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Whole Body MRI to Identify Atypical Neurofibromas in Patients With NF1

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03820778
Recruitment Status : Enrolling by invitation
First Posted : January 29, 2019
Last Update Posted : January 29, 2019
Sponsor:
Information provided by (Responsible Party):
Miriam Bornhorst, Children's National Research Institute

Study Description
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Brief Summary:
This study is being conducted to determine if Whole Body MRI (WBMRI) can be used to identify Atypical Neurofibromas (ANF) in Neurofibromatosis Type 1 (NF1) patients with high tumor burden. Each enrolled participant will have two (2) WBMRIs without sedation during the study period. Eligible participants must be Male or Female between the ages of 8-30 with diagnosed NF1; with one or more PN greater than 3cm in diameter and willing to comply with study procedures.

Condition or disease Intervention/treatment Phase
Neurofibromatosis 1 Neurofibroma Atypical Neurofibroma Atypical Neurofibromatosis Plexiform Neurofibroma Von Recklinghausen Disease Diagnostic Test: Whole Body MRI Not Applicable

Detailed Description:

This is a study to determine the feasibility of using Whole Body MRI (WBMRI) to detect Atypical Plexiform Neurofibromas (ANF) among Neurofibromatosis Type 1 (NF1) patients who are at highest risk for developing these tumors. In order to do this, the investigators will prospectively obtain WBMRI scans on patients with high plexiform tumor burden (which investigators will define as >=1 plexiform neurofibroma (PN) that is >3cm in diameter on MRI) at the same time as their regularly scheduled MRI to allow for radiographic comparison of the identified PNs. In this study, the investigators will plan to establish a method that effectively characterizes PNs in terms of their volume, radiographic appearance on WBMRI and association with clinical characteristics.

As an exploratory aim, the investigators plan to collect blood samples from each patient at the same time as the MRI to determine the feasibility of isolating cfDNA (circulating free DNA) from plasma of patients with high plexiform tumor burden.

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Diagnostic
Official Title: Whole Body MRI to Identify Atypical Neurofibromas in Patients With NF1 and High Plexiform Neurofibroma Tumor Burden
Actual Study Start Date : July 12, 2018
Estimated Primary Completion Date : July 12, 2022
Estimated Study Completion Date : July 12, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Neurofibromatosis

Arms and Interventions
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Arm Intervention/treatment
Experimental: Study Arm
Whole Body MRI along with standard of care regional MRI and blood draw at enrollment followed by Whole Body MRI along with standard of care regional MRI and blood draw after 4-6 months.
 Diagnostic Test: Whole Body MRI
WBMRI with axial and coronal STIR (short inversion time inversion recovery) images + DWI (diffuse weighted imaging) will be performed and compared to regional MRI to assess value of this diagnostic test for the identification of suspicious looking lesions (i.e. diffuse nodular lesions or atypical plexiform neurofibroma)


Outcome Measures
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Primary Outcome Measures :
  1. To determine if WBMRI can be used to identify ANF in NF1 patients with high tumor burden. [ Time Frame: 2-3 years ]
    We will review results of the WBMRI compared to regional MRIs (done with contrast) to determine if the WBMRI can accurately detect Atypical Neurofibromas.


Secondary Outcome Measures :
  1. Determine if clinical signs and symptoms correlate with tumor burden and/or the presence of ANF on WBMRI [ Time Frame: 2-3 years ]
    Using patient data collected during their routine clinic evaluation, we will determine if clinical signs and symptoms correlate with tumor burden and/or the presence of ANF on WBMRI


Other Outcome Measures:
  1. Determine if the cfDNA levels correlate with presence of ANF. [ Time Frame: 3-4 years ]
    We will measure cfDNA levels in each participant, and determine if these levels correlate with tumor burden and/or the presence of ANFs.


Eligibility Criteria
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Ages Eligible for Study:   8 Years to 30 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Males or females between 8-30 years of age
  2. Confirmed diagnosis of NF1
  3. Stated willingness to comply with all study procedures and availability for the duration of the study
  4. Prior MRI documentation confirming >=1 PN that is >3cm in diameter

Exclusion Criteria:

  1. Unable to undergo MRI without sedation
  2. Presence of metal or other devices that are contraindicated for MRI
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03820778


Locations
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United States, District of Columbia
Children's National Health System
Washington, District of Columbia, United States, 20010
Sponsors and Collaborators
Children's National Research Institute
Investigators
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Principal Investigator: Miriam Bornhorst, MD Children's National Health System
More Information
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Publications:

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Responsible Party: Miriam Bornhorst, Assistant Professor Division of Oncology and Center for Genetics Medicine, Children's National Research Institute
ClinicalTrials.gov Identifier: NCT03820778    
Other Study ID Numbers: Pro00010431
First Posted: January 29, 2019    Key Record Dates
Last Update Posted: January 29, 2019
Last Verified: January 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: Yes
Product Manufactured in and Exported from the U.S.: Yes
Additional relevant MeSH terms:
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Neurofibromatoses
Neurofibromatosis 1
Neurofibroma
Neurofibroma, Plexiform
Nerve Sheath Neoplasms
Neoplasms, Nerve Tissue
Neoplasms by Histologic Type
Neoplasms
Neoplastic Syndromes, Hereditary
 Neurocutaneous Syndromes
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Peripheral Nervous System Diseases
Neuromuscular Diseases
Peripheral Nervous System Neoplasms
Nervous System Neoplasms