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Whole Body Vibration Therapy in Boys With Duchenne Muscular Dystrophy

Primary Purpose

Duchenne Muscular Dystrophy

Status
Completed
Phase
Not Applicable
Locations
Canada
Study Type
Interventional
Intervention
Whole Body Vibration Therapy
Sponsored by
Children's Hospital of Eastern Ontario
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional prevention trial for Duchenne Muscular Dystrophy focused on measuring Whole body vibration therapy, muscle strength testing, bone health, ambulatory, endurance.

Eligibility Criteria

8 Years - 14 Years (Child)MaleDoes not accept healthy volunteers

Inclusion Criteria:

  1. Diagnosis of Duchenne muscular dystrophy confirmed by at least one of the following:

    • Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, and clinical presentation consistent with typical DMD
    • Positive gene deletion test (missing one or more exons) in the central rod domain (exons 25-60) of dystrophin, where reading frame can be predicted as "out-of-frame", and clinical presentation consistent with typical DMD
    • Complete dystrophin gene sequencing showing an alteration (point mutation, duplication, or other mutation resulting in a stop codon mutation) definitively associated with DMD, and clinical presentation consistent with typical DMD
  2. Age between 5 - 14 yrs old (inclusive)
  3. Positive Gower sign (indicating ability to rise from the floor & presence of proximal muscle weakness).
  4. Able to walk 10 meters in <12 seconds
  5. Able to stand upon WBVT plate (with knees flexed) for entire treatment protocol (i.e. 15-minutes)
  6. Stable absolute dose of glucocorticoids (i.e. prednisone or deflazacort) for at least 3 months prior
  7. Stable absolute doses of all medication that may affect muscle function (i.e. coenzyme Q10, green tea extract, creatine, arginine, glutamine, nutritional supplements, etc.) for at least 3 months prior
  8. Stable absolute dose of all medication that may affect bone metabolism (i.e. vitamin D and calcium supplementation) for at least 3 months prior

Exclusion Criteria:

  1. Clinical presentation, genetic testing and/or muscle biopsy consistent with Becker muscular dystrophy
  2. History of recent surgery (within past 6-months)
  3. History of a recent fracture (long-bone or vertebral) within past 6-months.
  4. Acute inflammatory processes of lower extremities (e.g. cellulitis, etc) due to risk of pain and/or worsening inflammatory process
  5. History of venous thrombosis (theoretically risk of inducing thromboembolic event).
  6. History of kidney or bladder stones
  7. History of uncontrolled seizures or severe migraines
  8. History of cardiac arrhythmia
  9. Intracranial pathology or hardware (e.g. ventriculoperitoneal shunt, cochlear implant).
  10. Use of any investigational or experimental products within last 6-months and/or concomitant participation in another study
  11. Inability or refusal to follow the study requirements (e.g. autism, severe cognitive or behaviour problems)
  12. Inability or refusal to provide informed consent (parent) and/or assent (child)

Sites / Locations

  • Children's Hospital of Eastern Ontario

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

No Intervention

Arm Label

Whole Body Vibration Therapy

Control group

Arm Description

Group will receive daily whole body vibration therapy for up to 9 minutes maximum at a maximum of 18 Hz.

Group will not receive whole body vibration therapy. This group will conduct all other tests and outcomes except whole body vibration therapy.

Outcomes

Primary Outcome Measures

Assess the safety of using whole body vibration therapy in boys with Duchenne muscular dystrophy. To assess whether whole body vibration therapy can improve muscle strength and prolong ambulation from baseline to 8 weeks of therapy. To asses.
Is WBVT safe, convenient and well-tolerated when administered daily to ambulatory to boys with DMD?

