Research OpportunityTitle: Phase I Trial of the Raf kinase and receptor Tyrosine Kinase Inhibitor Sorafineb (BAY 43-9006, Nexavar®) in Children and Young Adults with Neurofibromatosis Type 1 and Inoperable Plexiform Neurofibromas
The University of Alabama at Birmingham (UAB) is launching a clinical trial for qualified individuals with a diagnosis of neurofibromatosis type 1 (NF1).
Neurofibromatosis type 1 is associated with an increased risk of developing tumors of the central and peripheral nervous system. Between 20 and 44% of patients with NF1 develop plexiform neurofibromas. These tumors can cause significant disfigurement and compression of vital structures within the body. Currently, there is no standard treatment for large or complex plexiform neurofibromas except for surgery. However, surgery is often not a permanent solution for these tumors, and no effective drug therapy exists.
This is a research study that involves a medication called sorafinib, which is taken as an oral pill. Participation involves taking the medication and then having regular monitoring through blood work and MRIs. Among the goals of this phase I research study are (1) to determine what kind of side effects daily dosing of sorafenib causes in children and young adults with NF1 and plexiform neurofibromas; (2) to determine the highest dose of sorafenib that can be safely given to children and young adults with NF1 and plexiform neurofibromas; (3) to study how the body handles the drug by measuring the amount of drug in the bloodstream over time; (4) to determine whether sorafenib is helpful in the treatment of plexiform neurofibromas (causes the tumors to shrink or slows down their growth); and (5) to determine the effects of sorafenib on learning, attention, memory, and quality of life. Approximately 15 to 21 patients may take part in this study; the number of patients who may be enrolled on this study will depend on when the highest safe dose is found.
- Must have a diagnosis of neurofibromatosis type 1 (NF1)*
- Must be between 3 and 18 years of age
- Must have a plexiform (deep tissue) neurofibroma that either is growing bigger or is large enough to be medically significant
If you think you may be eligible for this study and you would like to talk to someone in person about your interest in qualifying for or participating in this research, please contact us by phone or e-mail.
University of Alabama at Birmingham
Contact: Christina Barger, M.S., CGC (Clinic Coordinator)
*This trial is specific to patients with neurofibromatosis type 1. There has been no evidence that individuals with neurofibromatosis type 2 or schwannomatosis would benefit from this clinical trial.