NCT02414165

Brief Summary

This is a multicenter, randomized, open-label phase 2/3 study of Toca 511 and Toca FC versus standard of care that comprises Investigator's choice of single agent chemotherapy (lomustine or temozolomide) or bevacizumab administered to subjects undergoing resection for first or second recurrence (including this recurrence) of GBM or AA. Subjects meeting all of the inclusion and none of the exclusion criteria will be randomized prior to surgery in a 1:1 ratio to receive either Toca 511 and Toca FC (Experimental arm, Arm T) or control treatment with one option of standard of care (Arm SOC). Stratification will be done by IDH1 mutation status. A second stratification factor is based on the patient's Karnofsky Performance Score (KPS) (70-80 vs 90-100). Further, to account for potential differences in treatment choices for the control arm in regions, the trial will be stratified by geographical region during the randomization process. Funding Source - FDA OOPD

Trial Health

60
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
403

participants targeted

Target at P75+ for phase_2

Timeline
Completed

Started Nov 2015

Typical duration for phase_2

Geographic Reach
4 countries

67 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

April 3, 2015

Completed
7 days until next milestone

First Posted

Study publicly available on registry

April 10, 2015

Completed
8 months until next milestone

Study Start

First participant enrolled

November 30, 2015

Completed
3.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 31, 2019

Completed
7 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 20, 2019

Completed
Last Updated

February 7, 2020

Status Verified

February 1, 2020

Enrollment Period

3.5 years

First QC Date

April 3, 2015

Last Update Submit

February 6, 2020

Conditions

Keywords

Recurrence

Outcome Measures

Primary Outcomes (1)

  • To compare the overall survival (OS) of subjects treated with Toca 511 combined with Toca FC to subjects treated according to standard of care after tumor resection for recurrence of glioblastoma or anaplastic astrocytoma

    Time from randomization date to death due to any cause

    30 December 2019

Secondary Outcomes (4)

  • Durable Response Rate (CR or PR ≥ 24 weeks)

    30 December 2019

  • Durable Clinical Benefit Rate (CR or PR ≥ 24 weeks or SD ≥ 18 months)

    30 December 2019

  • Duration of Durable Response

    30 December 2019

  • Overall Survival at 12 months

    30 December 2019

Study Arms (2)

Toca 511/Toca FC

EXPERIMENTAL

Resection followed by administration of 4 mL Toca 511 (vocimagene amiretrorepvec). Toca 511 is administered by injection into the wall of the subject's tumor resection cavity on Day 1 (approximately 40 injections of 0.1 mL) Toca FC is an extended-release formulation of flucytosine. Toca FC will be administered at 220 mg/kg/day orally for 7-day courses beginning at least 6 weeks after resection and repeated approximately every 6 weeks.

Biological: Toca 511Drug: Toca FC

Lomustine, Temozolomide, or Bevacizumab

ACTIVE COMPARATOR

Investigator selects one of the following: Bevacizumab: Beginning 6 weeks after tumor resection, bevacizumab will be administered by IV infusion at 10 mg/kg and repeated every 2 weeks. Refer to the prescribing information and to institutional guidelines for details on the administration procedure. Lomustine: Beginning 6 weeks after tumor resection, lomustine will be administered as a single oral dose of 110 mg/m2 and repeated every 6 weeks. Refer to the prescribing information and to institutional guidelines for details regarding the administration procedure. Temozolomide: Beginning 6 weeks after tumor resection, temozolomide will be administered per 1 of 2 options: * at a dose of 50 mg/m2 PO once daily continuously, or * at an initial dose of 150 mg/m2 IV or PO once daily for 5 consecutive days per 28-day treatment cycle that may be raised to 200 mg/ m2 once daily for 5 consecutive days in the following 28-day treatment cycles

Drug: LomustineDrug: TemozolomideBiological: Bevacizumab

Interventions

Toca 511BIOLOGICAL

Toca 511 consists of a purified retroviral replicating vector encoding a modified yeast cytosine deaminase (CD) gene. The CD gene converts the antifungal 5-flurocytosine (5FC) to the anticancer drug 5-FU in cells that have been infected by the Toca 511 vector.

