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Clinical Trials in the USA / NCT07038200
Recruiting Phase 3

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-1020 →Placebo

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
No
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)

FacilityCityRegionStatus
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.

More Avidity Biosciences, Inc. trials in the USA

Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping Active, not recruiting
Phase 2 Open-label Extension Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) Active, not recruiting
Global Open-Label Extension Study of Del-desiran for the Treatment of DM1 Enrolling by invitation

Other trials for the same condition

Safety and Preliminary Efficacy of ULSC in Facioscapulohumeral Muscular Dystrophy (FSHD) Recruiting
A First-in-human Study of EPI-321 in Facioscapulohumeral Muscular Dystrophy Active, not recruiting
Phase 2 Open-label Extension Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) Active, not recruiting
Phase 2 Study Evaluating Apitegromab for the Treatment of FSHD Recruiting
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