This trial is active, not recruiting.

Conditions hemangioblastoma, hippel lindau disease
Sponsor National Institute of Neurological Disorders and Stroke (NINDS)
Start date June 2000
Trial size 250 participants
Trial identifier NCT00005902, 00-N-0140, 000140


The purpose of this study is to learn more about the growth of brain and spinal cord tumors and cysts that develop in association with them in patients with von Hippel-Lindau disease. It will examine how fast the tumors grow and try to determine what factors (for example, puberty , pregnancy, menopause, blood proteins, etc.) affect their growth.

Patients between the ages of 8 and 75 years who are enrolled in NIH s study of von Hippel-Lindau disease may be eligible for this 5-year study. Participants will have magnetic resonance imaging (MRI) of the brain and spinal cord and a thorough neurological history and examination at the start of the study. A blood sample will be taken for analysis of factors (hormones or other proteins) that may predict tumor growth. Follow-up clinic visits every 6 months will include a physical and neurological examination, blood tests, and MRI scans of the brain and spine. If symptoms or tumor growth requires more frequent follow-up, scans will be done at 3-month intervals.

Surgical removal of brain and spinal cord tumors is currently the treatment of choice when these lesions cause neurological problems. A better understanding of which tumors are likely to grow and which will remain stable may help guide physicians in treatment decisions and avoid unnecessary procedures.

United States No locations recruiting
Other countries No locations recruiting

Eligibility Criteria

Male or female participants from 8 years up to 75 years old.

- INCLUSION CRITERIA: 1. Ages 8-75. 2. Confirmed diagnosis of von Hippel-Lindau disease. 3. Presence of one or more cerebral, cerebellar or spinal cord hemangioblastomas on screening MRI. 4. Provide written informed consent (or assent). 5. Karnofsky Performance Scale Score greater than or equal to 60. EXCLUSION CRITERIA: 1. Clinically unstable condition. 2. Being treated by any chemotherapy, immunotherapy, or steroids. 3. Allergy to CT or MRI contrast agents. 4. Contraindication to MRI scanning such as surgery that involves metal clips or wires which might be expected to cause tissue damage or produce image artifacts.

Additional Information

Official title A Prospective Natural History Study of VHL Patients With CNS Hemangioblastomas
Principal investigator John D Heiss, M.D.
Description Hemangioblastomas of the cerebellum, brainstem and spinal cord are frequent tumors in patients with von Hippel-Lindau (VHL) disease. Rarely, these lesions also affect the cerebrum. Patients often have multiple lesions, many of which are associated with cysts or syrinx. The current treatment for symptomatic lesions is surgical resection. Focused radiation is also being used in selected cases in an attempt to provide tumor control. The natural history of central nervous system (CNS) lesions in patients with VHL has not been addressed in a prospective study. It is not clear at which point these lesions will begin to grow, or develop cysts in the cerebellum or syrinx in the spinal cord, and systemic factors that influence tumor growth have not been identified. By identifying factors that predict or influence tumor progression or cyst development, we can more accurately recommend surgical or medical intervention at appropriate times and avoid unnecessary treatment for stable lesions. This study will collect prospective radiological and clinical data on growth of the central nervous system (CNS) hemangioblastomas and associated cysts. We will also prospectively collect information on systemic processes that may influence tumor progression, such as puberty, menopause, pregnancy, effects of hormone therapy, tumor load, serum VEGF levels, hemoglobin/hematocrit levels, and erythropoietin.
Trial information was received from ClinicalTrials.gov and was last updated in August 2016.
Information provided to ClinicalTrials.gov by National Institutes of Health Clinical Center (CC).