Patient case (clinical description): 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's own account: 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.

Clinical trial: KUL0401: An Open-label Pilot Study of Oxatomide in Steroid-Naive Duchenne Muscular Dystrophy

Eligibility criteria:
Subject Inclusion Criteria

          1. 5 to 10 years of age

          2. ambulatory

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

               -  Positive x-linked family history of DMD in older male relatives (onset by 5
                  years, wheelchair bound by 12 years), or;

               -  Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin
                  deficiency, and clinical picture consistent with typical DMD, 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 DMD.

          4. glucocorticosteroid-naive (i.e. has not been treated with prednisone or deflazacort
             within the past year)

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

          6. QMT biceps score variability no greater than 10% between screening visits

        Subject Exclusion Criteria

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

          2. Inability to suitably cooperate with strength assessments

          3. Symptomatic DMD carrier

          4. Use of oxatomide (or other anti-histamine drugs) within the last 6 months for DMD or
             any other disease

          5. Use of creatine monohydrate or glutamine within the last 6 months

          6. Use of carnitine, Coenzyme Q10, other amino acids or any herbal medications within the
             last 3 months

          7. History of symptomatic cardiomyopathy

          8. History of impairment of hepatic function

          9. History of significant concomitant illness or significant impairment of renal
             function.
Male
No healthy subjects accepted to join the trial.
Subject must be at least 5 Years old.
Subject must be at most 10 Years

Eligibility (NLI label): Entailment