NeurofibromatosisPhase 1NCT02211768
Transformation of Plexiform Neurofibromas to Malignant Peripheral Nerve Sheath Tumors in Neurofibromatosis Type 1
A Phase 1 study testing MRI, FDG-PET/CT scans and [18F]-FLT-PET/CT scans in people with Neurofibromatosis and MPNST, run by National Cancer Institute (NCI) at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 10 years and older
- Conditions the study is for
- Neurofibromatosis, MPNST
- What is being tested?
- MRI, FDG-PET/CT scans and [18F]-FLT-PET/CT scans
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 8 Dec 2014to 30 Dec 2019
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 1. Age:
- No upper age limit for patient enrollment.
- FLT PET: will only be performed in patients greater than or equal to 10 years old
- Research biopsies in consenting patients with MPNST: will only be performed in patients greater than or equal to 18 years old
- 2. Diagnosis:
- Patients who are diagnosed with NF1 using the NIH Consensus Conference criteria or have a confirmed NF1 mutation with analysis performed in a CLIA certified laboratory. NF1 mutation testing to confirm eligibility will not be performed on this protocol, but as part the POB separate screening study.
Show all 32Show fewer
- For the clinical diagnosis of NF1 all study subjects must have at two or more diagnostic criteria for NF1 listed below (NIH Consensus Conference):
- 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
- 3. NFI tumor manifestations
- Subjects must have:
- Diagnosis of NF1 with a lesion concerning for MPNST
- -Criteria include pain, growth of a known plexiform neurofibroma, abnormality on functional imaging study (FDG-PET) or change in clinical exam.
- OR
- Diagnosis of NF1 with a histologically confirmed MPNST.
- 4. Subjects must be eligible for and willing to participate and sign consent for NCI protocol 08-C-0079: Natural History Study and Longitudinal Assessment of Children, Adolescents, and Adults with Neurofibromatosis Type 1, for the clinical evaluation necessary for this study.
- 5. Prior and current therapy:
- For NF1 related benign tumor manifestations there is no standard effective medical treatment, and surgery is the only standard treatment. Chemotherapy and radiation therapy are additional treatment options for malignant NF1 related tumors. For the purpose of this study subjects who have not previously received medical or surgical treatment, patients who have previously received medical or surgical treatment, and subjects who are currently receiving medical treatment and or radiation for a NF1 related manifestation will be eligible.
- Patients must be recovered from acute toxicities of prior therapy in order to be able to safely undergo biopsies proposed on the trial. Prior and current treatment for NF1 related manifestations will be recorded on protocol 08-C-0079.
- Prior radiation therapy and chemotherapy in patients with MPNST must not have been administered within 4 weeks prior to enrollment.
- 6. Performance Status:
- ECOG less than or equal to 3. Subjects who are wheelchair bound because of paralysis will be considered ambulatory when they are up in their wheelchair. Subjects have to be able to travel to the NIH for evaluations.
- 7. Informed Consent:
- All patients or their legal guardians (if the patients is<18 years old) must sign an IRB-approved document of informed consent to demonstrate their understanding of the investigational nature and the risks of this study before any protocol-related studies are performed. When appropriate, pediatric subjects will be included in all discussions.
- 8. Hematologic criteria (applicable only in patients undergoing biopsy)
- Platelet count has to be greater than or equal to 100,000/microL
- Patients should have INR<1.4 and PT less than or greater to 40 seconds (unless due to lupus anticoagulant). In patients not meeting these parameters, clearance by hematology will be required prior to undergoing biopsy.
Who cannot join
- 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).
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
About this study
Official title: Transformation of Plexiform Neurofibromas to Malignant Peripheral Nerve Sheath Tumors in Neurofibromatosis Type 1: Clinical, Histopathologic, and Genomic Analysis
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.
Read more
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.
Read the detailed description
Background:
- NF1 is an autosomal dominant genetic disorder characterized by distinct features including the development of benign plexiform neurofibromas (PN) and malignant peripheral nerve sheath tumors (MPNST) tumors of the nervous system.
