- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00006435
Study of Plexiform Neurofibromas in Neurofibromatosis Type 1
Natural History of Plexiform in Neurofibromatosis Type I
Background:
-Plexiform neurofibromas in patients with NF1 are a significant cause of morbidity but little is known about the natural history of these lesions.
Objectives:
- The purpose of this study is to monitor the natural history of plexiform neurofibromas and to evaluate the usefulness of volumetric MRI tumor measurements in this disease.
- Other goals of the study are to provide a body of normative data on the growth rate of plexiform neurofibromas and to establish a tissue repository and pathology review center to allow future studies of the pathogenesis of neurofibromas and clinical trials of potential therapeutic agents.
Design
- This study is coordinated by Dr. Bruce Korf, and was initiated when he was at the Partners Center for Human Genetics, Boston, MA.
Study Overview
Status
Conditions
Detailed Description
Background:
-Plexiform neurofibromas in patients with NF1 are a significant cause of morbidity but little is known about the natural history of these lesions.
Objectives:
- The purpose of this study is to monitor the natural history of plexiform neurofibromas and to evaluate the usefulness of volumetric MRI tumor measurements in this disease.
- Other goals of the study are to provide a body of normative data on the growth rate of plexiform neurofibromas and to establish a tissue repository and pathology review center to allow future studies of the pathogenesis of neurofibromas and clinical trials of potential therapeutic agents.
Design
- This study is coordinated by Dr. Bruce Korf, and was initiated when he was at the Partners Center for Human Genetics, Boston, MA.
Study Type
Enrollment (Actual)
Contacts and Locations
Study Locations
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Maryland
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Bethesda, Maryland, United States, 20892
- National Institutes of Health Clinical Center, 9000 Rockville Pike
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Sampling Method
Study Population
Description
- INCLUSION
Diagnosis of Neurofibromatosis: All study subjects will fulfill two or more of the diagnostic criteria listed below for NF1.
Six or more caf(SqrRoot)(Copyright)-au-lait macules
- 1.5cm or larger in postpubertal individuals
- 0.5 cm or larger in prepubertal individuals
- Two or more neurofibromas of any type or 1 or more plexiform neurofibroma
- Freckling in the axilla or groin
- Optic glioma (tumor of the optic pathway)
- Two or more Lisch nodules (benign iris hamartomas)
A distinctive bony lesion
- Dysplasia of the sphenoid bone
- Dysplasia or thinning of long bone cortex
- A first degree relative with NF-1
- Plexiform Neurofibroma: A plexiform neurofibroma fulfilling entry criteria for the study will be defined as a diffuse soft tissue or nerve enlargement in a patient with NF1 that is causing, or has potential to cause, disfigurement or functional disability.
Distribution of Plexiform Neurofibromas by site: A total of 300 plexiform neurofibromas will be studied, consisting of 100 tumors in the following three groups (based on region of maximal involvement):
- Head and Neck
- Trunk and Limbs (externally visible)
- Trunk and Limbs (internal) [spinal plexiform neurofibromas involve two or more levels with connection between the levels or extending laterally along the nerve]
- Subject Ascertainment: Study subjects will be ascertained at any of the participating clinical centers. It is expected that these will include subjects already followed in these clinics, as well as newly diagnosed patients
EXCLUSION
- Presence of metallic implant(s) that will make the patient unable to have MRI studies
- Presence of medical or psychological condition that will make the patient unable to tolerate MRI studies or anesthesia (if needed)
- Inability to image tumor or define tumor margins by MRI (which may be determined after the initial study)
- Failure to obtain initial MRI within 60 days of enrollment
- Previous radiation therapy to site of plexiform neurofibroma
- Surgery involving the plexiform neurofibroma (excluding biopsy) within a six month period before enrollment
- Current antineoplastic therapy
- Entry of more than one member of the same family into the study is not permitted
Study Plan
How is the study designed?
Design Details
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Monitor natural history of plexiform neurofibromas
Time Frame: 3 years
|
Monitor natural history of plexiform neurofibromas
|
3 years
|
Usefulness of volumetric MRI measurements
Time Frame: 4 years
|
Usefulness of volumetric MRI measurements
|
4 years
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Establish tissue repository & pathology reviews center
Time Frame: 3 years
|
3 years
|
Body of data regarding growth rate of plexiform neurofibromas
Time Frame: 3 years
|
3 years
|
Collaborators and Investigators
Sponsor
Publications and helpful links
Helpful Links
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Nervous System Diseases
- Neoplasms by Histologic Type
- Neoplasms
- Genetic Diseases, Inborn
- Neuromuscular Diseases
- Neurodegenerative Diseases
- Neoplasms, Nerve Tissue
- Peripheral Nervous System Diseases
- Nervous System Neoplasms
- Heredodegenerative Disorders, Nervous System
- Neoplastic Syndromes, Hereditary
- Nerve Sheath Neoplasms
- Neurocutaneous Syndromes
- Peripheral Nervous System Neoplasms
- Neurofibromatoses
- Neurofibromatosis 1
- Neurofibroma
- Neurofibroma, Plexiform
Other Study ID Numbers
- 010027
- 01-C-0027
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Plexiform Neurofibromas
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AstraZenecaApproved for marketingNF type1 With Inoperable Plexiform NeurofibromasUnited States
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National Institutes of Health Clinical Center (CC)National Cancer Institute (NCI)CompletedUnspecified Childhood Solid Tumor, Protocol Specific | Neoplasm of Uncertain Malignant PotentialUnited States
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SpringWorks Therapeutics, Inc.AvailableNeurofibromatosis Type 1-Associated Plexiform Neurofibromas | Histiocytic Neoplasm | Other MAP-K Pathway Driven Diseases
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University of PittsburghNational Cancer Institute (NCI)Completed
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AstraZenecaCompletedHealthy Participants | Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PNs)United States
-
Shanghai Fosun Pharmaceutical Industrial Development...RecruitingNeurofibromatosis 1 | NF1 | Plexiform NeurofibromaChina
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-
Shanghai Ninth People's Hospital Affiliated to...RecruitingNeurofibromatosis 1 | Plexiform NeurofibromaChina
-
University of Alabama at BirminghamChildren's Hospital of Philadelphia; Congressionally Directed Medical Research...Not yet recruitingNeurofibromatosis 1 | Plexiform Neurofibroma