NCT01957059

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

A Phase I/II, Open-label, Dose Escalating With 48 Week Treatment Study to Assess the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of BMN053 (Previously Known as PRO053) in Subjects With Duchenne Muscular Dystrophy.

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: Male
• Age: 5 Years – 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of the study is to see whether BMN053 is safe and effective to use as medication for Duchenne muscular dystrophy (DMD) patients with a mutation around location 53 in the DNA for the dystrophin protein.

Detailed description

A Phase I/II, open-label, dose escalating with 48-week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of BMN 053 (previously known as PRO053) in subjects with Duchenne muscular dystrophy

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2013-06-01
• Primary completion: 2016-08-03
• Completion: 2016-08-03
• First posted: 2013-10-08
• Last updated: 2017-12-08

Locations

6 sites across 5 countries: Belgium, France, Italy, Netherlands, United Kingdom