Neoplasms

All Conditions

Nervous System Diseases

Skin and Connective Tissue Diseases

Rare Diseases

Glioma

Brain Neoplasms

Central Nervous System Neoplasms

Pinealoma

Ependymoma

Solitary Fibrous Tumors

Hemangiopericytoma

Hemangioblastoma

Choroid Plexus Neoplasms

Neoplasms, Fibrous Tissue

Neoplasms, Neuroepithelial

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Germ Cell and Embryonal

Neoplasms by Histologic Type

Neoplasms, Glandular and Epithelial

Neoplasms, Nerve Tissue

Nervous System Neoplasms

Brain Diseases

Central Nervous System Diseases

Cerebral Ventricle Neoplasms

Neoplasms, Vascular Tissue

Neoplasms, Connective Tissue

Neoplasms, Connective and Soft Tissue

Hemangioma

Hemangioma, Capillary

Germ Cells Tumors

Pineocytoma

Neuroepithelioma

The goal is to determine whether the new 2021 WHO classification, based on cIMPACT-NOW recommendations, results in more homogeneous patient groups than the old 2016 classification. Furthermore, it will help derive provisional guidelines on how patients with these newly defined tumour entities are best treated. These recommendations will be based on the experience of EORTC investigators with chosen treatments and their experience as reported in this data collection report.

Observational

EORTC HQ

Jordi Bruna Escuder

Michael Weller

Every new classification depends on its prognostic power and on the type of treatment given. With the rapid evolution of diagnostic methods and the advance in new treatments, there is much less reliable information available on how patients with newly defined brain tumour entities should be treated and what to expect from the current treatments.

The goal is to determine whether the new 2021 WHO classification, based on cIMPACT-NOW recommendations, results in more homogeneous patient groups than the old 2016 classification. Furthermore, it will help derive provisional guidelines on how patients with these newly defined tumour entities are best treated. These recommendations will be based on the experience of EORTC investigators with chosen treatments and their experience as reported in this data collection report.

* Tumour tissue specifically stored for the purpose of this project
* Residual biological material ("leftovers") and/or derivatives

Observational Study for Assessing Treatment and Outcome of Patients With Primary Brain Tumours Using cIMPACT-NOW and 2021 WHO Classification

Observational Study for Assessing Treatment and Outcome of Patients With Primary Brain Tumours Diagnosed According to cIMPACT-NOW Recommendations and the 2021 WHO Classification

Date of surgery that allowed for the first pathological diagnosis up to the date of death (any cause). Patients will be followed up for 5 years after enrolment.

From the date of start of the anti-tumor therapy until the date of first objective progression as determined by the local investigator or the date of patient's death whichever occurs first. Patients will be followed up for 5 years after enrolment.

Recorded from the date start of an anti-tumor therapy until disease progression or start of a new anti-tumor therapy. Patients will be followed up for 5 years after enrolment.

European Organisation for Research and Treatment of Cancer - EORTC

Symptoms and General Pathology

Diseases and Abnormalities at or Before Birth

Ear, Nose, and Throat Diseases

Recurrence

Meningioma

Neurofibromatoses

Neurofibromatosis 1

Neurofibroma

Neurofibromatosis 2

Meningeal Neoplasms

Nerve Sheath Neoplasms

Syndrome

Neoplastic Syndromes, Hereditary

Neurocutaneous Syndromes

Heredodegenerative Disorders, Nervous System

Neurodegenerative Diseases

Genetic Diseases, Inborn

Peripheral Nervous System Diseases

Neuromuscular Diseases

Peripheral Nervous System Neoplasms

Neurofibromatosis

Neurofibromatosis Type 1

Neurofibromatosis Type 2

Antineoplastic Agents

All Drugs and Chemicals

Sunitinib

Cytochrome P-450 CYP3A Inhibitors

Angiogenesis Inhibitors

Growth Inhibitors

Protein Kinase Inhibitors

Enzyme Inhibitors

Cytochrome P-450 Enzyme Inhibitors

Sunitinib will be administered at a dose of 50 mg orally once daily for four consecutive weeks, followed by a two-week rest period. Intra-patient dose reduction may be required depending on the type and severity of individual toxicity encountered. Imaging studies will be performed after every other cycle. Patients may continue on study as long as they are tolerating treatment and in the absence of disease progression.

The study drug will be administered on an outpatient basis. The starting dose will be 50 mg daily for 28 days (4 consecutive weeks) followed by 14 days off for patients not on CYP3A4 inducers or inhibitors. A cycle equals 42 days.

