Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12614000173695
Ethics application status
Approved
Date submitted
18/01/2014
Date registered
12/02/2014
Date last updated
12/02/2014
Type of registration
Prospectively registered

Titles & IDs
Public title
Pilot Study of Progressive Resistance Training for Sedentary Adults with Charcot-Marie-Tooth
Scientific title
Pilot study to evaluate the effect of progressive resistance training on muscle strength in sedentary adults with Charcot-Marie-Tooth
Secondary ID [1] 283929 0
Nil Known
Universal Trial Number (UTN)
Trial acronym
CMT
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Charcot-Marie-tooth 290945 0
Condition category
Condition code
Neurological 291293 291293 0 0
Other neurological disorders
Human Genetics and Inherited Disorders 291464 291464 0 0
Other human genetics and inherited disorders
Musculoskeletal 291465 291465 0 0
Other muscular and skeletal disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
The proposed project is for an eight-week RCT on resistance training study in persons with Charcot-Marie-Tooth (CMT), a muscular atrophy disease. Traditionally, people with CMT have been discouraged from performing exercise without strong supporting scientific evidence. The resulting lower activity levels and decreased muscle mass (from the disease process) means persons with CMT population may have increased incidence of metabolic syndrome, diabetes and cardiovascular disease. This pilot study will provide information on the effectiveness of progressive resistance training (PRT) for people with CMT. This research will provide data for larger studies that answer more specific questions about CMT and exercise. The study is a blinded RCT. The total duration of the study will be approximately 12 weeks with each participant performing pre-tests then being randomized into one of two intervention groups for 8 weeks, followed by post testing. One group will perform 8 weeks of individual power training exercise sessions supervised by an exercise physiologist (Exercise group) and the other group (Control group) will maintain their usual activity levels.

The power training group will require participants to attend three training sessions each week for 8 weeks at University of Sydney, Lidcombe campus. The training sessions would last for 60minutes each session (e.g. 3 x 60 minutes training sessions per week for 8 weeks). The exercises will focus on the upper and lower extremities, using Keiser pneumatic strength training equipment. The exercises will include leg press, chest press, knee extension, Ankle dorsiflexion, Hip abduction, seated row etc. The intensity of the training will be built up over the first few sessions to high intensity (80% 1RM) and then maintained at this intensity level for the remainder of the program.
Intervention code [1] 288624 0
Behaviour
Intervention code [2] 288725 0
Lifestyle
Comparator / control treatment
The Control group will maintain their usual activity levels.
Control group
Active

Outcomes
Primary outcome [1] 291287 0
Leg Press Muscle strength (Hamstring, gluteals, quadriceps): The 1 Repetition Maximum (1RM) test will be used to determine the dynamic maximal muscle strength. This technique has been used extensively to monitor gains in muscle strength in strength training intervention in older adults and gives a good reliability.
Timepoint [1] 291287 0
at baseline and after 8weeks
Primary outcome [2] 291455 0
Chest Press Muscle strength (Pectoralis, triceps). The 1 Repetition Maximum (1RM) test will be used to determine the dynamic maximal muscle strength.
Timepoint [2] 291455 0
At Baseline and after 8 weeks.
Secondary outcome [1] 306429 0
Body composition analysis: Dual-energy X-ray absorptiometry (DXA scan). DXA scan for whole body composition of bone mass, muscle mass and strength ratios.
Timepoint [1] 306429 0
only at baseline
Secondary outcome [2] 306430 0
Physical Performance: measured by Short Physical Performance Battery (SPPB) which involves scores on balance test, sit to stand test, and habitual walking speed.
Timepoint [2] 306430 0
at baseline and after 8 weeks
Secondary outcome [3] 306431 0
Habitual physical activity and sedentary behaviour: Actigraph
accelerometers set at 10 s epochs will be worn during all waking hours for 7 days on the non-dominant hip as well as on the non-dominant wrist at all times to monitor sleep quality.
Timepoint [3] 306431 0
at baseline and after 8 weeks
Secondary outcome [4] 306432 0
Walking Biomechanics: Surface Electromyography (EMG). EMG electrodes will be placed on the skin over 8 leg muscles on the dominant leg of the participants. The participants will then be asked to perform standard movements to allow the measurement of a standard value for the muscle activity to be referenced to. The subjects will be asked to walk on a treadmill at a comfortable pace for 1 minute. The treadmill is instrumented with sensors that allow the measurement of walking parameters.
Timepoint [4] 306432 0
at baseline and after 8 weeks
Secondary outcome [5] 306433 0
Questionnaire: The Short Form (36) Health Survey (SF-36)

SF-36 is a patient-reported survey of patient health and is a measure of health status.
Timepoint [5] 306433 0
at baseline and after 8 weeks
Secondary outcome [6] 306719 0
Questionnaire: Centre for Epidemiological Studies Depression Scale (CES-D).

