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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)

RecruitingPhase 1Ages 18 Years - 79 YearsAllInsmed Gene Therapy LLC
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In plain words

What the trial is, and whether it could suit you

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 โ‰ฅ 18 to \<80 years of age with ALS who carry superoxide dismutase type 1 (SOD1) mutations or harbor no known ALS-related genetic mutation.

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
Key Inclusion Criteria: - * Participant with body mass index (BMI) โ‰ฅ18 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 โ‰ฅ 24. * ALS disease duration โ‰ค 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.
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What it tests

  • INS1202
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Where

7 sites
USA004
La Jolla, United States
USA002
Palo Alto, United States
USA003
Baltimore, United States
USA009
Worcester, United States
USA001
Columbia, United States
USA007
Columbus, United States
USA006
Philadelphia, United States
๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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