Von Hippel-Lindau Disease Clinical Trial
Official title:
Pilot Study of the Effect of Vorinostat on Nervous System Hemangioblastomas In Von Hippel-Lindau Disease
Verified date | September 12, 2018 |
Source | National Institutes of Health Clinical Center (CC) |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 7 |
Est. completion date | September 12, 2018 |
Est. primary completion date | September 12, 2018 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility |
- 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. |
Country | Name | City | State |
---|---|---|---|
United States | National Institutes of Health Clinical Center, 9000 Rockville Pike | Bethesda | Maryland |
Lead Sponsor | Collaborator |
---|---|
National Institute of Neurological Disorders and Stroke (NINDS) |
United States,
Friedrich CA. Von Hippel-Lindau syndrome. A pleomorphic condition. Cancer. 1999 Dec 1;86(11 Suppl):2478-82. Review. — View Citation
Harries RW. A rational approach to radiological screening in von Hippel-Lindau disease. J Med Screen. 1994 Apr;1(2):88-95. — View Citation
Maher ER, Willatt L, Cuthbert G, Chapman C, Hodgson SV. Three cases of 16q duplication. J Med Genet. 1991 Nov;28(11):801-2. Erratum in: J Med Genet 1992 Feb;29(2):133. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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. | ongoing |
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