A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Ma… (NCT07712562) | Clinical Trial Compass
Not Yet RecruitingPhase 1/2
A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure Syndromes
United States60 participantsStarted 2026-08
Plain-language 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.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* 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.
Inclusion Criteria for Severe Aplastic Anemia (SAA) (Cohorts A and B):
* 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:
* Received one ≥ 3-month course of ATG and/or CSA-based IST.
* Refractory SAA, defined as failure to achieve CR or PR ≥ 3 months after starting ATG and/or CSA-based IST.
Inclusion Criteria for Relapsed Severe Aplastic Anemia (Cohort B):
* Relapsed SAA, defined as relapse following a CR or PR that was achieved ≥ 3 months after starting ATG- and/or cyclosporine A (CSA)-based IST.
Inclusion Criteria for Relapsed or Refractory Transfusion-Dependent Non-Severe Aplastic Anemia (Cohort C):
* Current or prior diagnosis of NSAA
* No current or prior diagnosis of SAA.
* Received at least 1 prior course of IST such as ATG- or CSA, with or without a TPO-R agonist (lasting ≥ 3 months).
* Meets criteria for transfusion dependence (either RBC or platelet):
* RBC transfusion dependence: transfusion of ≥ 2 units of RBCs in the past 56 days
* Platelet transfusion dependence: transfusion of ≥ 1 unit of apheresis platelets in the past 28 days
Participants entering the Extension Treatment Period must meet the following criteria:
* Signed in…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Overall Response Rate of Dibotatug (defined as proportion of subjects with a Complete Response or Partial Response) [Efficacy]
Timeframe: By 6 months
2
Incidence and severity of adverse events as assessed by CTCAE v6.0 [Safety and Tolerability]