Bone Marrow Failure SyndromesPhase 1/2BMFNCT07712562
A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure Syndromes
A Phase 1/2 study testing Dibotatug (DR-01) in people with Bone Marrow Failure Syndromes and Aplastic Anemias, run by Dren Bio at 10 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Bone Marrow Failure Syndromes, Aplastic Anemias, Severe Aplastic Anemia (SAA)
- What is being tested?
- Dibotatug (DR-01)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 10 sitesUnited States
- Study dates
- Aug 2026to Oct 2030
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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 informed consent form (ICF) for the Extension Treatment Period.
- CR or PR by Week 24 during the Main Treatment Period
Who cannot join
- 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.
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- 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).
Trial locations and contacts
10 sites in 1 country. Contact the study team or a site near you to ask about taking part.
10 sites in 1 country
United States10 sites
Dren Investigational Site
Atlanta, Georgia, 30342
Dren Bio Central Contact, 415-737-5277, DR-01-HEM-001_inquiries@drenbio.com
Dren Investigational Site
Cleveland, Ohio, 44195
Dren Bio Central Contact, 415-737-5277, DR-01-HEM-001_inquiries@drenbio.com
Dren Investigational Site
Columbus, Ohio, 43210
Dren Bio Central Contact, 415-737-5277, DR-01-HEM-001_inquiries@drenbio.com
Dren Investigational Site
Duarte, California, 91010
Dren Bio Central Contact, 415-737-5277, DR-01-HEM-001_inquiries@drenbio.com
Dren Investigational Site
Fairfax, Virginia, 22031
Dren Bio Central Contact, 415-737-5277, DR-01-HEM-001_inquiries@drenbio.com
Dren Investigational Site
Houston, Texas, 77030
Dren Bio Central Contact, 415-737-5277, DR-01-HEM-001_inquiries@drenbio.com
Dren Investigational Site
Miami, Florida, 33136
Dren Bio Central Contact, 415-737-5277, DR-01-HEM-001_inquiries@drenbio.com
Dren Investigational Site
New York, New York, 10065
Dren Bio Central Contact, 415-737-5277, DR-01-HEM-001_inquiries@drenbio.com
Dren Investigational Site
Palo Alto, California, 94304
Dren Bio Central Contact, 415-737-5277, DR-01-HEM-001_inquiries@drenbio.com
Dren Investigational Site
Seattle, Washington, 98109
Dren Bio Central Contact, 415-737-5277, DR-01-HEM-001_inquiries@drenbio.com
About this study
Official title: A Phase 1/2, Open-Label Study to Evaluate the Safety and Efficacy of Dibotatug in Adults With Bone Marrow Failure Syndromes
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.
Read the detailed 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 continue to enroll in Stage 2 for a total of up to 60 participants.
- Conditions
- Bone Marrow Failure Syndromes, Aplastic Anemias, Severe Aplastic Anemia (SAA), Severe Aplastic Anemia, Relapse, Severe Aplastic Anemia, Refractory, Non-severe Aplastic Anemia
- Being tested
- Dibotatug (DR-01)
- Keywords
- DR-01, Dibotatug, aplastic anemia, transfusion dependent, hematologic diseases, hematology, Blood disorder, pancytopenia, stem cell disorder, red blood cell disease, white blood cell disease
- Medical subject headings
- Bone Marrow Failure Disorders, Anemia, Aplastic, Recurrence, Hematologic Diseases, Pancytopenia, Leukocyte Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Dibotatug (DR-01)Drug
Dibotatug (DR-01) administered by IV infusion
| Study group | Role | What they receive |
|---|---|---|
| Dibotatug (DR-01) | Experimental | Subjects in this arm will receive 20 weeks of dosing with dibotatug. Subjects with a CR or PR by Week 24 have the option to continue dosing through Week 48; Dibotatug will be administered via IV infusion. Receives: Drug: Dibotatug (DR-01) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: By 6 months
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.
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jul 2026
- Study startAug 2026 (estimated)
- Primary completionApr 2029 (estimated)
- Study completionOct 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Bone Marrow Failure SyndromesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Single Arm Basket trial - 3 cohorts will receive the same treatment paradigm in parallel
- Masking
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Dren Bio
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Wan-Jen Hong, MD, Study director, Dren Bio
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07712562
- Sponsor's protocol ID
- DR-01-HEM-001
- Acronym
- BMF
- Status last verified
- Jul 2026
- First submitted
- 14 Jul 2026
- Last updated
- 24 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
- Expanded access
- Available
Study at a glance
From the registry record, last updated by the sponsor on 24 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2, Open-Label Study to Evaluate the Safety and Efficacy of Dibotatug in Adults With Bone Marrow Failure Syndromes
- ClinicalTrials.gov ID
- NCT07712562
- Acronym / sponsor ID
- BMF / DR-01-HEM-001
- Conditions
- Bone Marrow Failure Syndromes, Aplastic Anemias, Severe Aplastic Anemia (SAA), Severe Aplastic Anemia, Relapse, Severe Aplastic Anemia, Refractory, Non-severe Aplastic Anemia
- Keywords
- DR-01, Dibotatug, aplastic anemia, transfusion dependent, hematologic diseases, hematology, Blood disorder, pancytopenia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Dren BioIndustry (pharma, biotech, devices)
- Study start
- Aug 2026 (estimated)
- Primary completion
- Apr 2029 (estimated)
- Study completion
- Oct 2030 (estimated)
- First posted
- 17 Jul 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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 continue to enroll in Stage 2 for a total of up to 60 participants.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Single Arm Basket trial - 3 cohorts will receive the same treatment paradigm in parallel
| Arm | Type | Receives |
|---|---|---|
| Dibotatug (DR-01) | Experimental | Drug: Dibotatug (DR-01)Subjects in this arm will receive 20 weeks of dosing with dibotatug. Subjects with a CR or PR by Week 24 have the option to continue dosing through Week 48; Dibotatug will be administered via IV infusion. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (19 listed)
- 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.
- 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.
Key exclusion criteria (19 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Dibotatug (DR-01)Dibotatug (DR-01) administered by IV infusion | Drug | Dibotatug (DR-01) |
Primary outcome
Overall Response Rate of Dibotatug (defined as proportion of subjects with a Complete Response or Partial Response) [Efficacy]
Time frame: 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]
Time frame: 52 weeks
Incidence and severity of adverse events as assessed by CTCAE v6.0.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jul 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Wan-Jen Hong, MD study director
Dren Bio
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Dibotatug (DR-01) in people with Bone Marrow Failure Syndromes and Aplastic Anemias, run by Dren Bio at 10 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 19 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 24 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Dren Bio Central Contact (415-737-5277, DR-01-HEM-001_inquiries@drenbio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Response Rate of Dibotatug (defined as proportion of subjects with a Complete Response or Partial Response) [Efficacy] (time frame: By 6 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Jul 2026.