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NCT03538899 · ClinicalTrials.gov registry record · Phase 1
Autologous Gene Therapy for Artemis-Deficient SCID
A Phase 1 study of Severe Combined Immunodeficiency, sponsored by University of California, San Francisco.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 24
- Enrollment target
- 1
- Study location
NCT03538899: Recruiting Phase 1 study of Severe Combined Immunodeficiency, sponsored by University of California, San Francisco.
NCT03538899 is a Phase 1 study of Severe Combined Immunodeficiency that is actively recruiting participants, run by University of California, San Francisco. The registered enrollment target is 24 participants, below the 393-participant average among 5 other Severe Combined Immunodeficiency trials with a reported enrollment target (94% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03538899, a Phase 1 study of Severe Combined Immunodeficiency, is actively recruiting participants, sponsored by University of California, San Francisco.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 24 participants
- Enrollment target
- 1
- Study location
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.
Primary Outcome
Patient survival status and (if applicable) cause of death will be recorded to assess overall survival.
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 |
What NCT03538899 shows while recruiting
NCT03538899 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 24 participants, a relatively small participant target, below the 393-participant average among 5 other Severe Combined Immunodeficiency trials with a reported enrollment target (94% lower).
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.
NCT03538899 reports a single indexed study location in California.
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.
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).
Who is sponsoring clinical trial NCT03538899?
This trial is sponsored by University of California, San Francisco, which has 1,713 total clinical trials registered on ClinicalTrials.gov.
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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