Multi-center Project: Spinal Abnormalities in Neurofibromatosis Type1 (NF1) Patients

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Brief Title

Multi-center Project: Spinal Abnormalities in Neurofibromatosis Type1 (NF1) Patients

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 Description

      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.
    


Study Type

Observational




Condition

Neurofibromatosis Type 1



Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

300

Start Date

March 2006

Completion Date

September 2009

Primary Completion Date

September 2009

Eligibility Criteria

        Inclusion Criteria:

          -  Meet NIH diagnostic criteria for NF1

          -  Radiographic documentation of scoliosis will be necessary for inclusion as a
             "scoliosis case"

          -  Age between 3 and 18 years

        Exclusion Criteria:

          -  Do not have NF1
      

Gender

All

Ages

3 Years - 18 Years

Accepts Healthy Volunteers

No

Contacts

Jacques D'Astous, M.D., , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00667836

Organization ID

IRB_00023261


Responsible Party

Principal Investigator

Study Sponsor

Shriners Hospitals for Children


Study Sponsor

Jacques D'Astous, M.D., Principal Investigator, Shriners Hospitals for Children


Verification Date

February 2019