Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06199531 | Safety and Efficacy of GS-100 Gene Therapy in Patients With NGLY1 Deficiency | PHASE3 | ACTIVE NOT_RECRUITING | 10 | — | — | Feb 13, 2024 | Mar 1, 2031 | May 4, 2026 | 2 | United States |
Incidence of adverse events (AEs) and serious AEs (SAEs)
Improvement in one or more domains of the 88-item Gross Motor Function Measure (GMFM-88) from Baseline to Week 52
| Arm | Type | Description |
|---|---|---|
| Cohort 1 | EXPERIMENTAL | GS-100 Low Dose Level: 4e14 total vector genomes (vg) for 6-18-year-olds (fully enrolled, 2 participants) |
| Cohort 2 | EXPERIMENTAL | GS-100 Mid Dose Level: 1e15 total vector genomes (vg) for 13-18-year-olds, 8.7e14 total vg for 2-5-year-olds (fully enrolled, 2 participants) |
| Cohort 3 | EXPERIMENTAL | GS-100 High Dose Level: 3e15 total vector genomes (vg) for 6-18-year-olds, 2.6e15 vg for 2-5-year-olds (fully enrolled, 2 participants) |
| Cohort 4 | EXPERIMENTAL | GS-100 Intermediate Dose Level: 2e15 total vector genomes (vg) for 6-18-year-olds, 1.75e15 total vg for 2-5-year-olds (fully enrolled, 1 participant) |
| Pivotal Cohort | EXPERIMENTAL | GS-100 Selected Dose Level: 1e15 total vector genomes (vg) for 6-18-year-olds, 8.7e14 total vg for 2-5-year-olds (fully enrolled, 3 participants) |
| Name | Type | Description |
|---|---|---|
| GS-100 | GENETIC | A single intracerebroventricular (ICV) dose of GS-100 |
Inclusion Criteria: * Patients must be 2 to 18 years of age, inclusive, at the time of signing the informed consent form (ICF) * Patients with a documented diagnosis of NGLY1 Deficiency based on detection of biallelic variants in the NGLY1 gene via molecular genetic sequencing * Elevated GNA levels...