- Development of MPNST typically results from malignant transformation in a preexisting PN. Associated symptoms may overlap and be difficult to distinguish from growth of a benign PN. Currently surgery is the only standard treatment for PN and MPNST.
- The 5-year overall survival rate for NF1 patients with a MPNST is poor; therefore, early detection of malignant transformation of a PN is an important goal.
- Fluoro-deoxy-glucose (FDG) positron emission tomography (PET) in NF1 has utility in detecting malignant transformation. However, concerning lesions can have high FDG uptake and be benign on biopsy.
- Fluoro-thymidine (FLT) PET measures cell cycling and proliferation. Malignant lesions have higher proliferation rates than benign tumors; therefore, FLT-PET may be sensitive and specific in the early detection of malignant transformation and assess response.
- Genetic analysis is an important component in evaluating the transformation of PN to MPNST. Biallelic NF1 and tumor suppressor gene mutations (p53, INK4A, p27kip1), increased Ras activity and abnormal growth factor signaling have been described, but there is no known signature for MPNST.
- Massively parallel ( next generation ) sequencing technology permits whole-genome, whole-exome and transcriptome sequencing of multiple tumors including MPNST.
Objectives:
- Determine the feasibility of FLT PET in patients with NF1 and MPNST or lesions concerning for MPNST, or MPNST.
- 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 correctly classifying a tumor as benign or malignant.
- Evaluate the feasibility of whole-exome sequencing and other genetic/genomic methods, including detection of epigenetic and/or expression changes and RNA Seq of tumor (MPNST or lesion concerning for MPNST and adjacent benign PN) biopsies using interventional radiology sampling techniques in consenting individuals with NF1 participating in 10-C-0086.
- Perform detailed clinical analysis of individuals with NF1 and MPNST or lesions concerning for MPNST.
- Perform detailed pathologic analysis of biopsy specimens from tumor areas to determine if increased uptake of FDG or FLT predicts for malignant transformation.
- Identify somatic genetic variants that distinguish PN from MPNST and from germline sequence by whole-exome sequencing potentially identifying targets for treatment.
Eligibility:
- NF1 patients with lesions concerning for malignancy or with active MPNST.
- Willingness to enroll on NCI protocol 08-C-0079: Natural History Study and Longitudinal Assessment of Children, Adolescents, and Adults with Neurofibromatosis Type 1.
Design:
- Up to 15 patients will be enrolled on this pilot study.
- Patients will undergo the following evaluations:
- Detailed clinical evaluation of NF1 manifestations on NCI protocol #08-C-0079
- Imaging studies including:
- MRI, FDG-PET/CT scans (as standard care) in all subjects; and
- [(18)F]-FLT-PET/CT (research study) in subjects 10 years of age or older
- Genetic counseling (if participating in the germline blood sampling and biopsy analysis portion of the study)
- Tissue analysis: ---Patients 18 years of age or older with MPNST will participate in tissue analysis, if consenting and appropriately preserved archival tissue is available, or if patient agrees to optional research biopsy (if consented and safe). Patients with lesions concerning for malignancy will undergo clinically indicated biopsies of concerning lesions and of adjacent benign PN (if consented and safe) for detailed pathologic analysis and whole-exome sequencing (co-enrollment on 10-C-0086 Comprehensive Omics Analysis of Pediatric Solid Tumors and Establishment of a Repository for Related Biological Studies). Biopsies to be directed by PET fusion imaging in interventional radiology.
- Whole-exome sequencing of a germline blood sample (optional) if participating in the tissue analysis (co-enrollment on 10-C-0086 Comprehensive Omics Analysis of Pediatric Solid Tumors and Establishment of a Repository for Related Biological Studies).
- To better characterize lesions concerning for MPNST and predict those at higher risk for malignant transformation, we will correlate clinical and imaging findings, including radiographic evaluation with FDG-PET/CT, and [(18)F]-FLT-PET/CT, pathologic evaluation of tumor biopsies (if available), and analysis of whole exome sequencing of germline blood samples (if consented) and of tumor samples (when available).
