RecruitingPhase 2ACTRN12605000115639

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.

Sponsor

The Children's Hospital at Westmead

Enrollment

10 participants

Start Date

Nov 12, 2004

Study Type

Interventional

Conditions

Charcot-Marie-Tooth disease

Summary

This is a trial of a new therapy (botulinum toxin) for CMT to see if it will help to minimise or delay foot deformity in children with CMT.

Eligibility

Sex: Both males and females

Inclusion Criteria1

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.

Interested in this trial?

Get notified about updates and connect with the research team.

Interventions

Children who are enrolled in this trial, will have CMT1A. They will have a course of 6 monthly botulinum toxin injections for 2 years into one leg. This leg will be chosen at random, and the other leg