Thomas Kaley, MD

The purpose of this study is to find out what effects, good and/or bad, sunitinib has on patients and their tumors. At this time, no drugs are routinely used to treat meningioma, hemangioblastoma or hemangiopericytoma. Only surgery and radiation therapy are known to be useful.

Sunitinib is a drug approved for advanced kidney cancer. Sunitinib is also being studied for other tumors. It may be useful in the treatment of brain tumors because it can prevent formation of new blood vessels that allow tumor cells to survive and grow.

This is a phase II study of Sunitinib in patients with recurrent or inoperable meningiomas. An exploratory study will be performed for patients with recurrent hemangiopericytoma or hemangioblastoma. There will be approximately 50 patients enrolled on this study (40 meningiomas and 10 hemangiopericytomas/hemangioblastomas). The treatment plan is to use daily SU11248 at a dose of 50 mg, using the established schedule of 4 weeks of treatment followed by two weeks of rest period, forming a six-week treatment cycle. A medical professional will see each patient at least every six weeks while on the medication for toxicity assessment and physical examination. Extent of disease evaluations will occur at baseline, two weeks, twelve weeks, 24 weeks, and every twelve weeks thereafter. These evaluations will include MRI of the brain (or CT head if a patient cannot undergo MRI) and MR perfusion.

Phase II Trial of Sunitinib (SU011248) in Patients With Recurrent or Inoperable Meningioma

Dana-Farber Cancer Institute

University of Virginia

University of Pittsburgh

Pfizer

Memorial Sloan Kettering Cancer Center

All patients treated with Sunitinib (SU011248)

All Patients

Aggressive Memingioma

Patients with WHO grade I meningioma, HPC and hemangioblastoma

Exploratory Cohort

Total

Median Age

Age, Customized

Sex: Female, Male

Dr. Thomas Kaley

Complete Response

Partial Response

Progression of Disease

Stable Disease

Overall Objective Response

Overall Study

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Heart and Blood Diseases

Pheochromocytoma

Carcinoma

Carcinoma, Renal Cell

Neuroendocrine Tumors

Von Hippel-Lindau Disease

Paraganglioma

Angiomatosis

Vascular Diseases

Ciliopathies

Congenital Abnormalities

Abnormalities, Multiple

Renal Cell Carcinoma

Neuroendocrine Tumor

Clear Cell Renal Cell Carcinoma

Pancreatic Neuroendocrine Tumor

Paragangliomas 1

Bevacizumab

Endothelial Growth Factors

Mitogens

89Zr-bevacizumab

Antineoplastic Agents, Immunological

Radiopharmaceuticals

Adult patients with Von Hippel-Lindau disease

Patients will be injected intravenously with 37 MBq, protein dose 5 mg 89Zr-bevacizumab at day 0. PET scans will be done at day 4.

89Zr bevacizumab PET scan

Sjoukje Oosting, MD

Von Hippel Lindau disease (VHLD) is an inherited syndrome characterized by vascular malformations, kidney cancer, adrenal gland and pancreas tumors. The VHL protein is not functional in the different disease associated lesions which results in production of high amounts of vascular endothelial growth factor (VEGF). Currently there are no clinical, radiographic or molecular markers that can predict the natural history of a given lesion. With 89Zr-bevacizumab positron emission tomography (PET) scanning, VEGF can be visualized and quantified.

The investigators hypothesize that 89Zr-bevacizumab PET imaging is a useful tool to predict the behaviour of disease associated lesions in patients with VHLD.

Adult patients with VHLD who have had routine magnetic resonance imaging (MRI) scans of central nervous system (CNS) and abdomen will undergo a 89Zr-bevacizumab PET scan. MRI will be repeated within 12 months.

Blood samples will be taken at day 1 and at the day of the MRI scan for analysis of VEGF pathway related biomarkers (such as plasma VEGF, PDGF, placental growth factor (PlGF), soluble VEGF receptors) and endothelial activation markers (such as Von Willebrand Factor (VWF), plasminogen activator inhibitor type 1 antigen (PAI-1), tissue-type plasminogen activator antigen (t-PA) and circulating endothelial cells (CECs)). DNA analysis for evaluation of polymorphisms in genes involved in angiogenesis is optional. In available biopsy or resection specimens, additional molecular staining of VEGF pathway related proteins will be performed (such as VEGF, VEGF receptors, HIF).

Visualizing Vascular Endothelial Growth Factor (VEGF) Producing Lesions in Von Hippel-Lindau Disease

Visualizing VEGF Producing Lesions in Von Hippel-Lindau Disease

VHL Alliance

University Medical Center Groningen

500 mg/day on a 5 day on, 2 day off schedule

500 mg (5 capsules) daily by mouth on Days 1-5, 8-12, 15-19, and 22-26 of each 28-day cycle (i.e. 5 days on, 2 days off schedule).

