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.

Status

Recruiting

Phases

Phase 2

Study type

Interventional

Source

ANZCTR

Registry ID

ACTRN12605000115639

Enrollment

Registered

2005-08-09

Start date

2004-11-12

Completion date

Unknown

Last updated

2020-01-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

None listed

Brief 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.

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 will act as the control.

Study design

Allocation

Randomised controlled trial

Intervention model

Single group

Primary purpose

Treatment

Masking

Blinded (masking used)

Eligibility

Sex/Gender

All

Age

0 to No maximum

Healthy volunteers

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

Outcome results