Duchenne Muscular Dystrophy < 18y in Norway: Genotype/Phenotype, Growth, Puberty, Bone Health and Quality of Life.

The study will give a consent based epidemiological overview of Norwegian patients with Duchenne muscular dystrophy younger than 18 years of age. Genotype of the population will be described. Longitudinal development of growth, bone health, and , when applicable, puberty over a two year period will be studied. Questionnaires regarding quality of life will also be an important part of the study.

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Key information

Conditions

Duchenne Muscular Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Duchenne Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Age range

Up to 18 year

Sex eligibility

Male

Study type

Observational

Primary location

Oslo University Hospital

Oslo, 0424, Norway

About this study

The participants who are not regularly seen at our regional hospital, are recruited via their local neuropediatrician. Patients who do not come to our region hospital will be seen at their local hospital.Each participant will be seen up to three times with one year between each visit. The visits include clinical examination, DXA scan, x-ray for bone age, and blood tests including several parameters related to bone health, growth and puberty. Standardized questionnaires regarding quality of life (patients and parents) will be used, and certain questions regarding the importance of achieving normal height and puberty will be added. Additional anamnestic data will be retrieved from the patients' medical records.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Diagnosis of Duchenne muscular dystrophy
Age under 18 years
Residing in Norway

Exclusion criteria

Treatment and study plan

Primary outcomes

Genotype

Time frame: 1 day (At first visit)

Specific description of mutation

Sponsors and collaborators

Lead sponsor

Oslo University Hospital

Other

Collaborators

The Research Council of Norway
University Hospital of North Norway

Registry information

Official study title

Duchenne Muscular Dystrophy in Norway. Genotype/Phenotype in Patients Younger Than 18 Years, With a Main Focus on Growth, Puberty, Bone Health and Quality of Life.

Important dates

Study start: 2013
Primary completion: 2017
Study completion: 2017
First posted: Oct 16, 2013
Registry last updated: Sep 25, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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