Spinal Abnormalities in Neurofibromatosis Type 1 (NF1)

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

112 participants

Study Classification

OBSERVATIONAL

Study Start Date

2006-12-31

Study Completion Date

2012-12-31

Brief Summary

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

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Detailed Description

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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.

Conditions

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Neurofibromatosis Type 1

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Neurofibromatosis Type 1

Children with Neurofibromatosis Type 1

No interventions assigned to this group

Eligibility Criteria

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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.)

Minimum Eligible Age

6 Years

Maximum Eligible Age

9 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No