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

May 19, 2026 updated by: Insmed Gene Therapy LLC

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

23

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria: -

  • 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.

Key Exclusion Criteria: -

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

Note: Other protocol-defined inclusion/exclusion criteria may apply.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cohort 1
Participants with sporadic amyotrophic lateral sclerosis (sALS) will be administered INS1202, IT injection at dose level 1 on Day 1.
Genetic: INS1202
Suspension for injection.
Experimental: Cohort 2
Participants with either sALS or SOD1-ALS, will be administered INS1202 via IT injection at dose level 2 on Day 1.
Genetic: INS1202
Suspension for injection.
Experimental: Cohort 3
Participants with either sALS or SOD1-ALS, will be administered INS1202 via IT injection at dose level 3 on Day 1.
Genetic: INS1202
Suspension for injection.

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure
Time Frame
Recommended Phase 2 Dose (RP2D) of INS1202-101
Time Frame: Up to 48 weeks
Up to 48 weeks
Viral Vector Shedding Following the IT Administration of INS1202 by Droplet Digital Polymerase Chain Reaction (ddPCR)
Time Frame: Baseline, Week 1, Week 2, Week 4, Week 16 and Week 32
Baseline, Week 1, Week 2, Week 4, Week 16 and Week 32

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Insmed Gene Therapy LLC

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 9, 2026

Primary Completion (Estimated)

March 31, 2030

Study Completion (Estimated)

March 31, 2030

Study Registration Dates

First Submitted

December 4, 2025

First Submitted That Met QC Criteria

December 4, 2025

First Posted (Actual)

December 17, 2025

Study Record Updates

Last Update Posted (Actual)

May 20, 2026

Last Update Submitted That Met QC Criteria

May 19, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • INS1202-101

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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