Rimeporide in Patients With Duchenne Muscular Dystrophy (RIM4DMD)

This is an interventional treatment trial for Muscular Dystrophy, Duchenne focused on measuring Rimeporide Read More

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.).