ANZCTR - Registration

Please note the ANZCTR will be unattended from Friday 20 December 2024 for the holidays. The Registry will re-open on Tuesday 7 January 2025. Submissions and updates will not be processed during that time.

Registering a new trial?

To achieve prospective registration, we recommend submitting your trial for registration at the same time as ethics submission.

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

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]

Nil Known

Universal Trial Number (UTN)

Trial acronym

CMT

Linked study record

Health condition

Health condition(s) or problem(s) studied:

Charcot-Marie-tooth

Condition category

Condition code

Neurological

Other neurological disorders

Human Genetics and Inherited Disorders

Other human genetics and inherited disorders

Musculoskeletal

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]

Behaviour

Intervention code [2]

Lifestyle

Comparator / control treatment

The Control group will maintain their usual activity levels.

Control group

Active

Outcomes

Primary outcome [1]

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]

at baseline and after 8weeks

Primary outcome [2]

Chest Press Muscle strength (Pectoralis, triceps). The 1 Repetition Maximum (1RM) test will be used to determine the dynamic maximal muscle strength.

Timepoint [2]

At Baseline and after 8 weeks.

Secondary outcome [1]

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]

only at baseline

Secondary outcome [2]

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]

at baseline and after 8 weeks

Secondary outcome [3]

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]

at baseline and after 8 weeks

Secondary outcome [4]

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]

at baseline and after 8 weeks

Secondary outcome [5]

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]

at baseline and after 8 weeks

Secondary outcome [6]

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]

at baseline and after 8 weeks

Secondary outcome [7]

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]

at baseline and after 8 weeks

Secondary outcome [8]

Questionnaire: EWART self efficacy Scale (EWART).

This is questionnaire examine the self-efficacy of participants in regards to physical activities.

Timepoint [8]

at baseline and after 8 weeks

Secondary outcome [9]

Questionnaire: Demograhic survey.

This questionnaire attains general information for subject identifications such as gender, residence, education, smoking status, hospital admission etc.

Timepoint [9]

at baseline only

Secondary outcome [10]

Stair Climb Power test. The Stair Climb Power Test (SCPT) is a functional test associated with leg muscle power.

Timepoint [10]

at Baseline and 8 weeks

Secondary outcome [11]

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]

at Baseline and 8 weeks

Secondary outcome [12]

Handgrip Strength (via Handgrip Dynamometer)

Timepoint [12]

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?

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

Accrual to date

Final

Recruitment in Australia

Recruitment state(s)

NSW

Funding & Sponsors

Funding source category [1]

Charities/Societies/Foundations

Name [1]

The Charcot-Marie-Tooth Australia Association (CMTAA)

Address [1]

Building 22
Concord Hospital
Concord, NSW
2139 Australia

Country [1]

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]

None

Name [1]

Address [1]

Country [1]

Ethics approval

Ethics application status

Approved

Ethics committee name [1]

The University of Sydney HREC

Ethics committee address [1]

Level 6, Jane Foss Russell
The University of Sydney
NSW 2006 Australia

Ethics committee country [1]

Australia

Date submitted for ethics approval [1]

26/08/2013

Approval date [1]

08/01/2014

Ethics approval number [1]

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

Prof Maria Fiatarone Singh

Address

The University of Sydney Faculty of Health Sciences Exercise, Health & Performance Faculty Research Group Room K221, 75 East St Lidcombe NSW 2141

Country

Australia

Phone

+61 2 9351 9755

Fax

+61 2 9351 9204

maria.fiataronesingh@sydney.edu.au

Contact person for public queries

Name

Nidhi Saigal

Address

The University of Sydney Faculty of Health Sciences Exercise, Health & Performance Faculty Research Group Room K220, 75 East St Lidcombe NSW 2141

Country

Australia

Phone

+61 2 9351 9138

Fax

+61 2 9351 9204

nidhi.jain@sydney.edu.au

Contact person for scientific queries

Name

Maria Fiatarone Singh

Address

The University of Sydney Faculty of Health Sciences Exercise, Health & Performance Faculty Research Group Room K221, 75 East St Lidcombe NSW 2141

Country

Australia

Phone

+61 2 9351 9755

Fax

+61 2 9351 9204

maria.fiataronesingh@sydney.edu.au

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

Source	Title	Year of Publication	DOI
Embase	Relationship between physical performance and quality of life in Charcot-Marie-Tooth disease: a pilot study.	2016	https://dx.doi.org/10.1111/jns.12191

N.B. These documents automatically identified may not have been verified by the study sponsor.

Trial Review

Registering a new trial?

Documents added manually

Documents added automatically