EspeRare_RIM

About this trial

Last updated 6 years ago

Study ID

EspeRare_RIM_001

Status

Completed

Type

Interventional

Phase

Phase 1

Placebo

Accepting

6 to 14 Years

Male

Not accepting

Healthy Volunteers

Trial Timing

Ended 7 years ago

In Duchenne Muscular Dystrophy (DMD) there is an imbalance between the levels of calcium and sodium in the muscles cells which is thought to be important in the damage which occurs overtime. Sodium/proton type 1 exchanger (NHE-1) inhibition is an innovative pathway that has proved to efficiently prevent the accumulation of muscle damage (inflammation and fibrosis) in animal models of muscular dystrophies and heart failure. Based on prior safety and efficacy results in animal and humans, NHE-1 inhibition with Rimeporide represents a new therapeutic approach with no restriction on age and on genetic subtypes which could be combined to other treatments that restore or augment dystrophin.This study examines the safety and tolerability and effects on the muscles of rimeporide, in patients aged 6 to 14 years with Duchenne Muscular Dystrophy (DMD).

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.).

Rimeporide in Patients With Duchenne Muscular Dystrophy