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Phase 1/2 Study of Dibotatug (DR-01) for Bone Marrow Failure Syndromes

This study is testing a drug called Dibotatug (DR-01) in adults with bone marrow failure syndromes, including severe aplastic anemia (SAA). The goal is to see how safe Dibotatug (DR-01) is and how well it works. Researchers will look at how many participants have a complete or partial response to the treatment within 6 months. They will also track any side effects for up to 52 weeks. You may be able to join if you are 18 or older and have a current or past diagnosis of SAA, or if you have non-severe aplastic anemia that requires transfusions and has come back or not responded to previous treatments. The study plans to enroll up to 60 participants.

Study design
This is an open-label, Phase 1/2 study, meaning you and your doctors will know you are receiving Dibotatug (DR-01). It plans to enroll up to 60 participants across different groups of bone marrow failure syndromes.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
The study will track side effects for up to 52 weeks and measure treatment response for 6 months.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07712562

A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure Syndromes

Not Yet Recruiting
PHASE1Ages 18+InterventionalTreatment
Dren Bio
~60 participants
Updated 2026-07-24 on ClinicalTrials.gov
What's tested:Dibotatug (DR-01)

At a glance

Recruiting sites
0 of 10 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Overall Response Rate of Dibotatug (defined as proportion of subjects with a Complete Response or Partial Response) [Efficacy]
Measured over By 6 months
+1 more outcome measured

Conditions

Bone Marrow Failure Syndromes
Aplastic Anemias
Severe Aplastic Anemia (SAA)
Severe Aplastic Anemia, Relapse
Severe Aplastic Anemia, Refractory
Non-severe Aplastic Anemia

Explore related trials

Bone Marrow Failure SyndromeNon-Severe Aplastic AnemiaSevere Aplastic Anemia

NCT07712562

Where you'd take part

This study runs at 10 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Dren Investigational Site

    Duarte, Californiastudy coordinator listed

  • Dren Investigational Site

    Palo Alto, Californiastudy coordinator listed

  • Dren Investigational Site

    Miami, Floridastudy coordinator listed

  • Dren Investigational Site

    Atlanta, Georgiastudy coordinator listed

  • Dren Investigational Site

    New York, New Yorkstudy coordinator listed

  • Dren Investigational Site

    Cleveland, Ohiostudy coordinator listed

  • Dren Investigational Site

    Columbus, Ohiostudy coordinator listed

  • Dren Investigational Site

    Houston, Texasstudy coordinator listed

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Wan-Jen Hong, MD · STUDY_DIRECTOR · Dren Bio

Who to contact

Dren Bio Central Contact
[email protected]
415-737-5277
Email the study team

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Eligibility criteria

Inclusion

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.
Refractory SAA, defined as failure to achieve CR or PR ≥ 3 months after starting ATG and/or CSA-based IST.
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.
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
Signed informed consent form (ICF) for the Extension Treatment Period.
CR or PR by Week 24 during the Main Treatment Period

Exclusion

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.
Diagnosis of PNH or a clonal hematologic bone marrow disorder such as LGLL. Existence of PNH clones, LGLL cells, or clonal hematopoiesis of indeterminate potential (CHIP) clones without a clinical diagnosis is not exclusionary.
Use of a T-cell depleting agent (e.g., ATG, alemtuzumab, thymoglobulin) within 3 months prior to Day 1.
Use of a B-cell depleting agent (e.g., rituximab, ocrelizumab, ofatumumab, ublituximab) within 28 days prior to Day 1.
Use of any of the following within 14 days of Day 1, unless used as an established therapy at screening and there is evidence of either progressive cytopenia or lack of count improvement over the 3 months before screening:
Calcineurin inhibitor (e.g., cyclosporine), TPO-R agonist (e.g., eltrombopag), Androgen (e.g., danazol), Oral Janus kinase (JAK) inhibitor
Regardless of their use as an established therapy at screening, use of the above medications is prohibited for all participants from 3 months after Day 1.
Current infection not adequately responding to appropriate therapy or requiring hospitalization.
Current uncontrolled or invasive infection with cytomegalovirus (CMV), Epstein Barr virus (EBV), varicella zoster virus (VZV), herpes simplex virus (HSV), or human T-lymphotropic virus-1 (HTLV-1), defined by polymerase chain reaction (PCR) at screening. Note that low level CMV, EBV, or VZV viremia is not exclusionary.
Human immunodeficiency virus (HIV) infection.
Current or prior infection with hepatitis B virus (HBV)
Current hepatitis C virus (HCV) infection
Latent tuberculosis (TB) infection as indicated by IFN-γ release assay without documentation of appropriate treatment (appropriate therapy as defined by the World Health Organization \[WHO\] and/or the United States Centers for Disease Control and Prevention).
Estimated glomerular filtration rate \< 30 mL/min/1.73 m2 at screening (using the Chronic Kidney Disease Epidemiology Collaboration formula; Levey 2009).
Total bilirubin \> 1.5 × upper limit of normal (ULN) (\> 3 × ULN if known Gilbert's disease).
Aspartate aminotransferase or alanine aminotransferase \> 2.5 × ULN (except in participants with known iron overload).

What this trial measures

  • Overall Response Rate of Dibotatug (defined as proportion of subjects with a Complete Response or Partial Response) [Efficacy]By 6 months

    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]52 weeks

    Incidence and severity of adverse events as assessed by CTCAE v6.0.