- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04774289
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)
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
Status
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
Enrollment (Actual)
Contacts and Locations
Study Locations
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Maryland
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Bethesda, Maryland, United States, 20892
- National Cancer Institute (NCI)
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Sampling Method
Study Population
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
How is the study designed?
Design Details
- Observational Models: Cohort
- Time Perspectives: Retrospective
Cohorts and Interventions
Group / Cohort |
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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
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ongoing
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Collaborators and Investigators
Sponsor
Investigators
- Principal Investigator: Brigitte C Widemann, M.D., National Cancer Institute (NCI)
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 (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Nervous System Diseases
- Neoplasms, Connective and Soft Tissue
- Neoplasms by Histologic Type
- Neoplasms by Site
- Genetic Diseases, Inborn
- Neuromuscular Diseases
- Neurodegenerative Diseases
- Neoplasms, Nerve Tissue
- Peripheral Nervous System Diseases
- Nervous System Neoplasms
- Heredodegenerative Disorders, Nervous System
- Neoplastic Syndromes, Hereditary
- Neoplasms, Connective Tissue
- Sarcoma
- Neurocutaneous Syndromes
- Neoplasms, Fibrous Tissue
- Fibrosarcoma
- Neoplasms
- Neurofibromatoses
- Neurofibromatosis 1
- Neurofibroma
- Nerve Sheath Neoplasms
- Neurofibrosarcoma
- Neurofibroma, Plexiform
- Peripheral Nervous System Neoplasms
Other Study ID Numbers
- 10000271
- 000271-C
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
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.
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