Dovitinib

Eric Jonasch, MD

The goal of this clinical research study is to learn if dovitinib can safely be given to patients who have VHL with a measurable hemangioblastoma (tumor of the central nervous system). The effects of this drug on the disease will also be studied.

The Study Drug:

Dovitinib is designed to perform several anti-tumor functions, including cutting off the blood supply to tumors.

Study Drug Administration:

If you are found to be eligible to take part in this study, you will take 5 dovitinib capsules by mouth each day on Days 1-5, 8-12, 15-19, and 22-26 of each 28-day cycle.

You should take the dovitinib capsules with about a cup (8 ounces) of water and at least 1 hour before breakfast or at least 2 hours following breakfast.

If you have any side effects from the drug, tell the study doctor right away. The study doctor may then lower the dose or keep the dose level the same.

If you miss a dose of dovitinib on Days 1-4 (or 8-11, 15-18, or 22-25), you should not make up the dose on the same day. You should continue taking the drug as scheduled the following day. If you miss a dose on Day 5 (or 12, 19, or 26), you should skip the dose, rest 2 days, and begin dosing again as scheduled on Day 8 (or 15, 22, or 1 of the next cycle). The study doctor will tell you about any additional steps that should be taken if you miss a dose.

Every 4 weeks on this study is called a study "cycle."

Study Visits:

On Day 1 of Cycle 1, you will have an ECG.

On Day 14 (+/- 3 days) of Cycles 1 and 2:

-Blood (about 3 teaspoons) will be drawn for routine tests. This blood testing may be done at your local doctor's office and faxed to MD Anderson. You will be provided instructions about how to fax laboratory test results.

On Day 1 of Cycles 3 and Beyond (+/- 3 days), blood (about 3 teaspoons) will be drawn for routine tests.

Every 8 weeks (+/- 3 days):

* Any changes to your medical history since your last visit will be recorded.
* You will have a physical exam, including measurement of your vital signs and weight.
* You will be asked about any drugs or treatments you may be receiving.
* Your performance status will be recorded.
* You will be asked about any side effects that you have had since your last visit.

At the End of Cycles 2 and 4:

* You will have CT scans and MRI scans to check the status of the disease.
* If the doctors know or suspect that VHL is affecting your eyes, you will have an eye exam.

Length of Study:

You may continue taking the study drugs for as long as you are benefiting. You will be taken off study if the disease gets worse or intolerable side effects occur.

Early Withdrawal /End of Treatment Visit:

* About 30 days after you stop taking study drug, you will have the following procedures:
* You will have a physical exam, including measurement of your vital signs and weight.
* Your performance status will be recorded.
* You will be asked about any drugs or treatments you may be receiving.
* You will be asked about any side effects that you may have had since your last visit.
* Blood (about 3 teaspoons) will be drawn for routine tests.
* You will have CT scans and MRI scans to check the status of the disease.
* If the doctors know or suspect that VHL is affecting your eyes, you will have an eye exam.

About 24 weeks after your last dose of study drug, you will have the following procedures:

* You will have a physical exam, including measurement of your vital signs and weight.
* You will have follow-up imaging scans to check the status of the disease.
* Blood (about 3 teaspoons) will be collected for routine tests.
* You will be asked about any drugs or treatments you may be receiving.
* You will be asked about any side effects that you may have had since your last visit.

This is an investigational study. Dovitinib is not FDA approved or commercially available. It is currently being used for research purposes only.

Up to 25 patients will take part in this study. All will be enrolled at MD Anderson.

TKI 258 in Von Hippel-Lindau Syndrome (VHL)

A Pilot Trial of TKI 258 (Dovitinib) in Von Hippel-Lindau Syndrome

Most frequent \& Most Serious Adverse Events: Safety of treatment with Dovitinib for 6 months in participants with VHL who have a measurable hemangioblastoma undergoing surveillance evaluated by toxicity scored using Common Toxicity Criteria (CTC) Version 4.0.

Efficacy of treatment with dovitinib for 6 months in patients with VHL who have a measurable lesion evaluated by response using RECIST (Response Evaluation Criteria in Solid Tumors): Complete Response, Partial Response, Progressive Disease or Stable Disease.

