Neoplasms

All Conditions

Nervous System Diseases

Musculoskeletal Diseases

Ear, Nose, and Throat Diseases

Rare Diseases

Glioblastoma

Glioma

Neurilemmoma

Brain Neoplasms

Central Nervous System Neoplasms

Medulloblastoma

Meningioma

Nervous System Neoplasms

Spinal Cord Neoplasms

Pinealoma

Astrocytoma

Ependymoma

Craniopharyngioma

Adamantinoma

Neuroma, Acoustic

Germ Cells Tumors

Schwannoma

Pineocytoma

Acoustic Neuroma

Pineoblastoma

Oligoastrocytoma

tozuleristide 24 or 36 mg administered intravenously 1-24 hours prior to surgery

tozuleristide

imaging device attached to surgical microscope

Canvas imaging system

Standard of care surgical resection of tumor

Surgical resection of tumor

Clinical Trial Navigator

Ray Chu, MD

Chirag Patil, MD

Jeremy Rudnick, MD

Jethro Hu, MD

Serguei Bannykh, MD, PhD

John Yu, MD

The purpose of this study is to examine the use of a single dose of tozuleristide (24 or 36 mg) and the Canvas imaging system during surgical resection of primary central nervous system (CNS) tumors: Primary gadolinium enhancing (high grade) CNS tumors, primary non-gadolinium enhancing CNS tumors, and primary vestibular schwannoma.

The primary objectives of the study is to see how well tozuleristide and the Canvas imaging system during surgical resection will show fluorescence among primary enhancing/high grade CNS tumors; and among the tumors that demonstrate tozuleristide fluorescence, to estimate the true positive rate and true negative rate of fluorescence in tissue biopsies, as well as sensitivity and specificity of tozuleristide fluorescence for distinguishing tumor from non-tumoral tissue.

The secondary objectives of the study include evaluating the safety of tozuleristide and the Canvas imaging system, and to determine if the presence of remaining fluorescence at the time of surgery corresponds to remaining tumor evident on post-operative MRI images, or if the absence of fluorescence corresponds to evidence of no gross residual tumor on post-operative magnetic resonance imaging (MRI).

Fluorescence Detection of Adult Primary Central Nervous System Tumors With Tozuleristide and the Canvas System

A Phase 2 Study of Fluorescence Detection of Adult Primary Central Nervous System Tumors in Subjects Receiving Tozuleristide and Imaged With the Canvas System

Recorded and graded according to the National Cancer Institute's Common Terminology Criteria for Adverse Events Version 5

Blaze Bioscience Inc.

John Yu

Director, Surgical Neuro-Oncology

Diseases and Abnormalities at or Before Birth

Symptoms and General Pathology

Skin and Connective Tissue Diseases

Neurofibromatoses

Neurofibromatosis 1

Neurofibroma

Neurofibromatosis 2

Neuroma

Nerve Sheath Neoplasms

Neoplasms, Nerve Tissue

Neoplasms by Histologic Type

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

Neuroendocrine Tumors

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Germ Cell and Embryonal

Vestibulocochlear Nerve Diseases

Retrocochlear Diseases

Ear Diseases

Otorhinolaryngologic Diseases

Otorhinolaryngologic Neoplasms

Cranial Nerve Diseases

Neurofibromatosis

Neurofibromatosis Type 1

Neurofibromatosis Type 2

Neuroendocrine Tumor

Neuroepithelioma

Antineoplastic Agents

All Drugs and Chemicals

Axitinib

Protein Kinase Inhibitors

Enzyme Inhibitors

5 mg axitinib orally twice daily, with increase to 7 mg orally twice daily and 10 mg orally twice daily after 2 and 4 weeks, respectively, provided no adverse reactions (i.e., not exceeding grade 2 toxicities) and normotensive and not receiving antihypertension medications. Axitinib will be given continuously in 28-day cycles until disease progression or unacceptable toxicity.

Theodore Nicolaides, MD

The purpose of this study is to determine if the study drug, AXITINIB, has any effect on tumors found in patients with Neurofibromatosis Type 2 (NF2).

NF2 is a condition that mainly affects the skin and nervous system. It causes non-cancerous tumors to grow on the nerves around a person's body. Some signs of NF2 include a gradual loss of hearing and tumors growing on the skin, the brain and the spinal cord, which can lead to complications.

AXITINIB is an oral drug (taken by mouth) that is approved by the United States Food and Drug Administration (FDA) for the treatment of other types of tumors. However, in this research study, AXITINIB is considered investigational because it is not approved by the FDA for treatment of NF2. Much is known regarding how well it is tolerated (handled), but investigators do not know if it is effective in treating NF2.

This research study will test whether AXITINIB may shrink tumors commonly found in patients with NF2 or stop them from growing. This will help to decide if AXITINIB should be used to treat NF2 patients in the future. AXITINIB is a drug that has been used to treat various forms of cancer. It has not been studied for the treatment of tumors in NF2 patients. Investigators have selected AXITINIB for this clinical trial in patients with NF2 and NF2-related tumors because a very similar drug, bevacizumab, can shrink Vestibular Schwannomas (VS) in some NF2 patients.