Secondary Outcome Measures

Does WBVT result in any change in muscle strength.
Does WBVT results in any measurable change in muscle strength measured by the maximum resistance of deltoid, hip flexor and knee extensor (measured with microFET2 dynamometer) and grip strength (measured by Jamar hand-held dynamometer)
Does WBVT result in any muscle function change.
Does WBVT results in any measurable change in muscle function as measured by timed functional testing (timed 10m walk test; timed 4-stair climb; timed Gower manoeuvre, 6-minute walk test)?
Does WBVT result in any measurable change in muscle endurance.
Does WBVT result in any measurable change in muscle endurance (total number of steps taken each day, measured by pedometer)?
Quality of life changes.
Does WBVT result in any change in patient and family reported quality of life report? Measured by the Peds Q of L questionnaire.
Gait changes.
Does WBVT result in any change in patient's gait (as measured by Gangway gait analysis and Leonardo force plate analysis)
Bone health
Does WBVT result in any change in bone health indices (as measured by pQCT and routine skeletal imaging)

Full Information

First Posted
September 13, 2013
Last Updated
August 28, 2018
Sponsor
Children's Hospital of Eastern Ontario
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1. Study Identification

Unique Protocol Identification Number
NCT01954940
Brief Title
Whole Body Vibration Therapy in Boys With Duchenne Muscular Dystrophy
Official Title
THE EFFECT OF WHOLE BODY VIBRATION THERAPY UPON MUSCLE STRENGTH & FUNCTION IN AMBULATORY BOYS WITH DUCHENNE MUSCULAR DYSTROPHY
Study Type
Interventional

2. Study Status

Record Verification Date
August 2018
Overall Recruitment Status
Completed
Study Start Date
March 2013 (undefined)
Primary Completion Date
March 2017 (Actual)
Study Completion Date
March 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Children's Hospital of Eastern Ontario

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
Whole-body vibration therapy (WBVT) is a novel, non-pharmacological intervention aimed at improving muscle strength and endurance as well as bone density. It holds promise for children with neuromuscular disorders such as Duchenne muscular dystrophy (DMD) since muscle weakness results not only from muscle breakdown but also physical inactivity and muscle disuse atrophy. Weak DMD patients may increasingly limit their physical activity due to fear of falling or loss of independence (e.g. difficulty rising to stand without assistance). Prolonging the length of time boys with DMD are ambulatory is important for delaying complications of this disease (lung hypoventilation, scoliosis) as well as maintaining bone health. We propose to conduct a pilot study of WBVT in young boys with Duchenne muscular dystrophy (DMD). The primary outcome will be to document safety and feasibility of WBVT in this patient population. The secondary outcomes will evaluate changes in muscle strength and endurance. Bone health will also be examined as part of routine clinical care. The study will include 20 ambulatory boys with DMD; patients will be randomized (1:1 allocation) into 2 groups: WBVT treatment or no WBVT treatment (controls). Treatment groups will consist of 10 boys undergoing daily WBVT in an 8-week, open-label trial.
Detailed Description
Post-Study Completion Note: Given competition for enrollment in other DMD trials and burden from daily home WBVT training, it was not feasible to study WBVT in the trial setting, nor is it likely to be a feasible modality for optimizing musculoskeletal health in routine care.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy
Keywords
Whole body vibration therapy, muscle strength testing, bone health, ambulatory, endurance.

