Clinical trial: An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort (NCT02295748)

Eligibility criteria:
Inclusion Criteria:

          -  Subject is capable of understanding and complying with protocol requirements.

          -  Subject or, when applicable, the subject's legally acceptable representative signs and
             dates a written, informed consent form and any required privacy authorization prior to
             the initiation of any study procedures.

          -  If above the age of 7, the subject signs and dates a written, informed assent form
             (IAF) and any required privacy authorization prior to the initiation of any study
             procedures. Subjects under age 7 at the time of study entry who turn age 7 will sign
             and date a written informed assent form (IAF) at the visit following their 7th
             birthday, if required by the site's IRB.

          -  Subject participated in and received at least one dose of study medication in the
             MP-104-CL-005 protocol.

          -  The subject must have confirmed diagnosis of Duchenne Muscular Dystrophy defined as:

               1. onset of weakness before 5 years of age;

               2. proximal muscle weakness;

               3. increased serum creatine kinase more than 10 times the upper limit of normal
                  (ULN);

               4. muscle biopsy and dystrophin analyses consistent with DMD or DNA mutation and
                  analysis by PCR or Southern blot techniques to detect gene deletions.

          -  The subject weighs at least 13 kg and has a body mass index (BMI) of ≤ 40 kg/m2.

          -  Willingness and ability to comply with scheduled visits, oral drug administration, and
             study procedures including blood sample draws for safety labs.

          -  Up to date on all childhood vaccinations, including varicella vaccine (chicken pox).

          -  Baseline health is judged to be stable based on medical history, physical examination,
             laboratory profiles, vital signs, or ECGs at screening, as deemed by the Investigator.

          -  Continuous non smoker who has not used nicotine containing products for at least 3
             months prior to the first dose.

          -  The subject is able to take tablets.

        Exclusion Criteria:

          -  The subject has received any investigational compound and/or has participated in
             another clinical study within 90 days prior to study treatment with the exception of
             MP-104-CL-005, observational cohort studies or non-interventional studies.

          -  The subject is an immediate family member, study site employee, or is in a dependent
             relationship with a study site employee who is involved in the conduct of this study
             (e.g. spouse, parent, child, sibling) or may consent under duress.

          -  Any significant finding on the Columbia suicide severity rating scale (C SSRS) for
             subjects (ages 12-16, inclusive), in the opinion of the PI, warrants exclusion from
             this study.

          -  The subject has, in the judgment of the, clinically significant abnormal clinical
             chemistry laboratory parameters that may affect safety at Day 0.

          -  The subject has, in the judgment of the Investigator, a history or current medical
             condition that could affect safety including, but not limited to:

               1. Major renal or hepatic impairment

               2. Immunosuppression or other contraindications for corticosteroid treatment

               3. History of chronic systemic fungal or viral infections

               4. Diabetes mellitus

               5. Idiopathic hypocalciuria

               6. Symptomatic cardiomyopathy at Day 0

          -  The subject has a history of hypersensitivity or allergic reaction to steroids or
             their formulations including, but not limited to lactose, sucrose, etc.

          -  Inability to take tablets as assessed by site investigator.

          -  Subject is mentally or legally incapacitated or has significant emotional problems at
             the time of screening visit or expected during the conduct of the study.

          -  History of any illness that, in the opinion of the PI, might confound the results of
             the study or poses an additional risk to the subject by their participation in the
             study.

          -  Positive urine drug or alcohol results at Day 0.

          -  Hemoglobin level below the lower limit of normal at Day 0.
Male
No healthy subjects accepted to join the trial.
Subject must be at least 4 Years old.


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? 1