Health Trials

Clinical trials

2

Search and review clinical trials. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Recruiting

A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)

The primary objective of this study is to evaluate the safety and tolerability of a single dose of INS1201 via IT administration in ambulatory male participants with DMD.

Participants needed: 12
Trial details
Phase: Phase 1Age: 2-4Biological sex: MaleType: InterventionalSponsor: Insmed Gene Therapy LLCUpdated: Jul 28, 2026Locations: 10
Eligibility criteria

Participant must be male at birth, 3 to <5 years of age, inclusive (Part 1) and... [+4]

Prior treatment with gene or cell-based therapy at any time. [+12]

Read more
Status: Recruiting

A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)

The primary objective of this dose-finding study is to evaluate the safety, tolerability and pharmacodynamics of single dose of INS1202 via IT administration in participants ≥ 18 to \<80 years of age with ALS who carry superoxide dismutase type 1 (SOD1) mutations or harbor no known ALS-related genetic mutation.

Participants needed: 23
Trial details
Phase: Phase 1Age: 18-79Biological sex: AllType: InterventionalSponsor: Insmed Gene Therapy LLCUpdated: Jul 6, 2026Locations: 5
Eligibility criteria

Participant with body mass index (BMI) ≥18 kilograms per square meter (kg/m²). [+6]

Previous treatment for ALS with cellular or gene therapies. [+1]

Read more