A Phase I/II, Open-label, Dose Escalating With 48 Week Treatment Study to Assess the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of BMN053 (Previously Known as PRO053) in Subjects With Duchenne Muscular Dystrophy.

Trial Profile

A Phase I/II, Open-label, Dose Escalating With 48 Week Treatment Study to Assess the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of BMN053 (Previously Known as PRO053) in Subjects With Duchenne Muscular Dystrophy.

Discontinued
Phase of Trial: Phase I/II

Latest Information Update: 03 Nov 2016

At a glance

  • Drugs PRO 053 (Primary) ; PRO 053 (Primary)
  • Indications Duchenne muscular dystrophy
  • Focus Therapeutic Use
  • Sponsors BioMarin Nederland; BioMarin Pharmaceutical
  • Most Recent Events

    • 10 Jun 2017 Biomarkers information updated
    • 31 May 2016 According to a BioMarin media release, the company intends to discontinue clinical and regulatory development of Kyndrisa as well as the three other first-generation follow-on products, BMN 044, BMN 045 and BMN 053, currently in Phase 2 studies for distinct forms of Duchenne muscular dystrophy.
    • 31 May 2016 Status changed from active, no longer recruiting to discontinued, according to a BioMarin media release.
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