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NCT02725580 · ClinicalTrials.gov registry record · Phase 1
Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease
A Phase 1 study, sponsored by Emily de los Reyes.
- Completed
- Registry status
- Phase 1
- Development phase
- 13
- Enrollment target
NCT02725580: Completed Phase 1 study, sponsored by Emily de los Reyes.
NCT02725580 is a Phase 1 study that has completed, run by Emily de los Reyes. The registered enrollment target is 13 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (78% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02725580, a Phase 1 study, has completed, sponsored by Emily de los Reyes.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 13 participants
- Enrollment target
Study Summary
This is a phase 1/2, open-label, single dose study to evaluate the safety and efficacy of AT-GTX-501 delivered intrathecally into the lumbar spinal cord region of participants with mild to moderate variant late infantile neuronal ceroid lipofuscinosis associated with mutation(s) in the CLN6 gene (vLINCL6 disease).
Primary Outcome
An adverse event (AE) is defined as any untoward medical occurrence in a participant administered a pharmaceutical product, biologic, or medical device (medicinal products). An SAE is an AE occurring during any study phase (for example, baseline, treatment, or follow-up) that fulfils any of the following: results in death; is life-threatening; requires inpatient hospitalization or prolongs existing hospitalization; results in persistent or significant incapacity or substantial disruption of the
Interventions
- GENETIC AT-GTX-501
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 13 participants |
| Start Date | 2016-05 |
| Est. Completion | 2024-10 |
| Phase | Phase 1 |
What the finished NCT02725580 record still lists
NCT02725580 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 13 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (78% lower).
The record links to 0 conditions, and to 1 intervention - of which AT-GTX-501 is the first listed.
NCT02725580 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT02725580 about?
NCT02725580 is a clinical study titled "Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease". This is a phase 1/2, open-label, single dose study to evaluate the safety and efficacy of AT-GTX-501 delivered intrathecally into the lumbar spinal cord region of participants with mild to moderate variant late infantile neuronal ceroid lipofuscinosis associated with mutation(s) in the CLN6 gene (vL...
What is the current status of trial NCT02725580?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 13 participants. The study started on 2016-05. Estimated completion is 2024-10.
What interventions are being tested in trial NCT02725580?
The interventions under investigation include: AT-GTX-501 (GENETIC).
Who is sponsoring clinical trial NCT02725580?
This trial is sponsored by Emily de los Reyes, which has 4 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT02725580's enrollment target sits among peer trials
13 1847th of 2000 higher than 149 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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