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NCT06199531 · ClinicalTrials.gov registry record · Phase 1
Safety and Efficacy of GS-100 Gene Therapy in Patients With NGLY1 Deficiency
A Phase 1 study of NGLY1 Deficiency, sponsored by Grace Science.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 6
- Enrollment target
- 3
- Study locations
NCT06199531 is a Phase 1 study of NGLY1 Deficiency that is actively recruiting participants, run by Grace Science. The registered enrollment target is 6 participants. The trial reports 3 study locations across 3 states.
The verdict
NCT06199531, a Phase 1 study of NGLY1 Deficiency, is actively recruiting participants, sponsored by Grace Science.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 6 participants
- Enrollment target
- 3
- Study locations
Study Summary
A non-randomized, open-label, dose escalation study of a single intracerebroventricular (ICV) administration of a gene replacement therapy in subjects who are 2 to 18 years old with NGLY1 Deficiency.
Conditions Studied
Interventions
- GENETIC GS-100
Study Locations (3)
California
- Oakland Children's Hospital (UCSF Benioff) - Oakland
New York
- Columbia University Irving Medical Center - New York
Texas
- Texas Children's Hospital (Baylor College of Medicine) - Houston
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 6 participants |
| Start Date | 2024-02-13 |
| Est. Completion | 2028-01-31 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06199531
The ClinicalTrials.gov registry entry for NCT06199531 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 6 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Grace Science, which has 1 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with NGLY1 Deficiency appearing as the primary indexed condition, and to 1 intervention - of which GS-100 is the first listed.
NCT06199531 reports 3 study locations spanning 3 distinct geographic areas - top geographies include California, New York, Texas.
Frequently Asked Questions
What is clinical trial NCT06199531 about?
NCT06199531 is a clinical study titled "Safety and Efficacy of GS-100 Gene Therapy in Patients With NGLY1 Deficiency". A non-randomized, open-label, dose escalation study of a single intracerebroventricular (ICV) administration of a gene replacement therapy in subjects who are 2 to 18 years old with NGLY1 Deficiency.
What is the current status of trial NCT06199531?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 6 participants. The study started on 2024-02-13. Estimated completion is 2028-01-31.
What conditions does trial NCT06199531 study?
This clinical trial studies the following conditions: NGLY1 Deficiency.
What interventions are being tested in trial NCT06199531?
The interventions under investigation include: GS-100 (GENETIC).
Who is sponsoring clinical trial NCT06199531?
This trial is sponsored by Grace Science, which has 1 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06199531 being conducted?
This trial has 3 study locations across California, New York, Texas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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