Date: 2013-01-00
Type of information: Initiation of preclinical development
phase: 2b
Announcement: initiation of the study
Company: Prosensa (The Netherlands)
Product: PRO045
Action
mechanism: The antisense oligonucleotide PRO045 induces exon 45 skipping in the dystrophin gene and is intended for approximately 8% of all Duchenne Muscular Dystrophy (DMD) patients, including patients with deletions of exon 44, exon 46, exons 46-47, exons 46-48, exons 46-49, and exons 46-51. The underlying chemistry and mechanism of PRO045 are similar to drisapersen.
Disease: Duchenne muscular dystrophy
Therapeutic area: Genetic diseases - Neuromuscular diseases - Rare diseases
Country: Belgium, France, Italy, The Netherlands, United Kingdom
Trial
details: This Phase IIb, study is an open-label study to assess the efficacy, safety, pharmacodynamics and pharmacokinetics of multiple subcutaneous doses of PRO045 in subjects With Duchenne muscular dystrophy. The purpose of the study is to see whether PRO045 is safe and effective to use as medication for Duchenne muscular dystrophy patients with a mutation around location 45 in the DNA for the dystrophin protein. (NCT01826474)The study consists of two phases; a dose escalation phase (with subsequent dose-titration) and a 48-week treatment phase.
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