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.
Age ≥ 18 years old
Women of childbearing potential and males must agree to use 2 methods of effective contraception, with at least 1 method being highly effective.
Participants with SAA must have a current or prior diagnosis of SAA or very SAA.
Received one ≥ 3-month course of ATG and/or CSA-based IST.
Diagnosis of Fanconi anemia, dyskeratosis congenita, or other congenital BMF syndrome.
Prior HCT.
Planning to receive HCT as treatment for AA.
Evidence of a clonal disorder with poor risk cytogenetics per Revised International Prognostic Scoring System (IPSS-R) for MDS.
Single group
Dibotatug (DR-01)
DrugDibotatug (DR-01) administered by IV infusion
Overall Response Rate of Dibotatug (defined as proportion of subjects with a Complete Response or Partial Response) [Efficacy]
Overall Response Rate (ORR), defined as the proportion of subjects with Complete Response (CR) or Partial Response (PR) based on disease-specific response criteria.
Incidence and severity of adverse events as assessed by CTCAE v6.0 [Safety and Tolerability]
Incidence and severity of adverse events as assessed by CTCAE v6.0.
This trial is not recruiting at the moment. You can still explore other options:
