New hope for severe aplastic anemia: bone marrow transplant from partial matches shows promise

NCT ID NCT06517641

Disease control Sponsor: Medical College of Wisconsin Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a bone marrow transplant from a partially matched donor (like a parent or sibling) for people newly diagnosed with severe aplastic anemia, a condition where the bone marrow stops making enough blood cells. Participants receive chemotherapy and immune-suppressing drugs before and after the transplant to help the new marrow take hold. The goal is to see if this approach improves survival and reduces complications like graft-versus-host disease.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: bone marrow transplant with chemotherapy drugs (ATG, fludarabine, cyclophosphamide) and immune-suppressing drugs (tacrolimus, mycophenolate mofetil)
What this could lead to: If successful, this could offer a new, effective treatment option for severe aplastic anemia patients who lack a fully matched donor, potentially improving survival and reducing complications.
What could go wrong: This is a phase II trial with only 60 participants, so results are preliminary. The transplant carries risks like graft-versus-host disease, graft failure, and infection, and patients may need long-term immune-suppressing drugs.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 60 people

The number the study aims to enrol. It can still change while the study runs.

Started

May 2025

Expected to finish

Feb 2029

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

3 to 75 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Age 3 years to 75 years 2. Confirmed diagnosis of acquired SAA defined as: a. Bone marrow cellularity \< 25% or variable marrow cellularity but with \< 30% residual hematopoietic cells deemed HYPOcellular for age AND b. Two (2) out of 3 of the following (in peripheral blood). i. Neutrophils \< 0.5 x109/L ii. Platelets \< 20 x109/L iii. Reticulocyte count \< 20 x109/L (\< 60 x 109/L using an automated analysis) 3. 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. 4. Available donor as defined in the protocol. 5. Participant and/or legal guardian must sign informed consent. 6. Adequate organ function defined by institutional transplant standards or defined as below: 1. 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. 2. Hepatic: Total bilirubin \< 2.0 mg/dL unless Gilbert's disease is present 3. Renal: For participants \> 13.0 years of age at the time of enrollment: estimated creatinine clearance (CrCl) \> 60 mL/minute (per institutional standard). For participants \< 13.0 years of age at enrollment: glomerular filtration rate (GFR) estimated by the updated Schwartz formula ≥ 90 mL/min/1.73 m2. If the estimated GFR is \< 90 mL/min/1.73 m2, then renal function must be measured by 24-hour creatinine clearance or nuclear GFR, and must be \> 50 mL/min/1.73 m2. 4. Pulmonary: i. For participants \> 13.0 years of age: Diffusing capacity of the lung for carbon monoxide (DLCO, corrected/adjusted for hemoglobin \[Hb\]) \> 50%, or Spirometry with forced expiratory volume 1 (FEV1) \> 50% predicted (without administration of bronchodilator) and forced vital capacity (FVC) \> 50% predicted. ii. For participants \< 13.0 years of age unable to perform pulmonary function tests (PFTs) due to age or developmental ability: (1) no evidence of dyspnea at rest and (2) no need for supplemental oxygen and (3) O2 saturation \> 92% on room air at sea level (with lower levels allowed at higher elevations per established center standard of care \[e.g., Utah, 4,200 feet above sea level, does not give supplemental oxygen unless below 90%\]). 7. Karnofsky or Lansky performance status ≥ 60%. 8. Females and males of childbearing potential must agree to practice 2 effective methods of contraception at the same time or agree to abstinence. Exclusion Criteria: 1. 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. 2. 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). 3. Formal diagnosis of MDS by World Health Organization (WHO) 2022 or International Consensus Classification (ICC). 4. 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. 5. 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. 6. 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. 7. Prior allogeneic stem cell transplant. 8. Prior solid organ transplant. 9. Known life-threatening reaction (i.e., anaphylaxis) to Thymoglobulin® (Sanofi) that would prohibit use for the participant as this study requires use of the Thymoglobulin® (Sanofi) preparation of ATG. 10. Uncontrolled bacterial, viral, or fungal infection at the time of enrollment. Uncontrolled is defined as currently taking medication and with progression or no clinical improvement on adequate medical treatment. 11. Female participants who are pregnant, as detected using a pregnancy test as per institutional practice, or breast-feeding. 12. Prior malignancies except resected basal cell carcinoma or treated cervical carcinoma in situ. Cancer treated with curative intent \> 5 years previously will be allowed. Cancer treated with curative intent ≤ 5 years previously will not be allowed unless approved by the Protocol Chairs and/or Protocol Officer. Of note, participants with seropositivity for the human immunodeficiency virus (HIV) may be considered if viral load is undetectable. Similarly, carriers of hepatitis B (HepB) or hepatitis C (HepC) may not have a detectable viral load of HepB virus or HepC virus. Participants with HIV that is well-controlled on combination antiretroviral therapy and no AIDS related complications within the past 12 months are eligible. Infections other than HIV: * Prior infections must be controlled * HepB participants are eligible if on effective suppressive therapy and otherwise meet inclusion/exclusion criteria * HepC participants are eligible if otherwise meet inclusion/exclusion criteria

