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Sponsored by: |
Shriners Hospitals for Children |
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Information provided by: | Shriners Hospitals for Children |
ClinicalTrials.gov Identifier: | NCT00667836 |
We propose to establish a multi-center study to investigate the outcome of scoliosis and spinal abnormalities in patients with NF1.
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.
Condition |
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Neurofibromatosis Type 1 |
Study Type: | Observational |
Study Design: | Cohort, Prospective |
Official Title: | Multi-Center Project: Spinal Abnormalities in Neurofibromatosis Type1 (NF1) Patients |
Ages Eligible for Study: | 3 Years to 18 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Sampling Method: | Probability Sample |
All NF1 patients
Inclusion Criteria:
Exclusion Criteria:
Contact: Susan J Geyer, CCRP | 801-536-3561 | sgeyer@shrinenet.org |
Contact: Jeanne Siebert, RN, CCRp | 801-536-3601 | jsiebert@shrinenet.org |
United States, Utah | |
Shriners Hospitals for Children, Salt Lake City | Recruiting |
Salt Lake City, Utah, United States, 84103 | |
Contact: Susan J Geyer, CCRP 801-536-3561 sgeyer@shrinenet.org | |
Contact: Jeanne Siebert, RN, CCRP 801-536-3601 jsiebert @shrinenet.org | |
Principal Investigator: Jacques D'Astous, M.D. |
Principal Investigator: | Jacques D'Astous, M.D. | Shriners Hospitals for Children, Salt Lake City |
Responsible Party: | Shriners Hospitals for Children, Salt Lake City ( Jacques D'Astous, M.D. , PI ) |
Study ID Numbers: | IRB_00023261 |
Study First Received: | April 24, 2008 |
Last Updated: | April 24, 2008 |
ClinicalTrials.gov Identifier: | NCT00667836 |
Health Authority: | United States: Institutional Review Board |
Neurofibromatosis Type 1 NF1 Spinal Anomalies |
Neurodegenerative Diseases Neurofibromatosis 1 Neurofibromatosis type 1 Neoplastic Syndromes, Hereditary Heredodegenerative Disorders, Nervous System Neurofibroma Genetic Diseases, Inborn |
Neuromuscular Diseases Peripheral Nervous System Diseases Neurofibromatoses Congenital Abnormalities Nerve Sheath Neoplasms Neurocutaneous Syndromes |
Neoplasms Neoplasms by Histologic Type Nervous System Diseases Neoplasms, Nerve Tissue |