A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD

NCT07038200 · tracked via the Priya Life Science USA tracker

Sponsor

Avidity Biosciences, Inc.

Phase

Phase 3

Started

2025-06-10

Last updated

2026-07-27

Condition(s) studied

Facioscapulohumeral Muscular DystrophyFSHDFSHD - Facioscapulohumeral Muscular DystrophyFSHD1FSHD2Fascioscapulohumeral Muscular DystrophyFascioscapulohumeral Muscular Dystrophy Type 1Fascioscapulohumeral Muscular Dystrophy Type 2Facioscapulohumeral Muscular Dystrophy 1Facioscapulohumeral DystrophyFacio-Scapulo-Humeral DystrophyFacioscapulohumeral Muscular Dystrophy 2Facioscapulohumeral Muscular Dystrophy Type 1 (FSHD1)FSH Muscular DystrophyLandouzy Dejerine DystrophyLandouzy-Dejerine Muscular DystrophyLandouzy-Dejerine Syndrome

Investigational drug(s) / intervention(s)

AOC-1020Placebo

AOC-1020: Del-brax will be administered by intravenous (IV) infusion.

Placebo: Placebo will be administered by intravenous (IV) infusion.

Study summary

A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)

Eligibility

Sex

ALL

Min age

16 Years

Max age

70 Years

Healthy volunteers

Inclusion Criteria: * Clinical and genetic diagnosis of FSHD1 or FSHD2 * Ability to walk independently at pre-specified walking speed (orthoses and ankle braces allowed) for at least 10 meters at screening * Adequate muscle strength based on QMT composite score Exclusion Criteria: * Breastfeeding, pregnancy, or intent to become pregnant during the study * Unwilling or unable to comply with contraceptive requirements * Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study * Blood Pressure \> 140/90 mmHg at Screening * Treatment with another investigational drug or biological agent within 1 month of Screening or 5 half-lives of the drug, whichever is longer * Treatment with an oligonucleotide within 9 months of Screening

Primary outcome measure(s)

Change from Baseline to Week 78 in Quantitative Muscle Testing (QMT) total composite score (PPN) — Baseline through Week 78
Assessment of muscle strength using standardized quantitative measurements

Trial sites (46)

Facility	City	Region	Status
University of California Irvine	Orange	California	Active Not Recruiting
Stanford University	Palo Alto	California	Active Not Recruiting
University of Colorado	Denver	Colorado	Active Not Recruiting
University of Florida	Gainesville	Florida	Active Not Recruiting
University of Iowa	Iowa City	Iowa	Active Not Recruiting
Kansas University Medical Center	Kansas City	Kansas	Active Not Recruiting
Kennedy Krieger Institute	Baltimore	Maryland	Active Not Recruiting
University of Massachusetts	Worcester	Massachusetts	Active Not Recruiting
University of Rochester Medical Center	Rochester	New York	Active Not Recruiting
Duke University	Durham	North Carolina	Active Not Recruiting
Ohio State University	Columbus	Ohio	Active Not Recruiting
University of Pennsylvania	Philadelphia	Pennsylvania	Active Not Recruiting
University of Texas Health Science Center at San Antonio	San Antonio	Texas	Active Not Recruiting
Virginia Commonwealth University	Richmond	Virginia	Active Not Recruiting
University of Calgary - Cumming School of Medicine	Calgary	Alberta	Recruiting
University of Alberta Hospital	Edmonton	Alberta	Recruiting
The Ottawa Hospital	Ottawa	Ontario	Recruiting
Genge Partners Inc	Montreal	Quebec	Recruiting
Aarhus University Hospital	Aarhus	Denmark	Not Yet Recruiting
Rigshospitalet	Copenhagen	Denmark	Recruiting
Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone	Marseille	France	Not Yet Recruiting
CHU de Montpellier - Hopital Gui de Chauliac	Montpellier	France	Not Yet Recruiting
CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires	Nice	France	Not Yet Recruiting
AP-HP Hopital Pitie-Salpetriere	Paris	France	Recruiting
Universitaetsklinikum Bonn	Bonn	Germany	Recruiting
Georg-August-Universitaet Goettingen Stiftung oeffentlichen Rechts Universitaetsmedizin Goettingen	Göttingen	Germany	Recruiting
Klinikum der Ludwig-Maximilians-Universitaet Muenchen	München	Germany	Recruiting
Universitaetsklinikum Ulm	Ulm	Germany	Recruiting
Fondazione Serena ETS - Centro Clinico NeMO Milano	Milan	Italy	Recruiting
Azienda Ospedaliero Universitaria Pisana	Pisa	Italy	Recruiting
Azienda Ospedaliero-Universitaria Sant'Andrea	Roma	Italy	Recruiting
Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore	Roma	Italy	Recruiting
National Hospital Organization Osaka Toneyama Medical Center	Osaka	Japan	Recruiting
The University of Osaka Hospital	Osaka	Japan	Recruiting
National Hospital Organization Sendai Nishitaga Hospital	Sendai	Japan	Recruiting
National Center of Neurology and Psychiatry	Tokyo	Japan	Recruiting
Academisch Ziekenhuis Leiden	Leiden	Netherlands	Recruiting
Stichting Radboud Universitair Medisch Centrum	Nijmegen	Netherlands	Recruiting
Hospital Universitari de Bellvitge	Barcelona	Spain	Recruiting
Hospital Universitario Vall d'Hebron	Barcelona	Spain	Recruiting

+ 6 more sites — see the full list on the official registry below.

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07038200 on ClinicalTrials.gov ↗ ← All trials in the USA