Clinical trials: NGLY1 Deficiency
- Condition NGLY1 Deficiency
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
NGLY1 Deficiency
Safety and Efficacy of GS-100 Gene Therapy in Patients With NGLY1 Deficiency
A non-randomized, open-label, Phase 1/2/3 study of a single intracerebroventricular (ICV) administration of a gene replacement therapy (GS-100) in participants who are 2 to 18 years old with NGLY1 Deficiency.
- Trial name
- NCT06199531
- Testing
- GS-100
- Where
- 2 sites in United States
- Volunteers
- 10 people
- Sponsor
- Grace Science, LLC