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

Overview

Official 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

Brief title

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

Registration type

ClinicalTrials.gov

NCT number

NCT07290062

Status

Recruiting

Study category

Interventional, Drug trial, Genetic

Sponsor / center

Insmed Gene Therapy LLC

Research center

Not stated in the official study record.

Collaborators

Not stated in the official study record.

Study type

Interventional study

Phase

Phase 1

Enrollment

23

Start date

01-09-2026

Primary completion date

03-31-2030

Estimated / actual completion date

03-31-2030

Last source update

09-23-2026

What is being studied?

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.

Intervention(s)

Who may be eligible?

This is a simplified summary. The official study team or research center determines eligibility.

Population / disease status

Familial / genetic ALS, Sporadic ALS, Diagnosed ALS

Diagnosis / conditions

Amyotrophic Lateral Sclerosis

Age

18 Years to 79 Years

Disease duration

> 36 months accepted

Respiratory criteria

Not stated in the official study record.

ALSFRS-R criteria

The official eligibility criteria include language relevant to this topic; review the full criteria below.

Genetic criteria

The official eligibility criteria include language relevant to this topic; review the full criteria below.

Medication requirements

The official eligibility criteria include language relevant to this topic; review the full criteria below.

Major inclusion and exclusion criteria from the official record

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.

Genetics

Gene-specific study?

Yes

Gene or variant

SOD1

Genetic test required?

Not stated in the official study record.

Known carrier required?

Not stated

Confirmed pathogenic variant required?

Not stated in the official study record.

Presymptomatic carriers eligible?

Not stated in the official study record.

At-risk relative eligible?

Not stated

Genetic test provided?

Not stated in the official study record.

Genetic counseling provided?

Not stated in the official study record.

Results returned / offered?

Not stated

Family history required?

Not stated in the official study record.

Study design

Randomized?

Not stated in the official study record.

Allocation

Not stated in the official study record.

Intervention model

Not stated in the official study record.

Masking

Not stated in the official study record.

Placebo

Not stated in the official study record.

Primary purpose

Not stated in the official study record.

Endpoints

Primary outcome measures

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

Secondary outcome measures

• Recommended Phase 2 Dose (RP2D) of INS1202-101
• Viral Vector Shedding Following the IT Administration of INS1202 by Droplet Digital Polymerase Chain Reaction (ddPCR)

A biomarker outcome should not automatically be interpreted as a clinical outcome.

Locations

Contact

Central contact: Insmed Medical Information · medicalinformation@insmed.com · 1-844-446-7633