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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified April 2008 by Shriners Hospitals for Children.
Recruitment status was  Recruiting
Sponsor:
Information provided by:
Shriners Hospitals for Children
ClinicalTrials.gov Identifier:
NCT00667836
First received: April 24, 2008
Last updated: NA
Last verified: April 2008
History: No changes posted
  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

Ages Eligible for Study:   3 Years to 18 Years
Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: 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

No publications provided

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
Study First Received: April 24, 2008
Last Updated: April 24, 2008
Health Authority: United States: Institutional Review Board

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

Additional relevant MeSH terms:
Congenital Abnormalities
Neurofibromatoses
Neurofibromatosis 1
Neurofibroma
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 Diseases
Neuromuscular Diseases

ClinicalTrials.gov processed this record on August 28, 2014