A clinical study to provide drisapersen (study medication) to patients withDuchenne disease (muscular disease) and to assess the efficacy, safety and tolerability.

Phase 1

• Conditions: Duchenne Muscular Dystrophy (DMD); MedDRA version: 18.1Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

• Registration Number: EUCTR2014-005296-81-BE
• Lead Sponsor: BioMarin Pharmaceutical Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2015-11-19
• Last Update Posted: 8 August 2016

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 104

Inclusion Criteria

1. Ambulant subjects with DMD resulting from a mutation/deletion within the dystrophin gene, confirmed by a Deoxyribonucleic acid (DNA) diagnostic technique covering all dystrophin gene exons (e.g. MLPA, CGH, SCAIP, H-RMCA) and correctable by drisapersen sodium-induced exon 51 skipping.

2. Male subjects aged =5 years at Screening.

3. Able to Rise from Floor in = 7 seconds on at least 2 of the 3 pre-treatment visits (screening 1, screening 2, and baseline).

4. Able to walk at least 300m on the 6MWD on at least 2 of the 3 pre-treatment visits (screening 1, screening 2, and baseline).

5. Glucocorticosteroid use which is stable for at least 3 months prior to the first screening visit. Subjects must have been receiving glucocorticosteroids for at least 6 months prior to the first screening visit.

6. Willing and able to adhere to the study visit schedule and other protocol requirements.

7. Written informed consent signed (by parent(s)/legal guardian and/or the subject, according to the local regulations).

8. In France, a subject will be eligible for inclusion in this study only if either affiliated to, or a beneficiary of, a social security category.
Are the trial subjects under 18? yes
Number of subjects for this age range: 104
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. More than a 30º (degree) flexion of either ankle, measure by goniometry.

2. Any additional mutations for DMD that cannot be treated by drisapersen sodium.

3. Current or history of liver or renal disease or impairment.

4. Screening platelet count below the lower limit of normal (LLN).

5. Acute illness within 4 weeks prior to first study drug administration which may interfere with the study assessments.

6. History of significant medical disorder which may confound the interpretation of efficacy or safety data (e.g. inflammatory disease, severe mental retardation and/or behavioral problems).

7. Severe cardiomyopathy which, in the opinion of the Investigator prohibits participation in this study. If a subject has a left ventricular ejection fraction <45% at screening, the Investigator should discuss inclusion of the subject with the Medical Monitor.

8. Chronic use of anti-coagulants, anti-thrombotics or anti-platelet agents within 1 month of the first administration of study drug.

9. Use of idebenone or other forms of coenzyme Q10 within 1 month prior to the start of the screening for the study.

10. Use of any investigational product or participation in another trial with an investigational product, within the half-life of that investigational product or a minimum of 6 months prior to the start of screening for the study.

11. Previous use of drisapersen sodium or eteplirsen.

12. NOTE: Subjects who fail on an entry criterion (apart from those subjects deemed to be ineligible for safety reasons) may be allowed to be re-screened at a later date, following discussion with the Medical Monitor.

Study & Design

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