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A Phase 1/2/3 Open-label, Single Arm, Dose-finding Study to Investigate Long-term Safety, Tolerability and Efficacy of GS-100, an Adeno-associated Virus Serotype 9 (AAV9) Vector-mediated Gene Transfer of Human NGLY1, in Patients With NGLY1 Deficiency
Conditions
Interventions
GS-100
Locations
3
United States
Oakland Children's Hospital (UCSF Benioff)
Oakland, California, United States
Columbia University Irving Medical Center
New York, New York, United States
Texas Children's Hospital (Baylor College of Medicine)
Houston, Texas, United States
Start Date
February 13, 2024
Primary Completion Date
January 31, 2028
Completion Date
January 31, 2028
Last Updated
April 24, 2025
Lead Sponsor
Grace Science, LLC
Data Source & Attribution
This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.
Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.
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