Clinical Trials Directory

Trials / Withdrawn

WithdrawnNCT01890798

Drisapersen Duchenne Muscular Dystrophy (DMD) Treatment Protocol

A Continued Access Protocol for Eligible US Subjects With Duchenne Muscular Dystrophy Who Previously Participated in an Approved Drisapersen Study

Status
Withdrawn
Phase
Phase 3
Study type
Interventional
Enrollment
0 (actual)
Sponsor
GlaxoSmithKline · Industry
Sex
Male
Age
5 Years
Healthy volunteers
Not accepted

Summary

This is a single arm, open-label continued access protocol of drisapersen for the treatment of male subjects with Duchenne muscular dystrophy (DMD) having dystrophin mutations correctable by drisapersen-induced DMD Exon 51 skipping. The purpose of this continued access protocol is to offer pre-approval access to drisapersen for the treatment of subjects with DMD who previously participated in eligible drisapersen studies. The protocol will collect safety data required to assure subject safety and periodic efficacy data on muscle function.

Conditions

Interventions

TypeNameDescription
DRUGDrisapersenDrisapersen will be supplied as 3 millilitre (mL) vials containing 1mL sterile solution for subcutaneous injection. The strength of drisapersen solution will be 200 mg/mL.

Timeline

Start date
2014-01-01
Primary completion
2014-01-01
Completion
2015-07-01
First posted
2013-07-02
Last updated
2014-03-24

Source: ClinicalTrials.gov record NCT01890798. Inclusion in this directory is not an endorsement.