NCT01037309

Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)

A Phase I/IIa, Open Label, Escalating Dose, Pilot Study to Assess the Effect, Safety, Tolerability and Pharmacokinetics of Multiple Subcutaneous and Intravenous Doses of PRO044 in Patients With Duchenne Muscular Dystrophy

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 18 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: Male
• Age: 5 Years – 16 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 DMD patients with a mutation around location 44 in the DNA for the dystrophin protein.

Detailed description

To assess the effect of PRO044 at different dose levels in subjects with Duchenne muscular dystrophy To assess the safety and tolerability of PRO044 at different dose levels in subjects with Duchenne muscular dystrophy To determine the pharmacokinetics of PRO044 at different dose levels after subcutaneous and intravenous administration in subjects with Duchenne muscular dystrophy.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	PRO044 SC	Subcutaneous injection, once a week, for five weeks
DRUG	PRO044 IV	Intravenous injection, once a week, for five weeks

Timeline

• Start date: 2009-12-01
• Primary completion: 2013-05-01
• Completion: 2013-10-01
• First posted: 2009-12-23
• Last updated: 2018-10-16
• Results posted: 2015-04-09

Locations

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

Regulatory

• FDA-regulated drug study