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completed

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

key information

study id #: NCT01963897

condition: Duchenne Muscular Dystrophy

status: completed

purpose:

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.

mechanism of action: No pharmaceutical intervention

results: https://clinicaltrials.gov/ct2/show/results/NCT01963897

last updated: November 22, 2018

study details

start date: August 2013

estimated completion: June 2017

size / enrollment: 73

study description:
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.

primary outcomes:

  • Genotype [ Time Frame: 1 day (At first visit) ]
    Specific description of mutation

inclusion criteria:

• Eligible Sexes:

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

study contacts

sponsor: Oslo University Hospital

investigators: Magnhild Rasmussen, Dr.med.

trial center locations: Norway