Also known as: vocimagene amiretrorepvec, RRV, retroviral replicating vector
Toca 511/Toca FC

Toca FC is an extended-release formulation of flucytosine and is supplied as 500 mg tablets

Also known as: flucytosine, 5-FC, 5-fluorocytosine
Toca 511/Toca FC
Also known as: CCNU, CeeNU
Lomustine, Temozolomide, or Bevacizumab
Also known as: Temodar
Lomustine, Temozolomide, or Bevacizumab
BevacizumabBIOLOGICAL
Also known as: Avastin
Lomustine, Temozolomide, or Bevacizumab

Eligibility Criteria

Age18 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Subject has given written informed consent
  • Subject is between 18 years old and 75 years old, inclusive
  • Subjects must have histologically proven GBM or AA and:
  • Must have received first-line multimodal therapy with surgery followed by temozolomide (unless MGMT promoter unmethylated) and radiation (subjects with GBM must have received temozolomide and radiation concurrently)
  • Must be in first or second recurrence (including this recurrence)
  • Recurrence must be confirmed by diagnostic biopsy with local pathology review or contrast-enhanced MRI. If first recurrence of GBM is documented by MRI, an interval of at least 12 weeks after the end of prior radiation therapy is required unless there is either: i) histopathologic confirmation of recurrent tumor, or ii) new enhancement on MRI outside of the radiotherapy treatment field
  • Subjects must have measurable disease preoperatively, defined as at least 1 contrast-enhancing lesion, with 2 perpendicular measurements of at least 1 cm, as per RANO criteria
  • Subjects must be at least 4 weeks post last dose of temozolomide
  • Prior gamma knife, stereotactic radiosurgery, or other focal high-dose radiotherapy is allowed but the subject must have either histopathologic confirmation of recurrent tumor, or new enhancement on MRI outside of the radiotherapy treatment field
  • Based on the pre-operative evaluation by neurosurgeon, the subject is a candidate for ≥ 80% resection of enhancing region
  • IDH mutation status of the primary tumor must be available or tumor samples must be available for pre randomization testing
  • Laboratory values adequate for patient to undergo surgery, including:
  • Platelet count ≥ 60,000/mm3
  • Hgb ≥ 10 g/dL
  • Absolute neutrophil count (ANC) ≥ 1,500/mm3
  • +8 more criteria

You may not qualify if:

  • History of more than 2 prior recurrences (including this recurrence) of GBM or AA
  • History of other malignancy, unless the patient has been disease free for at least 5 years. Adequately treated basal cell carcinoma or squamous cell skin cancer is acceptable regardless of time, as well as localized prostate carcinoma or cervical carcinoma in situ after curative treatment
  • Histologically confirmed oligodendroglioma or mixed glioma
  • Known 1p/19q co deletion
  • A contrast enhancing brain tumor that is any of the following:
  • Multi focal (defined as 2 separate areas of contrast enhancement measuring at least 1 cm in 2 planes that are not contiguous on either fluid attenuated inversion recovery (FLAIR) or T2 sequences);
  • Associated with either diffuse subependymal or leptomeningeal dissemination; or
  • \> 5 cm in any dimension
  • The subject has or had any active infection requiring systemic antibiotic, antifungal or antiviral therapy within the past 4 weeks
  • The subject has any bleeding diathesis, or must take anticoagulants, or antiplatelet agents, including nonsteroidal anti inflammatory drugs (NSAIDs), at the time of the scheduled resection that cannot be stopped for surgery
  • The subject is human immunodeficiency virus (HIV) positive
  • The subject has a history of allergy or intolerance to flucytosine
  • The subject has a gastrointestinal disease that would prevent him or her from being able to swallow or absorb flucytosine
  • The subject received cytotoxic chemotherapy within the past 4 weeks (6 weeks for nitrosoureas) of the planned surgery date
  • The subject received any investigational treatment within the past 30 days or prior immunotherapy or antibody therapy within the past 45 days.
  • +9 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (67)

