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Bone marrow transplant or drugs? landmark trial for rare blood disease

NCT ID NCT05600426

Disease control Sponsor: Boston Children's Hospital Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jul 31, 2026 · Updated 4 times

Summary

This phase 3 trial compares two standard treatments for severe aplastic anemia (SAA) in patients up to age 25 who lack a matched sibling donor: unrelated donor bone marrow transplant (URD BMT) versus immune suppressive therapy (IST). The study measures which approach better prevents treatment failure or death. It also looks at quality of life and fertility. Both treatments are already used in practice, but this is the first head-to-head test as initial therapy.

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 or immune suppressive drugs (cyclosporine, anti-thymocyte globulin, methotrexate, fludarabine, cyclophosphamide, low-dose radiation)
What this could lead to
If this trial succeeds, it could show that upfront unrelated donor bone marrow transplant is better than immune therapy for curing severe aplastic anemia in young patients without a matched sibling donor.
What could go wrong
This is a relatively small phase 3 trial (53 participants), and both treatments have serious risks like infection, graft-versus-host disease, or treatment failure. Results may not apply to all patients.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

53 people

The number who actually took part.

Started

Jan 2023

Expected to finish

Dec 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

0 to 25 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: To be eligible to participate in the randomized trial, an individual must meet all the following criteria: 1. Provision of signed and dated informed consent form for the randomized trial by patient and/or legal guardian. 2. Age ≤25 years old at time of randomized trial consent. 3. Confirmed diagnosis of idiopathic SAA, defined as: 1. Bone marrow cellularity \<25%, or \<30% hematopoietic cells. 2. Two of three of the following (in peripheral blood): neutrophils \<0.5 x 10\^9/L, platelets \<20 x 10\^9/L, absolute reticulocyte count \<60 x 10\^9/L or hemoglobin \<8 g/dL. 4. No suitable fully matched related donor available (minimum 6/6 match for HLA-A and B at intermediate or high resolution and DRB1 at high resolution using DNA based typing). 5. At least 2 unrelated donors noted on NMDP search who are well matched (9/10 or 10/10 for HLA-A, B, C, DRB1, and DQB1 using high resolution). 6. In the treating physician's opinion, no obvious contraindications precluding them from BMT or IST. Exclusion Criteria: 1. Presence of Inherited bone marrow failure syndromes (IBMFS). The diagnosis of Fanconi anemia must be excluded by diepoxybutane (DEB) or equivalent testing on peripheral blood or marrow. Telomere length testing should be sent on all patients to exclude Dyskeratosis Congenita (DC), but if results are delayed or unavailable and there are no clinical manifestations of DC, patients may enroll. If patients have clinical characteristics suspicious for Shwachman-Diamond syndrome, this disorder should be excluded by pancreatic isoamylase testing or gene mutation analysis (note: pancreatic isoamylase testing is not useful in children \<3). Other testing per center may be performed to exclude IBMFS. 2. Clonal cytogenetic abnormalities or Fluorescence In-Situ Hybridization (FISH) pattern consistent with pre- myelodysplastic syndrome (pre-MDS) or MDS on marrow examination. 3. Known severe allergy to ATG. 4. Prior allogeneic or autologous stem cell transplant. 5. Prior solid organ transplant. 6. Infection with human immunodeficiency virus (HIV). 7. Active Hepatitis B or C. This only needs to be excluded in patients where there is clinical suspicion of hepatitis (e.g., elevated LFTs). 8. Female patients who are pregnant or breast-feeding. 9. Prior malignancies except resected basal cell carcinoma or treated cervical carcinoma in situ. 10. Disease modifying treatment prior to study enrollment, including but not limited to use of androgens, eltrombopag, romiplostim, or immune suppression. Note: Supportive care measures such as G-CSF, blood transfusion support and antibiotics are allowable

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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

Contacts and locations

Locations

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Arkansas

    Little Rock, Arkansas, 72202, United States

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • British Columbia Children's

    Vancouver, British Columbia, V6H 3V4, Canada

  • Children's Hospital & Research Center Oakland

    Oakland, California, 94609, United States

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital NOLA

    New Orleans, Louisiana, 70118, United States

  • Children's Hospital of Atlanta/Emory

    Atlanta, Georgia, 30322, United States

  • Children's Hospital of Orange County

    Orange, California, 92868, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Children's Hospital of the King's Daughter

    Norfolk, Virginia, 23507, United States

  • Children's Medical Center Dallas

    Dallas, Texas, 75235, United States

  • Children's National Hospital

    Washington D.C., District of Columbia, 20010, United States

  • Cohen Children's Medical Center of New York

    New Hyde Park, New York, 11040, United States

  • Columbia University Medical Center

    New York, New York, 10032, United States

  • Duke University

    Durham, North Carolina, 27705, United States

  • Fred Hutchinson Cancer Center

    Seattle, Washington, 98109, United States

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601, United States

  • Helen DeVos Children's Hospital

    Grand Rapids, Michigan, 49503, United States

  • Indiana University Hospital/Riley Hospital for Children

    Indianapolis, Indiana, 46202, United States

  • Johns Hopkins All Children's Hospital

    St. Petersburg, Florida, 33701, United States

  • Levine Children's Hospital

    Charlotte, North Carolina, 28203, United States

  • Loma Linda

    Loma Linda, California, 92354, United States

  • Maine Health

    Scarborough, Maine, 04074, United States

  • Mayo Clinic Rochestser

    Rochester, Minnesota, 55902, United States

  • Methodist Healthcare

    San Antonio, Texas, 78229, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Nemours Children's Hospital, Delaware

    Wilmington, Delaware, 19803, United States

  • Nicklaus Children's Hospital

    Miami, Florida, 33155, United States

  • Oregon Health & Science University

    Portland, Oregon, 97239, United States

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016, United States

  • Rady Children's Hospital San Diego

    San Diego, California, 92123, United States

  • Roswell Park Comprehensive Cancer Center

    Buffalo, New York, 14263, United States

  • St. Jude Children's Research Hospital

    Memphis, Tennessee, 38105, United States

  • Stanford

    Palo Alto, California, 94304, United States

  • Texas Children's Hospital

    Houston, Texas, 77030, United States

  • UCLA

    Los Angeles, California, 90095, United States

  • University of Alabama at Birmingham

    Birmingham, Alabama, 35249, United States

  • University of California San Francisco

    San Francisco, California, 94158, United States

  • University of Chicago

    Chicago, Illinois, 60637, United States

  • University of Florida

    Gainesville, Florida, 32610, United States

  • University of Miami

    Miami, Florida, 33136, United States

  • University of Michigan

    Ann Arbor, Michigan, 48109, United States

  • University of Mississippi Medical Center

    Jackson, Mississippi, 39216, United States

  • University of North Carolina

    Chapel Hill, North Carolina, 27599, United States

  • University of Utah/Primary Children's Hospital

    Salt Lake City, Utah, 84112, United States

  • University of Wisconsin

    Madison, Wisconsin, 53792, United States

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37203, United States

  • Washington University in St. Louis

    St Louis, Missouri, 63110, United States

  • Winnipeg CancerCare Manitoba

    Winnipeg, Manitoba, R3E 0V9, Canada

  • Yale University

    New Haven, Connecticut, 06520, United States

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