- Conditions
- Neurofibromatosis, MPNST
- Being tested
- MRI, FDG-PET/CT scans, [18F]-FLT-PET/CT scans
- Keywords
- Genetic Analysis, Biopsy, FDG-PET, FLT-PET
- Medical subject headings
- Neurofibromatoses, Neurofibrosarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MRI, FDG-PET/CT scansProcedure or surgery
Standard of care to be obtained as part of the study
[18F]-FLT-PET/CT scansDrug
FLT PET to be performed as a research imaging study as it is not considered standard imaging in NF1
| Study group | Role | What they receive |
|---|---|---|
| 1/FDG and FLT PET scans | Experimental | Subjects will undergo FDG-PET and FLT-PET scans at least one day apart Receives: Procedure: MRI, FDG-PET/CT scans; Drug: [18F]-FLT-PET/CT scans |
| 2/FDG-PET scan | Other | Subjects will undergo FDG-PET scan Receives: Procedure: MRI, FDG-PET/CT scans |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate the feasibility of whole-exome sequencing and other genetic/genomic methods
Genomic profile of subject's tumors to catalogue mutations associated with the development of a PN and then a MPNST
Measured: 3 years
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
Proportion of patients identified with benign or malignant tumor using FLT PET versus FDG PET
Measured: 3 years
Determine the feasibility of FLT PET in patients with NF1 and lesions concerning for MPNST, or MPNST
Sensitivity of FLT PET in distinguishing benign from malignant lesions
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Aug 2014 (estimated)
- Study start8 Dec 2014
- Primary completion5 Sep 2017
- Study completion30 Dec 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for NeurofibromatosisStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Brigitte C Widemann, M.D., Principal investigator, National Cancer Institute (NCI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02211768
- Sponsor's protocol ID
- 140163
- Other IDs
- 14-C-0163
- Status last verified
- 23 Jun 2026
- First submitted
- 6 Aug 2014
- Last updated
- 29 Oct 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: All IPD recorded in the medical record will be shared with intramural investigators upon request.
Publications
Benz MR, Czernin J, Dry SM, Tap WD, Allen-Auerbach MS, Elashoff D, Phelps ME, Weber WA, Eilber FC. Quantitative F18-fluorodeoxyglucose positron emission tomography accurately characterizes peripheral nerve sheath tumors as malignant or benign. Cancer. 2010 Jan 15;116(2):451-8. doi: 10.1002/cncr.24755.
PubMed 19924789Meany H, Dombi E, Reynolds J, Whatley M, Kurwa A, Tsokos M, Salzer W, Gillespie A, Baldwin A, Derdak J, Widemann B. 18-fluorodeoxyglucose-positron emission tomography (FDG-PET) evaluation of nodular lesions in patients with Neurofibromatosis type 1 and plexiform neurofibromas (PN) or malignant peripheral nerve sheath tumors (MPNST). Pediatr Blood Cancer. 2013 Jan;60(1):59-64. doi: 10.1002/pbc.24212. Epub 2012 May 29.
PubMed 22645095Salskov A, Tammisetti VS, Grierson J, Vesselle H. FLT: measuring tumor cell proliferation in vivo with positron emission tomography and 3'-deoxy-3'-[18F]fluorothymidine. Semin Nucl Med. 2007 Nov;37(6):429-39. doi: 10.1053/j.semnuclmed.2007.08.001.
PubMed 17920350
Study at a glance
From the registry record, last updated by the sponsor on 29 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Transformation of Plexiform Neurofibromas to Malignant Peripheral Nerve Sheath Tumors in Neurofibromatosis Type 1: Clinical, Histopathologic, and Genomic Analysis
- ClinicalTrials.gov ID
- NCT02211768
- Acronym / sponsor ID
- 140163
- Conditions
- Neurofibromatosis, MPNST
- Keywords
- Genetic Analysis, Biopsy, FDG-PET, FLT-PET
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- 8 Dec 2014 (actual)
- Primary completion
- 5 Sep 2017 (actual)
- Study completion
- 30 Dec 2019 (actual)
- First posted
- 7 Aug 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Background:
- NF1 is an autosomal dominant genetic disorder characterized by distinct features including the development of benign plexiform neurofibromas (PN) and malignant peripheral nerve sheath tumors (MPNST) tumors of the nervous system.