The CES_D is a widely used 20 item self-report scale which measures the current level of depressive symptomatology in the general population, with an emphasis on depressed mood during the past week.
Timepoint [6] 306719 0
at baseline and after 8 weeks
Secondary outcome [7] 306720 0
Questionnaire: Current Level of overall body pain measured by Visual analog scale (VAS).

VAS is a psychometric response scale and it is a measurement instrument for subjective characteristics or attitudes that cannot be directly measured.
Timepoint [7] 306720 0
at baseline and after 8 weeks
Secondary outcome [8] 306721 0
Questionnaire: EWART self efficacy Scale (EWART).

This is questionnaire examine the self-efficacy of participants in regards to physical activities.
Timepoint [8] 306721 0
at baseline and after 8 weeks
Secondary outcome [9] 306722 0
Questionnaire: Demograhic survey.

This questionnaire attains general information for subject identifications such as gender, residence, education, smoking status, hospital admission etc.
Timepoint [9] 306722 0
at baseline only
Secondary outcome [10] 306816 0
Stair Climb Power test. The Stair Climb Power Test (SCPT) is a functional test associated with leg muscle power.
Timepoint [10] 306816 0
at Baseline and 8 weeks
Secondary outcome [11] 306817 0
6 Minute Walk Test. The six-minute walk test (6MWT) measures the distance an individual is able to walk over a total of six minutes on a hard, flat surface.
Timepoint [11] 306817 0
at Baseline and 8 weeks
Secondary outcome [12] 306818 0
Handgrip Strength (via Handgrip Dynamometer)
Timepoint [12] 306818 0
at Baseline and 8 weeks

Eligibility
Key inclusion criteria
Sixteen individuals with CMT Type 1 or X will participate in this project.The following inclusion criteria enable the volunteer to participate in the CMT Study:

-Participants will have either type 1 or X CMT.
-They will be aged between 18-60yrs
-Be able to walk 100m at a gait speed over 0.4 ms-1 and without an assistive device [ankle-foot orthoses are allowed, no bilateral assistance]
-Participants must be able to get to Cumberland by own transport.
Minimum age
18 Years
Maximum age
60 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
The following conditions permanently exclude the subject from
participation in the CMT STUDY:
- Participants with recent exacerbation of CMT condition
- Those who are not sedentary (i.e. currently participating in structured aerobic or weight training activities)
- Those who have medical conditions that would preclude them from intense exercise.

Study design
Purpose of the study
Educational / counselling / training
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
To assess subject eligibility for CMT Study, a telephone screening would be administered. The questions on the form were designed to address all inclusion and exclusion criteria. A 30 minute phone call from the interviewer allowed all volunteers to answer the questions and ask any questions the subjects had about the study. After the forms were reviewed by the researchers, all potential subjects were informed of
their eligibility and if further screening was required. Written informed consent will be required prior to any testing or randomisation.

Allocation was concealed using sealed opaque envelopes given to eligible participants after completion of all baseline testing. Participants will be randomised into an intervention group, or a control group following completion of baseline assessments.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Randomization will be performed by a researcher, who is not involved in testing and counselling of participants, using computer- generated randomly permuted blocks. Participants will be informed of their group allocation by means of sealed opaque envelopes given to them after completion of all baseline testing.
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?


The people assessing the outcomes
Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint/s
Efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
27/02/2014
Actual
Date of last participant enrolment
Anticipated
30/05/2014
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
16
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW

Funding & Sponsors
Funding source category [1] 288572 0
Charities/Societies/Foundations
Name [1] 288572 0
The Charcot-Marie-Tooth Australia Association (CMTAA)
Country [1] 288572 0
Australia
Primary sponsor type
Charities/Societies/Foundations
Name
The Charcot-Marie-Tooth Australia Association (CMTAA)
Address
Building 22
Concord Hospital
Concord, NSW
2139 Australia
Country
Australia
Secondary sponsor category [1] 287283 0
None
Name [1] 287283 0
Address [1] 287283 0
Country [1] 287283 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 290435 0
The University of Sydney HREC
Ethics committee address [1] 290435 0
Level 6, Jane Foss Russell
The University of Sydney
NSW 2006 Australia
Ethics committee country [1] 290435 0
Australia
Date submitted for ethics approval [1] 290435 0
26/08/2013
Approval date [1] 290435 0
08/01/2014
Ethics approval number [1] 290435 0
2013/851

