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

Status Terminated

Clinical Trial 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.

Clinical Trial 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. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT02329769
Study type	Interventional
Source	BioMarin Pharmaceutical
Contact
Status	Terminated
Phase	Phase 2
Start date	December 2014
Completion date	August 31, 2016

View Details

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