Clinical trial: Finding the Optimum Regimen for Duchenne Muscular Dystrophy (NCT01603407)

Eligibility criteria:
Inclusion Criteria:

          -  Evidence of signed and dated informed consent form.

          -  Confirmed diagnosis of Duchenne muscular dystrophy

          -  Age greater than or equal to 4 years and less than 8 years old

          -  Ability to rise independently from floor, from supine to standing

          -  Willingness and ability to comply with scheduled visits, drug administration plan and
             study procedures

          -  Ability to maintain reproducible FVC measurements.

        Exclusion Criteria:

          -  History of major renal or hepatic impairment, immunosuppression or other
             contraindications to corticosteroid therapy.

          -  History of chronic systemic fungal or viral infections. Acute bacterial
             infection(including TB) would exclude from enrolment until the infection had been
             appropriately treated and resolved.

          -  Diabetes mellitus.

          -  Idiopathic hypercalcuria.

          -  Lack of chicken pox immunity and refusal to undergo immunization.

          -  Evidence of symptomatic cardiomyopathy at screening assessment (one to three months
             prior to the baseline visit). Asymptomatic cardiac abnormality on investigation would
             not be an exclusion.

          -  Current or previous treatment (greater than four consecutive weeks of oral therapy)
             with corticosteroids or other immunosuppressive treatments for DMD or other recurrent
             indications (e.g., asthma), unless approved by FOR-DMD Team (i.e., concurrent
             participation in another allowed DMD trial).

          -  Inability to take tablets, as assessed by the site investigator by the end of the
             screening period (the screening period ranges from one to three months prior to the
             baseline visit).

          -  Allergy/sensitivity to study drugs or their formulations including lactose and/or
             sucrose intolerance.

          -  Severe behavioral problems, including severe autism.

          -  Previous or ongoing medical condition, medical history, physical findings or
             laboratory abnormalities that could affect safety, make it unlikely that treatment and
             follow up will be correctly completed or impair the assessment of study results, in
             the judgment of the site investigator.

          -  Weight of less than 13 kilograms.

          -  Exposure to any investigational drug currently or within 3 months prior to start of
             study treatment.
Male
No healthy subjects accepted to join the trial.
Subject must be at least 4 Years old.
Subject must be at most 7 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? Contradiction