Actively Recruiting
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)
Led by Avidity Biosciences, Inc. · Updated on 2026-05-29
200
Participants Needed
46
Research Sites
8 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This clinical trial is focused on people diagnosed with Facioscapulohumeral Muscular Dystrophy (FSHD), specifically types FSHD1 or FSHD2. The study aims to evaluate the safety and effectiveness of an intravenous drug called del-brax (also known as AOC 1020) compared to a placebo. This is a randomized, double-blind, placebo-controlled Phase 3 trial sponsored by Avidity Biosciences, Inc. It seeks to better understand how del-brax affects muscle strength and disease progression in FSHD patients. Participants will be randomly assigned to receive either del-brax or a placebo through intravenous infusions every six weeks for a total of 13 doses over a 72-week treatment period. After the last dose at Week 72, a final assessment will take place at Week 78. Those who finish the treatment phase may have the option to join an open-label extension study if approved. Participants who do not join the extension will be monitored for safety for an additional 12 weeks. An independent committee will regularly review safety and effectiveness data throughout the study. During the trial, participants will visit the clinical site regularly for infusions and assessments. Researchers will measure muscle strength using the Quantitative Muscle Testing (QMT) composite score, along with other tests such as walking speed, timed mobility, patient-reported physical function, pain levels, and biomarkers related to FSHD. The total study duration is approximately 78 weeks, including screening, treatment, and follow-up periods. Safety and tolerability will be closely monitored throughout and after the treatment phase.
CONDITIONS
Brief Title
A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Clinical and genetic diagnosis of FSHD1 or FSHD2
- Ability to walk independently at a pre-specified walking speed for at least 10 meters (orthoses and ankle braces allowed)
- Adequate muscle strength based on Quantitative Muscle Testing (QMT) composite score
You will not qualify if you...
- Breastfeeding, pregnancy, or intent to become pregnant during the study
- Unwilling or unable to comply with contraceptive requirements
- Abnormal lab values, conditions, or diseases making participation unsuitable
- Blood pressure greater than 140/90 mmHg at screening
- Treatment with another investigational drug or biological agent within 1 month or 5 half-lives, whichever is longer
- Treatment with an oligonucleotide within 9 months of screening
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - Up to 6 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person) for screening and enrollment
Duration - 72 weeks
Participants receive an intravenous infusion of del-brax or placebo every 6 weeks for a total of 13 doses.
13 visits every 6 weeks for intravenous infusions
Duration - 6 weeks
Participants are assessed for safety and final outcomes after treatment completion.
1 final visit at Week 78
Trial Site Locations
Total: 46 locations
1
University of California Irvine
Orange, California, United States, 92868
Active, Not Recruiting
2
Stanford University
Palo Alto, California, United States, 94304
Active, Not Recruiting
3
University of Colorado
Denver, Colorado, United States, 80045
Active, Not Recruiting
4
University of Florida
Gainesville, Florida, United States, 32608
Active, Not Recruiting
5
University of Iowa
Iowa City, Iowa, United States, 52242
Active, Not Recruiting
6
Kansas University Medical Center
Kansas City, Kansas, United States, 66205
Active, Not Recruiting
7
Kennedy Krieger Institute
Baltimore, Maryland, United States, 21205
Active, Not Recruiting
8
University of Massachusetts
Worcester, Massachusetts, United States, 01655
Active, Not Recruiting
9
University of Rochester Medical Center
Rochester, New York, United States, 14642
Active, Not Recruiting
10
Duke University
Durham, North Carolina, United States, 27708
Active, Not Recruiting
11
Ohio State University
Columbus, Ohio, United States, 43221
Active, Not Recruiting
12
University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
Active, Not Recruiting
13
University of Texas Health Science Center at San Antonio
San Antonio, Texas, United States, 78229
Active, Not Recruiting
14
Virginia Commonwealth University
Richmond, Virginia, United States, 23298
Active, Not Recruiting
15
University of Calgary - Cumming School of Medicine
Calgary, Alberta, Canada
Actively Recruiting
16
University of Alberta Hospital
Edmonton, Alberta, Canada
Actively Recruiting
17
The Ottawa Hospital
Ottawa, Ontario, Canada
Actively Recruiting
18
Genge Partners Inc
Montreal, Quebec, Canada
Actively Recruiting
19
Aarhus University Hospital
Aarhus, Denmark
Not Yet Recruiting
20
Rigshospitalet
Copenhagen, Denmark
Actively Recruiting
21
Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone
Marseille, France
Not Yet Recruiting
22
CHU de Montpellier - Hopital Gui de Chauliac
Montpellier, France
Not Yet Recruiting
23
CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires
Nice, France
Not Yet Recruiting
24
AP-HP Hopital Pitie-Salpetriere
Paris, France
Not Yet Recruiting
25
Universitaetsklinikum Bonn
Bonn, Germany
Actively Recruiting
26
Georg-August-Universitaet Goettingen Stiftung oeffentlichen Rechts Universitaetsmedizin Goettingen
Göttingen, Germany
Not Yet Recruiting
27
Klinikum der Ludwig-Maximilians-Universitaet Muenchen
München, Germany
Not Yet Recruiting
28
Universitaetsklinikum Ulm
Ulm, Germany
Actively Recruiting
29
Fondazione Serena ETS - Centro Clinico NeMO Milano
Milan, Italy
Actively Recruiting
30
Azienda Ospedaliero Universitaria Pisana
Pisa, Italy
Actively Recruiting
31
Azienda Ospedaliero-Universitaria Sant'Andrea
Roma, Italy
Not Yet Recruiting
32
Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
Roma, Italy
Actively Recruiting
33
National Hospital Organization Osaka Toneyama Medical Center
Osaka, Japan
Actively Recruiting
34
The University of Osaka Hospital
Osaka, Japan
Actively Recruiting
35
National Hospital Organization Sendai Nishitaga Hospital
Sendai, Japan
Actively Recruiting
36
National Center of Neurology and Psychiatry
Tokyo, Japan
Actively Recruiting
37
Academisch Ziekenhuis Leiden
Leiden, Netherlands
Actively Recruiting
38
Stichting Radboud Universitair Medisch Centrum
Nijmegen, Netherlands
Actively Recruiting
39
Hospital Universitari de Bellvitge
Barcelona, Spain
Actively Recruiting
40
Hospital Universitario Vall d'Hebron
Barcelona, Spain
Actively Recruiting
41
Hospital Universitario Donostia
San Sebastián, Spain
Not Yet Recruiting
42
Hospital Universitari i Politecnic La Fe
Valencia, Spain
Actively Recruiting
43
National Hospital for Neurology & Neurosurgery
London, United Kingdom
Actively Recruiting
44
St. George's University Hospitals NHS Foundation Trust
London, United Kingdom
Actively Recruiting
45
Royal Victoria Infirmary
Newcastle upon Tyne, United Kingdom
Actively Recruiting
46
Royal Hallamshire Hospital
Sheffield, United Kingdom
Not Yet Recruiting
Research Team
A
Avidity Biosciences, Inc.
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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