NCT01826474

Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy

A Phase IIb, Open-label Study to Assess the Efficacy, Safety, Pharmacodynamics and Pharmacokinetics of Multiple Subcutaneous Doses of PRO045 in Subjects With Duchenne Muscular Dystrophy

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 15 (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 PRO045 is safe and effective to use as medication for Duchenne Muscular Dystrophy (DMD) patients with a mutation around location 45 in the DNA for the dystrophin protein.

Detailed description

A phase IIb, open-label, multiple-dose study. The study consists of two phases; a dose escalation phase (with subsequent dose-titration) and a 48-week treatment phase.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	PRO045, 0.15 mg/kg/week	Subcutaneous injection
DRUG	PRO045, 1.0 mg/kg/week	Subcutaneous injection
DRUG	PRO045, 3.0 mg/kg/week	Subcutaneous injection
DRUG	PRO045, 6.0 mg/kg/week	Subcutaneous injection
DRUG	PRO045, 9.0 mg/kg/week	Subcutaneous injection
DRUG	PRO045, selected dose	Subcutaneous injection

Timeline

• Start date: 2013-01-01
• Primary completion: 2016-08-31
• Completion: 2016-08-31
• First posted: 2013-04-08
• Last updated: 2017-12-08

Locations

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