Neurofibromatosis Type 1NCT00060008

Fludeoxyglucose F 18 Positron Emission Tomography and Magnetic Resonance Perfusion Imaging in Patients With Neurofibromatosis 1 and Plexiform Neurofibroma

A study testing fludeoxyglucose F 18 and gadopentetate dimeglumine in people with Neurofibromatosis Type 1 and Precancerous Condition, run by Children's Hospital of Philadelphia at 2 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

RATIONALE: New imaging procedures such as fludeoxyglucose F 18 positron emission tomography (FDG-PET) and magnetic resonance (MR) perfusion imaging may improve the ability to detect disease progression, help doctors predict a patient's response to treatment, and help plan the most effective treatment. Read more about this study

Who is it for?: Ages Up to 25 years
Conditions the study is for: Neurofibromatosis Type 1, Precancerous Condition
What is being tested?: fludeoxyglucose F 18 and gadopentetate dimeglumine
Type of treatment: Surgery or procedure
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 18 participants
Where: 2 sitesUnited States
Study dates: Apr 2002to May 2011
Phase: Interventional
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 Up to 25 years
All sexes
No healthy volunteers
Child, Adult

DISEASE CHARACTERISTICS:

Stratum 1:
Diagnosis of neurofibromatosis 1 (NF1) and plexiform neurofibromas
At high risk for progression, as defined by any of the following:
Anatomic location such that progression carries a high risk of impairment of function, pain, or disfigurement (e.g., neck/mediastinum, paraspinal nerve roots, orbit, and face)
Tumors that the patient, family, or caregiver believes have increased in size within the past year, but appear stable by standard clinical or radiographic measures
No plexiform neurofibromas that are small, cause no pain or functional impairment, or are not likely to cause pain or functional impairment over the succeeding 12 months
Stratum 2:
Diagnosis of NF1 and progressive plexiform neurofibromas
Neurofibroma progression documented by increase in lesion size on MRI
Currently being enrolled on a clinical therapeutic trial at Children's Hospital of Philadelphia

PATIENT CHARACTERISTICS:

Age

25 and under

Performance status

Not specified

Life expectancy

Not specified

Hematopoietic

Not specified

Hepatic

Not specified

Renal

Not specified

Other

Not pregnant or nursing
Negative pregnancy test

PRIOR CONCURRENT THERAPY:

Biologic therapy

Not specified

Chemotherapy

Stratum 1:
No prior or concurrent chemotherapy
No concurrent enrollment on a chemotherapy clinical trial
Stratum 2:
At least 4 weeks since prior chemotherapy

Endocrine therapy

Not specified

Radiotherapy

At least 6 weeks since prior radiotherapy (stratum 2)

Surgery

Prior surgery for progressive plexiform neurofibroma allowed if incompletely resected and measurable disease remains (stratum 2)

Trial locations and contacts

2 sites in 1 country.

United States2 sites

Abramson Cancer Center of the University of Pennsylvania
Philadelphia, Pennsylvania, 19104-4283
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104

Other trials in United States

About this study

Official title: Novel Imaging Modalities For Plexiform Neurofibromas

PURPOSE: This diagnostic trial is studying how well FDG-PET and MR perfusion imaging work in finding disease progression and determining response to treatment in patients with neurofibromatosis 1 and plexiform neurofibroma.

Read the detailed description

OBJECTIVES:

Determine whether fludeoxyglucose F 18 positron emission tomography (FDG-PET) and MR perfusion studies can predict plexiform neurofibroma growth rates in patients with neurofibromatosis 1.
Determine whether FDG-PET and MR perfusion studies can predict the likelihood of response in patients who are undergoing investigational treatment for plexiform neurofibromas.
Identify neuroimaging characteristics that distinguish patients who have responded to therapy from those who have not after completion of treatment.

OUTLINE:

Stratum 1: Patients undergo MR perfusion scan with gadopentetate dimeglumine and fludeoxyglucose F 18 positron emission tomography (FDG-PET) at baseline and quantitative MRI evaluation at baseline and 1 year.
Stratum 2: Patients undergo quantitative MRI, MR perfusion scan with gadopentetate dimeglumine, and FDG-PET at baseline and 1 year.

PROJECTED ACCRUAL: A total of 48 patients (32 for stratum 1 and 16 for stratum 2) will be accrued for this study.

Conditions: Neurofibromatosis Type 1, Precancerous Condition
Being tested: fludeoxyglucose F 18, gadopentetate dimeglumine
Keywords: neurofibromatosis type 1, plexiform neurofibroma
Medical subject headings: Neurofibromatosis 1, Precancerous Conditions, Neurofibroma, Plexiform

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

fludeoxyglucose F 18Radiation
gadopentetate dimeglumineRadiation

Study groups in NCT00060008
Study group	Role	What they receive
18FDG-PET scan and MR perfusion	Experimental	Subjects will undergo MRI for quantitative (2D and 3D) evaluation of plexiform neurofibroma size, MR perfusion scan, and fludeoxyglucose (18FDG) PET scan at the time of study entry. Subjects who are treated for plexiform neurofibroma will undergo another 18FDG PET scan after one year of study entry. Receives: Radiation: fludeoxyglucose F 18; Radiation: gadopentetate dimeglumine

What is being measured

The primary outcome is the main question the study is designed to answer.

