Together we can light the way for Facioscapulohumeral Muscular Dystrophy (FSHD) research.
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 wasting and decreased muscle function. 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.
FSHD is not very common, and it progresses slowly. This means that for some people, the symptoms may not appear for a long time or may not be very severe. However, it can vary from person to person.
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
- Soreness of the muscles
- Problems with eyes
- Unequal (nonsymmetrical) weakness
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
- How participant and caregiver personally identifiable information will be used
- Information on how to withdraw from the study
Approximately 70 FSHD patients will take part in the FORTITUDE™ study in several countries around the world. After confirming eligibility, participants enrolled in the study will be randomly assigned to the investigational drug or placebo.
Two (2) out of every three (3) participants will receive the investigational study drug while the remainder will receive the placebo, both through intravenous infusions given approximately every 3 months.
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)
- Muscle Needle Biopsies
- Muscle Strength Testing
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 medication. 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.
Where We Are
Study Site Locations
Current study locations are listed below and more may be added at any time. If you are interested in participating in the FORTITUDE™ study, contact the site nearest you or email us at firstname.lastname@example.org to learn more. If you don’t see a site near you but would be willing to travel, travel assistance may be made available for participants and their caregivers.