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NCT03897361 · ClinicalTrials.gov registry record · Phase 1
Stem Cell Gene Therapy for Cystinosis
A Phase 1 study, sponsored by University of California, San Diego.
- Completed
- Registry status
- Phase 1
- Development phase
- 6
- Enrollment target
NCT03897361: Completed Phase 1 study, sponsored by University of California, San Diego.
NCT03897361 is a Phase 1 study that has completed, run by University of California, San Diego. The registered enrollment target is 6 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (90% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03897361, a Phase 1 study, has completed, sponsored by University of California, San Diego.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 6 participants
- Enrollment target
Study Summary
This study is a Phase 1/2 clinical trial that will assess the safety and efficacy of enriched gene-corrected hematopoietic stem cells isolated from patients affected with cystinosis. (Investigational Product: CTNS-RD-04 or CTNS-RD-04-LB, where the suffix "-LB" stands for LentiBOOST)
Primary Outcome
The total count of all adverse events (AEs) recorded for each dosed participant reported at any scheduled study visit starting with the first pre-dose safety visit (i.e., the first visit after informed consent and before the first administration of study drug) and continuing through the end of the treatment period. Events were coded using MedDRA and graded according to NCI CTCAE criteria. The outcome is the total count of AEs per participant.
Interventions
- GENETIC CTNS-RD-04 or CTNS-RD-04-LB (where the suffix "-LB" stands for LentiBOOST)
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 6 participants |
| Start Date | 2019-07-08 |
| Est. Completion | 2024-09-18 |
| Phase | Phase 1 |
What the finished NCT03897361 record still lists
NCT03897361 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 6 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (90% lower).
The record links to 0 conditions, and to 1 intervention - of which CTNS-RD-04 or CTNS-RD-04-LB (where the suffix "-LB" stands for LentiBOOST) is the first listed.
NCT03897361 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03897361 about?
NCT03897361 is a clinical study titled "Stem Cell Gene Therapy for Cystinosis". This study is a Phase 1/2 clinical trial that will assess the safety and efficacy of enriched gene-corrected hematopoietic stem cells isolated from patients affected with cystinosis. (Investigational Product: CTNS-RD-04 or CTNS-RD-04-LB, where the suffix "-LB" stands for LentiBOOST)
What is the current status of trial NCT03897361?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 6 participants. The study started on 2019-07-08. Estimated completion is 2024-09-18.
What interventions are being tested in trial NCT03897361?
The interventions under investigation include: CTNS-RD-04 or CTNS-RD-04-LB (where the suffix "-LB" stands for LentiBOOST) (GENETIC).
Who is sponsoring clinical trial NCT03897361?
This trial is sponsored by University of California, San Diego, which has 775 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03897361's enrollment target sits among peer trials
6 1974th of 2000 higher than 12 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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