At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- AOC-1020 (drug), Placebo (drug)
- How long the study runs
- Study runs about 37 months (dates as stated)
- About the drug or intervention
- AOC-1020 — drug: Del-brax will be administered by intravenous (IV) infusion. · Placebo — drug: Placebo will be administered by intravenous (IV) infusion.
- Patient visit burden
- Not specified by the sponsor
In plain English
This study is testing a medicine called Del-brax (also known as AOC 1020) in people with facioscapulohumeral muscular dystrophy (FSHD), a condition that causes muscle weakness. The study covers both main types of FSHD, called FSHD1 and FSHD2. It is run by a company called Avidity Biosciences, Inc.
Who can take part
- You have a clinical and genetic diagnosis of FSHD1 or FSHD2
- You can walk on your own (you may use splints or ankle braces) at a set walking speed for at least 10 metres during screening
- You have enough muscle strength, measured by a muscle strength test (a QMT composite score)
Who may not be able to
- You are breastfeeding, pregnant, or planning to become pregnant during the study
- You are unwilling or unable to follow the contraception requirements
- You have unusual blood test results, or other conditions or diseases, that would make you unsuitable for the study
- Your blood pressure is higher than 140/90 mmHg at screening
- You have taken another experimental drug or biological treatment within 1 month of screening, or within 5 half-lives of that drug (whichever is longer)
- You have had a treatment called an oligonucleotide within 9 months of screening
What taking part involves
- • Not stated — ask the trial team
Time commitment: Not stated — ask the trial team about how many visits there are, how long the study lasts, and what taking part involves.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Testing a treatment
- Ages
- 16 Years to 70 Years
- Who
- All
- Number of participants
- 200
- Started
- 2025-06-10
- Last checked
- 2026-07
Plain English Summary
What is this study?
- • Testing a new treatment for facioscapulohumeral muscular dystrophy
- • Phase3 - 200 participants
- • 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)
Who can take part?
- • Ages 16 Years to 70 Years
- • Diagnosed with facioscapulohumeral muscular dystrophy
Where?
- • London - National Hospital for Neurology & Neurosurgery
- • London - St. George's University Hospitals NHS Foundation Trust
- • Newcastle upon Tyne - Royal Victoria Infirmary
- • Sheffield - Royal Hallamshire Hospital
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
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)
More detail
The study duration is approximately 22 months, consisting of a screening period of up to 6 weeks, a 78-week active treatment period, and a 12-week follow-up period (18 weeks from the last dose). Participants will be randomized to receive an intravenous infusion of either delbrax or placebo at the clinical study site every 6 weeks for a total of 13 doses. The final dose will occur at Week 72, followed by a final assessment at Week 78. After completion of the Week 78 visit, eligible participants will have the option to enroll in an open-label extension (OLE) study, pending regulatory approval. Participants who decline participation in the OLE will be followed for 12 weeks for safety. An Independent Data Monitoring Committee (IDMC) comprising members independent and external to the Sponsor will review safety, tolerability, and efficacy (as needed) data of this study at regular intervals.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 16 Years - 70 Years
- Who can join: All genders
What the study is looking for
- ✓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...
- ✓Adequate muscle strength based on QMT composite score
Who cannot take part
- ✗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...
See the full criteria
Where Is This Study? (4 UK sites)
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
How to Get in Touch
Avidity Biosciences, Inc., A Novartis Company
Sponsor contactCONTACT
Avidity Biosciences, Inc., A Novartis Company
Sponsor contactCONTACT