Neurofibromatosis Type 1 (NF1) and Tibial Dysplasia (NF1TD)
|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.|
|ClinicalTrials.gov Identifier: NCT00303368|
Recruitment Status : Unknown
Verified May 2008 by Shriners Hospitals for Children.
Recruitment status was: Recruiting
First Posted : March 16, 2006
Last Update Posted : May 23, 2008
|Condition or disease|
|Neurofibromatosis Type 1 Tibial Dysplasia|
The three specific aims of this study are:
- Specific Aim 1 - To assess health status and health - related quality of life (HRQL) in 50 children and adolescents with NF1 and tibial dysplasia and in NF1 controls. We hypothesize that children and adolescents with NF1 and tibial dysplasia will experience an additional burden of morbidity due to tibial dysplasia and a downward trajectory of health status and HRQL over time.
- Specific Aim 2 - To assess the long term outcome of current treatment in 100 adult patients diagnosed with NF1 and tibial dysplasia in childhood. We hypothesize that better quality of life and function, in adults with NF1 and tibial dysplasia, are associated with amputation in childhood compared to multiple surgical procedures, the lack of fibular involvement, and fracture later in childhood. We also hypothesize that individuals with NF1 and tibial dysplasia have a higher risk of other bony dysplasias but are at no higher risk of fracture in other bones.
- Specific Aim 3 - To assess the natural history and short-term response to therapy in a cohort of at least 60 children with NF1 and tibial dysplasia and at least 60 children with tibial dysplasia presumably without NF1 prospectively diagnosed during the course of the four-year study period. We hypothesize that NF1 patients with earlier presentation, Crawford Class II A-C, male gender, and the lack of bracing prior to age two are more likely to fracture. We also postulate that individuals with and without NF1 have a similar outcome and response to treatment.
The results of this project will provide a rational basis for future clinical and therapeutic trials.
|Study Type :||Observational|
|Estimated Enrollment :||420 participants|
|Official Title:||Multicenter Study of Tibial Dysplasia in Neurofibromatosis Type I (NF1) Patients|
|Study Start Date :||March 2004|
|Estimated Study Completion Date :||December 2008|
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): NCT00303368
|Contact: Jeanne A Siebert, MBAfirstname.lastname@example.org|
|Contact: Susan J Geyer, BAemail@example.com|
|United States, Utah|
|Shriners Hospitals for Children, Intermountain Hospital||Recruiting|
|Salt Lake City, Utah, United States, 84103|
|Contact: Jeanne A Siebert, MBA 801-536-3601 firstname.lastname@example.org|
|Contact: Susan J Geyer, BA 801-536-3561 email@example.com|
|Principal Investigator: Jacques D'Astous, MD|
|Principal Investigator:||John C Carey, MD, MPH||University of Utah, Health Science Center|