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

Part of paid clinical trials in La Jolla, California.

Sponsor
Insmed Gene Therapy LLC
Study ID
NCT07290062
Phase
PHASE1
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - 79 Years
Healthy Volunteers
Not accepted

Interventions

  • INS1202 — GENETIC
    Suspension for injection.

Study Details

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.

Key Dates

Start date
Jan 9, 2026
Status verified
May 2026
Primary completion
Mar 31, 2030
Completion
Mar 31, 2030

Study Design

Enrollment
23 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Cohort 1
    Participants with sporadic amyotrophic lateral sclerosis (sALS) will be administered INS1202, IT injection at dose level 1 on Day 1.
  • Experimental: Cohort 2
    Participants with either sALS or SOD1-ALS, will be administered INS1202 via IT injection at dose level 2 on Day 1.
  • Experimental: Cohort 3
    Participants with either sALS or SOD1-ALS, will be administered INS1202 via IT injection at dose level 3 on Day 1.

Primary Outcome Measure

Incidence and Severity of Treatment Emergent Adverse Events (TEAEs) [ Time Frame: Up to 48 weeks ]

Central Contacts

Locations (5)

FacilityCityStateZIPSite coordinators
USA004La JollaCalifornia92037
USA002Palo AltoCalifornia94304
USA001ColumbiaMissouri65211
USA007ColumbusOhio43221
USA006PhiladelphiaPennsylvania19107

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