Brief Title
Whole Body MRI to Identify Atypical Neurofibromas in Patients With NF1
Official Title
Whole Body MRI to Identify Atypical Neurofibromas in Patients With NF1 and High Plexiform Neurofibroma Tumor Burden
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.
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 Type
Interventional
Primary Outcome
To determine if WBMRI can be used to identify ANF in NF1 patients with high tumor burden.
Secondary Outcome
Determine if clinical signs and symptoms correlate with tumor burden and/or the presence of ANF on WBMRI
Condition
Neurofibromatosis 1
Intervention
Whole Body MRI
Study Arms / Comparison Groups
Study Arm
Description: 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.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Diagnostic Test
Estimated Enrollment
10
Start Date
July 12, 2018
Completion Date
August 1, 2022
Primary Completion Date
August 1, 2020
Eligibility 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
Gender
All
Ages
8 Years - 30 Years
Accepts Healthy Volunteers
No
Contacts
Miriam Bornhorst, MD, ,
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT03820778
Organization ID
Pro00010431
Responsible Party
Principal Investigator
Study Sponsor
Children's National Research Institute
Study Sponsor
Miriam Bornhorst, MD, Principal Investigator, Children's National Health System
Verification Date
February 2021