RecruitingPhase 2

Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia

United States60 participantsStarted 2025-05-12

Plain-language summary

BMT CTN 2207 will investigate the use of marrow transplantation for treatment of severe aplastic anemia that has not previously been treated.

Who can participate

Age range3 Years – 75 Years

SexALL

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Inclusion criteria

✓. Age 3 years to 75 years
✓. Confirmed diagnosis of acquired SAA defined as:
✓. No suitable fully matched related donor as per Investigator's discretion (6/6 match for HLA A and B at intermediate or high-resolution and DRB1 at high-resolution using deoxyribonucleic acid \[DNA\]-based typing) available.
✓. Available donor as defined in the protocol.
✓. Participant and/or legal guardian must sign informed consent.
✓. Adequate organ function defined by institutional transplant standards or defined as below:
✓. Cardiac: Left ventricular ejection fraction (LVEF) at rest \> 40% with no clinical signs of cardiac failure. For participants aged \< 13 years, shortening fraction (SF) ≥ 26% by echocardiogram or multigated acquisition (MUGA) may be substituted for LVEF.
✓. Hepatic: Total bilirubin \< 2.0 mg/dL unless Gilbert's disease is present

Exclusion criteria

✕. Inherited bone marrow failure syndromes such as Fanconi anemia and short telomere syndromes must be ruled out according to center standards. It is recommended that functional testing for Fanconi Anemia (di-epoxybutane \[DEB\] chromosomal breakage analysis) and telomere length assessment be performed. If available, genetic panels for inherited bone marrow failure syndromes can be considered as an alternative to functional testing.
✕. Clonal cytogenetic abnormalities consistent with pre-MDS or MDS on marrow examination (e.g., monosomy 7 and other MDS-defining changes per recent pathology guidelines).
✕. Formal diagnosis of MDS by World Health Organization (WHO) 2022 or International Consensus Classification (ICC).
✕. Recipient positive for HLA antibodies against a mismatched HLA in the selected donor determined by the presence of donor specific HLA antibodies (DSA) to any mismatched HLA allele/antigen at any of the following loci (HLA-A, -B, -C, -DRB1, DRB3, DRB4, DRB5, -DQA1, -DQB1, -DPA1, -DPB1) with median fluorescence intensity (MFI) \>3000 by microarray-based single antigen bead testing. In patients receiving red blood cell or platelet transfusions, DSA evaluation must be performed or repeated post-transfusion and immediately prior to initiation of recipient preparative regimen to ensure there is confirmation of no DSA to the selected donor when conditioning starts.
✕. Prior desensitization attempt for HLA antibodies to chosen donor. Any intervention with the sole intent to reduce the level of HLA DSA, (e.g., plasmapheresis, intravenous immunoglobulin \[IVIG\], MMF, etc.) would constitute a desensitization attempt.
✕. Prior treatment for SAA (e.g., immunosuppressive therapy using ATG, calcineurin inhibitors \[CNIs\], thrombopoietin receptor agonists or androgens). Short courses of steroids or IVIG that were not explicitly administered for SAA therapy will be allowed.
✕. Prior allogeneic stem cell transplant.
✕. Prior solid organ transplant.

What they're measuring

Graft versus host disease (GVHD)-free failure-free survival (GFFS) at One year

Timeframe: 1 year after initiation of conditioning

Trial details

NCT IDNCT06517641

SponsorMedical College of Wisconsin

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2028-08-01

Contact for this trial

Jennifer Romeril

301-251-1161 bmtctn2207@emmes.com

View on ClinicalTrials.gov More Severe Aplastic Anemia trials