NCT07290062RecruitingPhase 1
A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)
A Phase 1, Multicenter, Open-label, Dose-Finding Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection of INS1202 in Patients With Amyotrophic Lateral Sclerosis
Source: ClinicalTrials.gov · Last updated 23 Sept 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 1
- Study type
- Interventional
- Enrollment
- 23 (estimated)
- First submitted
- 4 Dec 2025
- Start date
- 9 Jan 2026
- Primary completion date
- 31 Mar 2030
- Lead sponsor
- Insmed Gene Therapy LLC
- Diseases
- ALS (motor neurone disease)
- NCT number
- NCT07290062
What you commit to
- Study type: Interventional · Phase 1
- Planned participants: 23
- From 9 Jan 2026 to the primary completion date, 31 Mar 2030
- 7 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Previous treatment for ALS with cellular or gene therapies.
Other illness
- Any investigational medication or treatment (for ALS or other condition).
- Note: Other protocol-defined inclusion/exclusion criteria may apply.
Who the study is looking for
- Participant with body mass index (BMI) ≥18 kilograms per square meter (kg/m\^2).
- Participant with symptomatic ALS as diagnosed by Gold Coast diagnostic criteria.
- Sporadic ALS cohorts: Negative testing for known monogenic mutations associated with familial ALS.
- SOD1-ALS (Cohorts 2 and 3 only): Confirmed pathogenic SOD1 mutation, with negative testing for other genetic mutations associated with familial ALS.
- Any polymorphism or mutation in the coding region will require additional review by the Sponsor to determine compatibility with the study intervention.
- Baseline ALSFRS-R ≥ 24.
- ALS disease duration ≤ 42 months.
Age in the registry: 18 years to 79 years · Sex: All · Does not accept healthy volunteers
Sites (7)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| USA003 Coordinator of this site? Confirm its status | Baltimore | United States | Recruiting |
| USA001 Coordinator of this site? Confirm its status | Columbia | United States | Recruiting |
| USA007 Coordinator of this site? Confirm its status | Columbus | United States | Recruiting |
| USA004 Coordinator of this site? Confirm its status | La Jolla | United States | Recruiting |
| USA002 Coordinator of this site? Confirm its status | Palo Alto | United States | Recruiting |
| USA006 Coordinator of this site? Confirm its status | Philadelphia | United States | Recruiting |
| USA009 Coordinator of this site? Confirm its status | Worcester | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.