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

Insmed Gene Therapy LLC · NCT07290062

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Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.

Key facts

Compensation
Not posted. Ask the study team before screening.
Who can join
Ages 18–79 · All sexes
Healthy volunteers
Not accepted (a condition is required)
Study type
Interventional · Phase 1
Target enrollment
23
US sites recruiting
7
Study start
January 9, 2026
Record last updated
September 23, 2026

Status re-checked against ClinicalTrials.gov within the last 12 hours.

What the study is about

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.

Being studied: INS1202

Conditions: Amyotrophic Lateral Sclerosis

Eligibility criteria

Copied from the official record. The study team makes the final decision at screening.

Show full criteria

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.

Recruiting US locations

  • USA004

    La Jolla, CA 92037

    Site contact: rprevite@health.ucsd.edu

  • USA002

    Palo Alto, CA 94304

    Site contact: NEUROMUSCULARRESEARCH@STANFORD.EDU

  • USA003

    Baltimore, MD 21205

    Site contact: emosmil1@jhmi.edu

  • USA009

    Worcester, MA 01655

    Site contact: danielle.kokoski@umassmed.edu

  • USA001

    Columbia, MO 65211

    Site contact: heathermchatton@health.missouri.edu

  • USA007

    Columbus, OH 43221

    Site contact: ALSResearch@osumc.edu

  • USA006

    Philadelphia, PA 19107

    Site contact: Alexander.McGeary@jefferson.edu

Study contacts (from the official record)

  • Insmed Medical Information · 1-844-446-7633 · medicalinformation@insmed.com
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A Study to Investigate the Safety and Pharmacodynamics of a Single… (NCT07290062) | Bored Studies