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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
23
Suspension for injection.
USA004
La Jolla, California, United States
RECRUITINGUSA002
Palo Alto, California, United States
RECRUITINGUSA003
Baltimore, Maryland, United States
Incidence and Severity of Treatment Emergent Adverse Events (TEAEs)
Time frame: Up to 48 weeks
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
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USA009
Worcester, Massachusetts, United States
RECRUITINGUSA001
Columbia, Missouri, United States
RECRUITINGUSA007
Columbus, Ohio, United States
RECRUITINGUSA006
Philadelphia, Pennsylvania, United States
RECRUITING