A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)
NCT ID: NCT07290062
Last Updated: 2025-12-17
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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NOT_YET_RECRUITING
PHASE1
23 participants
INTERVENTIONAL
2025-12-31
2030-03-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
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Cohort 1
Participants with sporadic amyotrophic lateral sclerosis (sALS) will be administered INS1202, IT injection at dose level 1 on Day 1.
INS1202
Suspension for injection.
Cohort 2
Participants with either sALS or SOD1-ALS, will be administered INS1202 via IT injection at dose level 2 on Day 1.
INS1202
Suspension for injection.
Cohort 3
Participants with either sALS or SOD1-ALS, will be administered INS1202 via IT injection at dose level 3 on Day 1.
INS1202
Suspension for injection.
Interventions
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INS1202
Suspension for injection.
Eligibility Criteria
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Inclusion Criteria
* 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.
Exclusion Criteria
* Any investigational medication or treatment (for ALS or other condition).
18 Years
79 Years
ALL
No
Sponsors
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Insmed Gene Therapy LLC
INDUSTRY
Responsible Party
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Central Contacts
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Other Identifiers
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INS1202-101
Identifier Type: -
Identifier Source: org_study_id