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NCT03311503 · ClinicalTrials.gov registry record · Phase 1
Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning
A Phase 1 study of Gene Therapy and Severe Combined Immunodeficiency, X Linked, sponsored by David Williams.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 12
- Enrollment target
- 4
- Study locations
NCT03311503: Recruiting Phase 1 study of Gene Therapy and Severe Combined Immunodeficiency, X Linked, sponsored by David Williams.
NCT03311503 is a Phase 1 study of Gene Therapy and Severe Combined Immunodeficiency, X Linked that is actively recruiting participants, run by David Williams. The registered enrollment target is 12 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (80% lower). The trial reports 4 study locations across 4 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03311503, a Phase 1 study of Gene Therapy and Severe Combined Immunodeficiency, X Linked, is actively recruiting participants, sponsored by David Williams.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 12 participants
- Enrollment target
- 4
- Study locations
Study Summary
This is a phase I/II open label multi-center study in which patients will receive low dose targeted busulfan followed by infusion of autologous CD34+ selected bone marrow or mobilized peripheral blood cells transduced with the G2SCID vector. Subjects will be enrolled over 3 years and be followed for 2 years post-infusion on this protocol, then followed long-term on a separate long-term follow-up protocol. Enrollment of subjects will be agreed upon by representatives of both sites. Data will be collected uniformly from both sites through an electronic capture system and key laboratory studies will be centralized. Harvest, cellular manufacturing and infusion will occur at each site using the same SOPs. Key aspects of cellular product characterization will be centralized
Primary Outcome
Events will include death, infusion of unmanipulated back-up product for failure of hematopoietic recovery, and allogeneic transplant performed for poor immune reconstitution
Conditions Studied
Interventions
- BIOLOGICAL autologous CD34+ cell transduced with G2SCID vector
Study Locations (4)
California
- Mattel Children's Hospital - UCLA - Los Angeles
Georgia
- Emory University/Childrens Healthcare of Atlanta - Atlanta
Massachusetts
- Boston Childrens Hospital - Boston
Ohio
- Cincinnati Children's Hospital Medical Center - Cincinnati
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2018-02-26 |
| Est. Completion | 2028-01-01 |
| Phase | Phase 1 |
What NCT03311503 shows while recruiting
NCT03311503 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 12 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (80% lower).
The record links to 2 conditions, with Gene Therapy appearing as the primary indexed condition, and to 1 intervention - of which autologous CD34+ cell transduced with G2SCID vector is the first listed.
NCT03311503 reports a single indexed study location in California, Georgia, Massachusetts.
Frequently Asked Questions
What is clinical trial NCT03311503 about?
NCT03311503 is a clinical study titled "Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning". This is a phase I/II open label multi-center study in which patients will receive low dose targeted busulfan followed by infusion of autologous CD34+ selected bone marrow or mobilized peripheral blood cells transduced with the G2SCID vector. Subjects will be enrolled over 3 years and be followed for...
What is the current status of trial NCT03311503?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 12 participants. The study started on 2018-02-26. Estimated completion is 2028-01-01.
What conditions does trial NCT03311503 study?
This clinical trial studies the following conditions: Gene Therapy, Severe Combined Immunodeficiency, X Linked.
What interventions are being tested in trial NCT03311503?
The interventions under investigation include: autologous CD34+ cell transduced with G2SCID vector (BIOLOGICAL).
Who is sponsoring clinical trial NCT03311503?
This trial is sponsored by David Williams, which has 5 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03311503 being conducted?
This trial has 4 study locations across California, Georgia, Massachusetts, Ohio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT03311503's enrollment target sits among peer trials
12 1852nd of 2000 higher than 90 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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