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

NCT ID: NCT01963897

Last Updated: 2017-09-25

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

73 participants

Study Classification

OBSERVATIONAL

Study Start Date

2013-08-31

Study Completion Date

2017-06-30

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Duchenne Muscular Dystrophy

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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Inclusion Criteria

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

Exclusion Criteria

\-
Maximum Eligible Age

18 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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The Research Council of Norway

OTHER

Sponsor Role collaborator

University Hospital of North Norway

OTHER

Sponsor Role collaborator

Oslo University Hospital

OTHER

Sponsor Role lead

Responsible Party

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Magnhild Rasmussen

Dr.med.

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Magnhild Rasmussen, Dr.med.

Role: PRINCIPAL_INVESTIGATOR

Oslo University Hospital

Locations

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Oslo University Hospital

Oslo, , Norway

Site Status

Countries

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Norway

Other Identifiers

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2013/513

Identifier Type: -

Identifier Source: org_study_id