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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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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1 | Experimental | Participants with sporadic amyotrophic lateral sclerosis (sALS) will be administered INS1202, IT injection at dose level 1 on Day 1. |
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| Cohort 2 | Experimental | Participants with either sALS or SOD1-ALS, will be administered INS1202 via IT injection at dose level 2 on Day 1. |
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| Cohort 3 | Experimental | Participants with either sALS or SOD1-ALS, will be administered INS1202 via IT injection at dose level 3 on Day 1. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| INS1202 | Genetic | Suspension for injection. |
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| Measure | Description | Time Frame |
|---|---|---|
| Incidence and Severity of Treatment Emergent Adverse Events (TEAEs) | Up to 48 weeks |
| Measure | Description | 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 |
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Key Inclusion Criteria: -
Key Exclusion Criteria: -
Note: Other protocol-defined inclusion/exclusion criteria may apply.
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Insmed Medical Information | Contact | 1-844-446-7633 | medicalinformation@insmed.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| USA004 | Recruiting | La Jolla | California | 92037 | United States |
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| Label | URL |
|---|---|
| Click here to access a lay person friendly information about this clinical trial. | View source |
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| USA002 | Recruiting | Palo Alto | California | 94304 | United States |
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| USA001 | Recruiting | Columbia | Missouri | 65211 | United States |
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| USA007 | Recruiting | Columbus | Ohio | 43221 | United States |
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| USA006 | Recruiting | Philadelphia | Pennsylvania | 19107 | United States |
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| ID | Term |
|---|---|
| D000690 | Amyotrophic Lateral Sclerosis |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
| D019636 | Neurodegenerative Diseases |
| D057177 | TDP-43 Proteinopathies |
| D009468 | Neuromuscular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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