Barrow Neurological Institute at Dignity Health St. Joseph's Hospital and Medical Center

Phoenix, Arizona, 85013, United States

Location

University of California, Irvine

Irvine, California, 92868, United States

Location

University of California San Diego

La Jolla, California, 92093, United States

Location

University of California, Los Angeles

Los Angeles, California, 90095, United States

Location

St. Joseph Hospital

Orange, California, 92868, United States

Location

University of California San Francisco

San Francisco, California, 94143, United States

Location

Stanford University

Stanford, California, 94305, United States

Location

University of Colorado Cancer Center

Aurora, Colorado, 80045, United States

Location

Colorado Neurological Institute

Englewood, Colorado, 80113, United States

Location

Associated Neurologists of Southern Connecticut

Fairfield, Connecticut, 06824, United States

Location

Yale University/Yale Cancer Center

New Haven, Connecticut, 06520, United States

Location

University of Florida McKnight Brain Institute

Gainesville, Florida, 32611, United States

Location

University of Miami

Miami, Florida, 33136, United States

Location

Moffitt Cancer Center

Tampa, Florida, 33612, United States

Location

NorthShore University Health System

Evanston, Illinois, 60201, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Johns Hopkins University School of Medicine

Baltimore, Maryland, 21287, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Henry Ford Health System

Detroit, Michigan, 48202, United States

Location

Abbott Northwestern Hospital / Allina Health

Minneapolis, Minnesota, 55407, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

HCA Midwest / Sarah Cannon

Kansas City, Missouri, 64132, United States

Location

Washington University St. Louis

St Louis, Missouri, 63110, United States

Location

University of Nebraska Medical Center

Omaha, Nebraska, 68198, United States

Location

JFK Medical Center Neuroscience Institute

Edison, New Jersey, 08820, United States

Location

John Theurer Cancer Center at Hackensack University

Hackensack, New Jersey, 07601, United States

Location

Overlook Medical Center

Summit, New Jersey, 07901, United States

Location

North Shore University Hospital

Lake Success, New York, 11042, United States

Location

NYU Langone Medical Center

New York, New York, 10016, United States

Location

Weill Cornell Medical College

New York, New York, 10021, United States

Location

Columbia University

New York, New York, 10032, United States

Location

University of Rochester Medical Center

Rochester, New York, 14642, United States

Location

Stony Brook University Hospital

Stony Brook, New York, 11794, United States

Location

Cincinnati's Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Ohio State University

Columbus, Ohio, 43210, United States

Location

Thomas Jefferson University

Philadelphia, Pennsylvania, 19101, United States

Location

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Sanford Research

Sioux Falls, South Dakota, 57104, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

Baylor University Medical Center

Dallas, Texas, 75246, United States

Location

Houston Methodist Hospital Outpatient Center

Houston, Texas, 77030, United States

Location

University of Texas Health Science Center at Houston (UTHealth)

Houston, Texas, 77030, United States

Location

University of Virginia Health System

Charlottesville, Virginia, 22908, United States

Location

Inova Dwight and Martha Schar Cancer Institute

Fairfax, Virginia, 22031, United States

Location

Sentara Neurosurgery Specialists

Norfolk, Virginia, 23507, United States

Location

West Virginia University

Morgantown, West Virginia, 26506, United States

Location

University of Wisconsin

Madison, Wisconsin, 53792, United States

Location

University of Alberta

Edmonton, Alberta, T6G 2B7, Canada

Location

University of British Columbia / Vancouver General Hospital

Vancouver, British Columbia, V5Z 1M9, Canada

Location

CancerCare Manitoba

Winnipeg, Manitoba, R3E 0V9, Canada

Location

London Regional Cancer Centre

London, Ontario, N6A 5W9, Canada

Location

Ottawa Hospital Regional Cancer Centre

Ottawa, Ontario, K1H 8L6, Canada

Location

Sunnybrook Hospital / Sunnybrook Research Institute

Toronto, Ontario, M4N 3M5, Canada

Location

Toronto Western Hospital

Toronto, Ontario, M5T 2S8, Canada

Location

Montreal Neurological Institute

Montreal, Quebec, H3A 2B4, Canada

Location

Jewish General Hospital

Montreal, Quebec, H3T 1E2, Canada

Location

Sherbrooke Hospital University Centre (CHUS)

