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

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)

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

DRUG

AOC 1020

AOC 1020 will be administered via intravenous (IV) infusion

DRUG

Placebo

Placebo will be administered via intravenous (IV) infusion

Sponsors & Collaborators

• Avidity Biosciences, Inc.

  lead INDUSTRY

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Model

SEQUENTIAL

Eligibility

Min Age

16 Years

Max Age

70 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2023-04-04

Primary Completion

2026-03-18

Completion

2026-03-20

FDA Drug

Yes

Countries

• United States
• Canada
• United Kingdom

Study Locations