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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 is a Phase 1 study that has completed, run by Emily de los Reyes. The registered enrollment target is 13 participants.
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).
Interventions
- GENETIC AT-GTX-501
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 13 participants |
| Start Date | 2016-05 |
| Est. Completion | 2024-10 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT02725580
The ClinicalTrials.gov registry entry for NCT02725580 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 13 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Emily de los Reyes, which has 4 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which AT-GTX-501 is the first listed.
NCT02725580 reports 0 study locations.
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.
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