Vaccine Therapy in Treating Patients with Malignant Peripheral Nerve Sheath Tumor That is Recurrent or Cannot Be Removed by Surgery

NCT02700230 | Completed Phase 1 DMC FDA Drug

• 4 other identifiers: MC1372NCI-2016-00179W81XWH-15-1-011515-008308
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief Summary

This phase I trial studies the side effects and the best dose of a vaccine therapy in treating patients with malignant peripheral nerve sheath tumor that cannot be removed by surgery (unresectable) or has come back after a period of improvement (recurrent). Vaccines made from a gene-modified virus may kill tumor cells expressing a gene called neurofibromin 1 (NF1) without affecting surrounding normal cells and may also help the body build an effective immune response to kill tumor cells.

Conditions

Metastatic Malignant Peripheral Nerve Sheath Tumor; Neurofibromatosis Type 1; Recurrent Malignant Peripheral Nerve Sheath Tumor

Outcome Measures

Primary Outcomes (3)

• Best response using the World Health Organization response criteria

  Responses will be summarized by simple descriptive summary statistics delineating complete and partial responses as well as stable and progressive disease in this patient population (overall and dose level).

  From the start of the treatment until disease progression/recurrence (taking as reference for progressive disease the smallest measurements recorded since the treatment started), assessed up to 2 years

• Incidence of adverse events according to the National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0

  The number and severity of all adverse events (overall and by dose-level) will be tabulated and summarized in this patient population. The grade 3+ adverse events will also be described and summarized in a similar fashion. Overall toxicity incidence as well as toxicity profiles by dose level, patient and tumor site will be explored and summarized. Frequency distributions, graphical techniques and other descriptive measures will form the basis of these analyses.

  Up to 2 years after treatment

• Maximum tolerated dose defined as the highest safely tolerated dose level where at most 1 out of 6 patients experience dose-limiting toxicity (DLT) with the next higher dose having at least 2 patients out of a maximum of 6 patients experience DLT

  Assessed according to according to Common Terminology Criteria for Adverse Events version 4.0. The number and severity of all adverse events (overall and by dose-level) will be tabulated and summarized in this patient population.

  6 weeks

Secondary Outcomes (12)

• Absolute percentage change in quality of life measured using the Brief Pain Inventory (short form) and Brief Fatigue Inventory

  Baseline to up to 2 years

• Change in biodistribution of virally infected cells at various time points after infection with MV-NIS using single photon emission computed tomography/computed tomography

  Baseline to up to day 8

• Growth-rate between treated and untreated lesions

  Up to 2 years

• Humoral and cellular immune response to the injected virus

  Up to 2 years

• Incidence of measles virus shedding/persistence following intratumoral administration

  Up to 2 years

Study Arms (1)

Treatment (MV-NIS)

EXPERIMENTAL

Patients receive MV-NIS intratumorally on day 1. Patients also undergo SPECT/CT at baseline and at 3 and 8 days after MV-NIS. Patients may also undergo SPECT/CT at 15 and 28 days, and at 6 weeks based on whether there is uptake on prior imaging studies. Patients also undergo MRI, ultrasound imaging, blood sample collection and tissue biopsy throughout the trial.

Procedure: Computed Tomography; Other: Laboratory Biomarker Analysis; Biological: Oncolytic Measles Virus Encoding Thyroidal Sodium Iodide Symporter; Other: Quality-of-Life Assessment; Procedure: Single Photon Emission Computed Tomography; Procedure: Magnetic Resonance Imaging; Procedure: Ultrasound Imaging; Procedure: Biospecimen Collection; Procedure: Biopsy; Other: Questionnaire Administration

Interventions

Computed Tomography PROCEDURE

Undergo CT scan

Also known as: CAT, CAT Scan, Computerized Axial Tomography, Computerized Tomography, CT, CT SCAN, tomography

Treatment (MV-NIS)

Laboratory Biomarker Analysis OTHER

Correlative studies

Treatment (MV-NIS)

Oncolytic Measles Virus Encoding Thyroidal Sodium Iodide Symporter BIOLOGICAL

Given intratumorally

Also known as: MV-NIS

Treatment (MV-NIS)

Quality-of-Life Assessment OTHER

Ancillary studies

Also known as: Quality of Life Assessment

Treatment (MV-NIS)

Single Photon Emission Computed Tomography PROCEDURE

Undergo SPECT imaging

Also known as: Medical Imaging, Single Photon Emission Computed Tomography, Single Photon Emission Tomography, single-photon emission computed tomography, SPECT, SPECT imaging, SPECT SCAN, SPET, tomography, emission computed, single photon, Tomography, Emission-Computed, Single-Photon

Treatment (MV-NIS)

Magnetic Resonance Imaging PROCEDURE

Undergo MRI

Also known as: Magnetic Resonance, Magnetic Resonance Imaging Scan, Medical Imaging, Magnetic Resonance / Nuclear Magnetic Resonance, MR, MR Imaging, MRI, MRI Scan, Nuclear Magnetic Resonance Imaging, NMRI, Structural MRI, sMRI

Treatment (MV-NIS)

Ultrasound Imaging PROCEDURE

Undergo ultrasound imaging

Also known as: 2-Dimensional Grayscale Ultrasound Imaging, 2-Dimensional Ultrasound Imaging, 2D-US, Ultrasonography, Ultrasound, Ultrasound Test, Ultrasound, Medical, US

