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

NCT06199531 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2026-05-04

No results posted yet for this study

Summary

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.

Conditions

  • NGLY1 Deficiency

Interventions

GENETIC

GS-100

A single intracerebroventricular (ICV) dose of GS-100

Sponsors & Collaborators

  • Grace Science, LLC

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
2 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-02-13
Primary Completion
2027-03-31
Completion
2031-03-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06199531 on ClinicalTrials.gov