Effect of Vorinostat on Nervous System Hemangioblastomas in Von Hippel-Lindau Disease (Missense Mutation Only)

Part of paid clinical trials in Bethesda, Maryland.

Sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
Study ID
NCT02108002
Phase
PHASE1
Status
Completed

Conditions

  • Von Hippel-Lindau Disease

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

Study Details

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.

Key Dates

Start date
Apr 5, 2014
Status verified
Sep 2018
Primary completion
Sep 12, 2018
Completion
Sep 12, 2018

Study Design

Enrollment
7 participants (actual)
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
DIAGNOSTIC

Primary Outcome Measure

The presence and quantity of mutant VHL protein in resected hemangioblastoma specimens, including comparison of specimens without vorinostat treatment and those with presurgical vorinostat treatment. [ Time Frame: ongoing ]

Locations (1)

FacilityCityStateZIPSite coordinators
National Institutes of Health Clinical Center, 9000 Rockville PikeBethesdaMaryland20892-

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National Institutes of Health Clinical Center, 9000 Rockville Pike· Bethesda, MD

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