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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: Recruiting Phase 1 study of NGLY1 Deficiency, sponsored by Grace Science.
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, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (90% lower). The trial reports 3 study locations across 3 states. According to ClinicalTrials.gov, the official US trial registry.
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.
Primary Outcome
Incidence of adverse events (AEs) and serious AEs (SAEs)
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 |
What NCT06199531 shows while recruiting
NCT06199531 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 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 a single indexed study location in 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.
Learn More About Clinical Trials
Similar trials for NGLY1 Deficiency
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT06199531's enrollment target sits among peer trials
6 1975th 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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