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Clinical trialNCT03619551Active, not recruitingPhase 2Interventional

Conditioning SCID Infants Diagnosed Early

SponsorCenter for International Blood and Marrow Transplant ResearchEnrollment56SCID
View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

The investigators want to study if lower doses of chemotherapy will help babies with SCID to achieve good immunity with less short and long-term risks of complications after transplantation. This trial identifies babies with types of immune deficiencies that are most likely to succeed with this approach and offers them transplant early in life before they get severe infections or later if their infections are under control. It includes only patients receiving unrelated or mismatched related donor transplants. The study will test if patients receiving transplant using either a low dose busulfan or a medium dose busulfan will have immune recovery of both T and B cells, measured by the ability to respond to immunizations after transplant. The exact regimen depends on the subtype of SCID the patient has. Donors used for transplant must be unrelated or half-matched related (haploidentical) donors, and peripheral blood stem cells must be used. To minimize the chance of graft-versus-host disease (GVHD), the stem cells will have most, but not all, of the T cells removed, using a newer, experimental approach of a well-established technology. Once the stem cell transplant is completed, patients will be followed for 3 years. Approximately 9-18 months after the transplant, vaccinations will be administered, and a blood test measuring whether your child's body has responded to the vaccine will be collected.

Investigators

The overall officials registered on this study. Names that resolve to a FindMyKOL profile link to it; the rest are shown as registered.

A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

41 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

Canada
  • Centre Hospitalier Universitaire Sainte-Justine

    Montreal, Montreal, Canada

  • the Hospital for Sick Children

    Toronto, Ontario, Canada

  • Cancer Care Manitoba/university of Manitoba

    Winnipeg, Winnipeg, Canada

United States
  • Univeristy of Alabama at Birmingham

    Birmingham, Alabama, United States

  • Mayo Clinic Arizona and Phoenix Children'S Hospital

    Phoenix, Arizona, United States

  • Children'S Hospital Los Angeles

    Los Angeles, California, United States

  • Ucla Center for Health Sciences

    Los Angeles, California, United States

  • Rady Children'S Hospital, San Diego

    San Diego, California, United States

  • University of California San Francisco Medical Center - Peds

    San Francisco, California, United States

  • University of Colorado - Children'S Hospital

    Aurora, Colorado, United States

  • Children'S National Medical Center

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

  • Shands Healthcare & University of Florida

    Gainesville, Florida, United States

  • University of Miami/jackson Memorial Hospital

    Miami, Florida, United States

  • All Children'S Hospital

    St. Petersburg, Florida, United States

  • Children'S Healthcare of Atlanta at Egleston

    Atlanta, Georgia, United States

  • Comer Children'S Hospital/university of Chicago Medicine

    Chicago, Illinois, United States

  • Indiana University Hospital/riley Hospital for Children

    Indianapolis, Indiana, United States

  • University of Iowa Hospitals & Clinics

    Iowa City, Iowa, United States

  • Children'S Hospital / Lsuhsc

    New Orleans, Louisiana, United States

  • Dana Farber Cancer Institute - Peds

    Boston, Massachusetts, United States

  • the University of Michigan

    Ann Arbor, Michigan, United States

  • Helen Devos Children'S

    Grand Rapids, Michigan, United States

  • University of Minnesota Blood and Marrow Transplant Program - Pediatrics

    Minneapolis, Minnesota, United States

  • the Children'S Mercy Hospitals and Clinics

    Kansas City, Missouri, United States

  • Nebraska Medicine

    Omaha, Nebraska, United States

  • Hackensack University Medical Center

    Hackensack, New Jersey, United States

  • Memorial Sloan Kettering Cancer Center - Peds

    New York, New York, United States

  • Morgan Stanley Children'S Hospital of New York-Presbyterian - Columbia University Medical Center

    New York, New York, United States

  • Cohen Children'S Medical Center

    Queens, New York, United States

  • Levine Children'S Hospital

    Charlotte, North Carolina, United States

  • Duke University Medical Center; Pediatric Blood and Marrow Transplant

    Durham, North Carolina, United States

  • Cincinnati Children'S Hospital Medical Center

    Cincinnati, Ohio, United States

  • Oregon Health and Science University

    Portland, Oregon, United States

  • Children'S Hospital of Philadelphia

    Philadelphia, Pennsylvania, United States

  • Children'S Hospital of Pittsburgh of Upmc

    Pittsburgh, Pennsylvania, United States

  • Vanderbilt University Medical Center

    Nashville, Tennessee, United States

  • Children'S Medical Center Dallas

    Dallas, Texas, United States

  • Utah Blood and Marrow Transplant Program-Peds

    Salt Lake City, Utah, United States

  • Fred Hutchinson Cancer Research Center

    Seattle, Washington, United States

  • University of Wisconsin Hospital and Clinics

    Madison, Wisconsin, United States

1 more site on the registry record.ClinicalTrials.gov ↗

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

The drug and biological interventions registered on this study, normalized to their RxNorm ingredient where possible.

Linked drugs open the directory filtered to KOLs working with them.clinicaltrials.gov ↗

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start
22 Oct 2018
Primary completion
1 Dec 2027
Completion
1 Dec 2028
Last update posted
6 Aug 2026
Registered enrollment
56
Registry id
NCT03619551

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; FindMyKOL does not interpret them.