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

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

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

Brief 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 Summary:

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.

Sponsor: BioMarin Pharmaceutical

Current Primary Outcome:

• Efficacy of PRO044 (composite of several measures) [ Time Frame: After 48 weeks of treatment ]

  Efficacy parameters:

  Muscle Function

  • 6 Minute Walk Distance (6MWD)
  • North Star Ambulatory Assessment
  • Timed tests (10-meter walk/run, rising from floor, stair climb)
  • DMD Functional Outcomes Questionnaire (DMD-FOS) -for ambulant subjects only
  • Egen Klassification - for non-ambulant subjects.

  Muscle strength

  • Pulmonary Function (Spirometry)
  • Handheld myometry.

  Exploratory:

  • Performance Upper Limb (PUL).
  • Patient Reported Outcome measure (PROM).
• Safety and tolerability of PRO044 (treatement emergent adverse events) [ Time Frame: After 48 weeks of treatment ]
  Number of subjects with 1 or more treatement emergent adverse events following SC or IV PRO044 dosing

Original Primary Outcome: Same as current

Current Secondary Outcome: Assess the pharmacokinetics of PRO044 (composite of several measures) [ Time Frame: After 48 weeks of treatment ]

Original Secondary Outcome: Same as current

Information By: BioMarin Pharmaceutical

Dates:
Date Received: December 22, 2014
Date Started: December 2014
Date Completion: December 2017
Last Updated: October 27, 2016
Last Verified: April 2015