A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects

PHASE3TerminatedINTERVENTIONAL
Enrollment

53

Participants

Timeline

Start Date

May 1, 2013

Primary Completion Date

June 1, 2016

Study Completion Date

October 1, 2016

Conditions
Muscular Dystrophies
Interventions
DRUG

Drisapersen

Drisapersen will be supplied as 3 mL (milliliter) vials containing 1 mL sterile solution of 200 mg/mL strength. Each subject will receive drisapersen 3 mg/kg administered IV once a week, continuously throughout their duration of participation

DRUG

Drisapersen

Drisapersen will be supplied as 3 mL (milliliter) vials containing 1 mL sterile solution of 220 mg/mL strength. Each subject will receive drisapersen 6 mg/kg administered SC once a week, either continuously or intermittently (for 8 weeks, followed by 4 weeks of no dosing) throughout their duration of participation

DRUG

Drisapersen

Drisapersen will be supplied as 3 mL (milliliter) vials containing 1 mL sterile solution of 220 mg/mL strength. Each subject will receive drisapersen 6 mg/kg administered SC once a week, either continuously or intermittently (for 8 weeks, followed by 4 weeks of no dosing) throughout their duration of participation

Trial Locations (13)

10032

Columbia University Medical Center, New York

12502

Shriner's Hospital for Children Tampa, Tampa

21205

Kennedy Krieger Institute, Baltimore

27710

Duke University Medical Center, Durham

43205

Nationwide Children's Hospital, Columbus

45229

Cincinnati Children's Hospital Medical Center, Cincinnati

52242

University of Iowa, Iowa City

55455

University of Minnesota, Minneapolis

95817

UC Davis Medical Center, Sacramento

97239

Shriners Hospitals For Children, Portland

V6H 3V4

Children's & Women's Health Centre of BC, Vancouver

N6A 4G5

Children's Hospital London Health Sciences Centre, London

H3T 1C5

CHU Ste-Justine, Montreal

Sponsors
All Listed Sponsors
lead

BioMarin Pharmaceutical

INDUSTRY