A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD
FORTITUDE-3
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)
1 other identifier
interventional
200
10 countries
46
Brief 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)
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Jun 2025
Typical duration for phase_3
46 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
June 10, 2025
CompletedFirst Submitted
Initial submission to the registry
June 13, 2025
CompletedFirst Posted
Study publicly available on registry
June 26, 2025
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 1, 2028
ExpectedStudy Completion
Last participant's last visit for all outcomes
July 1, 2028
May 29, 2026
September 1, 2025
2.9 years
June 13, 2025
May 27, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Quantitative Muscle Testing (QMT) composite score
Baseline through Week 78
Secondary Outcomes (7)
10-Meter Walk/Run Test (10MWRT)
Baseline through Week 78
Timed Up-and-Go (TUG)
Baseline through Week 78
Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function Item Bank v2.0-Physical Function-Short Form 20a Questionnaire
Baseline through Week 78
Worst Pain Numeric Rating Scale (NRS)
Baseline through Week 78
Patient Global Impression of Severity/Change (PGI-S/PGI-C)
Baseline through Week 78
- +2 more secondary outcomes
Study Arms (2)
del-brax
EXPERIMENTALDel-brax (AOC 1020) will be administered 13 times
placebo
PLACEBO COMPARATORSaline will be administered 13 times
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (46)
University of California Irvine
Orange, California, 92868, United States
Stanford University
Palo Alto, California, 94304, United States
University of Colorado
Denver, Colorado, 80045, United States
University of Florida
Gainesville, Florida, 32608, United States
University of Iowa
Iowa City, Iowa, 52242, United States
Kansas University Medical Center
Kansas City, Kansas, 66205, United States
Kennedy Krieger Institute
Baltimore, Maryland, 21205, United States
University of Massachusetts
Worcester, Massachusetts, 01655, United States
University of Rochester Medical Center
Rochester, New York, 14642, United States
Duke University
Durham, North Carolina, 27708, United States
Ohio State University
Columbus, Ohio, 43221, United States
University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
University of Texas Health Science Center at San Antonio
San Antonio, Texas, 78229, United States
Virginia Commonwealth University
Richmond, Virginia, 23298, United States
University of Calgary - Cumming School of Medicine
Calgary, Alberta, Canada
University of Alberta Hospital
Edmonton, Alberta, Canada
The Ottawa Hospital
Ottawa, Ontario, Canada
Genge Partners Inc
Montreal, Quebec, Canada
Aarhus University Hospital
Aarhus, Denmark
Rigshospitalet
Copenhagen, Denmark
Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone
Marseille, France
CHU de Montpellier - Hopital Gui de Chauliac
Montpellier, France
CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires
Nice, France
AP-HP Hopital Pitie-Salpetriere
Paris, France
Universitaetsklinikum Bonn
Bonn, Germany
Georg-August-Universitaet Goettingen Stiftung oeffentlichen Rechts Universitaetsmedizin Goettingen
Göttingen, Germany
Klinikum der Ludwig-Maximilians-Universitaet Muenchen
München, Germany
Universitaetsklinikum Ulm
Ulm, Germany
Fondazione Serena ETS - Centro Clinico NeMO Milano
Milan, Italy
Azienda Ospedaliero Universitaria Pisana
Pisa, Italy
Azienda Ospedaliero-Universitaria Sant'Andrea
Roma, Italy
Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
Roma, Italy
National Hospital Organization Osaka Toneyama Medical Center
Osaka, Japan
The University of Osaka Hospital
Osaka, Japan
National Hospital Organization Sendai Nishitaga Hospital
Sendai, Japan
National Center of Neurology and Psychiatry
Tokyo, Japan
Academisch Ziekenhuis Leiden
Leiden, Netherlands
Stichting Radboud Universitair Medisch Centrum
Nijmegen, Netherlands
Hospital Universitari de Bellvitge
Barcelona, Spain
Hospital Universitario Vall d'Hebron
Barcelona, Spain
Hospital Universitario Donostia
San Sebastián, Spain
Hospital Universitari i Politecnic La Fe
Valencia, Spain
National Hospital for Neurology & Neurosurgery
London, United Kingdom
St. George's University Hospitals NHS Foundation Trust
London, United Kingdom
Royal Victoria Infirmary
Newcastle upon Tyne, United Kingdom
Royal Hallamshire Hospital
Sheffield, United Kingdom
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 13, 2025
First Posted
June 26, 2025
Study Start
June 10, 2025
Primary Completion (Estimated)
May 1, 2028
Study Completion (Estimated)
July 1, 2028
Last Updated
May 29, 2026
Record last verified: 2025-09