Phase 1 Muscular Dystrophy Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• People aged 15 years or older.
• People who have a confirmed genetic diagnosis of FSHD type 1 or FSHD type 2.
• People whose FSHD symptoms score at least 3 out of 10 on a standard clinical severity scale, and who are able to walk independently at the time of the study.
• People who are able to follow all study requirements, including undergoing MRI scans.
• People of reproductive age or capacity who are willing to use two forms of highly effective contraception during the study and for at least three months after it ends.
• People who are able to understand the study and sign a written consent form.
• People already taking medications or supplements that may affect muscle function, provided the dose has been stable for at least 3 months before the study starts and remains stable throughout — this includes immune-modifying medications and targeted biological therapies.
• People with reduced upper arm strength, as measured by a specific upper limb performance test scoring 5 or below.
• People whose vaccinations are current and up to date.
• People whose arm reach ability, measured by a specific test of the dominant arm, falls within a defined range (confirm with trial site).
• People with no medical reason preventing them from having an MRI scan.
• People with a blood thickness level (hematocrit) of 50% or below.
• People with a prostate-specific antigen (PSA) blood level of 4.0 ng/mL or below — or 3.0 ng/mL or below for those with a close family member who has had prostate cancer.
• People with a fasting blood sugar level below 126 mg/dL.

Who may not be able to join:

• People who have had an allergic reaction to any component of the study treatment, including a substance called dimethyl sulfoxide (DMSO).
• People who currently have cancer or have been diagnosed with cancer in the past year (though people with certain common skin cancers — basal cell or squamous cell — would not automatically be excluded).
• People with any other health condition that the study doctor or sponsor considers unsafe for participation.
• People who have taken part in another clinical trial involving an experimental treatment within the three months before joining this study.
• People with a known active serious infection, including HIV, hepatitis B, or hepatitis C.
• People with a known active or inactive tuberculosis infection.
• People who have used any product intended to boost muscle growth or activity within 4 weeks before the study baseline visit.
• People currently taking certain medications that interact with specific liver and kidney processing pathways (confirm with trial site for specific medication names).
• People who have recently started taking a cholesterol-lowering statin medication, or made significant changes to their statin dose, within 3 months before the baseline visit (long-term, unchanged statin use is generally permitted).
• People who have taken the medication rapamycin within 3 months before the baseline visit.
• People whose muscle condition appears to be caused by something other than FSHD, or who have an additional muscle disease alongside FSHD, based on prior testing.
• People who have had a muscle biopsy (a small tissue sample taken from muscle) within 30 days before the baseline visit.
• People with high blood pressure above 160/100 mmHg.
• People who consume more than 50 grams of alcohol per day (heavy alcohol use).
• People currently using testosterone or human growth hormone (HGH).
• People currently taking medications that interfere with growth hormone or sex hormone levels in the body.
• People who are pregnant or breastfeeding.
• People with severe heart or lung disease, active infection, or other serious conditions that would make it difficult to safely assess the study treatment.
• People who are expected to need surgery at any point during the trial.
• People with a history of consistently not following medical treatment plans.
• People who have received an organ transplant.
• People with a low white blood cell count (neutropenia), below a level determined by the trial site — a different threshold applies for people of African-American background (confirm with trial site).
• People with severely reduced kidney function, below a certain level as measured by a standard kidney test.
• People who have recently received, or are planning to receive, a vaccine made with a live weakened virus.
• People with a neurological condition such as Parkinson's disease, or a severe muscle or joint condition, that would make it difficult to accurately assess muscle strength.

Important: Always verify eligibility with the trial site directly before applying.