A Prospective Natural History Study of the Progression of Physical Impairment, Activity Limitation and Quality of Life in Duchenne Muscular Dystrophy (DMD)

A Prospective Natural History Study of the Progression of Physical Impairment, Activity Limitation and Quality of Life in Duchenne Muscular Dystrophy (DMD) - DMD Natural History

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

NL-OMON

Registry ID

NL-OMON39538

Enrollment

Registered

2013-01-25

Start date

2012-08-31

Completion date

Unknown

Last updated

2024-04-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne Muscular Dystrophy (DMD) muscular dystrophy

Interventions

None listed

Eligibility

Age

2 Years to 17 Years

Inclusion criteria

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

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

Design outcomes

Primary

Measure	Time frame
This is an exploratory observational study with no formal statistical hypotheses. All data will be summarised at each time point. In general, categorical data will be presented using counts and percentages, whilst continual variable will be presented using the mean, standard deviation, median, minimum, maximum and number of patients. Interim analyses will be conducted at yearly intervals during this study.	—

Measure

Time frame

This is an exploratory observational study with no formal statistical hypotheses. All data will be summarised at each time point. In general, categorical data will be presented using counts and percentages, whilst continual variable will be presented using the mean, standard deviation, median, minimum, maximum and number of patients. Interim analyses will be conducted at yearly intervals during this study.

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Countries

Netherlands

Outcome results