Amyotrophic Lateral Sclerosis
Conditions
Brief summary
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
GENETICINS1202
Suspension for injection.
Sponsors
Insmed Gene Therapy LLC
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 79 Years
Healthy volunteers
No
Inclusion 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/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence and Severity of Treatment Emergent Adverse Events (TEAEs) | Up to 48 weeks |
Secondary
| Measure | Time frame |
|---|---|
| Recommended Phase 2 Dose (RP2D) of INS1202-101 | Up to 48 weeks |
| Viral Vector Shedding Following the IT Administration of INS1202 by Droplet Digital Polymerase Chain Reaction (ddPCR) | Baseline, Week 1, Week 2, Week 4, Week 16 and Week 32 |
Countries
United States
Contacts
CONTACTInsmed Medical Information
Outcome results
None listed