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



Status:Terminated
Conditions:Neurology, Orthopedic
Therapuetic Areas:Neurology, Orthopedics / Podiatry
Healthy:No
Age Range:3 - 18
Updated:12/10/2017
Start Date:September 1, 2012
End Date:October 1, 2016

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

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.

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.

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
We found this trial at
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700 Childrens Drive
Columbus, Ohio 43205
(616) 722-2000
Nationwide Children's Hospital At Nationwide Children’s, we are creating the future of pediatric health care....
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3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229
 1-513-636-4200 
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1881 Combate de los Pozos
Buenos Aires, C1245
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Sacramento, California 95817
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