NCT01910649

A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess Effect, Safety, Tolerability and PK of Multiple SC Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for IV Dosing as an Alternative Route of Administration

A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess the Effect, Safety, Tolerability and Pharmacokinetics of Multiple Subcutaneous Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for Intravenous Dosing as an Alternative Route of Administration

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: Male
• Age: 5 Years – 16 Years
• Healthy volunteers: Not accepted

Summary

The purpose of the extension phase of this study is to determine whether Drisapersen is effective in the treatment of boys with Duchenne muscular dystrophy resulting from a mutation thought to be corrected by exon 51 skipping.

Conditions

• Muscular Dystrophies

Interventions

Type	Name	Description
DRUG	Drisapersen	Subcutaneous and Intravenous

Timeline

• Start date: 2008-03-01
• Primary completion: 2016-09-01
• Completion: 2016-09-01
• First posted: 2013-07-29
• Last updated: 2016-11-07