Study of Plexiform Neurofibromas in Neurofibromatosis Type 1

This study has been completed.
Sponsor:
Information provided by:
National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier:
NCT00006435
First received: November 3, 2000
Last updated: August 22, 2014
Last verified: August 2014
  Purpose

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.


Condition
Plexiform Neurofibromas
NF1

Study Type: Observational
Study Design: Time Perspective: Prospective
Official Title: Natural History of Plexiform in Neurofibromatosis Type I

Resource links provided by NLM:


Further study details as provided by National Institutes of Health Clinical Center (CC):

Primary Outcome Measures:
  • Monitor natural history of plexiform neurofibromas [ Time Frame: 3 years ] [ Designated as safety issue: No ]
  • Usefulness of volumetric MRI measurements [ Time Frame: 4 years ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Establish tissue repository & amp; pathology reviews center [ Time Frame: 3 years ] [ Designated as safety issue: No ]
  • Body of data regarding growth rate of plexiform neurofibromas [ Time Frame: 3 years ] [ Designated as safety issue: No ]

Enrollment: 24
Study Start Date: October 2000
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.

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria
  • INCLUSION CRITERIA:

Subjects must fulfill two or more of the following diagnostic criteria for NF1:

Six or more cafe-au-lait macules (1. 1.5 cm or larger in postpubertal individuals; 2. 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 (1. Dysplasia of the sphenoid bone; 2. Dysplasia or thinning of long bone cortex);

A first degree relative with NF-1.

EXCLUSION CRITERIA:

Presence of metallic implant that will make the patient unable to have MRI studies.

Presence of a 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.

  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: NCT00006435

Locations
United States, Maryland
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States, 20892
United States, Massachusetts
Partners Center for Human Genetics
Boston, Massachusetts, United States, 02115
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Sponsors and Collaborators
Investigators
Principal Investigator: Brigitte C Widemann, M.D. National Cancer Institute (NCI)
  More Information

Additional Information:
No publications provided

ClinicalTrials.gov Identifier: NCT00006435     History of Changes
Obsolete Identifiers: NCT00445991
Other Study ID Numbers: 010027, 01-C-0027
Study First Received: November 3, 2000
Last Updated: August 22, 2014
Health Authority: United States: Federal Government

Keywords provided by National Institutes of Health Clinical Center (CC):
Tissue Repository
Growth Rate
Volumetric MRI

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

ClinicalTrials.gov processed this record on August 26, 2014