- Development of MPNST typically results from malignant transformation in a preexisting PN. Associated symptoms may overlap and be difficult to distinguish from growth of a benign PN. Currently surgery is the only standard treatment for PN and MPNST.
- The 5-year overall survival rate for NF1 patients with a MPNST is poor; therefore, early detection of malignant transformation of a PN is an important goal.
- Fluoro-deoxy-glucose (FDG) positron emission tomography (PET) in NF1 has utility in detecting malignant transformation. However, concerning lesions can have high FDG uptake and be benign on biopsy.
- Fluoro-thymidine (FLT) PET measures cell cycling and proliferation. Malignant lesions have higher proliferation rates than benign tumors; therefore, FLT-PET may be sensitive and specific in the early detection of malignant transformation and assess response.
- Genetic analysis is an important component in evaluating the transformation of PN to MPNST. Biallelic NF1 and tumor suppressor gene mutations (p53, INK4A, p27kip1), increased Ras activity and abnormal growth factor signaling have been described, but there is no known signature for MPNST.
- Massively parallel ( next generation ) sequencing technology permits whole-genome, whole-exome and transcriptome sequencing of multiple tumors including MPNST.
Objectives:
- Determine the feasibility of FLT PET in patients with NF1 and MPNST or lesions concerning for MPNST, or MPNST.
- 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 correctly classifying a tumor as benign or malignant.
- Evaluate the feasibility of whole-exome sequencing and other genetic/genomic methods, including detection of epigenetic and/or expression changes and RNA Seq of tumor (MPNST or lesion concerning for MPNST and adjacent benign PN) biopsies using interventional radiology sampling techniques in consenting individuals with NF1 participating in 10-C-0086.
- Perform detailed clinical analysis of individuals with NF1 and MPNST or lesions concerning for MPNST.
- Perform detailed pathologic analysis of biopsy specimens from tumor areas to determine if increased uptake of FDG or FLT predicts for malignant transformation.
- Identify somatic genetic variants that distinguish PN from MPNST and from germline sequence by whole-exome sequencing potentially identifying targets for treatment.
Eligibility:
- NF1 patients with lesions concerning for malignancy or with active MPNST.
- Willingness to enroll on NCI protocol 08-C-0079: Natural History Study and Longitudinal Assessment of Children, Adolescents, and Adults with Neurofibromatosis Type 1.
Design:
- Up to 15 patients will be enrolled on this pilot study.
- Patients will undergo the following evaluations:
- Detailed clinical evaluation of NF1 manifestations on NCI protocol #08-C-0079
- Imaging studies including:
- MRI, FDG-PET/CT scans (as standard care) in all subjects; and
- [(18)F]-FLT-PET/CT (research study) in subjects 10 years of age or older
- Genetic counseling (if participating in the germline blood sampling and biopsy analysis portion of the study)
- Tissue analysis: ---Patients 18 years of age or older with MPNST will participate in tissue analysis, if consenting and appropriately preserved archival tissue is available, or if patient agrees to optional research biopsy (if consented and safe). Patients with lesions concerning for malignancy will undergo clinically indicated biopsies of concerning lesions and of adjacent benign PN (if consented and safe) for detailed pathologic analysis and whole-exome sequencing (co-enrollment on 10-C-0086 Comprehensive Omics Analysis of Pediatric Solid Tumors and Establishment of a Repository for Related Biological Studies). Biopsies to be directed by PET fusion imaging in interventional radiology.
- Whole-exome sequencing of a germline blood sample (optional) if participating in the tissue analysis (co-enrollment on 10-C-0086 Comprehensive Omics Analysis of Pediatric Solid Tumors and Establishment of a Repository for Related Biological Studies).
