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)
Status: Recruiting
Location: See all (46) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
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
Participation Requirements
Sex: All
Minimum Age: 16
Maximum Age: 70
Healthy Volunteers: f
View:
• 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
Locations
United States
California
University of California Irvine
ACTIVE_NOT_RECRUITING
Orange
Stanford University
ACTIVE_NOT_RECRUITING
Palo Alto
Colorado
University of Colorado
ACTIVE_NOT_RECRUITING
Denver
Florida
University of Florida
ACTIVE_NOT_RECRUITING
Gainesville
Iowa
University of Iowa
ACTIVE_NOT_RECRUITING
Iowa City
Kansas
Kansas University Medical Center
ACTIVE_NOT_RECRUITING
Kansas City
Massachusetts
University of Massachusetts
ACTIVE_NOT_RECRUITING
Worcester
Maryland
Kennedy Krieger Institute
ACTIVE_NOT_RECRUITING
Baltimore
North Carolina
Duke University
ACTIVE_NOT_RECRUITING
Durham
New York
University of Rochester Medical Center
ACTIVE_NOT_RECRUITING
Rochester
Ohio
Ohio State University
ACTIVE_NOT_RECRUITING
Columbus
Pennsylvania
University of Pennsylvania
ACTIVE_NOT_RECRUITING
Philadelphia
Texas
University of Texas Health Science Center at San Antonio
ACTIVE_NOT_RECRUITING
San Antonio
Virginia
Virginia Commonwealth University
ACTIVE_NOT_RECRUITING
Richmond
Other Locations
Canada
University of Calgary - Cumming School of Medicine
RECRUITING
Calgary
University of Alberta Hospital
RECRUITING
Edmonton
Genge Partners Inc
RECRUITING
Montreal
The Ottawa Hospital
RECRUITING
Ottawa
Denmark
Aarhus University Hospital
NOT_YET_RECRUITING
Aarhus
Rigshospitalet
RECRUITING
Copenhagen
France
Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone
NOT_YET_RECRUITING
Marseille
CHU de Montpellier - Hopital Gui de Chauliac
NOT_YET_RECRUITING
Montpellier
CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires
NOT_YET_RECRUITING
Nice
AP-HP Hopital Pitie-Salpetriere
NOT_YET_RECRUITING
Paris
Germany
Universitaetsklinikum Bonn
RECRUITING
Bonn
Georg-August-Universitaet Goettingen Stiftung oeffentlichen Rechts Universitaetsmedizin Goettingen
NOT_YET_RECRUITING
Göttingen
Klinikum der Ludwig-Maximilians-Universitaet Muenchen
NOT_YET_RECRUITING
München
Universitaetsklinikum Ulm
RECRUITING
Ulm
Italy
Fondazione Serena ETS - Centro Clinico NeMO Milano
RECRUITING
Milan
Azienda Ospedaliero Universitaria Pisana
RECRUITING
Pisa
Azienda Ospedaliero-Universitaria Sant'Andrea
NOT_YET_RECRUITING
Roma
Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
RECRUITING
Roma
Japan
National Hospital Organization Osaka Toneyama Medical Center
RECRUITING
Osaka
The University of Osaka Hospital
RECRUITING
Osaka
National Hospital Organization Sendai Nishitaga Hospital
RECRUITING
Sendai
National Center of Neurology and Psychiatry
RECRUITING
Tokyo
Netherlands
Academisch Ziekenhuis Leiden
RECRUITING
Leiden
Stichting Radboud Universitair Medisch Centrum
RECRUITING
Nijmegen
Spain
Hospital Universitari de Bellvitge
RECRUITING
Barcelona
Hospital Universitario Vall d'Hebron
RECRUITING
Barcelona
Hospital Universitario Donostia
NOT_YET_RECRUITING
San Sebastián
Hospital Universitari i Politecnic La Fe
RECRUITING
Valencia
United Kingdom
National Hospital for Neurology & Neurosurgery
RECRUITING
London
St. George's University Hospitals NHS Foundation Trust
RECRUITING
London
Royal Victoria Infirmary
RECRUITING
Newcastle Upon Tyne
Royal Hallamshire Hospital
NOT_YET_RECRUITING
Sheffield
Contact Information
Primary
Avidity Biosciences, Inc.
medinfo@aviditybio.com
858-771-7038
Time Frame
Start Date: 2025-06-10
Estimated Completion Date: 2028-07
Participants
Target number of participants: 200
Treatments
Experimental: del-brax
Del-brax (AOC 1020) will be administered 13 times
Placebo_comparator: placebo
Saline will be administered 13 times
Related Therapeutic Areas
Sponsors
Leads: Avidity Biosciences, Inc.