Conditioning SCID Infants Diagnosed Early
Public ClinicalTrials.gov record NCT03619551. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Randomized Trial of Low Versus Moderate Exposure Busulfan for Infants With Severe Combined Immunodeficiency (SCID) Receiving TCRαβ+/CD19+ Depleted Transplantation: A Phase II Study by the Primary Immune Deficiency Treatment Consortium (PIDTC) and Pediatric Blood and Marrow Transplant Consortium (PBMTC)
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT03619551
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 56 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 0 Years to 2 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Oct 22, 2018 (Actual)
- Primary completion
- Dec 1, 2027 (Estimated)
- Completion
- Dec 1, 2028 (Estimated)
- Last update posted
- Oct 20, 2026
2018 – 2028
United States locations
- U.S. sites
- 35
- U.S. states
- 25
- U.S. cities
- 34
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Univeristy of Alabama at Birmingham | Birmingham | Alabama | 35294 | — |
| Mayo Clinic Arizona and Phoenix Children's Hospital | Phoenix | Arizona | 85016 | — |
| Children's Hospital Los Angeles | Los Angeles | California | 90027 | — |
| UCLA Center for Health Sciences | Los Angeles | California | 90095 | — |
| Rady Children's Hospital, San Diego | San Diego | California | 92123 | — |
| University of California San Francisco Medical Center - Peds | San Francisco | California | 94143 | — |
| University of Colorado - Children's Hospital | Aurora | Colorado | 80045 | — |
| Children's National Medical Center | Washington D.C. | District of Columbia | 20010 | — |
| Shands HealthCare & University of Florida | Gainesville | Florida | 32610 | — |
| University of Miami/Jackson Memorial Hospital | Miami | Florida | 33136 | — |
| All Children's Hospital | St. Petersburg | Florida | 33701 | — |
| Children's Healthcare of Atlanta at Egleston | Atlanta | Georgia | 30329 | — |
| Comer Children's Hospital/University of Chicago Medicine | Chicago | Illinois | 60637 | — |
| Children's Hospital / LSUHSC | New Orleans | Louisiana | 70118 | — |
| Dana Farber Cancer Institute - Peds | Boston | Massachusetts | 02115 | — |
| The University of Michigan | Ann Arbor | Michigan | 48109 | — |
| Helen DeVos Children's | Grand Rapids | Michigan | 49503 | — |
| University of Minnesota Blood and Marrow Transplant Program - Pediatrics | Minneapolis | Minnesota | 55455 | — |
| The Children's Mercy Hospitals and Clinics | Kansas City | Missouri | 64108 | — |
| Nebraska Medicine | Omaha | Nebraska | 68198 | — |
| Hackensack University Medical Center | Hackensack | New Jersey | 07601 | — |
| Morgan Stanley Children's Hospital of New York-Presbyterian - Columbia University Medical Center | New York | New York | 10032 | — |
| Cohen Children's Medical Center | Queens | New York | 11040 | — |
| Levine Children's Hospital | Charlotte | North Carolina | 28203 | — |
| Duke University Medical Center; Pediatric Blood and Marrow Transplant | Durham | North Carolina | 27705 | — |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | — |
| Oregon Health and Science University | Portland | Oregon | 97239-3098 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania | 15224 | — |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | — |
| Children's Medical Center Dallas | Dallas | Texas | 75235 | — |
| Utah Blood and Marrow Transplant Program-Peds | Salt Lake City | Utah | 84112 | — |
| Fred Hutchinson Cancer Research Center | Seattle | Washington | 98109 | — |
| University of Wisconsin Hospital and Clinics | Madison | Wisconsin | 53792 | — |
| Children's Hospital of Wisconsin | Milwaukee | Wisconsin | 53226 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 3 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03619551, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Oct 20, 2026 · Synced Oct 21, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT03619551 live on ClinicalTrials.gov.