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CompletedNCT00006435Updated May 4, 2020

Study of Plexiform Neurofibromas in Neurofibromatosis Type 1

An observational study in Plexiform Neurofibromas and NF1, sponsored by National Cancer Institute (NCI). Completed at 1 site in United States. Open to participants aged Up to 100 Years. Per ClinicalTrials.gov, last updated 2020-05-04.

Sponsored by National Cancer Institute (NCI) · Observational

Study type
Observational
Model
Cohort
Time perspective
Prospective
Enrollment
24
Ages
Up to 100 Years
Sex
All
01

Study 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.
Read the 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.
02

Conditions studied

  • Plexiform Neurofibromas
  • NF1

Keywords

  • Tissue Repository
  • Growth Rate
  • Volumetric MRI
03

In context

Neurofibroma, Plexiform

63 studies on the registry are indexed under Neurofibroma, Plexiform; 19 are open to participants now.

This study's enrollment of 24 is below the median of 200 across 13 observational studies indexed under Neurofibroma, Plexiform.

Browse Neurofibroma, Plexiform studies →

Lead sponsor

National Cancer Institute (NCI) is the lead sponsor of 3,556 studies on the registry; 336 are open to participants now.

Of its 411 completed or terminated interventional studies of FDA-regulated products, 373 (91%) have results posted.

Browse National Cancer Institute (NCI) studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
Up to 100 Years
Sexes eligible
All
Accepts healthy volunteers
No
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)

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  • 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 criteria

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 ) 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
Terms in these criteria, in plain language(1)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Biopsy and archival tissue

A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.

Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.

05

Study design

Observational model
Cohort
Time perspective
Prospective
Enrollment
24 participants (actual)
06

What researchers measure

Primary outcomes

  1. Monitor natural history of plexiform neurofibromas

    Monitor natural history of plexiform neurofibromas

    Time frame: 3 years

  2. Usefulness of volumetric MRI measurements

    Usefulness of volumetric MRI measurements

    Time frame: 4 years

Secondary outcomes

  1. Establish tissue repository & pathology reviews center

    Time frame: 3 years

  2. Body of data regarding growth rate of plexiform neurofibromas

    Time frame: 3 years

07

Study locations

1 site
  • National Institutes of Health Clinical Center, 9000 Rockville Pike
    Bethesda, Maryland 20892, United States
08

References and documents

09

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Ran from May 2001 to primary completion in Sep 2007, 75 months.

How these figures are worked out, and which studies are left out of them.

Registered
Nov 3, 2000
First posted
Nov 6, 2000
StartActual
May 18, 2001
Primary completionActual
Sep 7, 2007
CompletionActual
May 1, 2020
Last update
May 4, 2020
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on May 4, 2020, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT00006435
Lead sponsor
National Cancer Institute (NCI)
Responsible party
Sponsor
First posted
Nov 6, 2000
Start date
May 18, 2001
Primary completion
Sep 7, 2007
Completion
May 1, 2020
Last update
May 4, 2020

Study contacts

Brigitte C Widemann, M.D.
principal investigator · National Cancer Institute (NCI)
View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in May 2020. You cannot join it, but the record below documents what was studied.

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