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.

This study is currently recruiting participants. (see Contacts and Locations)
Verified October 2013 by Oslo University Hospital
Sponsor:
Collaborators:
The Research Council of Norway
University Hospital of North Norway
Information provided by (Responsible Party):
Magnhild Rasmussen, Oslo University Hospital
ClinicalTrials.gov Identifier:
NCT01963897
First received: October 12, 2013
Last updated: October 21, 2013
Last verified: October 2013
  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.


Condition
Duchenne Muscular Dystrophy

Study Type: Observational [Patient Registry]
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 3 Years
Official 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.

Resource links provided by NLM:


Further study details as provided by Oslo University Hospital:

Primary Outcome Measures:
  • Genotype [ Time Frame: 1 day (At first visit) ] [ Designated as safety issue: No ]
    Specific description of mutation


Biospecimen Retention:   Samples With DNA

whole blood, serum


Estimated Enrollment: 70
Study Start Date: August 2013
Estimated Study Completion Date: June 2016
Estimated Primary Completion Date: June 2016 (Final data collection date for primary outcome measure)
  Eligibility

Ages Eligible for Study:   up to 18 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population

All patients with Duchenne muscular dystrophy under 18 years in Norway

Criteria

Inclusion Criteria:

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

Exclusion Criteria:

-

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01963897

Contacts
Contact: Ellen J Annexstad, Cand.med. 02770 ellann@ous-hf.no
Contact: Magnhild Rasmussen, Dr.med. 02770 marasmus@ous-hf.no

Locations
Norway
Oslo University Hospital Recruiting
Oslo, Norway, 0424
Contact: Ellen J Annexstad, Cand.med.    02770    ellann@ous-hf.no   
Contact: Magnhild Rasmussen, Dr.med.    02770    marasmus@ous-hf.no   
Sub-Investigator: Ellen J Annexstad, Cand.med.         
Principal Investigator: Magnhild Rasmussen, Dr.med.         
Sponsors and Collaborators
Oslo University Hospital
The Research Council of Norway
University Hospital of North Norway
Investigators
Principal Investigator: Magnhild Rasmussen, Dr.med. Oslo University Hospital
  More Information

No publications provided

Responsible Party: Magnhild Rasmussen, Dr.med., Oslo University Hospital
ClinicalTrials.gov Identifier: NCT01963897     History of Changes
Other Study ID Numbers: 2013/513
Study First Received: October 12, 2013
Last Updated: October 21, 2013
Health Authority: Norway:National Committee for Medical and Health Research Ethics

Keywords provided by Oslo University Hospital:
Genotype/phenotype correlations
Growth
Bone health
Puberty
Quality of life

Additional relevant MeSH terms:
Muscular Dystrophy, Duchenne
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on August 19, 2014