ARODUX
Are You Eligible?To evaluate the safety, tolerability, pharmacokinetics and pharmacokinetics of an investigational drug, ARO-DUX4 in adults with Facioscapulohumeral Muscular Dystrophy Type 1.
Research Objectives
Status
Recruitment
Total participants required
Study location
Study type
Patient group
About this clinical trial
Facioscapulohumeral muscular dystrophy (FSHD) is a rare inherited condition that causes progressive muscle weakness, primarily affecting the face, shoulders, and upper arms. The most common form, FSHD1, results from abnormal expression of the DUX4 gene, leading to muscle loss. There is no approved treatment, and management focuses on symptom relief through physical therapy and exercise. ARO-DUX4 is being developed as a potential treatment targeting the DUX4 gene in skeletal muscle to reduce its activity, potentially halting or reversing disease progression. This study aims to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-DUX4 in individuals with FSHD1.
Eligibility
- Between 18-70 years (inclusive)
- Genetically confirmed FSHD1
- Clinical Severity Score between 3-8 (Scale, 0-10)
- Lower extremity muscle with MFF between 10% – 40% (inclusive) that can be biopsied.
Lead investigators
- A/Prof Robert Henderson
- Dr Po Sheng Yang
Clinical trial coordinator
- Jacqui Langton
Technical title
A Phase 1/2a Dose-escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and/or Pharmacodynamics of ARO-DUX4 in Adult Patients with Facioscapulohumeral Muscular Dystrophy Type 1
Register your interest
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