Whole Body MRI for Neurofibromatoses

1
Effectiveness
1
Safety
Children's National Health System, Washington, United States
Neurofibromatoses+7 More
Whole Body MRI - DiagnosticTest
Eligibility
< 65
All Sexes
Eligible conditions
Neurofibromatoses

Study Summary

This study is evaluating whether whole body MRI can be used to identify atypical neurofibromas in neurofibromatosis type 1 patients with high tumor burden.

See full description

Eligible Conditions

  • Neurofibromatoses
  • Neurofibroma
  • Neurofibroma, Plexiform
  • Watson Syndrome
  • Atypical Neurofibroma
  • Von Recklinghausen Disease
  • Atypical Neurofibromatosis
  • Plexiform Neurofibromas (PN)
  • Neurofibromatosis 1

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether Whole Body MRI will improve 1 primary outcome, 1 secondary outcome, and 1 other outcome in patients with Neurofibromatoses. Measurement will happen over the course of 2-3 years.

2-3 years
Determine if clinical signs and symptoms correlate with tumor burden and/or the presence of ANF on WBMRI
To determine if WBMRI can be used to identify ANF in NF1 patients with high tumor burden.
3-4 years
Determine if the cfDNA levels correlate with presence of ANF.

Trial Safety

Safety Estimate

1 of 3

Trial Design

2 Treatment Groups

Control
Study Arm

This trial requires 10 total participants across 2 different treatment groups

This trial involves 2 different treatments. Whole Body MRI is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are not being studied for commercial purposes.

Study Arm
DiagnosticTest
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.
ControlNo treatment in the control group
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Whole Body MRI
2007
N/A
~40

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 3-4 years
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 3-4 years for reporting.

Who is running the study

Principal Investigator
M. B.
Prof. Miriam Bornhorst, Assistant Professor Division of Oncology and Center for Genetics Medicine
Children's National Research Institute

Closest Location

Children's National Health System - Washington, United States

Eligibility Criteria

This trial is for patients born any sex aged 65 and younger. There are 4 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Stated willingness to comply with all study procedures and availability for the duration of the study
Prior MRI documentation confirming >=1 PN that is >3cm in diameter
Males or females between 8-30 years of age
Confirmed diagnosis of NF1

Patient Q&A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What does whole body mri usually treat?

Add answer

Whole-body MRIs are helpful for the treatment of people with [neurofibromatoses] who are asymptomatic and are able to live well with the diagnosis. MRIs are also an important tool to help guide therapeutic decisions, assist in planning treatment, and to monitor those who receive therapy. They are generally safe to inject and take, with a low risk of adverse effects.

Unverified Answer

Does neurofibromatoses run in families?

Add answer

Familial neurofibromatosis (NF2), in particular, is the genetic predisposition behind many hereditary cancers. The authors suggest that NF2 deserves further investigation for genetic predispositions for malignant peripheral nerve sheath tumors.

Unverified Answer

What causes neurofibromatoses?

Add answer

The most common cause of neurofibromatosis is thought to be environmental stress, though there is some evidence that this cancer may be more strongly linked to genetics.\nquestion: Is the number of medications that an individual uses associated with the number of medications prescribed?\answer: In a recent study, findings suggest that taking more than four analgesics to relieve joint, back, or muscle pain may be associated with increased prescribing of multiple pain medications including nonsteroidal anti-inflammatory drugs (NSAIDs) and medications that relieve joint and/or muscle pain. Furthermore, this association is stronger for NSAIDs than pain-relievers, and the magnitude of the association is potentially clinically important.

Unverified Answer

What are common treatments for neurofibromatoses?

Add answer

There is a wide range of therapies for neurofibromatosis, each of which has a unique benefit and side effect profile for each patient and tumor type.\n

Unverified Answer

How many people get neurofibromatoses a year in the United States?

Add answer

Neurofibromatoses represent 12/10% of newly diagnosed cancers in the United States. Given the number of cancers diagnosed with neurofibromatoses annually, it is likely that a significant number of patients have a delayed diagnosis of neurofibromatosis. Given the current rate of neurofibromatosis diagnosis annually, this delayed diagnosis may delay or preclude a potentially curative therapy. These discrepancies must be considered when counseling patients, as this could be harmful to the patient as well as their family.

Unverified Answer

What is neurofibromatoses?

Add answer

The neurofibromatoses are extremely variable but they can be recognized as a result of a specific mutation in a tumor suppressor gene. They occur as a result of the unregulated division of cells at a particular point in development rather than cancer. In the development of neurofibromatoses the tumor cells proliferate to form tumors. The tumors are most often benign but when malignant degeneration occurs the condition can be called a malignant tumor. The most common form of malignant tumor is neurofibromatosis Type 1 or NF1. Other forms of neurofibromatosis are harder to diagnose and may develop into a malignancy.

Unverified Answer

What are the signs of neurofibromatoses?

Add answer

In addition to the signs of cutis or dermal tumors, cutaneous and subcutaneous tumors may be associated with neurofibromas. Inadequate knowledge of cutaneous manifestation of neurofibromatoses may delay diagnosis and management of these patients, who often have no history of neurofibromas or dermal tumors. Tumoral cutaneous manifestations may present as wart-like ulcers with margins resembling a wound, cutis, or tumor-like skin masses or, less commonly, as a tumor with palpable margin.

Unverified Answer

Can neurofibromatoses be cured?

Add answer

With careful and close monitoring, some individuals with NF1 and NF2 can manage their disease. Because of the long-term risk of malignancy, long-term surveillance and early referral for early therapeutic therapy to achieve maximal symptom reduction before the development of malignancy is an essential clinical decision.

Unverified Answer

What are the chances of developing neurofibromatoses?

Add answer

While NF is an occupational hazard, it should be considered that only 2.6% of people developed a neurofibroma between 1986 and 2001. The possibility of NF, or other neurofibromatosis type-1 (NF1) diseases, should be sought when patients have multiple tumors, multiple tumors in different areas, or multiple tumors and enlarged LNs. The prevalence of NF1 in Koreans is 0.28% and is about a quarter that of the US population (0.82%).

Unverified Answer

Is whole body mri safe for people?

Add answer

Given our high rate of malignancies with whole body mri, we recommend use of whole body mri only when the patient is suspected of having a malignant growth. Whole body mri should not be used for screening as suggested by some authors. We should be careful when interpreting the scan and use it wisely. Recent findings have shown the superiority of whole body mri for detecting malignant tumors. Whole body mri was particularly useful in detecting intracranial tumors and soft tissue tumors.

Unverified Answer

Is whole body mri typically used in combination with any other treatments?

Add answer

The most prominent finding of our study is the widespread use of mri as an initial imaging study in patients with rENF. It is important to recognize this imaging modality as helpful in rENF treatment as well as diagnostic procedures, in particular in this age of imaging-based medicine. Whole-body mri can play an important role in the initial rENF study.

Unverified Answer

What is the survival rate for neurofibromatoses?

Add answer

Survival rates depend largely upon the type of neurofibromatosis (NFs). The prognosis of patients with Schwannomatosis is excellent and similar to that of the general population. However, NF2 is associated with a particularly unfavorable prognosis.

Unverified Answer
See if you qualify for this trial
Get access to this novel treatment for Neurofibromatoses by sharing your contact details with the study coordinator.