Can lower chemo doses help babies with SCID build better immunity?

NCT ID NCT03619551

Ongoing Disease control Sponsor: Center for International Blood and Marrow Transplant Research Source: ClinicalTrials.gov ↗

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

Summary

This phase 2 trial studies whether lower doses of the chemotherapy drug busulfan can help infants with severe combined immunodeficiency (SCID) achieve good immune function after a stem cell transplant, while reducing short- and long-term risks. The trial includes 56 babies receiving transplants from unrelated or half-matched related donors, with most T and B cells removed from the donor cells to lower the risk of graft-versus-host disease. Participants are followed for 3 years to see if they can produce antibodies after vaccinations.

What this could mean

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

Active substance

busulfan (chemotherapy) and stem cell transplant with T-cell and B-cell removal

What this could lead to

If successful, this could lead to safer stem cell transplants for babies with SCID, helping them build their own immunity with fewer side effects.

What could go wrong

This is a small, early-phase trial with only 56 participants, so results may not apply to all SCID cases. The experimental cell removal process may not prevent graft-versus-host disease or other complications.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

Omenn syndrome severe combined immunodeficiency

As listed by the trial registrant

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

SCID

Contacts and locations

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Locations

All Children's Hospital

St. Petersburg, Florida, 33701, United States
Cancer Care Manitoba/University of Manitoba

Winnipeg, Winnipeg, MB, Canada
Cardinal Glennon Children's Medical Center

St Louis, Missouri, 63104, United States
Centre Hospitalier Universitaire Sainte-Justine

Montreal, Montreal, QC, Canada
Children's Healthcare of Atlanta at Egleston

Atlanta, Georgia, 30329, United States
Children's Hospital / LSUHSC

New Orleans, Louisiana, 70118, United States
Children's Hospital Los Angeles

Los Angeles, California, 90027, United States
Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15224, United States
Children's Hospital of Wisconsin

Milwaukee, Wisconsin, 53226, United States
Children's Medical Center Dallas

Dallas, Texas, 75235, United States
Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States
Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States
Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States
Cohen Children's Medical Center

Queens, New York, 11040, United States
Comer Children's Hospital/University of Chicago Medicine

Chicago, Illinois, 60637, United States
Dana Farber Cancer Institute - Peds

Boston, Massachusetts, 02115, United States
Duke University Medical Center; Pediatric Blood and Marrow Transplant

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

Seattle, Washington, 98109, United States
Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States
Helen DeVos Children's

Grand Rapids, Michigan, 49503, United States
Indiana University Hospital/Riley Hospital for Children

Indianapolis, Indiana, 46202, United States
Levine Children's Hospital

Charlotte, North Carolina, 28203, United States
Mayo Clinic Arizona and Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States
Memorial Sloan Kettering Cancer Center - Peds

New York, New York, 10065, United States
Morgan Stanley Children's Hospital of New York-Presbyterian - Columbia University Medical Center

New York, New York, 10032, United States
Nebraska Medicine

Omaha, Nebraska, 68198, United States
Oregon Health and Science University

Portland, Oregon, 97239-3098, United States
Rady Children's Hospital, San Diego

San Diego, California, 92123, United States
Shands HealthCare & University of Florida

Gainesville, Florida, 32610, United States
The Children's Mercy Hospitals and Clinics

Kansas City, Missouri, 64108, United States
The Hospital for Sick Children

Toronto, Ontario, M5G 1E8, Canada
The University of Michigan

Ann Arbor, Michigan, 48109, United States
UCLA Center for Health Sciences

Los Angeles, California, 90095, United States
Univeristy of Alabama at Birmingham

Birmingham, Alabama, 35294, United States
University of California San Francisco Medical Center - Peds

San Francisco, California, 94143, United States
University of Colorado - Children's Hospital

Aurora, Colorado, 80045, United States
University of Iowa Hospitals & Clinics

Iowa City, Iowa, 52242, United States
University of Miami/Jackson Memorial Hospital

Miami, Florida, 33136, United States
University of Minnesota Blood and Marrow Transplant Program - Pediatrics

Minneapolis, Minnesota, 55455, United States
University of Wisconsin Hospital and Clinics

Madison, Wisconsin, 53792, United States
Utah Blood and Marrow Transplant Program-Peds

Salt Lake City, Utah, 84112, United States
Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States
Westchester Medical Center

Valhalla, New York, 10595, United States