Moving FORWARD™:
Building on FORTITUDE™, Focused on the Future.

The FORWARD™ study is a Phase 3 global study focused on testing del-brax (formerly AOC 1020) which targets the underlying cause of Facioscapulohumeral Muscular Dystrophy (FSHD). If you live with FSHD, consider joining this important research effort by participating in the FORWARD™ study.
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About the Study

The FORWARD™ study will evaluate the safety, tolerability, and effectiveness of del-brax in eligible individuals with FSHD.

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What to Expect

Patients will be enrolled in the study for about 18 months. An Open-Label Extension study that would take place after FORWARD™ is being planned.

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Study Locations

This study will take place at multiple clinical sites in several countries. Click here to view our map and find a participating site location near you.

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Who Is Eligible to Participate in This Study?

You or a loved one may qualify if you or they:

  • Are 16 to 70 years old
  • Have a clinical and genetic diagnosis of FSHD (either type 1 or type 2). Genetic confirmation can be provided as part of the study
  • Are able to walk independently (orthoses and ankle braces allowed) for at least 10 meters
  • Are willing and able to follow contraceptive guidelines, and are not currently pregnant, breastfeeding, or planning to become pregnant during the study or 12 weeks after the last dose of study drug

Additional requirements to participate will apply. A study representative will discuss them with you prior to or during the Screening Visit.

Study Site Locations

What is Facioscapulohumeral Muscular Dystrophy (FSHD)?

FSHD, also known as facioscapulohumeral muscular dystrophy, is a rare condition that affects the muscles. It gets worse over time and can make it hard to do daily activities like throw a ball or drink from a straw.

People with FSHD have a genetic abnormality that causes DUX4 protein to be present in their skeletal muscle when it should not be. This abnormal expression of DUX4 leads to a series of downstream events that result in muscle weakness and decreased muscle function that severely affect patient’s ability to perform activities of daily living. Researchers think that one approach to treating FSHD could be to reduce the expression of the DUX4 mRNA and the DUX4 protein. It is thought that reducing the DUX4 protein in the muscle cell could disrupt the path for muscle wasting in FSHD.

Overall, FSHD is a relentlessly progressive disease leading to approximately 20% of individuals losing the ability to walk.

Source: https://www.mda.org/disease/facioscapulohumeral-muscular-dystrophy

Common symptoms may include:

  • Facial weakness
  • Abdominal muscle weakness
  • Hip weakness
  • Lower leg muscle weakness
  • Shoulder, back, and shoulder blade weakness
  • Joint and spinal abnormalities
  • Pain
  • Problems with eyes
  • Unequal (nonsymmetrical) weakness

About the FORWARD™ Study

The FORWARD™ global Phase 3 registrational study was designed in partnership with patients, caregivers, advocates, and key physicians in the field to ensure we are best meeting the needs of the FSHD community; and is focused on finding out how effective and safe del-brax is for treating FSHD in adolescents and adults. 

The effectiveness of del-brax will be assessed by measuring muscle strength and function, and from answers to questionnaires about FSHD symptoms.

What to Expect During the FORWARD™ Study

To participate in this study, patients will be asked to read and sign an Informed Consent Form (ICF).

The form explains:

  • The purpose of the study
  • Required study visits and procedures
  • Potential risks and benefits of participation
  • Confidentiality
  • How participant and caregiver personally identifiable information will be used
  • Information on how participation is voluntary
Approximately 200 FSHD patients will take part in the FORWARD™ study. After confirming eligibility, patients enrolled in the study will be randomly assigned to receive del-brax or placebo.

One (1) out of every two (2) patients will receive del-brax while the remainder will receive placebo, both through intravenous infusions.

The study is blinded, meaning that neither you nor the study team will know which study treatment you are receiving.

Find a Study Location
Safety is our top priority, and that’s why study teams will keep a close eye on all patients in the study. We’ll make sure patients are doing well through various procedures and checks. Some of the required procedures include:
  • Health Questions
  • Physical Examinations
  • Vital Signs
  • Pregnancy Testing
  • Blood and Urine Sample Collections
  • Electrocardiogram (ECG)
  • Muscle Strength & Function Testing

Patients who qualify for the treatment period of the FORWARD™ study will receive study treatment for approximately 18 months.

Prior to that, patients take part in a screening period of up to 6 weeks. 

Avidity Biosciences is planning an Open-Label Extension (OLE) study pending regulatory approval, in which all patients, regardless of whether they received del-brax or placebo, will have the option to enroll.

Patients who do not immediately take part in or decline the OLE will be followed as part of the FORWARD study for approximately 2 months after their last study visit in a Post-Treatment Follow-up Period.

Travel support and reimbursement for the patient and a caregiver will be provided while participating in the study.

Frequently Asked Questions

What is the purpose of a clinical trial?

The main purpose of this clinical study is to gather information on the safety and efficacy of del-brax. The data collected during the study helps to determine whether the investigational treatment should be approved for general use and can also provide essential information on potential side effects and risks.

Why participate in a clinical study?
Participation in clinical studies has greatly impacted the health and well-being of many individuals. People participate for several reasons, including the desire to be more involved in their own healthcare, the opportunity to try new and innovative therapies before they become widely available, and making a positive impact on the advancement of medical science and the health of future generations. 
Will patients be compensated for participating in the FORWARD™ study? 
Many clinical studies offer reimbursement for participation, which can help to cover the costs associated with the study such as transportation and time off work. It’s important to ask about reimbursement for time and travel and what expenses will be covered before deciding to participate in a clinical trial. Travel reimbursement is offered as part of this study for both the patient and caregiver. In addition to reimbursement for travel expenses, some study sites may have the ability to pay a per visit stipend payment.
What is an investigational treatment?

An investigational treatment has not been approved by regulatory authorities but is allowed to be administered to people for research purposes to understand if it might be a safe and effective treatment for a specific disease.

What is a placebo?
In this study, the placebo is something that looks like del-brax but does not contain any active ingredients.
What are the rights of a participant and what will happen if a participant decides not to participate?
It is your choice to take part in this research study or not. You can change your mind and stop participating at any time. If you choose to stop, you may be able to still receive care at the clinic. If you’re thinking about stopping or want more information, please talk to the study doctor. 
What are my responsibilities while participating in this study?

If you decide to take part in the study, you will be expected to follow the rules and instructions given to you by the study team. If you can’t follow these rules and instructions, you may be asked to leave the study. The study will be closely monitored by the study doctor and will be carried out by trained staff. You will need to share information about your health, both now and in the past, to protect your safety. 

It is also recommended that you let your regular doctor know that you’re participating in this study.

Where We Are

Study Site Locations

Current study locations are listed below and more may be added at any time. If you or someone you know may be interested in participating in the FORWARD™ study, please have your physician contact medinfo@aviditybio.com.

      For interested patients or caregivers:

      For more information on this study please visit clinicaltrials.gov.

      To share this study by email, click here.

      For physicians and healthcare providers:

      Are you a physician looking to refer a patient to the FORWARD™ study?

      Please contact a study representative at medinfo@aviditybio.com for more information.