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Autologous Gene Therapy for Artemis-Deficient SCID
NCT03538899 · View on ClinicalTrials.gov ↗
Study Summary
This study aims to determine if a new method can be used to treat Artemis-deficient Severe Combined Immunodeficiency (ART-SCID), a severe form of primary immunodeficiency caused by mutations in the DCLRE1C gene. This method involves transferring a normal copy of the DCLRE1C gene into stem cells of an affected patient. Participants will receive an infusion of stem cells transduced with a self-inactivating lentiviral vector that contains a normal copy of the DCLRE1C gene. Prior to the infusion they will receive sub-ablative, dose-targeted busulfan conditioning. The study will investigate if the procedure is safe, whether it can be done according to the methods described in the protocol, and whether the procedure will provide a normal immune system for the patient. A total of 24 newly diagnosed patients will be enrolled at the University of California San Francisco in this single-site trial and will be followed for 15 years post-infusion. It is hoped that this type of gene transfer may offer improved outcomes for ART-SCID patients who lack a brother or sister who can be used as a donor for stem cell transplantation or who have failed to develop a functioning immune system after a previous stem cell transplant.
Conditions Studied
Interventions
- DRUG Busulfan
- DRUG AProArt-CD34
- DEVICE CliniMACS® CD34 Reagent System cell sorter device
Study Locations (1)
California
- University of California, San Francisco (UCSF) Children's Hospital — San Francisco
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 24 participants |
| Start Date | 2018-05-31 |
| Est. Completion | 2038-06 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03538899
The ClinicalTrials.gov registry entry for NCT03538899 describes a study currently listed as recruiting. It is categorized as Phase 1, 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 24 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is University of California, San Francis, which has 1,574 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 Severe Combined Immunodeficiency appearing as the primary indexed condition, and to 3 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: NCT03538899 reports 1 study location spanning 1 distinct geographic area — top geographies include California. 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 NCT03538899 about?
NCT03538899 is a clinical study titled "Autologous Gene Therapy for Artemis-Deficient SCID". This study aims to determine if a new method can be used to treat Artemis-deficient Severe Combined Immunodeficiency (ART-SCID), a severe form of primary immunodeficiency caused by mutations in the DCLRE1C gene. This method involves transferring a normal copy of the DCLRE1C gene into stem cells of a...
What is the current status of trial NCT03538899?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 24 participants. The study started on 2018-05-31. Estimated completion is 2038-06.
What conditions does trial NCT03538899 study?
This clinical trial studies the following conditions: Severe Combined Immunodeficiency. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT03538899?
The interventions under investigation include: Busulfan (DRUG), AProArt-CD34 (DRUG), CliniMACS® CD34 Reagent System cell sorter device (DEVICE). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT03538899?
This trial is sponsored by University of California, San Francis, which has 1,574 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT03538899 being conducted?
This trial has 1 study location across California. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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