Whole Body Vibration Therapy in Boys With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy Clinical Trial

Official title:

THE EFFECT OF WHOLE BODY VIBRATION THERAPY UPON MUSCLE STRENGTH & FUNCTION IN AMBULATORY BOYS WITH DUCHENNE MUSCULAR DYSTROPHY

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01954940
• Other study ID #: # 12/17E
• Status: Completed
• Phase: N/A
• Start date: March 2013
• Est. completion date: March 2017

Study information

• Verified date: August 2018
• Source: Children's Hospital of Eastern Ontario
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 4
• Est. completion date: March 2017
• Est. primary completion date: March 2017
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 8 Years to 14 Years

Eligibility

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)

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Device:
• Whole Body Vibration Therapy

Locations

Country	Name	City	State
Canada	Children's Hospital of Eastern Ontario	Ottawa	Ontario

Sponsors (1)

Lead Sponsor	Collaborator
Children's Hospital of Eastern Ontario

Country where clinical trial is conducted

Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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?	8 weeks
Secondary	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)	8 weeks
Secondary	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)?	8 weeks
Secondary	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)?	8 weeks
Secondary	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.	8 weeks
Secondary	Gait changes.	Does WBVT result in any change in patient's gait (as measured by Gangway gait analysis and Leonardo force plate analysis)	8 weeks
Secondary	Bone health	Does WBVT result in any change in bone health indices (as measured by pQCT and routine skeletal imaging)	8 weeks