- To better characterize lesions concerning for MPNST and predict those at higher risk for malignant transformation, we will correlate clinical and imaging findings, including radiographic evaluation with FDG-PET/CT, and [(18)F]-FLT-PET/CT, pathologic evaluation of tumor biopsies (if available), and analysis of whole exome sequencing of germline blood samples (if consented) and of tumor samples (when available).
Study design
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1/FDG and FLT PET scans | Experimental | Procedure: MRI, FDG-PET/CT scans, Drug: [18F]-FLT-PET/CT scansSubjects will undergo FDG-PET and FLT-PET scans at least one day apart |
| 2/FDG-PET scan | Other | Procedure: MRI, FDG-PET/CT scansSubjects will undergo FDG-PET scan |
Who may qualify
- Ages
- 10 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (32 listed)
- No upper age limit for patient enrollment.
- Patients who are diagnosed with NF1 using the NIH Consensus Conference criteria or have a confirmed NF1 mutation with analysis performed in a CLIA certified laboratory. NF1 mutation testing to confirm eligibility will not be performed on this protocol, but as part the POB separate screening study.
- For the clinical diagnosis of NF1 all study subjects must have at two or more diagnostic criteria for NF1 listed below (NIH Consensus Conference):
- 3. NFI tumor manifestations
- Diagnosis of NF1 with a lesion concerning for MPNST
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MRI, FDG-PET/CT scansStandard of care to be obtained as part of the study | Procedure or surgery | 1/FDG and FLT PET scans, 2/FDG-PET scan |
| [18F]-FLT-PET/CT scansFLT PET to be performed as a research imaging study as it is not considered standard imaging in NF1 | Drug | 1/FDG and FLT PET scans |
Primary outcome
Evaluate the feasibility of whole-exome sequencing and other genetic/genomic methods
Time frame: 3 years
Genomic profile of subject's tumors to catalogue mutations associated with the development of a PN and then a MPNST
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
Time frame: 3 years
Proportion of patients identified with benign or malignant tumor using FLT PET versus FDG PET
Determine the feasibility of FLT PET in patients with NF1 and lesions concerning for MPNST, or MPNST
Time frame: 3 years
Sensitivity of FLT PET in distinguishing benign from malignant lesions
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Oct 2026.
- Background: PubMed 19924789Benz MR, Czernin J, Dry SM, Tap WD, Allen-Auerbach MS, Elashoff D, Phelps ME, Weber WA, Eilber FC. Quantitative F18-fluorodeoxyglucose positron emission tomography accurately characterizes peripheral nerve sheath tumors as malignant or benign. Cancer. 2010 Jan 15;116(2):451-8. doi: 10.1002/cncr.24755.
- Background: PubMed 22645095Meany H, Dombi E, Reynolds J, Whatley M, Kurwa A, Tsokos M, Salzer W, Gillespie A, Baldwin A, Derdak J, Widemann B. 18-fluorodeoxyglucose-positron emission tomography (FDG-PET) evaluation of nodular lesions in patients with Neurofibromatosis type 1 and plexiform neurofibromas (PN) or malignant peripheral nerve sheath tumors (MPNST). Pediatr Blood Cancer. 2013 Jan;60(1):59-64. doi: 10.1002/pbc.24212. Epub 2012 May 29.
- Background: PubMed 17920350Salskov A, Tammisetti VS, Grierson J, Vesselle H. FLT: measuring tumor cell proliferation in vivo with positron emission tomography and 3'-deoxy-3'-[18F]fluorothymidine. Semin Nucl Med. 2007 Nov;37(6):429-39. doi: 10.1053/j.semnuclmed.2007.08.001.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. All IPD recorded in the medical record will be shared with intramural investigators upon request.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Brigitte C Widemann, M.D. principal investigator
National Cancer Institute (NCI)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing MRI, FDG-PET/CT scans and [18F]-FLT-PET/CT scans in people with Neurofibromatosis and MPNST, run by National Cancer Institute (NCI) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 32 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 29 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluate the feasibility of whole-exome sequencing and other genetic/genomic methods (time frame: 3 years). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02211768, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Oct 2026.