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SUMMER 2024 InTouch | 9 Help us find a new approach to treat FSHD1 Facioscapulohumeral Muscular Dystrophy Type 1 (FSHD1) About this study The purpose of the study is to evaluate if the investigational study drug (what effects, good or bad, on the body) in patients with FSHD1, compared to placebo. A placebo is a substance with no active ingredients. It looks like the real study drug but is not (e.g., a ‘dummy drug’). This study will occur in two parts: Part 1 and Part 2. You will be assigned to a part depending on the time you join the study. Part 1 Part 1 will last approximately 21 weeks and will consist of: • Screening Period: you will undergotests to determine if you are eligible for the study; • Treatment Period: you will attend two visits at the study centre including one overnight stay; • Follow Up Period: you will attend six visits at the study centre. Part 2 Part 2 will last approximately 60 weeks and will consist of: • Screening Period: you will undergo tests to determine if you are eligible for the study; • Treatment Period: you will attend two visits at the study centre; RESEARCH NEWS • Follow Up Period: you will attend up to twelve visits at the study centre. If you are successfully enrolled into the study, you will be reimbursed for any reasonable travel, parking, meals and other expenses associated with participating. Why is this study important? Your participation in this clinical study may help us to better understand FSHD1 which could help others with FSHD1 in the future. About the study drug The investigational study drug is being developed as a potential treatment for FSHD1. The study drug targets the gene that causes FSHDI in the skeletal muscle and will decrease and regulate its activity. This is expected to stop worsening of the disease and potentially reverse any ongoing muscle affects controlled by this gene in patients, leading to improved muscle strength and function. However, it is not known whether the study drug will have any effect on FSHD1. The study drug will be administered intravenously (into a vein). The study will enroll up to: • 16 patients in Part 1 • 36 patients in Part 2 As a patient in the study, you would receive: • one dose of the study drug or placebo in Part 1; • two to four doses of the study drug or placebo in Part 2. Who is eligible to take part? You may be eligible if you: • Are aged between 18 - 70; • Have been diagnosed with FSHD1; • Consent to a muscle biopsy. What else do I need to consider? • Participation in the study is voluntary. • If you choose to take part in the study, you can stop participating at any time without this affecting your routine medical care. How do I get more Information? To find out more, contact the study team using the information below. By contacting us, you are under no obligation to take part in the study. Miriam Rodrigues, Auckland, NZ neurogenetics@adhb.govt.nz Before you begin the study, you will be given detailed information about the study and encouraged to ask questions to make sure you have a full understanding of the requirements of the study.