Date: 2013-11-07
Type of information: Initiation of preclinical development
phase: observational
Announcement: enrollment of the 100th patient
Company: Prosensa (The Netherlands) GSK (UK)
Product:
Action mechanism:
Disease: Duchenne muscular dystrophy (DMD)
Therapeutic area: Genetic diseases - Neuromuscular diseases - Rare diseases
Country: USA, Argentina, Belgium, Brazil, France, Germany, Italy, The Netherlands, Sweden, Turkey
Trial
details: This prospective study aims to characterize the natural history and progression of Duchenne Muscular Dystrophy to help inform the design of future studies, to capture biomarkers of safety and disease progression and to provide comparative data for the development of rare exons for which formal controlled trials are not feasible. Patients in the study are assessed every six months for a period of three years to measure their muscle strength and function, in addition to how the disease affects their quality of life as the condition evolves over time. There will be 7 study visits and subjects will be in the study for a maximum of 3 years. Visits will occur every 6 months (+/- 1 month). Investigators are observing the patients as they perform various physical tests, and assess their quality of life through survey questions. Furthermore, certain biomarkers are measured through blood and urine samples to investigate a possible correlation to disease progression. The study is being conducted in 16 hospitals across 10 countries with 14 sites already open in the U.S. and Europe. (NCT01753804)Up to 250 DMD subjects planned in the following categories :
75 % ambulant subjects aged between 3 and 18 years at study entry
25% non-ambulant subjects with a maximum age of 18 years at study entry
Urine and blood samples will be collected once a year to measure biomarkers that will allow to have a better overview of DMD.
Latest
news: * On November 7, 2013, Prosensa, the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, has announced the successful enrollment of the 100th patient into the Natural History Study of Duchenne muscular dystrophy. The goal of this observational study is to characterize DMD at various stages of progression using the same measures used in ongoing clinical studies, such as the “six minute walk test.” No medication is being tested in this study.
Prosensa and GSK, who are both funding the study, expect to enroll up to 250 DMD patients into the study with any type of mutation in the DMD gene between the ages of three and 18.
