Study of Plexiform Neurofibromas in Neurofibromatosis Type 1

NCT00006435 | Completed

• 2 other identifiers: 01002701-C-0027
• Study Type: observational
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Plexiform Neurofibromas; NF1

Keywords

Tissue Repository; Growth Rate; Volumetric MRI

Outcome Measures

Primary Outcomes (2)

• Monitor natural history of plexiform neurofibromas

  Monitor natural history of plexiform neurofibromas

  3 years

• Usefulness of volumetric MRI measurements

  Usefulness of volumetric MRI measurements

  4 years

Secondary Outcomes (2)

• Establish tissue repository & pathology reviews center

  3 years

• Body of data regarding growth rate of plexiform neurofibromas

  3 years

Eligibility Criteria

• Age: Up to 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

A total of 300 plexiform neurofibromas will be studied, consisting of 100 tumors in the head/neck and trunk/limbs (externally visible and internal)

You may not qualify if:

• 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

Sponsors & Collaborators

• National Cancer Institute (NCI): lead

Study Sites (1)

National Institutes of Health Clinical Center, 9000 Rockville Pike

Bethesda, Maryland, 20892, United States

Location

Related Links

• NIH Clinical Center Detailed Web Page

MeSH Terms

Conditions

Neurofibroma, Plexiform

Condition Hierarchy (Ancestors)

Neurofibroma; 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

Study Officials

• Brigitte C Widemann, M.D.

  National Cancer Institute (NCI)

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 3, 2000
• First Posted: November 6, 2000
• Study Start: May 18, 2001
• Primary Completion: September 7, 2007
• Study Completion: May 1, 2020
• Last Updated: May 4, 2020

Record last verified: 2020-05

Locations

US (1)