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Trial registered on ANZCTR


Registration number
ACTRN12613000552785
Ethics application status
Approved
Date submitted
2/05/2013
Date registered
16/05/2013
Date last updated
17/05/2013
Type of registration
Prospectively registered

Titles & IDs
Public title
Foot and ankle strength training for children with Charcot-Marie-Tooth disease
Scientific title
Efficacy and safety of progressive resistance foot strength training on muscle strength and volume, gait and quality of life in children with Charcot-Marie-Tooth disease.
Secondary ID [1] 281924 0
None
Universal Trial Number (UTN)
U1111-1139-4203
Trial acronym
FAST CMT Trial
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Charcot-Marie-Tooth disease 288321 0
Condition category
Condition code
Neurological 288676 288676 0 0
Other neurological disorders
Human Genetics and Inherited Disorders 288677 288677 0 0
Other human genetics and inherited disorders
Physical Medicine / Rehabilitation 289423 289423 0 0
Physiotherapy

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Intervention group: High-dose progressive resistance strength training
Type of intervention: Extending and flexing the foot with progressive loading of weight using an ankle cuff.
Frequency: 3 x 25 min sessions per week plus 30 minutes rest after each session
Mode: One supervised training session at hospital and five home-based training sessions with parental supervision per fortnight
Dose: 2-3 sets of 8 repetitions at 50-70% of 1RM for 24 weeks. 1RM is measured every fortnight to adjust the weight lifted.

Adherence is monitored by an exercise diary documenting the number of training sessions completed and the intensity experienced with the Borg scale.
Intervention code [1] 286486 0
Treatment: Other
Comparator / control treatment
Intervention group: Low-dose progressive resistance strength training
Type of intervention: Extending and flexing the foot with progressive loading of weight using an ankle cuff
Frequency: 3 x 25 min sessions per week plus 30 minutes rest after each session
Mode: One supervised training session at hospital, five home-based training sessions with parental supervision per fortnight
Dose: 2-3 sets of 8 repetitions at 5% of 1RM for 24 weeks. 1RM is measured every fortnight to adjust the weight lifted.

Adherence is monitored by an exercise diary documenting the number of training sessions completed and the intensity experienced with the Borg scale.
Control group
Dose comparison

Outcomes
Primary outcome [1] 288826 0
Dorsiflexion strength measured by hand-held dynamometry (Citec, C.I.T. Technics, Haren, the Netherlands)
Timepoint [1] 288826 0
Baseline, 6 months, 12 months, 24 months
Primary outcome [2] 288827 0
Safety measured by Magnetic Resonance Imaging of leg and foot muscles
Timepoint [2] 288827 0
Baseline, 6 months and 24 months
Secondary outcome [1] 301078 0
Disability measured with the CMT Pediatric Scale. The CMTPedS is a patient-centred, psychometrically robust 11-item scale consisting of seven areas of measurement (strength, dexterity, sensation, gait, balance, power, endurance).
Timepoint [1] 301078 0
Baseline, 6-months, 12-months and 24-months
Secondary outcome [2] 301079 0
Gait measured with 3D Gait Analysis
Timepoint [2] 301079 0
Baseline, 6 months and 24 months
Secondary outcome [3] 301080 0
Quality of life measured with the Child Health Questionnaire
Timepoint [3] 301080 0
Baseline, 6 months, 12 months, 24 months
Secondary outcome [4] 301081 0
Ankle stability measured with the Cumberland Ankle Instability Tool-Youth (CAITY)
Timepoint [4] 301081 0
Baseline, 6 months, 12 months, 24 months

Eligibility
Key inclusion criteria
Children aged 6-17 years with a confirmed diagnosis of Charcot-Marie-Tooth disease (CMT)
Minimum age
6 Years
Maximum age
17 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Strength training 3-months prior to recruitment; acute foot or lower limb injuries (e.g. fracture, ankle sprain); previous intra-articular foot surgery (e.g. fusion); an inability to actively dorsiflex (<10% of norm); non-ambulant; inability to comply with the research protocol (e.g. prolonged absence); hypertension; cardiovascular conditions which are exacerbated by exercise; diagnosis of inflammatory arthritis, diabetes or other peripheral neurological disorder; highly dependent on medical care; people with a cognitive impairment, an intellectual disability or a mental illness or insufficient knowledge of the English language to complete the required questionnaires during the study.

