Effect of Vorinostat on Nervous System Hemangioblastomas in Von Hippel-Lindau Disease (Missense Mutation Only)
Status:
Completed
Trial end date:
2018-09-12
Target enrollment:
Participant gender:
Summary
Background:
- Von Hippel-Lindau (VHL) disease is a rare gene disease. People with VHL often have a brain
tumor called hemangioblastoma. Standard treatment for these tumors is risky surgery.
Researchers want to find new ways to treat people who have the tumors. They want to see if a
drug that fights other cancers might slow the growth of hemangioblastomas in some people with
VHL. Some people with VHL have mutations that make abnormal proteins. Tumors form in such
people because the abnormal protein is broken down quickly. The cancer drug may work in these
tumors by preventing breakdown of protein.
Objective:
- To study how the drug vorinostat affects hemangioblastomas in people with VHL.
Eligibility:
- Adults at least 18 old with hemangioblastomas from VHL.
Design:
- Participants must already be in study 03-N-0164. They must have tumor surgery scheduled.
- Participants must stop taking most medications 14 days before surgery.
- One week before surgery, participants will enter the hospital. They will be screened
with medical history and physical and neurological exams. They will give blood and urine
samples. Participants will have an electrocardiogram. For this test, small sticky
patches are put on the arms, legs, and chest. Participants will lie still for a few
minutes while a machine records heart rate and rhythm.
- Participants will take one vorinostat by mouth each day for 7 days.
- Participants will have blood drawn during the week to check for any side effects.
- Participants will have their tumor removed in surgery. Researchers will study the tumor
tissue for the effects of the study drug.
- A nurse will call participants 1 month after surgery to check for side effects.
Phase:
Phase 1
Details
Lead Sponsor:
National Institute of Neurological Disorders and Stroke (NINDS)