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Treatment to Slow Tumor Progression in Children with Neurofibromatosis Type 1
Untitled Document
Name of the Trial
Phase II Randomized Study of Tipifarnib in Pediatric Patients With Neurofibromatosis
Type I and Progressive Plexiform Neurofibromas (NCI-01-C-0222H). See
the protocol summary.
Principal Investigator
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Dr. Brigitte Widemann
Principal Investigator |
Dr. Brigitte Widemann, NCI Center for Cancer Research
Why is This Trial Important?
Neurofibromatosis Type 1 (NF1), one of the most common genetic disorders, affects
1 in every 3,500 individuals. NF1 is caused by changes in the gene that contains
the instructions for making a protein called neurofibromin, which helps control
tissue growth.
People with NF1 have an increased risk of developing benign and malignant tumors
of the nervous system. About 25 percent of individuals with NF1 develop plexiform
neurofibromas (PNs), a benign tumor arising from the outer layer of nerves.
Although benign, PNs involve multiple nerve branches and can grow to a very
large size, causing pain, functional impairment, and even life-threatening complications.
In this phase II trial, PN tumor growth rates will be measured during treatment
with the drug tipifarnib and a placebo. Tipifarnib belongs to a family of molecularly
targeted agents called farnesyltransferase inhibitors, which can inactivate
certain proteins that promote tumor growth. The researchers hope that tipifarnib
will delay or interrupt PN growth.
"We decided to focus on PNs because there is no effective drug treatment
for these tumors," said Dr. Widemann. "Complete surgical resection
is the only standard treatment, but it isn't a viable option for most patients
because PNs tend be diffuse, invasive, and intertwined with vital structures
like major blood vessels or nerves. It's virtually impossible to remove the
PN completely."
Who Can Join This Trial?
Researchers seek to enroll 60 patients aged 3 to 25 who have been diagnosed
with NF1 and have measurable progressive PNs. See
the complete list of eligibility criteria.
Where Is This Trial Taking Place?
Multiple study sites across the United States are enrolling patients. See
the list of study sites.
Contact Information
See the list
of study contacts or call NCI's Cancer Information Service at 1-800-4-CANCER
(1-800-422-6237). The call is toll-free and confidential.
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