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

• Sponsor: Children's Hospital of Philadelphia (Other)
• Overall Status: Terminated
• CT.gov ID: NCT00060008
• Collaborator: (none)
• Enrollment: 18
• Locations: 2
• Arm: 1
• Duration (Months): 109
• Patients Per Site: 9
• Patients Per Site Per Month: 0.1

Study Details

Study Description

Brief 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.

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.

Condition or Disease	Intervention/Treatment	Phase
Neurofibromatosis Type 1; Precancerous Condition	Radiation: fludeoxyglucose F 18; Radiation: gadopentetate dimeglumine	N/A

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

• Study Type: Interventional
• Actual Enrollment: 18 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Diagnostic
• Official Title: Novel Imaging Modalities For Plexiform Neurofibromas
• Study Start Date: Apr 1, 2002
• Actual Primary Completion Date: May 1, 2011
• Actual Study Completion Date: May 1, 2011

Arms and Interventions

Arm	Intervention/Treatment
Experimental: 18FDG-PET scan and MR perfusion 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.	Radiation: fludeoxyglucose F 18; Radiation: gadopentetate dimeglumine

Outcome Measures

Primary Outcome Measures

1. Tumor Progression as Measured by Tumor Area and Volume at 1 Year. [One year]

   We correlated SUVmax and change in tumor volume over the subsequent year

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A to 25 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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)

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Abramson Cancer Center of the University of Pennsylvania	Philadelphia	Pennsylvania	United States	19104-4283
2	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania	United States	19104

Sponsors and Collaborators

• Children's Hospital of Philadelphia

Investigators

• Study Chair: Michael Fisher, MD, Children's Hospital of Philadelphia

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Children's Hospital of Philadelphia
• ClinicalTrials.gov Identifier: NCT00060008
• Other Study ID Numbers: 2001-8-2543; CHP-724; CDR0000299006
• First Posted: May 7, 2003
• Last Update Posted: Apr 4, 2014
• Last Verified: Apr 1, 2014

• Keywords provided by Children's Hospital of Philadelphia: neurofibromatosis type 1; plexiform neurofibroma
• Additional relevant MeSH terms: Neurofibromatoses; Neurofibromatosis 1; Neurofibroma; Precancerous Conditions; Neurofibroma, Plexiform; Fluorodeoxyglucose F18

Study Results

Participant Flow

Arm/Group Title	18FDG-PET Scan and MR Perfusion
Arm/Group Description	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. fludeoxyglucose F 18 gadopentetate dimeglumine
Period Title: Overall Study
STARTED	18
COMPLETED	18
NOT COMPLETED	0

Baseline Characteristics

Arm/Group Title	18FDG-PET Scan and MR Perfusion
Arm/Group Description	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. fludeoxyglucose F 18 gadopentetate dimeglumine
Overall Participants	18
Age, Customized (years) [Mean (Full Range) ]
Mean (Full Range) [years]	14.4
Sex: Female, Male (Count of Participants)
Female	9 50%
Male	9 50%

Outcome Measures

1. Primary Outcome

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

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	SUVmax < 2	SUVmax >2
Arm/Group Description	Subjects underwent MRI for quantitative (3D) evaluation of plexiform neurofibroma size and fludeoxyglucose (18FDG) PET scan at the time of study entry.	Subjects underwent MRI for quantitative (3D) evaluation of plexiform neurofibroma size and fludeoxyglucose (18FDG) PET scan at the time of study entry.
Measure Participants	11	4
Measure tumors	15	4
Median (Full Range) [percentage of change]	4	27

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	SUVmax < 2, SUVmax >2
	Comments
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.016
	Comments
	Method	Wilcoxon (Mann-Whitney)
	Comments

Adverse Events

Adverse Event Reporting Description	Adverse Events were not collected for this study.
Arm/Group Title	18FDG-PET Scan and MR Perfusion
Arm/Group Description	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. fludeoxyglucose F 18 gadopentetate dimeglumine
All Cause Mortality
	18FDG-PET Scan and MR Perfusion
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	18FDG-PET Scan and MR Perfusion
	Affected / at Risk (%)	# Events
Total	0/0 (NaN)
Other (Not Including Serious) Adverse Events
	18FDG-PET Scan and MR Perfusion
	Affected / at Risk (%)	# Events
Total	0/0 (NaN)

Limitations/Caveats

The sample size was modest. It is possible that with increased sample size, if the correlation estimate holds, it will become statistically significant.

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.