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

NCT05747924 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: AOC 1020-CS1
• Study Type: interventional
• Target: 90
• Locations: 3 countries
• Sites: 17

Brief 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

Muscular Dystrophy, Facioscapulohumeral; 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; 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

Keywords

FORTITUDE; Avidity; Avidity Biosciences; AOC 1020

Outcome Measures

Primary Outcomes (2)

• Incidence of treatment-emergent adverse events (Cohorts A & B)

  Through study completion, up to Day 365

• Change in plasma KHDC1L (Part C)

  Ratio to Baseline

  Across months 3 to 12

Secondary Outcomes (5)

• Plasma pharmacokinetic (PK) parameters of AOC 1020 (Cohorts A & B)

  Through study completion; up to Day 365

• Plasma pharmacokinetic (PK) parameters of AOC 1020 (Cohorts A & B)

  Through study completion; up to Day 365

• Plasma pharmacokinetic (PK) parameters of AOC 1020 (Cohorts A & B)

  Through study completion; up to Day 365

• Muscle drug concentration (Cohorts A & B)

  Day 120

• Change in circulating creatine kinase (Cohort C)

  Across months 3 to 12

Study Arms (5)

AOC 1020 Regimen 1

EXPERIMENTAL

Cohort A: AOC 1020 Dose Regimen 1; Five doses administered intravenously over 9 months

Drug: AOC 1020

AOC 1020 Regimen 2

EXPERIMENTAL

Cohort B1: AOC 1020 Dose Regimen 2; Five doses administered intravenously over 9 months

Drug: AOC 1020

AOC 1020 Regimen 3

EXPERIMENTAL

Cohort C: AOC 1020 Dose Regimen 3; Eight doses administered intravenously over approximately 10 months

Drug: AOC 1020

Placebo (Saline) Regimen 1

PLACEBO COMPARATOR

Cohort A \& B: Placebo; Five doses administered intravenously over 9 months

Drug: Placebo

Placebo (Saline) Regimen 2

PLACEBO COMPARATOR

Cohort C: Placebo; Eight doses administered intravenously over approximately 10 months

Drug: Placebo

Interventions

AOC 1020 DRUG

AOC 1020 will be administered via intravenous (IV) infusion

AOC 1020 Regimen 1; AOC 1020 Regimen 2; AOC 1020 Regimen 3

Placebo DRUG

Placebo will be administered via intravenous (IV) infusion

Also known as: Saline

Placebo (Saline) Regimen 1; Placebo (Saline) Regimen 2

Eligibility Criteria

• Age: 16 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• FSHD1 or FSHD2 diagnosis confirmed by documented genetic testing (testing provided by Sponsor)
• Ambulatory and able to walk 10 meters (with or without assistive devices such as one cane, walking stick or braces)
• At least 1 muscle region suitable for biopsy (testing provided by Sponsor)
• Muscle weakness in both upper and lower body, as determined by Investigator

You may not qualify if:

• Pregnant or intends to become pregnant while on study, or active breastfeeding
• Unwilling or unable to continue to comply with contraceptive requirements
• Body mass index (BMI) \>35.0 kg/m2 at Screening
• History of muscle biopsy within 30 days of the screening biopsy or planning to undergo any nonstudy muscle biopsies over the duration of the study
• History of bleeding disorders, significant keloid, or other skin or muscle conditions (e.g., severe muscle wasting) that, in the opinion of the Investigator, makes the participant unsuitable for serial muscle biopsy
• Anticipated survival less than 2 years
• Blood or plasma donation within 16 weeks of Study Day 1
• Any contraindication to MRI
• Any abnormal lab values, conditions or diseases that, in the opinion of the investigator or Sponsor, would make the participant unsuitable for the study or could interfere with participation or completion of the study
• Treatment with any investigative medication within 1 month (or 5 half-lives of the drug, whichever is longer) of Screening

Sponsors & Collaborators

• Avidity Biosciences, Inc.: lead

Study Sites (17)

University of California Los Angeles

Los Angeles, California, 90095, United States

Location

Stanford University

Palo Alto, California, 94304, United States

Location

University of California San Diego

San Diego, California, 92093, United States

Location

University of Colorado

Denver, Colorado, 80045, United States

Location

University of Florida

Gainesville, Florida, 32608, United States

Location

Rare Disease Research

Atlanta, Georgia, 30329, United States

Location

Kansas University Medical Center

Kansas City, Kansas, 66205, United States

Location

University of Rochester Medical Center

Rochester, New York, 14642, United States

Location

Duke University

Durham, North Carolina, 27708, United States

Location

Ohio State University

Columbus, Ohio, 43221, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

University of Texas Southwestern

Dallas, Texas, 75390, United States

Location

Virginia Commonwealth University

Richmond, Virginia, 23298, United States

Location

University of Washington

Seattle, Washington, 98104, United States

Location

University of Ottawa

Ottawa, Ontario, K1Y 4E9, Canada

Location

University College London

London, WIT 7HA, United Kingdom

Location

University of Sheffield

Sheffield, S10 2TN, United Kingdom

Location

Related Links

• Avidity Biosciences Website
• FORTITUDE Study Website

MeSH Terms

Conditions

Muscular Dystrophy, Facioscapulohumeral; Facioscapulohumeral Muscular Dystrophy 1B; Muscular Dystrophies; Facioscapulohumeral muscular dystrophy 1a

Interventions

Sodium Chloride

Condition Hierarchy (Ancestors)

Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 17, 2023
• First Posted: February 28, 2023
• Study Start: April 4, 2023
• Primary Completion: March 18, 2026
• Study Completion: March 20, 2026
• Last Updated: April 24, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

GB (2); US (14); CA (1)