Bone Marrow Failure Syndromes study: Dibotatug
Official title A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure Syndromes
ClinicalTrials.gov ID: NCT07712562
What this study is testing
What is Dibotatug (DR-01)?
Dibotatug (DR-01) is an investigational medicine, given as an IV infusion (through a vein), being studied as a potential treatment for bone marrow failure syndromes.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study is testing whether Dibotatug (DR‑01) is safe and how well it works in adults with bone marrow failure syndromes.
- Who it's for
- You may be able to join if you are an adult with a bone marrow failure condition like aplastic anemia.
- What's involved
- You will receive the drug Dibotatug (DR‑01) and have regular check‑ups to see how you feel and if the drug is safe.
- Time commitment
- The study lasts several months and includes multiple visits to the clinic for monitoring.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Age ≥ 18 years old
- Received one ≥ 3-month course of ATG and/or 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).
You likely can't join if
- 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.
- Use of a T-cell depleting agent (e.g., ATG, alemtuzumab, thymoglobulin) within 3 months prior to Day 1.
See the full eligibility criteria
- Age ≥ 18 years old
- Received one ≥ 3-month course of ATG and/or 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).
- RBC transfusion dependence: transfusion of ≥ 2 units of RBCs in the past 56 days
- Signed informed consent form (ICF) for the Extension Treatment Period.
- CR or PR by Week 24 during the Main Treatment Period
- 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...
- 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.
- 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...
- 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...
- 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).
The study team makes the final eligibility decision.
Where it is enrolling
- Atlanta, Georgia, United States
- New York, New York, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Atlanta, Georgia, United States; New York, New York, United States. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.