A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)

PHASE1/PHASE2TerminatedINTERVENTIONAL
Enrollment

9

Participants

Timeline

Start Date

June 30, 2013

Primary Completion Date

August 3, 2016

Study Completion Date

August 3, 2016

Conditions
Duchenne Muscular Dystrophy
Interventions
DRUG

Regimen Selection Phase Group 2

"All doses of BMN053 will be administered as IV infusions. The proposed doses are as follows:~• 3 mg/kg"

DRUG

Regimen Selection Phase Group 3

"All doses of BMN053 will be administered as IV infusions. The proposed doses are as follows:~• 4-6 mg/kg"

DRUG

Treatment Phase Group 4

All doses of PRO053 will be administered as IV infusions. The proposed doses will be decided upon completion of the Regimen Selection Phase of Groups 2 and 3

DRUG

Regimen Selection Phase Group 1 (COMPLETED)

All doses of BMN053 have been administered as subcutaneous injections.

DRUG

Dosing Extension

All doses of PRO053 will be administered as IV infusions. The proposed doses will be decided upon completion of the Regimen Selection Phase of Groups 2 and 3 and the Treatment Phase Group 4.

Trial Locations (6)

3000

UZ Leuven, Campus Gasthuisberg, Leuven

75651

Institut de Myologie, Paris

00168

Policlinico Universitario Agostino Gemelli, Rome

2333ZA

Leids Universitair Medisch Centrum, Leiden

WC1N 3JH

Great Ormond Street Hospital for Children, London

NE1 3BZ

Institute of Genetic Medicine International Centre for Life, Newcastle

Sponsors
All Listed Sponsors
lead

BioMarin Pharmaceutical

INDUSTRY