Incidence of Malignant Peripheral Nerve Sheath Tumor (MPNST) Development in Participants With Neurofibromatosis Type 1 (NF1) Receiving and Not Receiving Medical Therapies Directed at Plexiform Neurofibromas (PN)

October 25, 2023 updated by: National Cancer Institute (NCI)

Incidence of Malignant Peripheral Nerve Sheath Tumors Development in Patients With Neurofibromatosis Type 1 Receiving and Not Receiving Medical Therapies Directed at Plexiform Neurofibromas

Background:

NF1 is a genetic syndrome. Tumors appear early in life. Many people with NF1 develop PN. These tumors can become an aggressive cancer called MPNST. People with MPNST may benefit from treatment with a MEK inhibitor (MEKi). Researchers want to learn if there is an increased risk of MPNST formation from MEKi treatment in people with NF1. To do this, they will review data that has been collected in NIH NF1 studies.

Objective:

To describe the characteristics of people who have taken part in NF1 studies at NIH and to compare the risk of MPNST formation in those treated with MEKi or other PN-directed treatment.

Eligibility:

People with NF1 who were seen at NIH from Jan. 1, 1998, to Jan. 1, 2020.

Design:

Participants medical records will be reviewed. Participants who opted out of future use of their data will not be included.

Demographic data, like sex, race, and date of birth, will be collected.

Data about MEKi and non-MEKi treatments will be collected.

Clinical data, such as surgery and treatment details, will be collected.

The differences between all participants who were seen at NIH for any NF1 related study will be compared. Participants will be put into 4 groups:

History of MEKi therapy

Treatment with tumor directed therapy other than MEKi

Treatment with both MEKi and non-MEKi tumor directed therapies

No tumor directed medical therapy

Participants with NF1 who were treated for PN with either a MEKi treatment or a non-MEKi treatment will also be compared.

The study will last for 3 to 6 months.

Study Overview

Detailed Description

Study Description: This is a retrospective review that includes all patients with NF1 enrolled on natural history and treatment trials at the National Cancer Institute (NCI) from 1/1/1998 to 1/1/2020, excluding patients referred for treatment of MPNST.

Objective: To describe the clinical and demographic characteristics of NF1 study participants who come for evaluation at the NCI and to compare the risk of MPNST formation in patients treated with MEK 1/2 inhibitor (MEKi) or other PN-directed treatment.

Study Population: All participants with NF1 seen at the NIH from 1/1/1998 to 1/1/2020

Description of Sites/Facilities conducting research: This is a single site study initiated by the Pediatric Oncology Branch at the National Cancer Institute

Study Duration: Data abstraction to completion of data analyses will take 3-6 months.

Study Type

Observational

Enrollment (Actual)

296

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

    • Maryland
      • Bethesda, Maryland, United States, 20892
        • National Cancer Institute (NCI)

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 month and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

primary clinical

Description

  • Inclusion Criteria:
  • All participants seen at the NIH from 1/1/1998 to 1/1/2020 with a clinical or laboratory diagnosis of NF1
  • Participants beyond the neonatal period (4 weeks).

Exclusion Criteria:

-All participants seen at the NIH from 1/1/1998 to 1/1/2020 with a clinical or laboratory diagnosis of NF1 who opted out of future use.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Retrospective

Cohorts and Interventions

Group / Cohort
1
Participants with NF1 seen at the NIH from 1/1/1998 to 1/1/2020

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
risk of MPNST formation
Time Frame: ongoing
descriptive statistics and frequency distribution of selected variables
ongoing

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Investigators

  • Principal Investigator: Brigitte C Widemann, M.D., National Cancer Institute (NCI)

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 24, 2021

Primary Completion (Actual)

March 1, 2022

Study Completion (Actual)

December 8, 2022

Study Registration Dates

First Submitted

February 26, 2021

First Submitted That Met QC Criteria

February 26, 2021

First Posted (Actual)

March 1, 2021

Study Record Updates

Last Update Posted (Actual)

October 26, 2023

Last Update Submitted That Met QC Criteria

October 25, 2023

Last Verified

October 24, 2023

More Information

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 Neurofibromatosis 1

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