The Toca 5 Trial: Toca 511 & Toca FC Versus Standard of Care in Patients With Recurrent High Grade Glioma

NCT ID: NCT02414165

Last Updated: 2020-02-07

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2/PHASE3

Total Enrollment

403 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-11-30

Study Completion Date

2019-12-20

Brief Summary

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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

Detailed Description

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Conditions

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Glioblastoma Multiforme Anaplastic Astrocytoma

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Toca 511/Toca FC

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.

Group Type EXPERIMENTAL

Toca 511

Intervention Type BIOLOGICAL

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.

Toca FC

Intervention Type DRUG

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

Lomustine, Temozolomide, or Bevacizumab

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

Group Type ACTIVE_COMPARATOR

Lomustine

Intervention Type DRUG

Temozolomide

Intervention Type DRUG

Bevacizumab

Intervention Type BIOLOGICAL

Interventions

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Toca 511

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.

Intervention Type BIOLOGICAL

Toca FC

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

Intervention Type DRUG

Lomustine

Intervention Type DRUG

Temozolomide

Intervention Type DRUG

Bevacizumab

Intervention Type BIOLOGICAL

Other Intervention Names

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vocimagene amiretrorepvec RRV retroviral replicating vector flucytosine 5-FC 5-fluorocytosine CCNU CeeNU Temodar Avastin

Eligibility Criteria

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Inclusion Criteria

1. Subject has given written informed consent
2. Subject is between 18 years old and 75 years old, inclusive
3. Subjects must have histologically proven GBM or AA and:

1. 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)
2. Must be in first or second recurrence (including this recurrence)
3. 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
4. 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
5. Subjects must be at least 4 weeks post last dose of temozolomide
6. 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
7. Based on the pre-operative evaluation by neurosurgeon, the subject is a candidate for ≥ 80% resection of enhancing region
8. IDH mutation status of the primary tumor must be available or tumor samples must be available for pre randomization testing
9. 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
* Absolute lymphocyte count (ALC) ≥ 500/mm3
* Adequate liver function, including:

* Total bilirubin ≤ 1.5 x ULN (unless has Gilbert's syndrome)
* ALT ≤ 2.5 x ULN f. Estimated glomerular filtration rate of at least 50 mL/min by the Cockcroft Gault formula
10. Women of childbearing potential (≥12 months of non-therapy-induced amenorrhea or surgically sterile) must have had a negative serum pregnancy test within the past 21 days and must use a birth control method in addition to barrier methods (condoms).
11. Subject or subject's partner is willing to use condoms for 12 months after receiving Toca 511 or until there is no evidence of the virus in his/her blood, whichever is longer.
12. The subject has a KPS ≥ 70
13. The subject is willing and able to abide by the protocol

Exclusion Criteria

1. History of more than 2 prior recurrences (including this recurrence) of GBM or AA
2. 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
3. Histologically confirmed oligodendroglioma or mixed glioma
4. Known 1p/19q co deletion
5. 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
6. The subject has or had any active infection requiring systemic antibiotic, antifungal or antiviral therapy within the past 4 weeks
7. 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
8. The subject is human immunodeficiency virus (HIV) positive
9. The subject has a history of allergy or intolerance to flucytosine
10. The subject has a gastrointestinal disease that would prevent him or her from being able to swallow or absorb flucytosine
11. The subject received cytotoxic chemotherapy within the past 4 weeks (6 weeks for nitrosoureas) of the planned surgery date
12. The subject received any investigational treatment within the past 30 days or prior immunotherapy or antibody therapy within the past 45 days.
13. The subject is pregnant or breast feeding
14. The subject intends to undergo treatment with the Gliadel® wafer at the time of this surgery or has received the Gliadel® wafer \< 30 days from W1D1 (surgery)
15. The subject has received bevacizumab for their disease unless in the context of primary therapy for newly diagnosed glioma
16. For subjects planned to potentially receive bevacizumab, they have no evidence of uncontrolled hypertension (defined as a blood pressure of ≥ 150 mm Hg systolic and/or ≥ 100 mm Hg diastolic on medication) or active GI perforation
17. The subject has received systemic dexamethasone continuously at a dose \> 8 mg/day for 8 weeks prior to the date of the screening assessment
18. Severe pulmonary, cardiac or other systemic disease, specifically:

* New York Heart Association \> Grade 2 congestive heart failure within 6 months prior to study entry, unless asymptomatic and well controlled with medication
* Uncontrolled or significant cardiovascular disease, clinically significant ventricular arrhythmia (such as ventricular tachycardia, ventricular fibrillation, or Torsades des pointes), clinically significant pulmonary disease (such as ≥ Grade 2 dyspnea, according to CTCAE 4.03)
* Subjects who have any other disease, either metabolic or psychological, which as per Investigator assessment may affect the subject's compliance or place the subject at higher risk of potential treatment complications
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Tocagen Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Timothy Cloughesy, MD

