NCT02958202

Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)

A Multi Center, Multi National, Open Label, Extension Study to Evaluate the Long-term Efficacy and Safety of BMN 044 (PRO044) in Subjects With Duchenne Muscular Dystrophy

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 7 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: Male
• Age: 5 Years
• Healthy volunteers: Not accepted

Summary

The aim of this study is to provide continuing access to BMN 044 treatment for subjects previously treated with BMN 044. The information gained from this study is expected to further characterize the efficacy and safety of BMN 044 over a longer treatment period.

Detailed description

This is a phase 2 multi center, multi national, open label, long term extension study. Up to approximately 50 male subjects with Duchenne Muscular Dystrophy (DMD) who have previously been treated with BMN 044 will be enrolled. Subjects will receive either IV infusions or SC injections at pre-defined doses. Safety and tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and efficacy assessments will be conducted at regular intervals throughout the study. Subjects will be permitted to continue in this study until the subject meets any of the defined withdrawal criteria, BioMarin decides to halt the clinical development of BMN 044, or BMN 044 receives marketing authorization.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	BMN 044 IV 6 mg/kg
DRUG	BMN 044 IV 9 mg/kg
DRUG	BMN 044 SC 6 mg/kg

Timeline

• Start date: 2016-04-01
• Primary completion: 2016-09-01
• Completion: 2016-09-01
• First posted: 2016-11-08
• Last updated: 2018-01-26

Locations

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