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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06817382 | A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD) | PHASE1 | RECRUITING | 12 | — | — | Jul 22, 2025 | Mar 31, 2028 | May 18, 2026 | 10 | United States |
| Arm | Type | Description |
|---|---|---|
| Part 1: Cohort 1 | EXPERIMENTAL | Participants aged 3 to \<5 years will receive a single dose level 1 of INS1201 by IT injection on Day 1. |
| Part 1: Cohort 2 | EXPERIMENTAL | Participants aged 3 to \<5 years will receive a single dose level 2 of INS1201 by IT injection on Day 1. |
| Part 2: Cohort 3 | EXPERIMENTAL | Participants aged 2 to \<3 years will receive a single dose level 1 of INS1201 by IT injection on Day 1. |
| Part 2: Cohort 4 | EXPERIMENTAL | Participants aged 2 to \<3 years will receive a single dose level 2 of INS1201 by IT injection on Day 1. |
| Name | Type | Description |
|---|---|---|
| INS1201 | GENETIC | Suspension for IT injection. |
Inclusion Criteria * Participant must be male at birth, 3 to \<5 years of age, inclusive (Part 1) and 2 to \<3 years of age (Part 2), at the time of legally authorized representative (LAR) signing and dating the informed consent form. * Ambulatory -as defined as the ability to walk at least 10 mete...