Clinical trial: An Open-label Pilot Study of Coenzyme Q10 in Steroid-Treated Duchenne Muscular Dystrophy (NCT00033189)

Eligibility criteria:
Subject Inclusion Criteria

          1. Age: 5 - 11 years old

          2. Ambulant

          3. Diagnosis of DMD confirmed by at least one the following:

               -  Positive X-linked family history for typical Duchenne muscular dystrophy in older
                  male relatives (onset by age 5 yr., wheelchair-bound by age 12 yr.) OR

               -  Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin
                  deficiency, and clinical picture consistent with typical Duchenne dystrophy OR

               -  Gene deletion test positive (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 picture consistent with typical Duchenne dystrophy.

          4. On Glucocorticosteroids: Children must be on a steady dose of prednisone or
             deflazacort, on any schedule (Daily, alternate days, 10 days on, 10 days off or twice
             a week), for the last 6 months before starting the clinical trial. Dose of steroid or
             schedule cannot be altered during the study.

          5. Evidence of muscle weakness by MRC score or clinical functional evaluation

          6. Ability to provide reproducible repeat QMT bicep score within 10% of first assessment
             score.

          7. Ability to swallow tablets

        Subject Exclusion Criteria

          1. Failure to achieve one or more of the diagnostic inclusion criteria cited above.

          2. Symptomatic DMD carrier

          3. Previous (6 months or less) or current use of Coenzyme Q10 (for DMD or any other
             disease)

          4. Use of carnitine, other amino acids, creatine, glutamine, or any herbal medicines
             within the last 3 months.

          5. History of significant concomitant illness or significant impairment of renal or
             hepatic function.
Male
No healthy subjects accepted to join the trial.
Subject must be at least 5 Years old.
Subject must be at most 11 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