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

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

Plain Language Summary

Simplified for easier understanding

This study tests whether injections of botulinum toxin (similar to Botox) can slow down or prevent foot deformity in children with Charcot-Marie-Tooth disease (CMT), an inherited nerve condition. CMT can cause the foot to develop a high arch that gets worse over time. Researchers want to see if these injections can delay or prevent this problem. You may be eligible if: - Your child has been diagnosed with Charcot-Marie-Tooth disease type 1A - Your child has bilateral pes cavus (high arches in both feet) that is not yet fixed or rigid - The diagnosis has been confirmed clinically, and where possible with nerve tests or genetic testing You may NOT be eligible if: - There are no specific exclusion criteria listed for this study Talk to your doctor about whether this trial might be right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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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

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 will act as the control.


Locations(1)

Australia

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