Tumor Progression as Measured by Tumor Area and Volume at 1 Year.
We correlated SUVmax and change in tumor volume over the subsequent year
Measured: One year

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Apr 2002May 2011

First posted7 May 2003 (estimated)
Study startApr 2002
Primary completionMay 2011
Study completionMay 2011

Why it stopped: Study was closed early due to poor accrual.

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Neurofibromatosis Type 1Studies of the same condition, open and under way.
More trials run by Children's Hospital of PhiladelphiaThe 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 N/A
Primary purpose: Diagnosis
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 18 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00060008
Sponsor's protocol ID: 2001-8-2543
Other IDs: CHP-724, CDR0000299006
Status last verified: Apr 2014
First submitted: 6 May 2003
Last updated: 4 Apr 2014
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Neurofibromatosis Type 1 trials

Study at a glance

From the registry record, last updated by the sponsor on 4 Apr 2014. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Novel Imaging Modalities For Plexiform Neurofibromas
ClinicalTrials.gov ID: NCT00060008
Acronym / sponsor ID: 2001-8-2543
Conditions: Neurofibromatosis Type 1, Precancerous Condition
Keywords: neurofibromatosis type 1, plexiform neurofibroma
Study type: Interventional
Recruitment status: TerminatedReason stopped: Study was closed early due to poor accrual.
Enrolment: 18 participants (actual)
Lead sponsor: Children's Hospital of PhiladelphiaUniversity, hospital or other
Study start: Apr 2002
Primary completion: May 2011 (actual)
Study completion: May 2011 (actual)
First posted: 7 May 2003 (estimated)
Results: Posted 4 Apr 2014

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

OBJECTIVES:

Determine whether fludeoxyglucose F 18 positron emission tomography (FDG-PET) and MR perfusion studies can predict plexiform neurofibroma growth rates in patients with neurofibromatosis 1.
Determine whether FDG-PET and MR perfusion studies can predict the likelihood of response in patients who are undergoing investigational treatment for plexiform neurofibromas.
Identify neuroimaging characteristics that distinguish patients who have responded to therapy from those who have not after completion of treatment.

OUTLINE:

Stratum 1: Patients undergo MR perfusion scan with gadopentetate dimeglumine and fludeoxyglucose F 18 positron emission tomography (FDG-PET) at baseline and quantitative MRI evaluation at baseline and 1 year.
Stratum 2: Patients undergo quantitative MRI, MR perfusion scan with gadopentetate dimeglumine, and FDG-PET at baseline and 1 year.

PROJECTED ACCRUAL: A total of 48 patients (32 for stratum 1 and 16 for stratum 2) will be accrued for this study.

Study design

Primary purpose: Diagnosis
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
18FDG-PET scan and MR perfusion	Experimental	Radiation: fludeoxyglucose F 18, Radiation: gadopentetate dimeglumineSubjects will undergo MRI for quantitative (2D and 3D) evaluation of plexiform neurofibroma size, MR perfusion scan, and fludeoxyglucose (18FDG) PET scan at the time of study entry. Subjects who are treated for plexiform neurofibroma will undergo another 18FDG PET scan after one year of study entry.

Who may qualify

Ages: Up to 25 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

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
fludeoxyglucose F 18	Radiation	18FDG-PET scan and MR perfusion
gadopentetate dimeglumine	Radiation	18FDG-PET scan and MR perfusion

Primary outcome

Tumor Progression as Measured by Tumor Area and Volume at 1 Year.
Time frame: One year
We correlated SUVmax and change in tumor volume over the subsequent year

Results and safety

Results posted to the registry on 4 Apr 2014, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
18FDG-PET Scan and MR Perfusion	18	18

Primary outcome: Tumor Progression as Measured by Tumor Area and Volume at 1 Year.

Time frame: One year. Median (Full Range). Unit: percentage of change

Measure	SUVmax < 2 n = 11	SUVmax >2 n = 4
Value	4 (-0.6 to 63)	27 (16 to 32)

SUVmax < 2 vs SUVmax >2: p = 0.016, Wilcoxon (Mann-Whitney)

Adverse events

Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
18FDG-PET Scan and MR Perfusion	0 of 0	0 of 0	Not reported

Limitations and caveats (from the sponsor): The sample size was modest. It is possible that with increased sample size, if the correlation estimate holds, it will become statistically significant.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 4 Apr 2014.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Neurofibromatosis Type 1

Active, not recruitingPhase 1/2
Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma
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RecruitingPhase 3
A Study of the Drugs Selumetinib vs. Carboplatin and Vincristine in Patients With Low-Grade Glioma
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RecruitingPhase 1
Phase I Trial of TURALIO(R) (Pexidartinib, PLX3397) in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN) and Tenosynovial Giant Cell Tumor ...
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Active, not recruitingPhase 2
Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
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RecruitingObservational study
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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.

Michael Fisher, MD study chair

Children's Hospital of Philadelphia

2 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

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: stopped early, as of the sponsor's last update on 4 Apr 2014. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites 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.

Are results available for this study?

Yes. Results are posted in the registry (first posted 4 Apr 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Tumor Progression as Measured by Tumor Area and Volume at 1 Year. (time frame: One year). 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 NCT00060008.

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 4 Apr 2014.