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NCT Number: NCT07290062

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

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.

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Key information

Conditions

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

Age range

18 year–79 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

USA004, La Jolla, California, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

INS1202

Genetic

Suspension for injection.

Primary outcomes

  1. Incidence and Severity of Treatment Emergent Adverse Events (TEAEs)

    Time frame: Up to 48 weeks

Secondary outcomes

  1. Recommended Phase 2 Dose (RP2D) of INS1202-101

    Time frame: Up to 48 weeks

  2. 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

Study contacts

Contact information is provided by the study sponsor or research team.

Insmed Medical Information

CONTACT

[email protected]

1-844-446-7633

Sponsors and collaborators

Lead sponsor

Insmed Gene Therapy LLC

Industry

Registry information

Official study title

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

Acronym: ARMOR

Important dates

Study start
2026
Primary completion
2030
Study completion
2030
First posted
Dec 17, 2025
Registry last updated
Jul 6, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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