NCT06199531View on ClinicalTrials.gov

Safety and Efficacy of GS-100 Gene Therapy in Patients With NGLY1 Deficiency

PHASE1/PHASE2RecruitingINTERVENTIONAL
Enrollment

6

Participants

Timeline

Start Date

February 13, 2024

Primary Completion Date

January 31, 2028

Study Completion Date

January 31, 2028

Conditions
NGLY1 Deficiency
Interventions
GENETIC

GS-100

A single intracerebroventricular (ICV) dose of the study treatment will be administered to patients with confirmed mutations in the NGLY1 gene.

Trial Locations (3)

10032

RECRUITING

Columbia University Irving Medical Center, New York

77030

RECRUITING

Texas Children's Hospital (Baylor College of Medicine), Houston

94609

RECRUITING

Oakland Children's Hospital (UCSF Benioff), Oakland

Sponsors

Lead Sponsor

Grace Science, LLC
All Listed Sponsors
lead

Grace Science, LLC

INDUSTRY

NCT06199531 - Safety and Efficacy of GS-100 Gene Therapy in Patients With NGLY1 Deficiency | Biotech Hunter | Biotech Hunter