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Study of Brain and Spinal Cord Tumor Growth and Cyst Development in Patients With Von Hippel Lindau Disease

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ClinicalTrials.gov Identifier: NCT00005902
Recruitment Status : Active, not recruiting
First Posted : June 7, 2000
Last Update Posted : November 12, 2021
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Institute of Neurological Disorders and Stroke (NINDS) )

Tracking Information
First Submitted Date June 6, 2000
First Posted Date June 7, 2000
Last Update Posted Date November 12, 2021
Actual Study Start Date February 15, 2001
Primary Completion Date Not Provided
Current Primary Outcome Measures
 (submitted: June 26, 2020)
Determine the course of clinical and radiographic progression of VHL [ Time Frame: Baseline, 12-24 month intervals ]
The questions to be answered fall in the following general categories:1. Course of Clinical Progression2. Radiographic Progression3. Post-treatment outcome (surgery and radiation)4. Contributing factors5. Genetic variability and effects on disease progression
Original Primary Outcome Measures Not Provided
Change History
Current Secondary Outcome Measures
 (submitted: October 2, 2021)
Determine the course of clinical and radiographic progression of VHL disease following surgical interventions. [ Time Frame: 12-24 months intervals after baseline evaluation. ]
Patients with CNS VHL disease undergo surgery for removal of problem causing tumors and cysts. We are investigating the effect of such necessary surgeries on the clinical measures including neurological deficits, functional measures and reported pain measures. We will also evaluate whether the need for other CNS surgeries remains the same or decreases with time.
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Study of Brain and Spinal Cord Tumor Growth and Cyst Development in Patients With Von Hippel Lindau Disease
Official Title A Prospective Natural History Study of VHL Patients With CNS Hemangioblastomas
Brief Summary

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.

...

Detailed 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 erythropoeitin level and hemoglobin/hematocrit levels.

This natural history study has resulted in significant improvement in our understanding of the pathophysiology of VHL disease. Data from this study have resulted in a better understanding of the origin and growth patterns of hemangioblastomas, and endolymphatic sac tumors in patients with VHL disease. New therapies targeting hemangioblastomas in VHL disease are being devised based on information gathered from this study. We expect this natural history disease to continue to inspire new hypothesis driven studies while improving the understanding and management of hemangioblastomas in VHL disease.

Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population This study will enroll 250 subjects with von Hippel-Lindau (VHL) disease. Withdrawal/dropouts will not be replaced.
Condition
  • Hemangioblastoma
  • Hippel Lindau Disease
Intervention Not Provided
Study Groups/Cohorts 1
250 subjects with von Hippel-Lindau (VHL) disease.
Publications * Lonser RR, Butman JA, Huntoon K, Asthagiri AR, Wu T, Bakhtian KD, Chew EY, Zhuang Z, Linehan WM, Oldfield EH. Prospective natural history study of central nervous system hemangioblastomas in von Hippel-Lindau disease. J Neurosurg. 2014 May;120(5):1055-62. doi: 10.3171/2014.1.JNS131431. Epub 2014 Feb 28.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Active, not recruiting
Actual Enrollment
 (submitted: June 23, 2005)
250
Original Enrollment Same as current
Study Completion Date Not Provided
Primary Completion Date Not Provided
Eligibility Criteria
  • 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. Able to give informed consent (or assent), or have a parent able to provide informed consent if a child.
    5. Karnofsky Performance Scale Score greater than or equal to 60.
    6. Have the capacity to undergo serial MRI scanning of the CNS without IV sedation.

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.
Sex/Gender
Sexes Eligible for Study: All
Ages 8 Years to 75 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT00005902
Other Study ID Numbers 000140
00-N-0140
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement Not Provided
Responsible Party National Institutes of Health Clinical Center (CC) ( National Institute of Neurological Disorders and Stroke (NINDS) )
Study Sponsor National Institute of Neurological Disorders and Stroke (NINDS)
Collaborators Not Provided
Investigators
Principal Investigator: Prashant Chittiboina, M.D. National Institute of Neurological Disorders and Stroke (NINDS)
PRS Account National Institutes of Health Clinical Center (CC)
Verification Date September 24, 2021