Transformation of Plexiform Neurofibromas to Malignant Peripheral Nerve Sheath Tumors in Neurofib… (NCT02211768) | Clinical Trial Compass
CompletedPhase 1
Transformation of Plexiform Neurofibromas to Malignant Peripheral Nerve Sheath Tumors in Neurofibromatosis Type 1
United States10 participantsStarted 2014-12-08
Plain-language summary
Background:\<TAB\>
\- Many people with neurofibromatosis type 1 (NF1) get tumors of the nervous system. Finding malignant tumors early is important for removing them. Researchers want to find ways of doing this with scans and genetic testing.
Objectives:
\- To learn more about neurofibromatosis type 1.
Eligibility:
\- People age 10 and older with NF1 who have a benign tumor or have had a malignant one.
Design:
* Participants will be screened in another study with medical history, physical exam, and urine and blood tests. They will have a magnetic resonance imaging (MRI) scan.
* MRI: Participants will lie on a table that slides into a metal cylinder. They will be in the scanner for 60 90 minutes, lying still for 15 minutes at a time. Participants will get earplugs for the loud sounds. They will get a contrast agent (dye) through a thin plastic tube (catheter) inserted in an arm vein.
* As part of their regular care, participants will have:
* FDG-PET/CT scan. They will get radioactive glucose (sugar) through a catheter in an arm vein.
* \[18F\]-FLT-PET/CT scan. This is like the FDG scan but with a different radioactive chemical.
* Biopsy. A piece of tumor tissue is removed with a needle. A piece of tissue from a previous biopsy may also be studied.
* Participants may have genetic testing. Blood will be taken. It will be tested along with biopsy samples. Researchers will explain the risks and procedures. They may notify participants if testing shows health problems.
* After this study, participants will continue their regular cancer care.
Who can participate
Age range
10 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Six or more caf(SqrRoot)(Copyright)-au-lait spots (greater than or equal to 0.5 cm in prepubertal subjects or greater than or equal to 1.5 cm in postpubertal subjects)
. Greater than or equal to 2 neurofibromas or 1 plexiform neurofibroma
. Freckling in the axilla or groin
. Optic glioma
. Two or more Lisch nodules
. A distinctive bony lesion (dysplasia of the sphenoid bone or dysplasia or thinning of long bone cortex)
. A first-degree relative with NF1
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Since this trial has already been completed, would it be worth asking whether the results from the whole-exome sequencing or FLT PET findings have been published, and whether those findings might apply to my situation?
2This study compared FLT PET scans to FDG PET scans to tell apart benign plexiform neurofibromas from malignant tumors — based on what this research found, which type of PET scan would you recommend for evaluating my lesion today?
3Because this was a Phase 1 feasibility study focused on testing whether these imaging and genomic methods were even workable, what do we actually know so far about how accurate FLT PET is at detecting MPNST in NF1 patients, and are there follow-up studies I should know about?
4Given that this trial was looking at how plexiform neurofibromas transform into malignant peripheral nerve sheath tumors, are there signs in my case that my doctor would want to monitor closely, and would genetic or genomic testing similar to what this study used be available to me outside of a trial?
5Are there other active clinical trials or standard monitoring approaches for NF1 patients at risk of MPNST that I should be considering now that this study is complete?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Evaluate the feasibility of whole-exome sequencing and other genetic/genomic methods
Timeframe: 3 years
2
Evaluate the ability of FLT PET to distinguish benign PN from malignant lesions, and to determine if FLT PET is more accurate than FDG PET in classifying a tumor as benign or malignant
Timeframe: 3 years
3
Determine the feasibility of FLT PET in patients with NF1 and lesions concerning for MPNST, or MPNST
. Diagnosis of NF1 with a lesion concerning for MPNST
Exclusion criteria
. Allergy or relative contraindications to MRI contrast agents.
. Patients who require sedation for imaging studies will be excluded from the FLT PET scan research test. They will undergo only the standard of care MRI and FDG PET scan.
. Contraindication to MRI scanning, such as surgery that involves metal clips or wires or metal prostheses which might be expected to cause tissue damage or produce image artifacts.
. Patients with severe chronic renal insufficiency (glomerular filtration rate \< 30 mL/min/1.73 m(2)), hepatorenal syndrome or post-liver transplantation.
. History of prior fluorothymidine allergy or intolerance.
. Participants with severe claustrophobia not relieved by oral anxiolytic medication or patients weighing \>136 kg (weight limit for scanner table)
. Pregnant women are excluded from this study because of the effects of radioactive materials with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with radioactive materials, breastfeeding should be discontinued.
. Requirement for medications, which interfere with platelet function, such as aspirin, which cannot be stopped within 1 week prior to the biopsy (applicable only to patients undergoing biopsy).