A randomised phase II study to evaluate the effectiveness of botulinum toxin to delay, and possibly prevent, progressive foot deformities in children with Charcot-Marie-Tooth (CMT) disease.
Phase 2
Recruiting
- Conditions
- Charcot-Marie-Tooth diseaseNeurological - Other neurological disordersHuman Genetics and Inherited Disorders - Other human genetics and inherited disorders
- Registration Number
- ACTRN12605000115639
- Lead Sponsor
- The Children's Hospital at Westmead
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 10
Inclusion Criteria
Children will have a non-fixed bilateral pes cavus due to Charcot Marie Tooth disease type 1A. All patients will have a definite clinical diagnosis of Charcot-Marie-Tooth disease, which will be confirmed where possible by neurophysiologic testing and/or genetic testing of the patient or an affected first-degree relative. The diagnosis of pes cavus will be based upon clinical examination and foot X-rays.
Exclusion Criteria
No exclusion criteria
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method