Role: PRINCIPAL_INVESTIGATOR

University of California, Los Angeles

Locations

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

Phoenix, Arizona, United States

Site Status

University of California, Irvine

Irvine, California, United States

Site Status

University of California San Diego

La Jolla, California, United States

Site Status

University of California, Los Angeles

Los Angeles, California, United States

Site Status

St. Joseph Hospital

Orange, California, United States

Site Status

University of California San Francisco

San Francisco, California, United States

Site Status

Stanford University

Stanford, California, United States

Site Status

University of Colorado Cancer Center

Aurora, Colorado, United States

Site Status

Colorado Neurological Institute

Englewood, Colorado, United States

Site Status

Associated Neurologists of Southern Connecticut

Fairfield, Connecticut, United States

Site Status

Yale University/Yale Cancer Center

New Haven, Connecticut, United States

Site Status

University of Florida McKnight Brain Institute

Gainesville, Florida, United States

Site Status

University of Miami

Miami, Florida, United States

Site Status

Moffitt Cancer Center

Tampa, Florida, United States

Site Status

NorthShore University Health System

Evanston, Illinois, United States

Site Status

University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status

Johns Hopkins University School of Medicine

Baltimore, Maryland, United States

Site Status

University of Michigan

Ann Arbor, Michigan, United States

Site Status

Henry Ford Health System

Detroit, Michigan, United States

Site Status

Abbott Northwestern Hospital / Allina Health

Minneapolis, Minnesota, United States

Site Status

University of Minnesota

Minneapolis, Minnesota, United States

Site Status

HCA Midwest / Sarah Cannon

Kansas City, Missouri, United States

Site Status

Washington University St. Louis

St Louis, Missouri, United States

Site Status

University of Nebraska Medical Center

Omaha, Nebraska, United States

Site Status

JFK Medical Center Neuroscience Institute

Edison, New Jersey, United States

Site Status

John Theurer Cancer Center at Hackensack University

Hackensack, New Jersey, United States

Site Status

Overlook Medical Center

Summit, New Jersey, United States

Site Status

North Shore University Hospital

Lake Success, New York, United States

Site Status

NYU Langone Medical Center

New York, New York, United States

Site Status

Weill Cornell Medical College

New York, New York, United States

Site Status

Columbia University

New York, New York, United States

Site Status

University of Rochester Medical Center

Rochester, New York, United States

Site Status

Stony Brook University Hospital

Stony Brook, New York, United States

Site Status

Cincinnati's Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

University Hospitals Cleveland Medical Center

Cleveland, Ohio, United States

Site Status

Cleveland Clinic

Cleveland, Ohio, United States

Site Status

Ohio State University

Columbus, Ohio, United States

Site Status

Thomas Jefferson University

Philadelphia, Pennsylvania, United States

Site Status

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status

Sanford Research

Sioux Falls, South Dakota, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

Baylor University Medical Center

Dallas, Texas, United States

Site Status

Houston Methodist Hospital Outpatient Center

Houston, Texas, United States

Site Status

University of Texas Health Science Center at Houston (UTHealth)

Houston, Texas, United States

Site Status

University of Virginia Health System

Charlottesville, Virginia, United States

Site Status

Inova Dwight and Martha Schar Cancer Institute

Fairfax, Virginia, United States

Site Status

Sentara Neurosurgery Specialists

Norfolk, Virginia, United States

Site Status

West Virginia University

Morgantown, West Virginia, United States

Site Status

University of Wisconsin

Madison, Wisconsin, United States

Site Status

University of Alberta

Edmonton, Alberta, Canada

Site Status

University of British Columbia / Vancouver General Hospital

Vancouver, British Columbia, Canada

Site Status

CancerCare Manitoba

Winnipeg, Manitoba, Canada

Site Status

London Regional Cancer Centre

London, Ontario, Canada

Site Status

Ottawa Hospital Regional Cancer Centre

Ottawa, Ontario, Canada

Site Status

Sunnybrook Hospital / Sunnybrook Research Institute

Toronto, Ontario, Canada

Site Status

Toronto Western Hospital

Toronto, Ontario, Canada

Site Status

Montreal Neurological Institute

Montreal, Quebec, Canada

Site Status

Jewish General Hospital

Montreal, Quebec, Canada

Site Status

Sherbrooke Hospital University Centre (CHUS)

Sherbrooke, Quebec, Canada

Site Status

Rambam Health Care

Haifa, , Israel

Site Status

Hadassah Medical Center

Jerusalem, , Israel

Site Status

Tel Aviv Sourasky Medical Center

Tel Aviv, , Israel

Site Status

Seoul National University Hospital

Seoul, , South Korea

Site Status

Severance Hospital

Seoul, , South Korea

Site Status

Samsung Medical Center

Seoul, , South Korea

Site Status

Seoul St. Mary's Hospital

Seoul, , South Korea

Site Status

Countries

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United States Canada Israel South Korea

References

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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.

Reference Type DERIVED
PMID: 33119048 (View on PubMed)

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.

Reference Type DERIVED
PMID: 27252174 (View on PubMed)

Other Identifiers

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FD-R-5732

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

Tg 511-15-01

Identifier Type: -

Identifier Source: org_study_id

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