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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Safety and Efficacy of GS-100 Gene Therapy in Patients With NGLY1 Deficiency
NCTID
NCT06199531
(View at clinicaltrials.gov)
Description
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.
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Indication
NGLY1 Deficiency
Compound Name
GS-100 (AAV9-CAG-coNGLY1)
Sponsor
Grace Science, LLC
Funder Type
Industry
Status
Recruiting
Enrollment Count
12
Therapy Information
Target Gene/Variant
NGLY1
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Intracerebroventricular
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV9
Editor Type
none
Dose 1
Undisclosed low dose
Dose 2
Undisclosed medium dose
Dose 3
Undisclosed high dose
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2023-11-21
Completion Date
2028-01-31
Last Update
2024-06-04
Participation Criteria
Eligible Age
2 Years - 18 Years
Standard Ages
Child, Adult
Eligible Sex
ALL
Locations
No.of Trial Sites
2
Locations
United States
Regulatory Information
Has US IND
True
Recent Updates
Selected for START program (6/3/24)
Resources/Links
News and Press Releases
Grace Science, LLC Selected by FDA to Participate in the START Pilot Program for GS-100 Gene Therapy for NGLY1 Deficiency and Announcement of the Successful Treatment of the 2nd Patient
This Father Founded a Medical Research Startup to Save His Kids Life
Preclinical Publications
AAV9-NGLY1 gene replacement therapy improves phenotypic and biomarker endpoints in a rat model of NGLY1 Deficiency
GlcNAc-Asn is a biomarker for NGLY1 deficiency