NCT02636686

Extension Study of Drisapersen in DMD Subjects

An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of Drisapersen in Subjects With Duchenne Muscular Dystrophy.

• Status: No Longer Available
• Phase: —
• Study type: Expanded Access
• Enrollment: —
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: Male
• Age: 5 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

This is a phase IIIb, multi-centre, open-label extension study in male subjects with DMD who previously have been treated with drisapersen, aiming at assessing the safety and efficacy of drisapersen.

Detailed description

This is a phase IIIb, multi-centre, open-label extension study in male subjects with DMD who have previously been treated with drisapersen. This study aims to enroll up to approximately 220 subjects. The primary dosing arm is drisapersen 6 mg/kg as subcutaneous (SC) injection(s) once a week. All subjects starting with subcutaneous injections will receive a loading dose of twice weekly 6mg/kg drisapersen for the first three weeks of treatment. This study does not have a minimum duration of participation. Subjects will have varying times of study participation depending on when they enter from one of the eligible studies and will be permitted to continue the study until such a time that they withdraw based on protocol-defined criteria, or BioMarin stops the study. Subjects naïve to treatment are not eligible for participation in this study For subjects who have previously experienced significant safety or tolerability issues in one of the eligible studies, or who experience these during this study, there is the potential of an alternate intermittent dosing arm. This will be agreed in advance with the Medical Monitor. For subjects who have previously experienced significant injection site reactions in an earlier drisapersen study, or who experience similar reaction(s) during this study, there is the potential to be dosed intravenously.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	Drisapersen	Subjects will receive 6 mg/kg of drisapersen by subcutaneous injection once weekly. If subjects have experienced an intolerable injection site reaction(s), in consultation with the investigator, the subject may be allowed intermittent injections (8 weeks on/4 weeks off) or weekly intravenous infusions of 3 or 6 mg/kg

Timeline

• First posted: 2015-12-22
• Last updated: 2018-01-24

Locations

39 sites across 20 countries: United States, Argentina, Australia, Belgium, Bulgaria, Czechia, France, Germany, Israel, Italy, Japan, Netherlands, Norway, Poland, Russia, South Korea, Spain, Taiwan, Turkey (Türkiye), United Kingdom