Miami, FLNCT07712562Now EnrollingIRB Ready

Bone Marrow Failure Syndromes Clinical Trial in Miami, FL

Access cutting-edge bone marrow failure syndromes treatment through this phase 1/2 clinical trial at a research site in Miami. Study-provided care at no cost to qualified participants.

Sponsored by Dren Bio

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Expert Care in Miami

Access bone marrow failure syndromes specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related bone marrow failure syndromes treatment provided free

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Check if you qualify for this bone marrow failure syndromes clinical trial in Miami, FL

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Why Participate?

  • No-Cost Study Care

  • Local to Miami

    Convenient for FL residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Miami site if eligible
  4. 4Begin participation

About This Bone Marrow Failure Syndromes Study in Miami

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.

Sponsor: Dren Bio

Who Can Participate

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 informed consent form (ICF) for the Extension Treatment Period.
CR or PR by Week 24 during the Main Treatment Period

Exclusion Criteria

Diagnosis of Fanconi anemia, dyskeratosis congenita, or other congenital BMF syndrome.
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).

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Miami?

Yes, this clinical trial (NCT07712562) has an active research site in Miami, FL that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Bone Marrow Failure Syndromes Treatment Options in Miami, FL

If you're searching for bone marrow failure syndromes treatment options in Miami, FL, this clinical trial (NCT07712562) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Miami research site is actively enrolling participants for this phase 1/2 clinical trial. You'll receive care from experienced bone marrow failure syndromes specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all bone marrow failure syndromes clinical trials near you to find additional studies recruiting in your area.

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