A phase Ib, open label study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending oral doses of rimeporide in patients with Duchenne Muscular Dystrophy (DMD)

Phase 1

• Conditions: Distrofia Muscolare di Duchenne (DMD); MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

• Registration Number: EUCTR2015-002530-50-IT
• Lead Sponsor: ESPERARE FOUNDATIO

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Completion: 2017-12-20
• Study Start: 2018-08-22
• Last Update Posted: 10 December 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 20

Inclusion Criteria

1. Duchenne muscular dystrophy genetically confirmed;
2. Males between 6 and 14 years old;
3. Able to walk independently at least 75 meters;
4. Patients on a stable dose of corticosteroids at least 6 months prior to baseline;
5. Willing and able to comply with all protocol requirements and procedures;
6. Signed informed consents by the parent(s)/legal guardian(s);
7. France only: Affiliated to or a beneficiary of a social security system
Are the trial subjects under 18? yes
Number of subjects for this age range: 1
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. 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;
2. Current or history of liver disease or impairment,
3. 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;
4. Acute illness within 4 weeks of the first administration of study medication which may interfere with study assessments;
5. Significant change of dosage and/or dosing regimens for corticosteroids planned for the duration of study medication;
6. Use of beta blockers / and ACEI or ARB unless at stable dose for at least 3 months prior to baseline;
7. Use of protocol pump inhibitors unless at a stable dose for at least 3 months prior to baseline
8. Use of aldosterone antagonists (i.e. spironolactone, eplerenone) within 3 months prior to first administration of study medication;
9. Use of anticoagulants, antithrombotics or antiplatelet agents,
10. Previous treatment with idebenone or other forms of Coenzyme Q10 within 1 month of the first administration of study medication;
11. 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;
12. A baseline QTc>450msec,or history of risk factors for torsades de pointes (eg, heart failure, hypokalaemia, family history of long QT syndrome);
13. LVEF= 45% at screening or within the past 6 months and/or history of acute heart failure;
14. Ventilator dependent;
15. Known individual hypersensitivity to any of the ingredients/excipients of the study medication;
16. For patients willing to undergo MRI/MRS assessments: Patients with specific contraindication to MRI (e.g.: metallic foreign body, claustrophobia, etc.).

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified