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Extension Study of Drisapersen in DMD Subjects

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ClinicalTrials.gov Identifier: NCT02636686
Expanded Access Status : No longer available (Due to a company decision to stop the development of exon-skipping Duchenne muscular dystrophy therapy.)
First Posted : December 22, 2015
Last Update Posted : January 24, 2018
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Tracking Information
First Submitted Date December 9, 2015
First Posted Date December 22, 2015
Last Update Posted Date January 24, 2018
 
Descriptive Information
Brief Title Extension Study of Drisapersen in DMD Subjects
Brief 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.

Study Type Expanded Access
Expanded Access Type Treatment IND/Protocol
Intervention 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
Other Name: PRO051
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Expanded Access Status No longer available
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Argentina,   Australia,   Belgium,   Bulgaria,   Czechia,   France,   Germany,   Israel,   Italy,   Japan,   Korea, Republic of,   Netherlands,   Norway,   Poland,   Russian Federation,   Spain,   Taiwan,   Turkey,   United Kingdom,   United States
Removed Location Countries Czech Republic
 
Administrative Information
NCT Number NCT02636686
Responsible Party BioMarin Pharmaceutical
Study Sponsor BioMarin Pharmaceutical
Collaborators Not Provided
Investigators
Study Director: Derry Ridgway, MD BioMarin Pharmaceutical
PRS Account BioMarin Pharmaceutical
Verification Date January 2018