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)
Recruitment will be through referral from a Paediatric Neurologist, or those already enrolled in the Australasian Paediatric CMT Registry.

Allocation concealment procedures: Randomisation will occur using a computer-generated algorithm, maintained and assigned by our NHMRC Centre of Research Excellence phone-based system. All investigators will be concealed to the randomisation process and allocation sequence.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
The 1:1 randomisation sequence using random blocks will be determined by a computer-generated algorithm, maintained and assigned by our NHMRC Centre of Research Excellence phone-based system, with stratification according to age (6-11 years, 12-17 years)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s

The people assessing the outcomes
Intervention assignment
Parallel
Other design features
Phase
Not Applicable
Type of endpoint(s)
Safety/efficacy
Statistical methods / analysis
Analysis will be by intention-to-treat. Treatment effect between groups will be determined at 6-months (efficacy and safety), 12-months (maintenance of effect and safety) and 24-months (safety).

Based on previous studies and our pilot data, we estimate that a sample of 60 participants (30 per group) will provide 80% power (alpha 5%) to detect a difference between group means of 14% in dorsiflexion strength (SD 17%). We
assume a 50% correlation between pre- and post-test scores, loss to follow-up of 5% and non-compliance of 20%.

Recruitment
Recruitment status
Not yet 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)
NSW
Recruitment hospital [1] 559 0
The Children's Hospital at Westmead - Westmead
Recruitment postcode(s) [1] 6299 0
2145 - Westmead

Funding & Sponsors
Funding source category [1] 286710 0
Charities/Societies/Foundations
Name [1] 286710 0
Muscular Dystrophy Association - USA
Address [1] 286710 0
Muscular Dystrophy Association — USA
National Headquarters
3300 E. Sunrise Drive
Tucson, AZ 85718
Country [1] 286710 0
United States of America
Primary sponsor type
Individual
Name
Joshua Burns, PhD
Address
The Children's Hospital at Westmead
The University of Sydney
Locked Bag 4001
Westmead, 2145, NSW
Country
Australia
Secondary sponsor category [1] 285482 0
None
Name [1] 285482 0
Address [1] 285482 0
Country [1] 285482 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 288778 0
Sydney Childrens Hospitals Network Human Research Ethics Committee
Ethics committee address [1] 288778 0
Research Ethics Manager
Research Office
The Children's Hospital at Westmead
Locked Bag 4001
Westmead, 2145, NSW
Ethics committee country [1] 288778 0
Australia
Date submitted for ethics approval [1] 288778 0
23/01/2013
Approval date [1] 288778 0
18/04/2013
Ethics approval number [1] 288778 0
13/SCHN/21

Summary
Brief summary
We will conduct a 2-year randomised controlled trial to investigate the efficacy and safety of progressive resistance strength training of the foot and ankle in 60 children with Charcot-Marie-Tooth disease (CMT).

We will test the hypothesis that progressive resistance strength training is a safe intervention that will improve strength, disability, gait and quality of life in children affected by CMT.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 37722 0
A/Prof Joshua Burns
Address 37722 0
The Children's Hospital at Westmead
The University of Sydney
Locked Bag 4001
Westmead, 2145, NSW
Country 37722 0
Australia
Phone 37722 0
+61 2 9845 1228
Fax 37722 0
Email 37722 0
joshua.burns@health.nsw.gov.au
Contact person for public queries
Name 37723 0
Ms Amy Sman
Address 37723 0
The Children's Hospital at Westmead
The University of Sydney
Locked Bag 4001
Westmead, 2145, NSW
Country 37723 0
Australia
Phone 37723 0
+61 2 9845 3004
Fax 37723 0
Email 37723 0
amy.sman@health.nsw.gov.au
Contact person for scientific queries
Name 37724 0
A/Prof Joshua Burns
Address 37724 0
The Children's Hospital at Westmead
The University of Sydney
Locked Bag 4001
Westmead, 2145, NSW
Country 37724 0
Australia
Phone 37724 0
+61 2 98451228
Fax 37724 0
Email 37724 0
joshua.burns@health.nsw.gov.au

No information has been provided regarding IPD availability
Summary results
No Results