Summary
Brief summary
Charcot-Marie-Tooth (CMT) is the most frequently inherited peripheral neuropathy affecting around 1 in 2500 persons. CMT causes slow degeneration of the nerves in the extremities including feet, legs, arms and hands. Typically muscles weaken and atrophy due to the loss of activation by the affected nerves. In the past, persons with CMT have been discouraged from performing exercise and physical activity because clinicians believed that such activity might hasten the disease; these beliefs have never been validated by studies. However, it is now known that regular exercise or activity helps maintain good health and function in most populations. Inactivity and a poor muscle mass can also lead to metabolic syndrome, increasing morbidity and the risk of developing diabetes and cardiovascular disease. These metabolic diseases may even worsen the symptoms of Charcot-Marie-Tooth disease, for example diabetes can worsen CMT symptoms.

More evidence is required on exercise for persons with CMT, especially at moderate to high intensities. Recent research has shown that light and moderate intensity exercise is not detrimental to persons with CMT and offers many of the same benefits to persons with CMT as it does to the general population . The few previous studies have used home-based low intensity training (i.e. 20-30% maximum lift) for the upper limbs and moderate intensity for the legs (i.e. 40-50% maximum lift). In the neurologically normal population, muscle and strength gains are correlated with the intensity of exercise, i.e. high intensity training develops greater strength and muscle mass. A considerable amount of weakness and muscle atrophy in persons with CMT may be due to secondary disuse due inactivity. Higher intensity training could be beneficial for those with CMT to improve muscle mass, power and strength. The benefits of intense training might also provide persons with CMT from developing secondary metabolic disease or lead to improve functional ability in daily activities.

We propose to conduct a study investigating the benefits of a high intensity training gym-based program (i.e. power training) for individuals with CMT. The proposed project is an eight-week resistance training randomized controlled pilot trial. This pilot study will provide information on the effectiveness of progressive resistance training (PRT) for people with CMT. We expect power training may benefit individuals with CMT by increasing muscle mass (strength and metabolic benefits) and muscle power (increased function). This research will provide data for a larger National Health and Medical Research Council funding application that will have the statistical power to answer more specific questions about CMT and exercise.

Recently, many people with CMT have become aware that exercise might be beneficial for them, but do not understand how to accommodate exercise in their lives. Therefore, the results of this pilot study will be promoted to educate Exercise Sports Science Australia (ESSA) and Australian Accredited Exercise Physiologists (AEP) on the benefits of exercise for CMT. In time, we hope that people with CMT will be able to easily contact AEPs in the community for advice on exercise.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 45674 0
Prof Maria Fiatarone Singh
Address 45674 0
The University of Sydney Faculty of Health Sciences Exercise, Health & Performance Faculty Research Group Room K221, 75 East St Lidcombe NSW 2141
Country 45674 0
Australia
Phone 45674 0
+61 2 9351 9755
Fax 45674 0
+61 2 9351 9204
Email 45674 0
[email protected]
Contact person for public queries
Name 45675 0
Ms Nidhi Saigal
Address 45675 0
The University of Sydney Faculty of Health Sciences Exercise, Health & Performance Faculty Research Group Room K220, 75 East St Lidcombe NSW 2141
Country 45675 0
Australia
Phone 45675 0
+61 2 9351 9138
Fax 45675 0
+61 2 9351 9204
Email 45675 0
[email protected]
Contact person for scientific queries
Name 45676 0
Prof Maria Fiatarone Singh
Address 45676 0
The University of Sydney Faculty of Health Sciences Exercise, Health & Performance Faculty Research Group Room K221, 75 East St Lidcombe NSW 2141
Country 45676 0
Australia
Phone 45676 0
+61 2 9351 9755
Fax 45676 0
+61 2 9351 9204
Email 45676 0
[email protected]

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
SourceTitleYear of PublicationDOI
EmbaseRelationship between physical performance and quality of life in Charcot-Marie-Tooth disease: a pilot study.2016https://dx.doi.org/10.1111/jns.12191
N.B. These documents automatically identified may not have been verified by the study sponsor.