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

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.
Verified April 2008 by Shriners Hospitals for Children.
Recruitment status was:  Recruiting
Sponsor:
ClinicalTrials.gov Identifier:
NCT00667836
First Posted: April 28, 2008
Last Update Posted: April 28, 2008
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by:
Shriners Hospitals for Children
  Purpose

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
Neurofibromatosis Type 1

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Multi-Center Project: Spinal Abnormalities in Neurofibromatosis Type1 (NF1) Patients

Resource links provided by NLM:


Further study details as provided by Shriners Hospitals for Children:

Estimated Enrollment: 300
Study Start Date: March 2006
Estimated Study Completion Date: September 2009
Estimated Primary Completion Date: September 2009 (Final data collection date for primary outcome measure)
  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   3 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
All NF1 patients
Criteria

Inclusion Criteria:

  • Meet NIH diagnostic criteria for NF1
  • Radiographic documentation of scoliosis will be necessary for inclusion as a "scolosis case"
  • Age between 3 and 18 years

Exclusion Criteria:

  • Do not have NF1
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00667836


Contacts
Contact: Susan J Geyer, CCRP 801-536-3561 sgeyer@shrinenet.org
Contact: Jeanne Siebert, RN, CCRp 801-536-3601 jsiebert@shrinenet.org

Locations
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.         
Sponsors and Collaborators
Shriners Hospitals for Children
Investigators
Principal Investigator: Jacques D'Astous, M.D. Shriners Hospitals for Children
  More Information

Responsible Party: Jacques D'Astous, M.D. , PI, Shriners Hospitals for Children, Salt Lake City
ClinicalTrials.gov Identifier: NCT00667836     History of Changes
Other Study ID Numbers: IRB_00023261
First Submitted: April 24, 2008
First Posted: April 28, 2008
Last Update Posted: April 28, 2008
Last Verified: April 2008

Keywords provided by Shriners Hospitals for Children:
Neurofibromatosis Type 1
NF1
Spinal Anomalies

Additional relevant MeSH terms:
Neurofibromatoses
Neurofibroma
Neurofibromatosis 1
Nerve Sheath Neoplasms
Neoplasms, Nerve Tissue
Neoplasms by Histologic Type
Neoplasms
Neoplastic Syndromes, Hereditary
Neurocutaneous Syndromes
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Peripheral Nervous System Neoplasms
Nervous System Neoplasms
Peripheral Nervous System Diseases
Neuromuscular Diseases


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