Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
Conditioning SCID Infants Diagnosed Early
NCT03619551 · View on ClinicalTrials.gov ↗
Study Summary
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.
Conditions Studied
Interventions
- DRUG Busulfan
- DEVICE Cell processing for TCRαβ+/CD19+ depletion
Study Locations (20)
California
- Children's Hospital Los Angeles — Los Angeles
- UCLA Center for Health Sciences — Los Angeles
- Rady Children's Hospital, San Diego — San Diego
- University of California San Francisco Medical Center - Peds — San Francisco
Florida
- Shands HealthCare & University of Florida — Gainesville
- University of Miami/Jackson Memorial Hospital — Miami
- All Children's Hospital — St. Petersburg
Michigan
- The University of Michigan — Ann Arbor
- Helen DeVos Children's — Grand Rapids
Alabama
- Univeristy of Alabama at Birmingham — Birmingham
Arizona
- Mayo Clinic Arizona and Phoenix Children's Hospital — Phoenix
Colorado
- University of Colorado - Children's Hospital — Aurora
District of Columbia
- Children's National Medical Center — Washington D.C.
Georgia
- Children's Healthcare of Atlanta at Egleston — Atlanta
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 56 participants |
| Start Date | 2018-10-22 |
| Est. Completion | 2028-12-01 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03619551
The ClinicalTrials.gov registry entry for NCT03619551 describes a study currently listed as active not recruiting. It is categorized as Phase 2, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 56 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Center for International Blood and Marrow Transplant Research, which has 16 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with SCID appearing as the primary indexed condition, and to 2 interventions — of which Busulfan is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT03619551 reports 20 study locations spanning 14 distinct geographic areas — top geographies include California, Florida, Michigan. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT03619551 about?
NCT03619551 is a clinical study titled "Conditioning SCID Infants Diagnosed Early". 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 appr...
What is the current status of trial NCT03619551?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 56 participants. The study started on 2018-10-22. Estimated completion is 2028-12-01.
What conditions does trial NCT03619551 study?
This clinical trial studies the following conditions: SCID. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT03619551?
The interventions under investigation include: Busulfan (DRUG), Cell processing for TCRαβ+/CD19+ depletion (DEVICE). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT03619551?
This trial is sponsored by Center for International Blood and Marrow Transplant Research, which has 16 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT03619551 being conducted?
This trial has 20 study locations across Alabama, Arizona, California, Colorado, District of Columbia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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