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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
Led by Insmed Gene Therapy LLC · Updated on 2026-05-20
23
Participants Needed
5
Research Sites
N/A
Total Duration
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AI-Summary
What this Trial Is About
Researchers are investigating the safety, tolerability, and pharmacodynamics of a single dose of INS1202 given by intrathecal injection in adults aged 18 to under 80 years with amyotrophic lateral sclerosis (ALS). This study includes participants with ALS who either carry mutations in the superoxide dismutase type 1 (SOD1) gene or have no known ALS-related genetic mutations. The study is a phase 1, open-label dose-finding trial aimed at understanding how the drug behaves and its safety profile in this population. Participants will receive a single intrathecal injection of INS1202 at one of three dose levels on Day 1. The first dose level is given only to participants with sporadic ALS, while the second and third dose levels include participants with either sporadic ALS or SOD1-ALS. This dosing strategy helps evaluate different amounts of the study drug in these groups. During the study, participants will be monitored for up to 48 weeks to assess the incidence and severity of any adverse events related to the treatment. Researchers will also evaluate the recommended phase 2 dose and monitor viral vector shedding at multiple time points, including baseline, weeks 1, 2, 4, 16, and 32. This comprehensive monitoring ensures careful assessment of safety and drug behavior throughout the study period.
CONDITIONS
Brief Title
A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participant with body mass index (BMI) ≥18 kilograms per square meter (kg/m²).
- Participant with symptomatic ALS 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 familial ALS mutations.
- Any polymorphism or mutation in the coding region requires Sponsor review for study compatibility.
- Baseline ALS Functional Rating Scale-Revised (ALSFRS-R) score ≥24.
- ALS disease duration ≤42 months.
You will not qualify if you...
- Previous treatment for ALS with cellular or gene therapies.
- Any investigational medication or treatment for ALS or other conditions.
- Other protocol-defined inclusion or exclusion criteria may apply.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single day (Day 1)
Participants receive a single intrathecal injection of INS1202 at one of three dose levels depending on their cohort assignment.
1 treatment visit (in-person)
Duration - Up to 48 weeks
Participants are monitored for safety, pharmacodynamics, and viral vector shedding after treatment.
Visits at Baseline, Week 1, Week 2, Week 4, Week 16, and Week 32
Trial Site Locations
Total: 5 locations
1
USA004
La Jolla, California, United States, 92037
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2
USA002
Palo Alto, California, United States, 94304
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3
USA001
Columbia, Missouri, United States, 65211
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4
USA007
Columbus, Ohio, United States, 43221
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5
USA006
Philadelphia, Pennsylvania, United States, 19107
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Research Team
I
Insmed Medical Information
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
3
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