Neurofibromatosis Type 1NF1TDNCT00303368
Neurofibromatosis Type 1 (NF1) and Tibial Dysplasia
An observational study of Neurofibromatosis Type 1 and Tibial Dysplasia, run by Shriners Hospitals for Children at 1 site in United States.
Trial summary
The study is a multicenter four-year outcome study of the natural history of tibial dysplasia in patients with NF1 and selected patients without NF1. We will obtain information on the natural history, burden, functional and health status, health-related quality of life, and surgical interventions/outcomes of tibial… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Neurofibromatosis Type 1, Tibial Dysplasia
- How many people
- 395 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2004to Jan 2016
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Adults and children with NF1, with or without Tibial Dysplasia, ages 3+ and individuals ages birth through 18 with Tibial Dysplasia, with or without NF1.
Who may be able to join
- Aim 1:
- Group 1, NF1 with Tibial Dysplasia, Ages: 3-18
- Group 2 (control), NF1 without Tibial Dysplasia, Ages 3-18
- Aim 2: NF1 with Tibial Dysplasia, 19+
- Aim 3: Tibial Dysplasia with or without NF1, Ages: birth to 18
- Tissue procurement, any participant undergoing surgery at the tibial site for routine standard of care
Who cannot join
- Patients without a diagnosis of NF1 or Tibial Dysplasia
Trial locations and contacts
1 site in 1 country.
United States1 site
Shriners Hospitals for Children, Intermountain Hospital
Salt Lake City, Utah, 84103
About this study
Official title: Multicenter Study of Tibial Dysplasia in Neurofibromatosis Type I (NF1) Patients
The study is a multicenter four-year outcome study of the natural history of tibial dysplasia in patients with NF1 and selected patients without NF1. We will obtain information on the natural history, burden, functional and health status, health-related quality of life, and surgical interventions/outcomes of tibial dysplasia. The project will also establish a Core Facility (NOCF) for tissue samples for future studies.
Read the detailed description
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.
- Conditions
- Neurofibromatosis Type 1, Tibial Dysplasia
- Keywords
- Neurofibromatosis (NF1), Tibial Dysplasia (TD)
- Medical subject headings
- Neurofibromatosis 1, Neurofibromatoses
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Mar 2006 (estimated)
- Study startMar 2004
- Primary completionJan 2014
- Study completionJan 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neurofibromatosis Type 1Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Retrospective
- Enrolment
- 395 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shriners Hospitals for Children
- Sponsor type
- University, hospital or other
- Collaborators
- University of Utah
- Responsible party
- Principal investigator, Jacques D'Astous, Shriners Hospitals for Children
- Investigators
- John C Carey, MD, MPH, Principal investigator, University of Utah, Health Science Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00303368
- Sponsor's protocol ID
- 9165
- Acronym
- NF1TD
- Status last verified
- Feb 2019
- First submitted
- 14 Mar 2006
- Last updated
- 25 Feb 2019
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 25 Feb 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter Study of Tibial Dysplasia in Neurofibromatosis Type I (NF1) Patients
- ClinicalTrials.gov ID
- NCT00303368
- Acronym / sponsor ID
- NF1TD / 9165
- Conditions
- Neurofibromatosis Type 1, Tibial Dysplasia
- Keywords
- Neurofibromatosis (NF1), Tibial Dysplasia (TD)
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 395 participants (actual)
- Lead sponsor
- Shriners Hospitals for ChildrenUniversity, hospital or other
- Collaborators
- University of Utah
- Study start
- Mar 2004 (actual)
- Primary completion
- Jan 2014 (actual)
- Study completion
- Jan 2016 (actual)
- First posted
- 16 Mar 2006 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study is a multicenter four-year outcome study of the natural history of tibial dysplasia in patients with NF1 and selected patients without NF1. We will obtain information on the natural history, burden, functional and health status, health-related quality of life, and surgical interventions/outcomes of tibial dysplasia. The project will also establish a Core Facility (NOCF) for tissue samples for future studies.
Detailed description
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 design
- Observational model
- Other
- Time perspective
- Retrospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Adults and children with NF1, with or without Tibial Dysplasia, ages 3+ and individuals ages birth through 18 with Tibial Dysplasia, with or without NF1.
Key inclusion criteria (6 listed)
- Aim 1:
- Aim 2: NF1 with Tibial Dysplasia, 19+
- Aim 3: Tibial Dysplasia with or without NF1, Ages: birth to 18
- Tissue procurement, any participant undergoing surgery at the tibial site for routine standard of care
Key exclusion criteria (1 listed)
- Patients without a diagnosis of NF1 or Tibial Dysplasia
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Feb 2019.
Related trials for Neurofibromatosis Type 1
Active, not recruitingPhase 1/2
Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma
Low Grade Glioma, Recurrent Childhood Pilocytic Astrocytoma
RecruitingPhase 3
A Study of the Drugs Selumetinib vs. Carboplatin and Vincristine in Patients With Low-Grade Glioma
Low Grade Astrocytoma, Low Grade Glioma
RecruitingPhase 1
Phase I Trial of TURALIO(R) (Pexidartinib, PLX3397) in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN) and Tenosynovial Giant Cell Tumor ...
Neurofibroma, Plexiform, Precursor Cell Lymphoblastic Leukemia-Lymphoma
Active, not recruitingPhase 2
Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
Juvenile Myelomonocytic Leukemia, Neurofibromatosis Type 1
RecruitingObservational study
Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST)
Neurofibromatosis 1, Nerve Sheath Neoplasms
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
John C Carey, MD, MPH principal investigator
University of Utah, Health Science Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Neurofibromatosis Type 1 and Tibial Dysplasia, run by Shriners Hospitals for Children at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Feb 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Feb 2019.