Niraparib/TTFields in GBM
Primary Purpose
Glioblastoma, Recurrent Glioblastoma, GBM
Status
Active
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Niraparib
Optune
Planned surgical resection
Sponsored by
About this trial
This is an interventional treatment trial for Glioblastoma
Eligibility Criteria
Inclusion Criteria:
- Histopathologically or molecularly (per c-IMPACT NOW criteria) proven diagnosis of glioblastoma which is recurrent following radiation therapy (prior dose must have been between 40 and 75 Gy).
- Tumor O-6-methylguanine-deoxyribonucleic acid (DNA) methyltransferase (MGMT) methylation status must be available from any prior GBM tumor specimen.
- Patients must have measurable contrast-enhancing disease (defined by at least 1cm x 1cm) by magnetic resonance imaging (MRI) imaging within 28 days of starting study treatment.
- Patients may have had treatment for an unlimited number of prior relapses.
- Patients must have recovered from severe toxicity of prior therapy.
- Patients must be able to swallow oral medications.
- Karnofsky performance status >= 60.
- Life expectancy >3 months.
- Adequate hematologic parameters.
- Adequate hepatic function within 7 days prior to start of study treatment.
- Adequate renal function within 7 days prior to start of study treatment.
- Reproductive Status
- Women - negative serum or urine pregnancy test
- Men and Women - must agree to an adequate method to avoid pregnancy
- Participant must agree to not donate blood during the study or for 90 days after the last dose of niraparib.
- Participant must, in the opinion of the Investigator, be able to comply with study procedures, including use of the Optune device.
- Cohort B (surgical) patients only: patients must be undergoing surgery that is clinically indicated as determined by their care providers.
- Cohort B (surgical) patients only: patients must have a tumor tissue form indicating availability of archived tissue from a previous surgery for glioblastoma.
- Patients must be able to understand the study procedures and agree to participate in the study by providing written informed consent (or have legally authorized representative sign on patient's behalf if patient physically unable to sign consent due to neurologic deficit).
Exclusion Criteria:
- Age < 22 years.
- Prior treatment with tumor-treating fields therapy (Optune) within the past 6 months.
- Prior treatment with a PARP inhibitor.
- Known history or current diagnosis of myelodysplastic syndrome (MDS)/acute myeloid leukemia (AML).
- Patients with infratentorial tumor.
- Participant has had any known Grade 3 or 4 anemia, neutropenia or thrombocytopenia due to prior chemotherapy that persisted > 4 weeks and was related to the most recent treatment.
- Participant must not have a serious, uncontrolled medical disorder, nonmalignant systemic disease, or active, uncontrolled infection. Examples include, but are not limited to, uncontrolled ventricular arrhythmia, recent (within 90 days) myocardial infarction, uncontrolled major seizure disorder, unstable spinal cord compression, superior vena cava syndrome, or any psychiatric disorder that prohibits obtaining informed consent.
- Implanted pacemaker, defibrillator or deep brain stimulator, other implanted electronic devices in the brain. Non-programmable shunts are allowed. Patients with a programmable shunt are excluded.
- Skull defects.
- Known hypersensitivity to conductive hydrogels or known hypersensitivity to niraparib components or excipients.
- Patients with gastrointestinal disorders or abnormalities that would interfere with absorption of study treatment.
- Prisoners or subjects who are involuntarily incarcerated.
- Subjects who are compulsorily detained for treatment of either a psychiatric or physical (eg, infectious disease) illness.
- Participant must not be simultaneously enrolled in any interventional clinical trial.
Sites / Locations
- Hospital of the University of Pennsylvania
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Active Comparator
Arm Label
Cohort A
Cohort B
Arm Description
Cohort A is for subjects with recurrent glioblastoma who do not have clinical indication for surgical resection of the recurrent tumor. Subjects in Cohort A will initiate and continue TTFields therapy for 5-7 days prior to starting niraparib.
Cohort B is for subjects with recurrent glioblastoma who have a clinical indication for surgical resection of the recurrent tumor. Subjects in Cohort B will receive TTFields for 5-7 days prior to planned surgical resection, undergo surgical resection, resume TTFields postoperatively, and initiate niraparib 5- 7 days after starting TTFields postoperatively.
Outcomes
Primary Outcome Measures
Disease control, defined as achievement of either CR, PR, or SD, as defined by modified Response Assessment in Neuro-Oncology (mRANO) criteria.
Complete response (CR) is seen as the disappearance of all enhancing measurable and non-measurable disease sustained for at least 4 weeks.
Partial response (PR) is ≥50% decrease in sum of products of perpendicular diameters or ≥65% decrease in total volume of all measurable enhancing lesions compared with baseline, sustained for at least 4 weeks.
Stable disease (SD), must be present on two consecutive MRI scans, with the 2nd/confirmatory MRI performed at least 16 weeks after starting treatment.
Secondary Outcome Measures
Number of AEs (Adverse Events)
Assessed by the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0, all events will be recorded from the time a subject has signed the informed consent form.
Duration of disease control.
Achieving a confirmed best response to treatment of stable disease (SD), partial response (PR), or complete response (CR).
