Facioscapulohumeral Muscular Dystrophy (FSHD) is a rare genetic disease that causes progressive muscle weakness affecting the face, shoulders, arms, trunk, legs, and hips. Most people with FSHD begin to notice symptoms as a teenager or young adult.
If FSHD is suspected, genetic testing can confirm a diagnosis of FSHD1 (95% of cases) or FSHD2 (5% of cases). Both types of FSHD are caused by a protein called DUX4 that is present in everyone, but is normally "shut off" at an early age. In people with FSHD, DUX4 remains "on" or present and becomes toxic to muscle cells, causing them to die and not be able to repair or regenerate. Currently there are no approved treatments that can slow, stop, or reverse the symptoms of FSHD.
About the Study
REACH is a Phase 3 study that is comparing the safety and effectiveness of a potential new treatment for FSHD against a placebo (tablet with no active ingredient). Researchers are trying to determine if the study medicine can stabilize and/or improve participants' muscle function by suppressing DUX4, the protein that causes FSHD1 and FSHD2. REACH will enroll approximately 230 participants with FSHD at study sites across North America and Europe.
Losmapimod is an oral medication taken twice a day that works by reducing the expression of the DUX4 gene in muscle cells by blocking certain proteins.
Fulcrum Therapeutics previously completed two Phase 2 clinical studies that showed losmapimod slowed the progression of FSHD and reduced participants' symptoms, including improved arm strength. These studies found that losmapimod is safe and well-tolerated by participants. Based on these findings, researchers have determined that losmapimod is a good candidate for continued research and development.
About Fulcrum Therapeutics
Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need.
What To Expect
If you are interested in participating, the study team will schedule a screening visit to determine if you are eligible. At the screening visit, the study team will ask about your medical history, medications, and FSHD symptoms. If needed, they will collect a blood sample for genetic testing to confirm a diagnosis of FSHD. They will also complete additional laboratory tests, measure your heart activity, scan your muscles, and measure your shoulder range of motion.
If you are eligible, you will be randomly assigned to receive either the oral study medicine or placebo (tablet with no active ingredient). Neither you nor the study doctor will know what you have been assigned. Half of the study participants will receive the study medicine, and the other half will receive the placebo.
You will be enrolled in the study for about 53 weeks and will need to attend at least 8 study visits during this time. You will be reimbursed for travel and your time for attending study visits.
After you complete the REACH study, you may have the opportunity to continue in an open label extension (OLE) study. In an OLE, all participants receive the study medicine, regardless of whether they were initially assigned to the study medicine or placebo.
As an eligible participant, you will receive the study medicine or placebo and study tests at no cost to you. You may be reimbursed for travel and your time for completing study visits.
Taking part in this study may or may not make your FSHD symptoms better.
Knowledge gained from this study will contribute to the development of an effective treatment for FSHD and may help others living with FSHD in the future.
Your study doctor will discuss potential risks and other available treatment options for your condition prior to the start of the study.
So far - So good!
You appear to meet the basic requirements for this FSHD study. However, we will need to confirm you have genetic testing for FSHD. Please provide your contact information so a member of our study team can contact you regarding the next steps in the process.
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