Together we can light the way for Facioscapulohumeral Muscular Dystrophy (FSHD) research.
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 been diagnosed with FSHD
- Are able to walk 10 meters with or without assistive devices; if you require 2 canes or a walker to walk 10 meters, you would not be eligible
- Have not been diagnosed with congenital FSHD
- Are not pregnant, breastfeeding, or planning a pregnancy in the next 1-2 years
Additional requirements to participate will apply. A study representative will discuss them with you during the Screening Visit.
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.
Overall, FSHD is a chronically 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 FORTITUDE™ Study
What to Expect During the FORTITUDE™ Study
To participate in this study, participants 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 to withdraw from the study
For every three participants enrolling in the study, two will receive investigational therapy and 1 will receive placebo (2:1 randomization), both through intravenous infusions.
The study is blinded, meaning that neither you nor the study team will know which study treatment you are receiving.
- Health Questions
- Physical Examinations
- Vital Signs
- Pregnancy Testing
- Blood and Urine Sample Collections
- Electrocardiogram (ECG)
- MRI
- Muscle Needle Biopsies
- Muscle Strength Testing
Participants will be enrolled in the FORTITUDE™ study for approximately 13.5 months, including screening.
Avidity Biosciences has also initiated an Open-Label Extension (OLE) study, in which all participants will receive active study medication for an ongoing period.
Participants who do not immediately take part in or decline the OLE will be followed for another 12 weeks in an Extended Follow-up Period.
Travel support and reimbursement for the participant 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 a clinical trial is to gather information on the safety and efficacy of an investigational therapy. The data collected during the trial helps to determine whether the investigational medication should be approved for general use and can also provide essential information on potential side effects and risks.
Why participate in a clinical trial?
Participation in clinical trials 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 I be compensated for participating in a clinical trial?
What is an investigational medication?
What is a placebo?
What are my rights as a participant and what will happen if I decide not to participate?
What are my responsibilities while participating in this study?
It is also recommended that you let your regular doctor know that you’re participating in this study.
How can I learn more about the investigational medication and this study?
More information on this clinical trial can be found on clinicaltrials.gov.
Where We Are
Study Site Locations
For interested participants or caregivers:
To share this study by email, click here.
For physicians and healthcare providers:
Are you a physician looking to refer a patient to the FORTITUDE™ study?
Please contact a study representative at medinfo@aviditybio.com for more information.