Objective radiographic response (ORR)
ORR is defined by mRANO criteria, and duration of response.
Progression-free survival (PFS)
Progression-free survival (PFS) is defined as the time from date of enrollment until the earliest date of disease progression (as determined by mRANO criteria) or death due to any cause.
Overall survival (OS)
Overall survival (OS) is defined as the time from date of enrollment until death from any cause.
Full Information
NCT ID
NCT04221503
First Posted
December 17, 2019
Last Updated
January 30, 2023
Sponsor
University of Pennsylvania
Collaborators
Tesaro, Inc., NovoCure Ltd.
1. Study Identification
Unique Protocol Identification Number
NCT04221503
Brief Title
Niraparib/TTFields in GBM
Official Title
A Phase II Study Evaluating the Efficacy and Safety of Niraparib and Tumor-Treating Fields in Recurrent Glioblastoma
Study Type
Interventional
2. Study Status
Record Verification Date
January 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
December 30, 2019 (Actual)
Primary Completion Date
December 2025 (Anticipated)
Study Completion Date
December 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University of Pennsylvania
Collaborators
Tesaro, Inc., NovoCure Ltd.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
Yes
Device Product Not Approved or Cleared by U.S. FDA
Yes
5. Study Description
Brief Summary
Evaluating the efficacy and safety of niraparib and Tumor-Treating Fields (TTFields) in recurrent glioblastoma (GBM).
Detailed Description
Tumor-treating fields (TTFields) causes downregulation of BRCA1 signaling and reduced deoxyribonucleic acid (DNA) double-strand break repair capacity. Tumors that are deficient in the homologous recombination DNA damage repair pathway are highly sensitive to blockade of the repair of single strand DNA breaks via poly-ADP ribose polymerase (PARP) inhibition. This is a study of niraparib, a PARP inhibitor, in combination with tumor-treating fields for recurrent glioblastoma. We hypothesize that tumor-treating fields will induce a state of "BRCAness" in the glioma tumor cells, thus sensitizing them to PARP inhibition and resulting in tumor cell death.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Glioblastoma, Recurrent Glioblastoma, GBM
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
30 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Cohort A
Arm Type
Experimental
Arm Description
Cohort A is for subjects with recurrent glioblastoma who do not have clinical indication for surgical resection of the recurrent tumor. Subjects in Cohort A will initiate and continue TTFields therapy for 5-7 days prior to starting niraparib.
Arm Title
Cohort B
Arm Type
Active Comparator
Arm Description
Cohort B is for subjects with recurrent glioblastoma who have a clinical indication for surgical resection of the recurrent tumor. Subjects in Cohort B will receive TTFields for 5-7 days prior to planned surgical resection, undergo surgical resection, resume TTFields postoperatively, and initiate niraparib 5- 7 days after starting TTFields postoperatively.
Intervention Type
Drug
Intervention Name(s)
Niraparib
Other Intervention Name(s)
ZEJULA
Intervention Description
Niraparib ([3S]-3-[4-{7-(aminocarbonyl)-2H-indazol-2-yl} phenyl] piperidine [tosylate monohydrate salt]) is an orally available, potent, highly selective poly (adenosine diphosphate [ADP]-ribose) polymerase (PARP) -1 and -2 inhibitor. The niraparib drug product is provided as 100-mg capsules filled with a dry blend of niraparib tosylate monohydrate, lactose monohydrate, and magnesium stearate in a hard gelatin capsule.
Intervention Type
Device
Intervention Name(s)
Optune
Other Intervention Name(s)
Tumor Treatment Fields (TTFields)
Intervention Description
Optune, which is manufactured by Novocure, is a portable battery or power supply operated device which produces alternating electrical fields, called tumor treatment fields ("TTFields") within the human body. TTFields are applied to the patient by electrically-insulated surface transducer arrays. TTFields disrupt the rapid cell division exhibited by cancer cells.
Intervention Type
Procedure
Intervention Name(s)
Planned surgical resection
Other Intervention Name(s)
Tumor Resection
Intervention Description
Surgery of supratentorial glioblastoma (GBM).
Primary Outcome Measure Information:
Title
Disease control, defined as achievement of either CR, PR, or SD, as defined by modified Response Assessment in Neuro-Oncology (mRANO) criteria.
Description
Complete response (CR) is seen as the disappearance of all enhancing measurable and non-measurable disease sustained for at least 4 weeks.
Partial response (PR) is ≥50% decrease in sum of products of perpendicular diameters or ≥65% decrease in total volume of all measurable enhancing lesions compared with baseline, sustained for at least 4 weeks.
Stable disease (SD), must be present on two consecutive MRI scans, with the 2nd/confirmatory MRI performed at least 16 weeks after starting treatment.
Time Frame
When termination of the study or 5 years after removal from protocol therapy, whichever occurs first.
Secondary Outcome Measure Information:
Title
Number of AEs (Adverse Events)
Description
Assessed by the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0, all events will be recorded from the time a subject has signed the informed consent form.
Time Frame
When termination of the study or 5 years after removal from protocol therapy, whichever occurs first.
