Summary
AOC 1001-CS2 (MARINA-OLE) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) study to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients
Official Title
A Phase 2 Extension Study to Evaluate the Long-Term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients
Details
This study will continue to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of AOC 1001 in participants that enrolled in the randomized, placebo-controlled, First-In-Human Phase 1/2 AOC 1001-CS1 (MARINA) clinical study.
Participants from AOC 1001-CS1 are eligible to enroll in AOC 1001-CS2 if they have satisfactorily completed AOC 1001-CS1.
The total duration of active treatment in AOC 1001-CS2 is up to 58 months. Once participants have completed active treatment, they will be followed through an 8-week safety follow-up period. The sponsor may extend active treatment beyond 58 months at a future timepoint.
As of September 2024, the dosing regimen was updated to every 8 weeks.
Keywords
DM1, Muscular Dystrophies, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Disorders, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, Neurodegenerative Diseases, Myotonic Dystrophy Type 1 (DM1), DM, Dystrophy Myotonic, Steinert Disease, MARINA-OLE, MARINA, Avidity Biosciences, Avidity, AOC 1001, Myotonic Muscular Dystrophy, Atrophic Muscular Disorders, Nervous System Heredodegenerative Disorders, Inborn Genetic Diseases