Sherbrooke, Quebec, J1H 5N4, Canada

Location

Rambam Health Care

Haifa, 3109601, Israel

Location

Hadassah Medical Center

Jerusalem, 91120, Israel

Location

Tel Aviv Sourasky Medical Center

Tel Aviv, 6423906, Israel

Location

Seoul National University Hospital

Seoul, 03080, South Korea

Location

Severance Hospital

Seoul, 03722, South Korea

Location

Samsung Medical Center

Seoul, 06351, South Korea

Location

Seoul St. Mary's Hospital

Seoul, 06591, South Korea

Location

Related Publications (2)

  • Cloughesy TF, Petrecca K, Walbert T, Butowski N, Salacz M, Perry J, Damek D, Bota D, Bettegowda C, Zhu JJ, Iwamoto F, Placantonakis D, Kim L, Elder B, Kaptain G, Cachia D, Moshel Y, Brem S, Piccioni D, Landolfi J, Chen CC, Gruber H, Rao AR, Hogan D, Accomando W, Ostertag D, Montellano TT, Kheoh T, Kabbinavar F, Vogelbaum MA. Effect of Vocimagene Amiretrorepvec in Combination With Flucytosine vs Standard of Care on Survival Following Tumor Resection in Patients With Recurrent High-Grade Glioma: A Randomized Clinical Trial. JAMA Oncol. 2020 Dec 1;6(12):1939-1946. doi: 10.1001/jamaoncol.2020.3161.

  • Cloughesy TF, Landolfi J, Hogan DJ, Bloomfield S, Carter B, Chen CC, Elder JB, Kalkanis SN, Kesari S, Lai A, Lee IY, Liau LM, Mikkelsen T, Nghiemphu PL, Piccioni D, Walbert T, Chu A, Das A, Diago OR, Gammon D, Gruber HE, Hanna M, Jolly DJ, Kasahara N, McCarthy D, Mitchell L, Ostertag D, Robbins JM, Rodriguez-Aguirre M, Vogelbaum MA. Phase 1 trial of vocimagene amiretrorepvec and 5-fluorocytosine for recurrent high-grade glioma. Sci Transl Med. 2016 Jun 1;8(341):341ra75. doi: 10.1126/scitranslmed.aad9784.

MeSH Terms

Conditions

GlioblastomaAstrocytomaRecurrence

Interventions

vocimagene amiretrorepvecFlucytosineLomustineTemozolomideBevacizumab

Condition Hierarchy (Ancestors)

GliomaNeoplasms, NeuroepithelialNeuroectodermal TumorsNeoplasms, Germ Cell and EmbryonalNeoplasms by Histologic TypeNeoplasmsNeoplasms, Glandular and EpithelialNeoplasms, Nerve TissueDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

CytosinePyrimidinonesPyrimidinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsNitrosourea CompoundsUreaAmidesOrganic ChemicalsNitroso CompoundsDacarbazineTriazenesImidazolesAzolesAntibodies, Monoclonal, HumanizedAntibodies, MonoclonalAntibodiesImmunoglobulinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsSerum GlobulinsGlobulins

Study Officials

  • Timothy Cloughesy, MD

    University of California, Los Angeles

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 3, 2015

First Posted

April 10, 2015

Study Start

November 30, 2015

Primary Completion

May 31, 2019

Study Completion

December 20, 2019

Last Updated

February 7, 2020

Record last verified: 2020-02

Locations