Facioscapulohumeral Muscular Dystrophy (FSHD)
Condition Description
Facioscapulohumeral muscular dystrophy (FSHD) is a rare genetic muscle disease that affects the muscles of your child's face, shoulders, upper arms, and lower legs. These muscles weaken and shrink (atrophy).
Active Trials
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TRIAL TITLE: A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Adult Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)
DESCRIPTION: A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Adult Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)
SPONSOR: Avidity Biosciences
INDICATION: Facioscapulohumeral Muscular Dystrophy (FSHD)
STUDY PROTOCOL: AOC 1020-CS1
PHASE: 1/2
STATUS: Active
RECRUITING PATIENTS: No
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TRIAL TITLE: A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Adult Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)
DESCRIPTION: AOC 1020-CS1 is a first-in-human, 3-part, multi-center, Phase 1/2, randomized, double-blind, placebo-controlled study designed to evaluate safety, tolerability, pharmacokinetics and to explore pharmacodynamics and efficacy of single and multiple-doses of AOC 1020 administered intravenously in adult participants with FSHD Type 1 (FSHD1) and FSHD Type 2 (FSHD2).
SPONSOR: Avidity Biosciences
INDICATION: Facioscapulohumeral Muscular Dystrophy (FSHD)
STUDY PROTOCOL: AOC 1020-CS2
PHASE: 1/2
STATUS: Active
RECRUITING PATIENTS: No