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INS1201 · 1 trial · 1 indication
| 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...
INS1201 is an investigational gene therapy being developed by Insmed Incorporated for Duchenne Muscular Dystrophy, a genetic muscle-wasting disorder. It is administered as a single intrathecal injection, meaning it is delivered into the fluid surrounding the spinal cord. INS1201 is in Phase 1 clinical development and has not been approved by the FDA.
INS1201 is being studied as a treatment for Duchenne Muscular Dystrophy in ambulatory males. The Phase 1 trial enrolls males aged 2 years and older in the United States. Because it remains investigational, INS1201 is not approved for any indication and is available only through clinical research.
INS1201 is being developed by Insmed Incorporated, a biopharmaceutical company whose common stock trades on Nasdaq under the ticker INSM. Insmed is the sponsor of the Phase 1 study evaluating the gene therapy in ambulatory males with Duchenne Muscular Dystrophy.
INS1201 is in Phase 1 clinical development. Its single registered trial is a Phase 1 study of a one-time intrathecal injection in ambulatory males with Duchenne Muscular Dystrophy. The study is currently active but not recruiting, and INS1201 has not received FDA approval.
INS1201 is being evaluated in one clinical trial, NCT06817382, titled a study to investigate the safety and biodistribution of a single intrathecal injection of INS1201 in ambulatory males with Duchenne Muscular Dystrophy. The Phase 1 trial has a planned enrollment of 12 participants, is controlled, and is active but not recruiting.