Study of ARO-DUX4 in Adult Patients With Facioscapulohumeral Muscular Dystrophy Type 1

Launched by ARROWHEAD PHARMACEUTICALS · Nov 13, 2023

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called ARO-DUX4 for adults with Facioscapulohumeral Muscular Dystrophy Type 1 (FSHD1), a condition that affects muscle strength and movement. The main goals of this study are to check how safe the treatment is, how well it is tolerated, and how it works in the body. Participants will receive either the treatment or a placebo (a substance with no active medication) in two parts of the study. In the first part, they will receive one dose, while in the second part, they will receive four doses. Throughout the trial, participants will have muscle biopsies, which are small samples taken from their muscles to help researchers learn more about the treatment's effects.

To be eligible for this study, participants need to be adults aged between 18 and 75 who have a confirmed diagnosis of FSHD1 and show a certain level of muscle weakness. They also need to be able to have a muscle biopsy and must not have certain health conditions like uncontrolled high blood pressure or serious heart issues. Participants can expect to visit the study site for assessments and treatments, and those who complete the initial parts of the trial may have the option to continue receiving the treatment in a follow-up study. It's important for participants to know that they will need to use effective contraception during the study and for a period afterward if they are able to conceive.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Genetically confirmed FSHD1 based on Screening evaluation or source verifiable medical record
• • Clinical severity score between 3 and 8 (scale, 0 to 10)
• • Must have eligible lower extremity muscle for biopsy as determined from MRI by a central reader
• • A 12-lead electrocardiogram (ECG) at Screening with no abnormalities that may compromise participant's safety in the study
• • Participants of childbearing potential and their partners must use highly effective contraception during the study and for at least 12 weeks following the end of study or last dose of study medication, whichever is later. Males must not donate sperm during the study from Day 1 until at least 12 weeks following the end of study or last dose of study medication, whichever is later.
• Exclusion Criteria:
• • Human Immunodeficiency Virus (HIV) infection as shown by presence of anti-HIV antibody (seropositive) at Screening
• • Seropositive for hepatitis B (HBV) or hepatitis C (HCV) at Screening
• • Uncontrolled hypertension
• • Severe cardiovascular disease
• • History of thrombolic events
• • Platelet count less that the lower limit of normal at Screening
• • History or presence of: a hypercoagulable state, nephrotic range proteinuria, antiphospholipid antibody syndrome, myeloproliferative disease, inability to ambulate, use of hormone-based contraceptives.
• • Any contraindication to muscle biopsy or MRI
• • Note: additional inclusion/exclusion criteria may apply per protocol