee, the study will continu…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: All participants must meet all of the following criteria to be included in the study: 1\. Age ≥ 18 years old 2-3. Women of childbearing potential and males must agree to use 2 methods of effective contraception, with at least 1 method being highly effective. Inclusion Criteria for Severe Aplastic Anemia (SAA) (Cohorts A and B) 4. Participants with SAA must have a current or prior diagnosis of SAA or very SAA. Inclusion Criteria for Refractory Severe Aplastic Anemia (Cohort A) Participants with refractory SAA must have: 5\. Received one ≥ 3-month course of ATG and/or CS…