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Conditions

The condition(s) this trial relates to.

acquired aplastic anemia aplastic anemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Severe aplastic anemia

How to take part

Only the study team decides who joins. These are the ways to reach them.

The places running it

25 sites. The list below names each one and where it is.
The official record

ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

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A doctor treating you

A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

Blood and Marrow Transplant Center at Northside Hospital
RECRUITING

Atlanta, Georgia, 30342, United States
City of Hope
RECRUITING

Duarte, California, 91010, United States
Dana Farber Cancer Institute
RECRUITING

Boston, Massachusetts, 02215, United States
Duke University Health System
RECRUITING

Durham, North Carolina, 27705, United States
Emory Winship Cancer Institute
RECRUITING

Atlanta, Georgia, 30322, United States
Fred Hutchinson Cancer Center
RECRUITING

Seattle, Washington, 98109, United States
Huntsman Cancer Institute
RECRUITING

Salt Lake City, Utah, 84112, United States
Johns Hopkins University
RECRUITING

Baltimore, Maryland, 21218, United States
Karmanos Cancer Institute
RECRUITING

Detroit, Michigan, 48201, United States
Levine Cancer Institute
RECRUITING

Charlotte, North Carolina, 28204, United States
Massachusetts General Hospital
RECRUITING

Boston, Massachusetts, 02114, United States
Mayo Clinic
RECRUITING

Rochester, Minnesota, 55905, United States
Medical College of Wisconsin
RECRUITING

Milwaukee, Wisconsin, 53226, United States
Memorial Sloan Kettering Cancer Center
RECRUITING

New York, New York, 10021, United States
Moffitt Cancer Center
RECRUITING

Tampa, Florida, 33612, United States
Stanford University
RECRUITING

Stanford, California, 94305, United States
The Ohio State University
RECRUITING

Columbus, Ohio, 43210, United States
UNC Chapel Hill
RECRUITING

Chapel Hill, North Carolina, 27599, United States
University of Alabama at Birmingham
RECRUITING

Birmingham, Alabama, 35294, United States
University of California, Los Angeles
RECRUITING

Los Angeles, California, 90095, United States
University of Kansas Medical Center
RECRUITING

Westwood, Kansas, 66205, United States
University of Oklahoma
RECRUITING

Oklahoma City, Oklahoma, 73117, United States
University of Pennsylvania
RECRUITING

Philadelphia, Pennsylvania, 19104, United States
Vanderbilt University
RECRUITING

Nashville, Tennessee, 37235, United States
Washington University School of Medicine, Barnes-Jewish Hospital
RECRUITING

St Louis, Missouri, 63110, United States

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