Multi-center Project: Spinal Abnormalities in Neurofibromatosis Type1 (NF1) Patients
NCT ID: NCT00667836
Last Updated: 2019-02-25
Study Results
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Basic Information
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COMPLETED
300 participants
OBSERVATIONAL
2006-03-31
2009-09-30
Brief Summary
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The three specific aims of this study are:
Specific Aim 1 - To assess health status and health-related quality of life (HRQL) in children and adolescents with NF1 and scoliosis. We hypothesize that children and adolescents with NF1 and scoliosis will experience an additional burden of morbidity due to scoliosis and a downward trajectory of health status and HRQL over time.
Specific Aim 2 - To assess the natural history and short-term response to therapy in a cohort of children with NF1 and scoliosis prospectively diagnosed during the course of the four-year study period. We hypothesize that some NF1 patients with idiopathic scoliosis will modulate to the dystrophic form. We also hypothesize that NF1 patients with earlier presentation are more likely to have or modulate to the dystrophic form.
Specific Aim 3 - To assess biochemical markers of bone metabolism in NF1 individuals. We hypothesize that NF1 individuals will have statistically significant differences in biochemical markers of bone metabolism compared to controls. We also hypothesize that NF1 individuals with scoliosis will have differences in biochemical markers of bone metabolism compared to NF1 individuals without scoliosis.
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Detailed Description
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Specific Aim 1 - To assess health status and health-related quality of life (HRQL) in children and adolescents with NF1 and scoliosis. We hypothesize that children and adolescents with NF1 and scoliosis will experience an additional burden of morbidity due to scoliosis and a downward trajectory of health status and HRQL over time.
Specific Aim 2 - To assess the natural history and short-term response to therapy in a cohort of children with NF1 and scoliosis prospectively diagnosed during the course of the four-year study period. We hypothesize that some NF1 patients with idiopathic scoliosis will modulate to the dystrophic form. We also hypothesize that NF1 patients with earlier presentation are more likely to have or modulate to the dystrophic form.
Specific Aim 3 - To assess biochemical markers of bone metabolism in NF1 individuals. We hypothesize that NF1 individuals will have statistically significant differences in biochemical markers of bone metabolism compared to controls. We also hypothesize that NF1 individuals with scoliosis will have differences in biochemical markers of bone metabolism compared to NF1 individuals without scoliosis.
Conditions
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Study Design
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COHORT
PROSPECTIVE
Eligibility Criteria
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Inclusion Criteria
* Radiographic documentation of scoliosis will be necessary for inclusion as a "scoliosis case"
* Age between 3 and 18 years
Exclusion Criteria
3 Years
18 Years
ALL
No
Sponsors
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Shriners Hospitals for Children
OTHER
Responsible Party
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Jacques D'Astous
Principal Investigator
Principal Investigators
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Jacques D'Astous, M.D.
Role: PRINCIPAL_INVESTIGATOR
Shriners Hospitals for Children
Locations
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Shriners Hospitals for Children, Salt Lake City
Salt Lake City, Utah, United States
Countries
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Other Identifiers
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IRB_00023261
Identifier Type: -
Identifier Source: org_study_id
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