The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial registered on ANZCTR


Trial ID
ACTRN12605000115639
Ethics application status
Approved
Date submitted
5/08/2005
Date registered
9/08/2005
Date last updated
22/08/2007
Type of registration
Retrospectively registered

Titles & IDs
Public title
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.
Scientific title
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.
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Charcot-Marie-Tooth disease 203 0
Condition category
Condition code
Human Genetics and Inherited Disorders 226 226 0 0
Other human genetics and inherited disorders
Neurological 227 227 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
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.
Intervention code [1] 130 0
Treatment: Drugs
Comparator / control treatment
The other leg will act as the control.
Control group
Active

Outcomes
Primary outcome [1] 274 0
The primary outcome will be changes in foot shape and posture in the treated leg compared to the untreated leg.
This will be measured by Xrays, Foot posture index and Clinical photographs.
Timepoint [1] 274 0
Xrays - taken at baseline, and at conclusion (2 years).
Foot posture index - baseline, then every 6, 12, 18 months and at conclusion.
Clinical photographs - baseline, then every 6, 12, 18 months and at conclusion.
Secondary outcome [1] 613 0
To ensure that the use of botulinum toxin will not have detrimental effects on foot posture and deformity, using:
X-rays – done pre- and post-trial.
Timepoint [1] 613 0
Foot posture index and clinical photographs – done pre-trial , then every 6 months till completion of the trial.

Eligibility
Key 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.
Minimum age
Not stated
Maximum age
Not stated
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
No exclusion criteria

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Sealed envelopes prepared in advance
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
There is no stratification. The chief investigator and the physician injecting the botulinum toxin are aware of the leg selection for treatment. The investigators doing the follow-up assessments are blinded to the treated leg. The selection of the limb is done by the chief investigator by coin flip.
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint(s)
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)

Funding & Sponsors
Funding source category [1] 288 0
Other
Name [1] 288 0
Institute for Neuromuscular Research
Address [1] 288 0
Country [1] 288 0
Australia
Primary sponsor type
Hospital
Name
The Children's Hospital at Westmead
Address
Country
Australia
Secondary sponsor category [1] 219 0
None
Name [1] 219 0
None
Address [1] 219 0
Country [1] 219 0

Ethics approval
Ethics application status
Approved

Summary
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.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 35871 0
Address 35871 0
Country 35871 0
Phone 35871 0
Fax 35871 0
Email 35871 0
Contact person for public queries
Name 9319 0
Sian Rudge
Address 9319 0
Clinical Sciences
The Children's Hospital at Westmead
Locked Bag 4001
Westmead NSW 2145
Country 9319 0
Australia
Phone 9319 0
+61 2 98451229
Fax 9319 0
+61 2 98451317
Email 9319 0
sianr@chw.edu.au
Contact person for scientific queries
Name 247 0
Sian Rudge
Address 247 0
Clinical Sciences
The Children's Hospital at Westmead
Locked Bag 4001
Westmead NSW 2145
Country 247 0
Australia
Phone 247 0
+61 2 98451229
Fax 247 0
+61 2 98451317
Email 247 0
sianr@chw.edu.au