Spinal Abnormalities in Neurofibromatosis Type 1 (NF1)

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Brief Title

Spinal Abnormalities in Neurofibromatosis Type 1 (NF1)

Official Title

Spinal Abnormalities in Neurofibromatosis Type 1 (NF1)

Brief Summary

      The purpose of this study is to determine the incidence and clinical history of
      neurofibromatosis type 1-related spinal abnormalities.
    

Detailed Description

      Neurofibromatosis type 1 (NF1) is a common genetic disorder that is associated with spinal
      abnormalities which are varied and may include scoliosis, neurofibromas, meningoceles, and
      vertebral defects. Skeletal abnormalities occur in more than one third of individuals with
      the disorder. These abnormalities are unpredictable and the pathogenesis, natural history,
      and clinical outcome remain relatively unclear.

      The primary objective of this study is to determine the incidence and clinical history of
      NF1-related spinal abnormalities in children with NF1, over a 3-year period.

      In the study, researchers will enroll children between ages 6 and 9 years who have been
      diagnosed with NF1 to look at changes in the spine. Participants in the study will be
      followed yearly for a total of 4 evaluations. Evaluations may include bone scans, spinal
      x-rays, magnetic resonance imaging (MRI), computed tomography (CT) scans, and urine samples.

      Information gained from this study may lead to a better understanding of the causes of bone
      disease in NF1, and improved treatment.
    


Study Type

Observational


Primary Outcome

Scoliosis and it's progression

Secondary Outcome

 Differences in other bone health variables as measured by thoracic MRIs, Dexa (xray measuring bone density), pQCT (a cross sectional picture of the tibia), urine analysis, and scoli series (xrays to look for scoliosis).

Condition

Neurofibromatosis Type 1


Study Arms / Comparison Groups

 Neurofibromatosis Type 1
Description:  Children with 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

112

Start Date

December 2006

Completion Date

December 2012

Primary Completion Date

December 2012

Eligibility Criteria

        Inclusion Criteria:

          -  Diagnosis of NF1

          -  Age greater than 6 years at time of enrollment

          -  Age less than 9 year and 1 day at time of enrollment

          -  Tanner Stage not greater than 1

        Exclusion Criteria:

          -  Prior surgical repair of spine

          -  Short-segment (4-6 vertebrae) curve with a Cobb angle of 45°or more

          -  Hormone replacement therapy

          -  Chronic steroid use

          -  Tibial pseudarthrosis

          -  Other chronic medical problems known to influence bone health (i.e. diabetes mellitus,
             cerebral palsy, etc.)
      

Gender

All

Ages

6 Years - 9 Years

Accepts Healthy Volunteers

No

Contacts

David Viskochil, MD, PhD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00844129

Organization ID

15464

Secondary IDs

R01NS050509

Responsible Party

Principal Investigator

Study Sponsor

University of Utah

Collaborators

 National Institute of Neurological Disorders and Stroke (NINDS)

Study Sponsor

David Viskochil, MD, PhD, Principal Investigator, Division of Medical Genetics, Department of Pediatrics, University of Utah


Verification Date

April 2018