Treatment to Slow Tumor Progression in Children with Neurofibromatosis Type 1
Name of the Trial
Why Is This Trial Important?
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), benign tumors 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?
Where Is This Trial Taking Place?
Whom to Contact
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