Overview

This trial has been terminated.

Condition duchenne muscular dystrophy
Treatment observational study
Sponsor BioMarin Pharmaceutical
Start date September 2012
End date December 2016
Trial size 269 participants
Trial identifier NCT01753804, PRO-DMD-01

Summary

To characterize the natural history and progression of Duchenne Muscular Dystrophy (DMD) 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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Observational model case-only
Time perspective prospective
Arm
All participants will follow the same protocol, including muscle strength and function testing, and blood and urine collection, for a maximum of 7 visits over 3 years.
observational study
There is no medication or device tested in this study. This is an obversational study on the progression of the disease.

Primary Outcomes

Measure
6 minute walk distance
time frame: Change from visit 1 walking distance

Eligibility Criteria

Male participants from 3 years up to 18 years old.

Inclusion Criteria: - Diagnosis of DMD resulting from a mutation in the DMD gene confirmed by a state of the art DNA diagnostic technique covering all DMD gene exons. - Age 3 - 18 years - Willing and able to comply with protocol requirements - Life expectancy of at least 3 years - Able to give informed assent and/or consent in writing signed by the subject and/or parent(s)/legal guardian (according to local regulations) Exclusion Criteria: - Current participation in a clinical study with an Investigational Medicinal Product (IMP) - Participation within the previous 1 month in a clinical study with an IMP

Additional Information

Official title A Prospective Natural History Study of Progression of Physical Impairment, Activity Limitation and Quality of Life in Duchenne Muscular Dystrophy.
Principal investigator Nathalie Goemans, MD
Description This is a prospective study. All DMD patients that fulfil the inclusion/exclusion criteria are eligible although the study is weighted towards ambulant subjects aged 3 years or older. 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). 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 Subjects will be asked to perform muscle testing assessment with a clinical evaluator, such as walking for 6 minutes, climb stairs, breathe in a tube, see how they can move their arms and legs. They will be asked questions about how they feel overall and perform daily activities. These measurements will be assessed every 6 months. Urine and blood samples will be collected once a year to measure biomarkers that will allow to have a better overview of DMD.
Trial information was received from ClinicalTrials.gov and was last updated in October 2016.
Information provided to ClinicalTrials.gov by BioMarin Pharmaceutical.