NCT02329769

Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)

A Phase II, Open Label, Extension Study to Assess the Effect of PRO044 in Patients With Duchenne Muscular Dystrophy

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 15 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: Male
• Age: 9 Years – 20 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to see whether PRO044 is safe and effective to use as medication for Duchenne Muscular Dystrophy (DMD) patients with a mutation around location 44 in the DNA for the dystrophin protein.

Detailed description

A Phase II, open-label, extesion study. Following a Screening period of up to one month, subjects previously treated with PRO044, and eligible for enrolment in PRO044-CLIN-02, will be allocated to one of three groups to receive either 6 mg/kg or 9 mg/kg PRO044 weekly by IV infusion or 6 mg/kg weekly by SC injection for 48 weeks. Safety and tolerability, pharmacokinetics (PK), pharmacodynamic (PD) and efficacy assessments will be conducted at regular intervals throughout the study.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	PRO044 SC 6 mg/kg
DRUG	PRO044 IV 6 mg/kg
DRUG	PRO044 IV 9 mg/kg

Timeline

• Start date: 2014-12-01
• Primary completion: 2016-07-01
• Completion: 2016-08-31
• First posted: 2015-01-01
• Last updated: 2017-12-08

Locations

5 sites across 4 countries: Belgium, Italy, Netherlands, Sweden