Clinical trial: Rimeporide in Patients With Duchenne Muscular Dystrophy (NCT02710591)

Eligibility criteria:
Inclusion Criteria:

          -  Duchenne muscular dystrophy genetically confirmed;

          -  Males between 6 and 14 years old;

          -  Able to walk independently at least 75 meters;

          -  Patients on a stable dose of corticosteroids at least 6 months prior to baseline;

          -  Patients able to swallow capsules size 4 according to the parents and investigator
             opinion;

          -  Willing and able to comply with all protocol requirements and procedures;

          -  Signed informed consents by the parent(s)/legal guardian(s);

          -  France only: Affiliated to or a beneficiary of a social security system

        Exclusion Criteria:

          -  Patients with significant renal disease or impairment, with Glomerular Filtration Rate
             estimated using plasma cystatin C level using the Filler formula less than
             90ml/min/1.73m2

          -  Current or history of liver disease or impairment,

          -  History of any significant medical disorder which may confound the interpretation of
             either efficacy or safety data e.g. inflammatory, coagulation disease, unstable
             cardiac or respiratory disease

          -  Acute illness within 4 weeks of the first administration of study medication which may
             interfere with study assessments;

          -  Significant change of dosage and/or dosing regimens for corticosteroids planned for
             the duration of study medication;

          -  Use of beta blockers / and ACEI or ARB unless at stable dose for at least 3 months
             prior to baseline;

          -  Use of Proton Pump Inhibitors unless at a stable dose for at least 3 months prior to
             baseline

          -  Use of aldosterone antagonists (i.e. spironolactone, eplerenone) within 3 months prior
             to first administration of study medication;

          -  Use of anticoagulants, antithrombotics or antiplatelet agents,

          -  Use of antibiotics with predominant renal secretion (e.g., cephalosporins),
             immunosuppressive agents exception corticosteroids, continuous treatment with
             non-steroidal, anti-inflammatory drugs (NSAIDs), or lithium;

          -  Previous treatment with idebenone or other forms of Coenzyme Q10 within 1 month of the
             first administration of study medication;

          -  Previous treatment with investigational drugs within 4 weeks (or 7 half-life if longer
             than 4 weeks) of the first administration of study medication including placebo;

          -  A baseline QTc>450msec,or history of risk factors for torsades de pointes (eg, heart
             failure, hypokalaemia, family history of long QT syndrome);

          -  LVEF≤ 45% at screening or within the past 6 months and/or history of acute heart
             failure;

          -  Ventilator dependent;

          -  Known individual hypersensitivity to any of the ingredients/excipients of the study
             medication;

          -  Patients with specific contraindication to MRI (e.g.: metallic foreign body,
             claustrophobia, etc.).
Male
No healthy subjects accepted to join the trial.
Subject must be at least 6 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