Treatment to Slow Tumor Progression in Children with Neurofibromatosis Type 1
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.
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.
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.