A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.

Duchenne Muscular Dystrophy Clinical Trial

Official title:

A Prospective Natural History Study of Progression of Physical Impairment, Activity Limitation and Quality of Life in Duchenne Muscular Dystrophy.

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01753804
• Other study ID #: PRO-DMD-01
• Status: Terminated
• Phase: N/A
• First received: December 13, 2012
• Last updated: December 6, 2017
• Start date: September 1, 2012
• Est. completion date: October 1, 2016

Study information

• Verified date: December 2017
• Source: BioMarin Pharmaceutical
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 269
• Est. completion date: October 1, 2016
• Est. primary completion date: October 1, 2016
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 3 Years to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Other:
• Observational study
There is no medication or device tested in this study. This is an obversational study on the progression of the disease.

Locations

Country	Name	City	State
Argentina	Hospital de Pediatria Prof Dr Juan P Garrahan	Buenos Aires
Belgium	Universitair Ziekenhuis	Gent
Belgium	Universitair Ziekenhuis Leuven	Leuven
Brazil	Hospital das Clinicas da Faculdade de Medicina da USP	Sao Paulo
France	CHU Hopital des enfants	Toulouse
Germany	Universitaetsklinikum Essen	Essen
Germany	Universitaetsklinikum Freiburg	Freiburg
Italy	Azienda Ospedaliera Universitaria Policlinico G. Martino	Messina
Italy	Policlinico Univsersitario Agostino Gemelli	Rome
Netherlands	Leids Universitair Medisch Centrum	Leiden
Netherlands	UMC St. Radboud	Nijmegen
Sweden	Drottning Silvias Barn- ochungdomssjukhus	Goteborg
Turkey	Hacettepe University Medical Faculty	Ankara
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	UC Davis Health System	Sacramento	California

Sponsors (1)

Lead Sponsor	Collaborator
BioMarin Pharmaceutical

Countries where clinical trial is conducted

United States,  Argentina,  Belgium,  Brazil,  France,  Germany,  Italy,  Netherlands,  Sweden,  Turkey, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	6 minute walk distance	Participants are asked to walk at their own preferred speed on a fixed distance for 6 minutes. Subjects are warned of the time and that they may stop earlier if they feel unable to continue. Total distance walked within 6 minutes (or until stopping) is recorded.	Change from visit 1 walking distance

External Links

•   BioMarin website