New hope for sickle cell: Half-Matched transplants show promise

NCT ID NCT03263559

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

First seen Jun 27, 2026 · Last updated Jul 09, 2026 · Updated 2 times

Summary

This phase 2 trial tested a half-matched (haploidentical) bone marrow transplant in 95 children and adults with severe sickle cell disease. The goal was to see if this approach could help patients live without major complications for two years. Participants received a reduced-intensity conditioning regimen before the transplant. The study found that many patients achieved event-free survival, suggesting this could be a viable option for those without a fully matched donor.

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 from a half-matched donor
What this could lead to: If successful, this approach could offer a treatment option for severe sickle cell disease patients who lack a fully matched donor.
What could go wrong: This is a phase 2 trial with 95 participants, so results are still early. Risks include graft failure, infection, and graft-versus-host disease. Lifelong immunosuppression may be needed.

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

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Conditions

The condition(s) this trial relates to.

sickle cell disease

As listed by the trial registrant

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

SICKLE CELL DISEASE

Contacts and locations

Locations

Cardinal Glennon Children's Hospital

St Louis, Missouri, 63104, United States
Children's Hospital of Michigan

Detroit, Michigan, 48201, United States
Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States
Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States
Duke University Medical Center

Durham, North Carolina, 27705, United States
Fred Hutchinson Cancer Research Center

Seattle, Washington, 98109, United States
H. Lee Moffitt Cancer Center

Tampa, Florida, 33612, United States
Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States
Helen Devos Children's at Spectrum Health

Grand Rapids, Michigan, 49503, United States
Indiana University Medical Center

Indianapolis, Indiana, 46202, United States
Johns Hopkins University

Baltimore, Maryland, 21287, United States
Levine Children's Hospital

Charlotte, North Carolina, 28203, United States
Methodist Healthcare/West Cancer Center

Memphis, Tennessee, 38104, United States
Nationwide Children's Hospital

Columbus, Ohio, 43205-2696, United States
Nicklaus Children's Hospital/University of Miami Children's Hospital

Miami, Florida, 33155, United States
Northside Hospital

Atlanta, Georgia, 30342, United States
Northwell Health/Monter Cancer Center

Lake Success, New York, 11042, United States
Riley Children's Hospital at IU Health

Indianapolis, Indiana, 46020, United States
Roswell Park Cancer Center

Buffalo, New York, 14203, United States
St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States
Texas Children's Hospital (Baylor)

Houston, Texas, 77030, United States
Texas Transplant Institute

San Antonio, Texas, 78229, United States
The Medical College of Wisconsin

Wauwatosa, Wisconsin, 53226, United States
UCSF Benioff Children's Hospital Oakland

Oakland, California, 94609, United States
University Hospitals of Cleveland/Case Western

Cleveland, Ohio, 44106, United States
University of Alabama at Birmingham

Birmingham, Alabama, 35294, United States
University of Colorado - Denver/Children's Hospital of Colorado

Aurora, Colorado, 80045, United States
University of Florida College of Medicine

Gainesville, Florida, 32610, United States
University of Pittsburgh

Pittsburgh, Pennsylvania, 15260, United States
University of Texas/MD Anderson Cancer Center

Houston, Texas, 77030, United States
Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States
Washington University, St. Louis

St Louis, Missouri, 63110, United States

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