RecruitingPHASE3INTERVENTIONAL

A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD

This study aims to find out if a new medicine, del-brax (also known as AOC 1020), is a safe and helpful treatment for people living with Facioscapulohumeral Muscular Dystrophy (FSHD). FSHD is a condition that causes muscle weakness, mainly in the face, shoulders, and upper arms. The study is in its final phase, meaning it has already shown some promise in earlier stages. Participants will either receive the new medicine or a dummy treatment (placebo) through an infusion every six weeks over a period of about 72 weeks. The main goal is to see if del-brax can improve symptoms or slow the progression of FSHD. Researchers will also be carefully monitoring for any side effects. This research could lead to a new treatment option for FSHD.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Avidity Biosciences, Inc.

Enrolment target

200

Start

10 Jun 2025

Estimated completion

01 Jul 2028

What is this study about?

This study is looking into a new treatment called del-brax, which is also known as AOC 1020. It's designed for people who have Facioscapulohumeral Muscular Dystrophy, or FSHD for short. FSHD is a condition that makes muscles weaker over time, often starting in the face, shoulders, and upper arms.

The main purpose of this study is to discover if del-brax is safe to use and if it can help people with FSHD. This is a "Phase 3" study, which means it's one of the final steps before a new medicine might be approved for wider use. Researchers will compare del-brax with a 'placebo', which looks exactly like the medicine but doesn't contain any active ingredients. This helps them understand whether any changes felt by participants are truly due to the medicine or other factors.

By taking part, you would be helping doctors and scientists learn more about FSHD and potentially find an important new way to manage the condition. All information gathered is carefully reviewed to ensure the study is as safe and effective as possible for everyone involved.

Key takeaways

This study is testing a new medicine called del-brax for FSHD.
It's a 'Phase 3' study, meaning it's a final step in testing before potential approval.
Participants will receive infusions every 6 weeks for about 72 weeks.
The study aims to see if del-brax is safe and effective in treating FSHD.
You might receive the active medicine or a placebo (dummy treatment).
Participation involves regular clinic visits for infusions and assessments.

Who may be eligible?

To be considered for this study, you need a confirmed diagnosis of FSHD, either type 1 or type 2, from your doctor. You also need to be able to walk on your own for at least 10 meters (about the length of a small bus) at a reasonable speed. Even if you use special ankle or leg braces, you might still be able to join.

There are also some requirements about your physical health, including your muscle strength based on certain tests. You must be between 16 and 70 years old to take part. The study is open to everyone, regardless of whether you are male or female.

However, you can't join if you are pregnant, breastfeeding, or planning to become pregnant during the study. You also can't be taking part in another drug study or have recently been treated with certain other specific medications. Researchers check for other health conditions or blood pressure readings that might make participating unsafe for you.

Quick self-check

Do you have a confirmed FSHD diagnosis (Type 1 or Type 2)?
Can you walk independently for at least 10 meters, even with braces?
Are you between 16 and 70 years old?
Are you not pregnant, breastfeeding, or planning to become pregnant during the study?
Are you able to meet specific contraceptive requirements?
Have you spoken with your doctor about any other medications you are currently taking?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to join this study, it would involve a screening period of up to 6 weeks to make sure it's suitable for you. After that, the main part of the study lasts for about 72 weeks, which is roughly a year and a half. During this time, you would visit the clinic every 6 weeks for an intravenous infusion – this means the study medicine or the placebo would be given directly into your vein, like a drip. There will be 13 doses in total.

Your final infusion will be at Week 72, followed by a final check-up visit at Week 78. So, in total, you'd be involved for about a year and a half, or 78 weeks. After this, you might have the option to continue receiving the medicine in a special 'open-label extension' study, if approved. If you choose not to, the study team will still check in with you for 12 weeks to make sure you are well.

Potential risks and benefits

Taking part in any medical study carries both potential benefits and risks. You might benefit from receiving a new medicine that could help your FSHD, or you could benefit from closer medical monitoring and care from experts. However, there's no guarantee the treatment will work for you, and like all medicines, del-brax could have side effects, which the study team will monitor closely. You also might receive the placebo, which has no active medicine. It's very important to remember that you are free to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (46)

University of California Irvine
Orange, United States· Active not recruiting
Stanford University
Palo Alto, United States· Active not recruiting
University of Colorado
Denver, United States· Active not recruiting
University of Florida
Gainesville, United States· Active not recruiting
University of Iowa
Iowa City, United States· Active not recruiting
Kansas University Medical Center
Kansas City, United States· Active not recruiting
Kennedy Krieger Institute
Baltimore, United States· Active not recruiting
University of Massachusetts
Worcester, United States· Active not recruiting
University of Rochester Medical Center
Rochester, United States· Active not recruiting
Duke University
Durham, United States· Active not recruiting
Ohio State University
Columbus, United States· Active not recruiting
University of Pennsylvania
Philadelphia, United States· Active not recruiting

+34 more sites — see the official record for the full list.

Common questions

What is FSHD?

FSHD stands for Facioscapulohumeral Muscular Dystrophy. It's a genetic condition that causes muscles to weaken, most commonly in the face, shoulders, and upper arms.

What is a placebo?

A placebo is a dummy treatment that looks exactly like the study medicine but doesn't contain any active ingredients. It helps researchers fairly compare how well the new medicine works.

How will I receive the study medicine?

You will receive the study medicine or placebo through an intravenous infusion, which means it will be given directly into a vein, like a drip, at the study clinic.

How long will I be in the study?

The study lasts for about 78 weeks, which is roughly a year and a half, including the screening and treatment periods.

Can I leave the study if I change my mind?

Yes, you can leave the study at any time, for any reason, without it affecting your regular medical care.

How to find out more

Avidity Biosciences, Inc.

medinfo@aviditybio.com 858-771-7038 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.