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: 0
• Participant gender: All

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
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: National Institute of Neurological Disorders and Stroke (NINDS)
• Treatments: Vorinostat

Criteria

• INCLUSION CRITERIA

1. Adult patients (age greater than or equal to 18 years)

2. Known VHL disease arising from a missense mutation.

3. Demonstrated clinical progression of CNS hemangioblastoma.

4. Enrolled in 03-N-0164, Evaluation of Neurosurgical Disorders.

5. Able to provide written informed consent.

EXCLUSION CRITERIA

1. Patients who have been previously treated with vorinostat.

2. Significant medical illnesses that in the investigator s opinion cannot be adequately controlled with appropriate therapy or would compromise the patient s ability to tolerate this therapy.

3. History of a second cancer (except non-melanoma skin cancer or carcinoma in-situ of the cervix), unless in complete remission and off of all therapy for that disease for a minimum of 3 years.

4. Active infection or serious concurrent medical illness.

5. Pregnancy and breast-feeding.

6. Presence of any disease that will obscure toxicity or dangerously alter drug metabolism (such as uncontrolled diabetes, liver disease, bleeding disorder)

7. Currently receiving other investigational agents.

8. History of allergic reactions attributed to compounds of similar chemical or biologic composition to vorinostat, such as valproate.

9. Currently taking another HDACi, such as valproate.