Clinical trial: Whole Body Vibration Therapy in Boys With Duchenne Muscular Dystrophy (NCT01954940)

Eligibility criteria:
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)
Male
No healthy subjects accepted to join the trial.
Subject must be at least 8 Years old.
Subject must be at most 14 Years

Patient description (medical language): An 8-year-old boy is brought to the clinic by his parents because of weakness and difficulty of standing up from a sitting position. The mother is healthy but had a brother who died in his 20th after being disabled and using wheelchairs in the last few years of his life. Physical examination shows 3/5 lower extremity muscle strength and enlarged calf muscles. The other physical examination and vital signs are unremarkable. Muscle biopsy showed absence of dystrophin protein. The patient is diagnosed with DMD.

Patient description (patient's own words): I brought my  8-year-old son to the doctor's clinic. He was struggling to stand up from a chair for the past few days. My wife is healthy but she was pretty worried since his brother died in his 20s after being disabled and using a wheelchair for the last years of his life. The doctor did an exam which highlighted weakness in the leg muscles and unusually large calf muscles. The rest of the exam was fine, and his vitals were normal. My son did a muscle biopsy, and it showed that the protein important for muscle strength and function is missing. The doctor diagnosed him with Duchenne muscular dystrophy.

Does the patient match the trial criteria? Entailment