Treatment (MV-NIS)

Biospecimen Collection PROCEDURE

Undergo blood sample collection

Also known as: Biological Sample Collection, Biospecimen Collected, Specimen Collection

Treatment (MV-NIS)

Biopsy PROCEDURE

Undergo tissue biopsy

Also known as: Bx

Treatment (MV-NIS)

Questionnaire Administration OTHER

Ancillary studies

Treatment (MV-NIS)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age \>= 18 years
• Pathologically confirmed MPNST, with or without underlying diagnosis of neurofibromatosis type 1 (diagnostic criteria for neurofibromatosis type 1)
• Measurable disease as defined by at least one tumor that is measurable in two dimensions on CT or magnetic resonance imaging (MRI) scan (minimum size 1.0 cm for at least one lesion)
• MPNST for which standard therapy is not curative, including patients with surgically unresectable lesions, progression (WHO criteria) or recurrence of an MPNST in a previously radiated field (if it has been at least 4 weeks prior to registration since the last dose of radiation); Note: patients with metastatic disease also are eligible for participation
• Patient may have more than one site of recurrent or metastatic disease but only one lesion that is \>= 1 cm in size will be injected (if in the lung, the lesion must be \>= 2 cm and adjacent to the pleura in the lung)
• The following laboratory values obtained =\< 14 days prior to registration
• Absolute neutrophil count (ANC) \>= 1500
• Platelet (PLT) \>= 100,000
• Hemoglobin (HgB) \>= 9.0 g/dL
• Total bilirubin =\< institutional upper limit of normal (ULN)
• Serum glutamic oxaloacetic transaminase (SGOT) (aspartate aminotransferase \[AST\]) =\< 1.5 x upper limit of normal (ULN)
• Creatinine =\< 1.0 mg/dL
• International normalized ratio (INR) =\< 2.0
• Negative pregnancy test done =\< 7 days prior to registration, for women of childbearing potential only
• Eastern Cooperative Oncology Group (ECOG) performance status (PS) 0, 1 or 2

You may not qualify if:

• Any of the following because this study involves an agent that has known genotoxic, mutagenic and teratogenic effects
• Pregnant women
• Nursing women
• Men or women of childbearing potential who are unwilling to employ adequate contraception during treatment and 8 weeks following the completion of treatment
• Co-morbid systemic illnesses or other severe concurrent disease which, in the judgment of the investigator, would make the patient inappropriate for entry into this study or interfere significantly with the proper assessment of safety and toxicity of the prescribed regimens
• Receiving therapeutic anticoagulation (Coumadin or low molecular weight heparin, heparin, apixaban, dabigatran, rivaroxaban, warfarin)
• Active infection =\< 5 days prior to registration
• History of tuberculosis or history of purified protein derivative (PPD) positivity
• Any of the following prior therapies:
• Chemotherapy =\< 3 weeks prior to registration
• Immunotherapy =\< 4 weeks prior to registration
• Biologic therapy =\< 4 weeks prior to registration
• Radiation therapy =\< 3 weeks prior to registration
• Failure to fully recover from acute, reversible effects defined as =\< grade 1 Common Terminology Criteria for Adverse Events (CTCAE) version (v.) 4.0 of prior chemotherapy regardless of interval since last treatment except alopecia and neuropathy
• Requiring blood product support

Sponsors & Collaborators

• Mayo Clinic: lead

Study Sites (1)

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Related Publications (1)

• Hassan A, Pestana RC, Parkes A. Systemic Options for Malignant Peripheral Nerve Sheath Tumors. Curr Treat Options Oncol. 2021 Feb 27;22(4):33. doi: 10.1007/s11864-021-00830-7.

  PMID: 33641042 DERIVED

Related Links

• Mayo Clinic Clinical Trials

MeSH Terms

Conditions

Neurofibrosarcoma; Neurofibromatosis 1

Interventions

X-Rays; Photons; Magnetic Resonance Spectroscopy; High-Energy Shock Waves; Specimen Handling; Biopsy

Condition Hierarchy (Ancestors)

Fibrosarcoma; Neoplasms, Fibrous Tissue; Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Sarcoma; Neurofibroma; Nerve Sheath Neoplasms; Neoplasms, Nerve Tissue; Peripheral Nervous System Neoplasms; Nervous System Neoplasms; Nervous System Diseases; Peripheral Nervous System Diseases; Neuromuscular Diseases; Neurofibromatoses; Neoplastic Syndromes, Hereditary; Neurocutaneous Syndromes; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Electromagnetic Radiation; Electromagnetic Phenomena; Magnetic Phenomena; Physical Phenomena; Radiation; Radiation, Ionizing; Elementary Particles; Light; Optical Phenomena; Radiation, Nonionizing; Spectrum Analysis; Chemistry Techniques, Analytical; Investigative Techniques; Ultrasonic Waves; Sound; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Cytodiagnosis; Cytological Techniques; Diagnostic Techniques, Surgical; Surgical Procedures, Operative

Study Officials

• Dusica Babovic-Vuksanovic, MD

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 25, 2016
• First Posted: March 7, 2016
• Study Start: March 22, 2017
• Primary Completion: April 17, 2024
• Study Completion: April 17, 2024
• Last Updated: September 27, 2024

Record last verified: 2024-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)