A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: INS1202.
- Who it may be relevant to
- Registry conditions: Amyotrophic Lateral Sclerosis. Basic parameters: 18 years — 79 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
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
Overview
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.
Interventions
- Genetic INS1202
Suspension for injection.
Primary outcome measures
- Incidence and Severity of Treatment Emergent Adverse Events (TEAEs) [Time frame: Up to 48 weeks]
Secondary outcome measures (2)
- Recommended Phase 2 Dose (RP2D) of INS1202-101 [Time frame: 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]
Eligibility 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Non-randomized
- Model
- Sequential
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 5 centers
- USA004 — La Jolla
- USA002 — Palo Alto
- USA001 — Columbia
- USA007 — Columbus
- USA006 — Philadelphia
Identifiers
NCT: NCT07290062 · INS1202-101