Clinical Trial: Multi-Center Project: Spinal Abnormalities in Neurofibromatosis Type1 (NF1) Patients

Study Status: Active, not recruiting
Recruit Status: Unknown status
Study Type: Observational

Official Title: Multi-Center Project: Spinal Abnormalities in Neurofibromatosis Type1 (NF1) Patients

Brief Summary:

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.

Detailed Summary:
Sponsor: Shriners Hospitals for Children

Current Primary Outcome:

Original Primary Outcome:

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Shriners Hospitals for Children

Dates:
Date Received: April 24, 2008
Date Started: March 2006
Date Completion: September 2009
Last Updated: April 24, 2008
Last Verified: April 2008