Novartis

M.D. Anderson Cancer Center

Dovitinib oral 500 mg/day on a 5 day on, 2 day off schedule (Days 1-5, 8-12, 15-19, and 22-26) of each 28-day cycle

Age, Continuous

Gender

Ethnicity (NIH/OMB)

Race (NIH/OMB)

United States

Region of Enrollment

Eric Jonasch, MD/Professor, Genitourinary Medical Oncology

Alanine aminotransferase increased

Alkaline phosphatase increased

Aspartate aminotransferase increased

Diarrhea

Dyspepsia

GGT Increased

Hyperglycemia

Hyperkalemia

Mucositis oral

Nausea

Rash maculo-papular

White Blood cell decreased

Most Frequent & Most Serious Adverse Events: Safety of Dovitinib for 6 Months

Number of VHL Participants With Response at 6 Months

Patients will receive Bevacizumab (Avastin) 10mg/kg IV every two weeks for 6 months

Avastin

J Marc Pipas, MD

Von Hippel-Lindau (VHL) disease is an inherited syndrome manifested by a variety of benign and malignant tumors. Hemangioblastomas are the most common lesion associated with VHL disease affecting 60-84% of patients with a mean age at diagnosis of 29 years. Standard treatment for this disease is by surgery or radiotherapy. No approved systemic therapy yet exists. Patients with VHL have an increased growth factor production, specifically vascular endothelial growth factor (VEGF), resulting in angiogenesis (growth of blood vessels). Studies show that Bevacizumab inhibits the growth of VEGF protein and will block the VEGF-driven angiogenesis and result in stabilization and regression of hemangioblastomas in VHL disease patients. The dose of bevacizumab will be 10 mg/kg every two weeks for up to 6 months.

Bevacizumab (Avastin) in Unresectable/Recurrent Hemangioblastoma From Von-Hippel-Lindau Disease

D0904 - A Pilot Study of Bevacizumab (Avastin) in Patients With Unresectable or Recurrent Hemangioblastoma From Von Hippel-Lindau Disease.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Observational Study for Assessing Treatment and Outcome of Patients With Primary Brain Tumours Using cIMPACT-NOW and 2021 WHO Classification	NCT05259605	Glioma Glioneuronal Tu... Choroid Plexus ... Pineal Tumors Germ Cell Tumor Tumor of the Se... Diffuse Midline... Ependymoma Embryonal Tumor Pineal Tumor Hemangiopericyt... Hemangioblastom... Melanocytic Tum...	Observational	18 Years -	European Organisation for Research and Treatment of Cancer - EORTC
Phase II Trial of Sunitinib (SU011248) in Patients With Recurrent or Inoperable Meningioma	NCT00589784	CNS Cancer Meningioma Intracranial He... Hemangioblastom... Neurofibromatos...	Sunitinib	18 Years -	Memorial Sloan Kettering Cancer Center
Visualizing Vascular Endothelial Growth Factor (VEGF) Producing Lesions in Von Hippel-Lindau Disease	NCT00970970	Von Hippel-Lind... Hemangioblastom... Renal Cell Carc... Pheochromocytom... Pancreatic Neur...	89Zr bevacizuma...	18 Years -	University Medical Center Groningen
TKI 258 in Von Hippel-Lindau Syndrome (VHL)	NCT01266070	Von Hippel-Lind...	Dovitinib	18 Years -	M.D. Anderson Cancer Center
Bevacizumab (Avastin) in Unresectable/Recurrent Hemangioblastoma From Von-Hippel-Lindau Disease	NCT01015300	Hemangioblastom... Von Hippel Lind...	Avastin	18 Years -	Dartmouth-Hitchcock Medical Center
Phase II Trial of Sunitinib (SU011248) in Patients With Recurrent or Inoperable Meningioma	NCT00589784	CNS Cancer Meningioma Intracranial He... Hemangioblastom... Neurofibromatos...	Sunitinib	18 Years -	Memorial Sloan Kettering Cancer Center
Observational Study for Assessing Treatment and Outcome of Patients With Primary Brain Tumours Using cIMPACT-NOW and 2021 WHO Classification	NCT05259605	Glioma Glioneuronal Tu... Choroid Plexus ... Pineal Tumors Germ Cell Tumor Tumor of the Se... Diffuse Midline... Ependymoma Embryonal Tumor Pineal Tumor Hemangiopericyt... Hemangioblastom... Melanocytic Tum...	Observational	18 Years -	European Organisation for Research and Treatment of Cancer - EORTC
Endolymphatic Sac Tumors in a Population of Patients With Von Hippel-Lindau Disease:The Natural History and Pathobiology, and a Prospective Non-Randomized Clinical Trial of Hearing Preservation Surgery in Patients With Early Stage Endolymphatic Sac Tumors	NCT00001668	Deafness Kidney Diseases Kidney Neoplasm... Neoplasms Retinal Disease...		-	National Institutes of Health Clinical Center (CC)

Spots Global Cancer Trial Database for hemangioblastoma