Pfizer, Inc., the manufacturer of the study drug, AXITINIB, will provide the AXITINIB being used in this study.

Primary Objective: To estimate the objective volumetric response rates to axitinib in adult NF2 patients with VS.

Secondary Objectives: To assess the toxicity of axitinib given daily in patients with NF2 and to examine the association of objective measures of response on MRI, i.e. volumetric tumor analysis with clinical measures of response, i.e. (audiogram), as well as quality of life assessments (NFTI-QOL). In addition, response in non-VS tumors, such as other schwannomas and meningiomas, may be explored.

Study of Axitinib in Patients With Neurofibromatosis Type 2 and Progressive Vestibular Schwannomas

Phase II Study of Axitinib in Patients With Neurofibromatosis Type 2 and Progressive Vestibular Schwannomas

The histoscore is given as the sum of the percentage of staining multiplied by an ordinal value corresponding to the intensity level (0 = none, 1 = weak, 2 = moderate, 3 = strong). With 4 intensity levels, the resulting score ranged from 0 (no staining in the tumor) to 300 (diffuse intense staining of the tumor).

NYU Langone Health

5 mg axitinib orally twice daily, with increase to 7 mg orally twice daily and 10 mg orally twice daily after 2 and 4 weeks. Axitinib will be given continuously in 28-day cycles until disease progression or unacceptable toxicity.

Age, Continuous

Sex: Female, Male

Race and Ethnicity were not collected from any participant.

Race and Ethnicity Not Collected

United States

Region of Enrollment

Theodore Nicolaides

10 tissue matched historical controls were analyzed. The Median (Full Range) of the histoscore was 122 (60 to 265)

Expression Levels of p-S6 Based Immunohistochemistry (Histoscore)

10 tissue matched historical controls were analyzed. The Median (Full Range) of the histoscore was 135 (100 to 240)

Expression Levels of p-ERK Based Immunohistochemistry (Histoscore)

10 tissue matched historical controls were analyzed. The Median (Full Range) of the histoscore was 12 (0 to 100)

Expression Levels of p-AKT Based Immunohistochemistry (Histoscore)

No controls - Given that only a single surgery is generally performed to resect meningiomas and schwannomas without prior biopsy, obtaining matched control of pre and post treatment samples from the same tumor was not feasible. The study team therefore relied on matched archival control tissue for comparisons of other outcome measures; Everolimus blood levels not being one of them.

Pre-Operative Everolimus Blood Levels

Post-Operative Everolimus Blood Levels

Overall Study

Neoplasms, Vascular Tissue

Meningeal Neoplasms

Anti-Infective Agents

Everolimus

Sirolimus

Temsirolimus

MTOR Inhibitors

Immunosuppressive Agents

Immunologic Factors

RAD001 taken by mouth 10mg daily for 10 days before surgery

RAD001

The primary objective is to estimate the proportions of vestibular schwannomas (VS) and meningiomas after 10 days of exposure to the study drug RAD001 at a dose of 10 mg daily, as determined by immunohistochemistry. This is a "phase 0" PK (pharmacokinetic) and PD (pharmacodynamic) study of RAD001 in patients with Neurofibromatosis Type 2-related and sporadic VS and meningiomas. Enrolled patients will take RAD001 prior to a scheduled VS or meningioma surgery, and blood and tissue samples will be obtained for further analysis.

Exploring the Activity of RAD001 in Vestibular Schwannomas and Meningiomas

1) To estimate the proportions of VS and meningiomas with complete inhibition of phospho-S6 after10 days of exposure to RAD001 at a dose of 10 mg daily, as determined by immunohistochemistry. This endpoint was chosen based on prior pharmacodynamic data from a published trial, showing complete inhibition of phospho-S6 in solid tumor tissue of patients treated with RAD001 and our own preliminary data confirming baseline phospho-S6 expression in VS and meningiomas.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Fluorescence Detection of Adult Primary Central Nervous System Tumors With Tozuleristide and the Canvas System	NCT04743310	Central Nervous...	tozuleristide Canvas imaging ... Surgical resect...	18 Years -	Cedars-Sinai Medical Center
Study of Axitinib in Patients With Neurofibromatosis Type 2 and Progressive Vestibular Schwannomas	NCT02129647	Neurofibromatos... Vestibular Schw...	Axitinib	18 Years -	NYU Langone Health
Exploring the Activity of RAD001 in Vestibular Schwannomas and Meningiomas	NCT01880749	Neurofibromatos... Vestibular Schw... Meningiomas	RAD001	18 Years -	NYU Langone Health
Exploring the Activity of RAD001 in Vestibular Schwannomas and Meningiomas	NCT01880749	Neurofibromatos... Vestibular Schw... Meningiomas	RAD001	18 Years -	NYU Langone Health
Fluorescence Detection of Adult Primary Central Nervous System Tumors With Tozuleristide and the Canvas System	NCT04743310	Central Nervous...	tozuleristide Canvas imaging ... Surgical resect...	18 Years -	Cedars-Sinai Medical Center

Spots Global Cancer Trial Database for vestibular schwannomas