7. Study Design

Primary Purpose
Prevention
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
4 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Whole Body Vibration Therapy
Arm Type
Experimental
Arm Description
Group will receive daily whole body vibration therapy for up to 9 minutes maximum at a maximum of 18 Hz.
Arm Title
Control group
Arm Type
No Intervention
Arm Description
Group will not receive whole body vibration therapy. This group will conduct all other tests and outcomes except whole body vibration therapy.
Intervention Type
Device
Intervention Name(s)
Whole Body Vibration Therapy
Primary Outcome Measure Information:
Title
Assess the safety of using whole body vibration therapy in boys with Duchenne muscular dystrophy. To assess whether whole body vibration therapy can improve muscle strength and prolong ambulation from baseline to 8 weeks of therapy. To asses.
Description
Is WBVT safe, convenient and well-tolerated when administered daily to ambulatory to boys with DMD?
Time Frame
8 weeks
Secondary Outcome Measure Information:
Title
Does WBVT result in any change in muscle strength.
Description
Does WBVT results in any measurable change in muscle strength measured by the maximum resistance of deltoid, hip flexor and knee extensor (measured with microFET2 dynamometer) and grip strength (measured by Jamar hand-held dynamometer)
Time Frame
8 weeks
Title
Does WBVT result in any muscle function change.
Description
Does WBVT results in any measurable change in muscle function as measured by timed functional testing (timed 10m walk test; timed 4-stair climb; timed Gower manoeuvre, 6-minute walk test)?
Time Frame
8 weeks
Title
Does WBVT result in any measurable change in muscle endurance.
Description
Does WBVT result in any measurable change in muscle endurance (total number of steps taken each day, measured by pedometer)?
Time Frame
8 weeks
Title
Quality of life changes.
Description
Does WBVT result in any change in patient and family reported quality of life report? Measured by the Peds Q of L questionnaire.
Time Frame
8 weeks
Title
Gait changes.
Description
Does WBVT result in any change in patient's gait (as measured by Gangway gait analysis and Leonardo force plate analysis)
Time Frame
8 weeks
Title
Bone health
Description
Does WBVT result in any change in bone health indices (as measured by pQCT and routine skeletal imaging)
Time Frame
8 weeks

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
8 Years
Maximum Age & Unit of Time
14 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Diagnosis of Duchenne muscular dystrophy confirmed by at least one of the following: Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, and clinical presentation consistent with typical DMD Positive gene deletion test (missing one or more exons) in the central rod domain (exons 25-60) of dystrophin, where reading frame can be predicted as "out-of-frame", and clinical presentation consistent with typical DMD Complete dystrophin gene sequencing showing an alteration (point mutation, duplication, or other mutation resulting in a stop codon mutation) definitively associated with DMD, and clinical presentation consistent with typical DMD Age between 5 - 14 yrs old (inclusive) Positive Gower sign (indicating ability to rise from the floor & presence of proximal muscle weakness). Able to walk 10 meters in <12 seconds Able to stand upon WBVT plate (with knees flexed) for entire treatment protocol (i.e. 15-minutes) Stable absolute dose of glucocorticoids (i.e. prednisone or deflazacort) for at least 3 months prior Stable absolute doses of all medication that may affect muscle function (i.e. coenzyme Q10, green tea extract, creatine, arginine, glutamine, nutritional supplements, etc.) for at least 3 months prior Stable absolute dose of all medication that may affect bone metabolism (i.e. vitamin D and calcium supplementation) for at least 3 months prior Exclusion Criteria: Clinical presentation, genetic testing and/or muscle biopsy consistent with Becker muscular dystrophy History of recent surgery (within past 6-months) History of a recent fracture (long-bone or vertebral) within past 6-months. Acute inflammatory processes of lower extremities (e.g. cellulitis, etc) due to risk of pain and/or worsening inflammatory process History of venous thrombosis (theoretically risk of inducing thromboembolic event). History of kidney or bladder stones History of uncontrolled seizures or severe migraines History of cardiac arrhythmia Intracranial pathology or hardware (e.g. ventriculoperitoneal shunt, cochlear implant). Use of any investigational or experimental products within last 6-months and/or concomitant participation in another study Inability or refusal to follow the study requirements (e.g. autism, severe cognitive or behaviour problems) Inability or refusal to provide informed consent (parent) and/or assent (child)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Leanne Ward, MD
Organizational Affiliation
Children's Hospital of Eastern Ontario
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children's Hospital of Eastern Ontario
City
Ottawa
State/Province
Ontario
ZIP/Postal Code
K1H 8L1
Country
Canada

12. IPD Sharing Statement

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Whole Body Vibration Therapy in Boys With Duchenne Muscular Dystrophy

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