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

NCT07290062 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: INS1202-101
• Study Type: interventional
• Target: 23
• Locations: 1 country
• Sites: 5

Brief Summary

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.

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (1)

• Incidence and Severity of Treatment Emergent Adverse Events (TEAEs)

  Up to 48 weeks

Secondary Outcomes (2)

• Recommended Phase 2 Dose (RP2D) of INS1202-101

  Up to 48 weeks

• Viral Vector Shedding Following the IT Administration of INS1202 by Droplet Digital Polymerase Chain Reaction (ddPCR)

  Baseline, Week 1, Week 2, Week 4, Week 16 and Week 32

Study Arms (3)

Cohort 1

EXPERIMENTAL

Participants with sporadic amyotrophic lateral sclerosis (sALS) will be administered INS1202, IT injection at dose level 1 on Day 1.

Genetic: INS1202

Cohort 2

EXPERIMENTAL

Participants with either sALS or SOD1-ALS, will be administered INS1202 via IT injection at dose level 2 on Day 1.

Genetic: INS1202

Cohort 3

EXPERIMENTAL

Participants with either sALS or SOD1-ALS, will be administered INS1202 via IT injection at dose level 3 on Day 1.

Genetic: INS1202

Interventions

INS1202 GENETIC

Suspension for injection.

Cohort 1; Cohort 2; Cohort 3

Eligibility Criteria

• Age: 18 Years - 79 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• Previous treatment for ALS with cellular or gene therapies.
• Any investigational medication or treatment (for ALS or other condition).

Sponsors & Collaborators

• Insmed Gene Therapy LLC: lead

Study Sites (5)

USA004

La Jolla, California, 92037, United States

RECRUITING

USA002

Palo Alto, California, 94304, United States

RECRUITING

USA001

Columbia, Missouri, 65211, United States

RECRUITING

USA007

Columbus, Ohio, 43221, United States

RECRUITING

USA006

Philadelphia, Pennsylvania, 19107, United States

RECRUITING

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

Insmed Medical Information

CONTACT

1-844-446-7633; medicalinformation@insmed.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 4, 2025
• First Posted: December 17, 2025
• Study Start: January 9, 2026
• Primary Completion (Estimated): March 31, 2030
• Study Completion (Estimated): March 31, 2030
• Last Updated: April 29, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (5)