Title
Duration of disease control.
Description
Achieving a confirmed best response to treatment of stable disease (SD), partial response (PR), or complete response (CR).
Time Frame
When termination of the study or 5 years after removal from protocol therapy, whichever occurs first.
Title
Objective radiographic response (ORR)
Description
ORR is defined by mRANO criteria, and duration of response.
Time Frame
When termination of the study or 5 years after removal from protocol therapy, whichever occurs first.
Title
Progression-free survival (PFS)
Description
Progression-free survival (PFS) is defined as the time from date of enrollment until the earliest date of disease progression (as determined by mRANO criteria) or death due to any cause.
Time Frame
When termination of the study or 5 years after removal from protocol therapy, whichever occurs first.
Title
Overall survival (OS)
Description
Overall survival (OS) is defined as the time from date of enrollment until death from any cause.
Time Frame
When termination of the study or 5 years after removal from protocol therapy, whichever occurs first.
Other Pre-specified Outcome Measures:
Title
Objective response rate (ORR) associations.
Description
Association between pre-treatment tumor genomics, transcriptomics, proteomics, and prior bevacizumab use and ORR.
Time Frame
When termination of the study or 5 years after removal from protocol therapy, whichever occurs first.
Title
Progression-free survival (PFS) associations
Description
Association between pre-treatment tumor genomics, transcriptomics, proteomics, and prior bevacizumab use and PFS.
Time Frame
When termination of the study or 5 years after removal from protocol therapy, whichever occurs first.
Title
Overall survival (OS) associations
Description
Association between pre-treatment tumor genomics, transcriptomics, proteomics, and prior bevacizumab use and OS.
Time Frame
When termination of the study or 5 years after removal from protocol therapy, whichever occurs first.
10. Eligibility
Sex
All
Minimum Age & Unit of Time
22 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Histopathologically or molecularly (per c-IMPACT NOW criteria) proven diagnosis of glioblastoma which is recurrent following radiation therapy (prior dose must have been between 40 and 75 Gy).
Tumor O-6-methylguanine-deoxyribonucleic acid (DNA) methyltransferase (MGMT) methylation status must be available from any prior GBM tumor specimen.
Patients must have measurable contrast-enhancing disease (defined by at least 1cm x 1cm) by magnetic resonance imaging (MRI) imaging within 28 days of starting study treatment.
Patients may have had treatment for an unlimited number of prior relapses.
Patients must have recovered from severe toxicity of prior therapy.
Patients must be able to swallow oral medications.
Karnofsky performance status >= 60.
Life expectancy >3 months.
Adequate hematologic parameters.
Adequate hepatic function within 7 days prior to start of study treatment.
Adequate renal function within 7 days prior to start of study treatment.
Reproductive Status
Women - negative serum or urine pregnancy test
Men and Women - must agree to an adequate method to avoid pregnancy
Participant must agree to not donate blood during the study or for 90 days after the last dose of niraparib.
Participant must, in the opinion of the Investigator, be able to comply with study procedures, including use of the Optune device.
Cohort B (surgical) patients only: patients must be undergoing surgery that is clinically indicated as determined by their care providers.
Cohort B (surgical) patients only: patients must have a tumor tissue form indicating availability of archived tissue from a previous surgery for glioblastoma.
Patients must be able to understand the study procedures and agree to participate in the study by providing written informed consent (or have legally authorized representative sign on patient's behalf if patient physically unable to sign consent due to neurologic deficit).
Exclusion Criteria:
Age < 22 years.
Prior treatment with tumor-treating fields therapy (Optune) within the past 6 months.
Prior treatment with a PARP inhibitor.
Known history or current diagnosis of myelodysplastic syndrome (MDS)/acute myeloid leukemia (AML).
Patients with infratentorial tumor.
Participant has had any known Grade 3 or 4 anemia, neutropenia or thrombocytopenia due to prior chemotherapy that persisted > 4 weeks and was related to the most recent treatment.
Participant must not have a serious, uncontrolled medical disorder, nonmalignant systemic disease, or active, uncontrolled infection. Examples include, but are not limited to, uncontrolled ventricular arrhythmia, recent (within 90 days) myocardial infarction, uncontrolled major seizure disorder, unstable spinal cord compression, superior vena cava syndrome, or any psychiatric disorder that prohibits obtaining informed consent.
Implanted pacemaker, defibrillator or deep brain stimulator, other implanted electronic devices in the brain. Non-programmable shunts are allowed. Patients with a programmable shunt are excluded.
Skull defects.
Known hypersensitivity to conductive hydrogels or known hypersensitivity to niraparib components or excipients.
Patients with gastrointestinal disorders or abnormalities that would interfere with absorption of study treatment.
Prisoners or subjects who are involuntarily incarcerated.
Subjects who are compulsorily detained for treatment of either a psychiatric or physical (eg, infectious disease) illness.
Participant must not be simultaneously enrolled in any interventional clinical trial.
Facility Information:
Facility Name
Hospital of the University of Pennsylvania
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
12. IPD Sharing Statement
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Niraparib/TTFields in GBM
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