NCT05747924

Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)

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 Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 90 (actual)
• Sponsor: Avidity Biosciences, Inc. · Industry
• Sex: All
• Age: 16 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

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 Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

Detailed 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 participants with FSHD Type 1 (FSHD1) and FSHD Type 2 (FSHD2). Cohort A comprises a placebo-controlled dose titration cohort (Cohort A1) which includes a nested single and multiple dose schedule. Cohort B comprises a placebo-controlled, nested single ascending dose (SAD)/multiple ascending dose (MAD) cohort (Cohort B1). Cohort C comprises a randomized, placebo-controlled, expansion cohort (Cohort C1). For each of Cohorts A, B, and C the study duration is 12 months as the active treatment period is approximately 9 months for Cohorts A \& B and approximately 10 months for Cohort C followed by a 12-week follow-up period for Cohorts A \& B and a 7-week follow-up period for Cohort C. Once participants have completed active treatment with follow-up through 12 months, they may have the option to participate in a planned open-label extension. If patients do not enroll in the open-label extension, they will be followed for 12-weeks after their last dose of study medication.

Conditions

• FSHD
• FSHD1
• FSHD2
• FMD
• FMD2
• Fascioscapulohumeral Muscular Dystrophy
• Fascioscapulohumeral Muscular Dystrophy Type 1
• Fascioscapulohumeral Muscular Dystrophy Type 2
• Dystrophies, Facioscapulohumeral Muscular
• Dystrophy, Facioscapulohumeral Muscular
• Facioscapulohumeral Muscular Dystrophy 1
• Facioscapulohumeral Muscular Dystrophy 2
• Facio-Scapulo-Humeral Dystrophy
• Atrophy, Facioscapulohumeral
• Atrophies, Facioscapulohumeral
• Facioscapulohumeral Atrophy
• Muscular Dystrophies
• Muscular Dystrophy, Facioscapulohumeral
• FSH Muscular Dystrophy
• Landouzy Dejerine Dystrophy
• Landouzy-Dejerine Muscular Dystrophy
• Dystrophies, Landouzy-Dejerine
• Dystrophy, Landouzy-Dejerine
• Landouzy-Dejerine Syndrome
• Muscular Dystrophy, Landouzy Dejerine
• Progressive Muscular Dystrophy
• FSH

Interventions

Type	Name	Description
DRUG	AOC 1020	AOC 1020 will be administered via intravenous (IV) infusion
DRUG	Placebo	Placebo will be administered via intravenous (IV) infusion

Timeline

• Start date: 2023-04-04
• Primary completion: 2026-10-01
• Completion: 2027-04-01
• First posted: 2023-02-28
• Last updated: 2025-04-01

Locations

17 sites across 3 countries: United States, Canada, United Kingdom

Regulatory

• FDA-regulated drug study