Niraparib: Participants will receive niraparib 200 mg orally once daily starting on Day 1 of RT. Following completion of RT, participants will continue niraparib adjuvant therapy orally once daily on Days 1 to 28 of each 28-day cycle until progression by BICR
Temozolomide: Participants randomized to the comparator arm (Arm B) will receive SOC TMZ 75 mg/m2 orally once daily with RT starting on Day 1 of RT. Following completion of RT, participants will complete a 4-week rest period, and then receive adjuvant TMZ 150 to 200 mg/m2 orally once daily on Days 1 to 5 of each 28-day cycle until progression by BICR or for a maximum of 6 cycles.
Study summary
The goal of this Phase 3 clinical trial is to compare the efficacy of niraparib versus temozolomide (TMZ) in adult participants with newly-diagnosed, MGMT unmethylated glioblastoma multiforme (GBM). The main question it aims to answer is:
Does niraparib improve overall survival (OS) compared to TMZ?
Participants will be randomly assigned to one of two treatment arms: niraparib or TMZ.
* study drug (Niraparib) or
* comparator drug (Temozolomide - which is the standard approved treatment for MGMT unmethylated glioblastoma).
The study medication will be taken daily while receiving standard of care radiation therapy (RT) for 6-7 weeks.
Participants may continue to take the niraparib or TMZ adjuvantly as long as the cancer does not get worse or completion of 6 cycles of treatment (TMZ). A total of 450 participants will be enrolled in the study.
Participants' tasks will include:
* Complete study visits as scheduled
* Complete a diary to record study medication
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria:
* 1\. Histologic documentation of a newly-diagnosed intracranial GBM, per 2021 WHO classification guidelines through local pathology review.
* 2\. Age ≥18 years at the time of signing informed consent.
* 3\. Sufficient tissue available for retrospective central pathology review, retrospective central confirmation of MGMT promoter methylation status and genomic analysis. If insufficient tissue is available,pproval may be granted on a case-by-case basis after a review.
* 4\. Unmethylated MGMT promoter region determined locally by a validated PSQ or qMS-PCR assay compliant to local regulations. Numerical cut-off for an MGMT unmethylated tumor will be defined in the protocol.
* 5\. Suitability for SOC RT to 60 Gy in 30 fractions using ESTRO-EANO 'single phase' targeting approach \[Niyazi, 2023\], per investigator's judgment.
* 6\. No prior treatment for GBM (including brachytherapy or BCNU wafers), other than surgical resection or biopsy.
* 7\. Female participants: Not pregnant, planning to get pregnant, or breastfeeding and one of the following conditions apply: is of nonchildbearing potential or is of childbearing potential AND using a contraceptive method that is highly effective (with a failure rate of \<1% per year) from screening through at least 180 days after the last dose of study intervention. Breastfeeding is contraindicated during the study and for one month after the last dose of study intervention.
* 8\. Male participants: Must agree to the following during the study intervention period and for at least 6 months after the last dose of study intervention: refrain from donation sperm PLUS be abstinent from heterosexual activity or agree to use a male condom and be advised of the benefit for a female partner to use a contraceptive method that is highly effective (with a failure rate of \<1% per year).
* 9\. The participant must be capable of providing signed informed consent, including compliance with the requirements and restrictions listed in the ICF and in this protocol.
* 10\. Karnofsky performance status of ≥70.
* 11\. Adequate organ function
* 12\. Normal blood pressure (BP) or adequately treated and controlled hypertension (defined as systolic BP ≤140 mmHg and diastolic BP ≤90 mmHg).
* 13\. Stable or decreased dose of dexamethasone, requiring no more than 5 mg daily equivalent dose, within 7 days before randomization.
* 14\. Ability to swallow oral medications whole.
Exclusion Criteria:
* 1\. Presence of metastatic or predominant leptomeningeal disease.
* 2\. Current active pneumonitis or any history of pneumonitis requiring steroids (any dose) or immunomodulatory treatment within 90 days of planned start of the study.
* 3\. Participant is at an increased bleeding risk due to concurrent conditions (e.g., major injuries or major surgery within the past 28 days prior to start of study treatment with the exception of tumor resection).
* 4\. Any clinically significant gastrointestinal abnormalities that may alter absorption such as malabsorption syndrome or major resection of the stomach and/or bowels.
* 5\. Has cirrhosis or current unstable liver or biliary disease per investigator assessment defined by the presence of ascites, encephalopathy, coagulopathy, hypoalbuminemia, esophageal/gastric varices, or persistent jaundice. NOTE: Stable noncirrhotic chronic liver disease (including Gilbert's syndrome or asymptomatic gallstones), hepatobiliary involvement of malignancy, or chronic stable HBV infection (in a participant for whom HDV infection has been excluded) or chronic HCV infection is acceptable if the participant otherwise meets entry criteria.
* 6\. Known human immunodeficiency virus (HIV) unless participants meet all of the following criteria:
* Cluster of differentiation 4 ≥350/µL and viral load \<400 copies/mL.
* No history of acquired immunodeficiency syndrome-defining opportunistic infections within 12 months prior to enrollment.
* No history of HIV-associated malignancy for the past 5 years.
* Concurrent antiretroviral therapy as per the most current National Institutes of Health (NIH) Guidelines for the Use of Antiretroviral Agents in Adults and Adolescents Living with HIV \[NIH, 2021\] started \>4 weeks prior to study enrollment.
* 7\. MDS/AML or with features suggestive of MDS/AML.
* 8\. History of another malignancy within 2 years prior to registration. Participants with a past history of adequately treated carcinoma-in-situ, basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or superficial transitional cell carcinoma of the bladder are eligible. Participants with a history of other malignancies are eligible if they have been treated with curative intent or continuously disease free for at least 2 years after definitive primary treatment.
* 9\. Prior history of posterior reversible encephalopathy syndrome (PRES).
* 10\. Any psychological, familial, sociological, or geographical condition potentially hampering compliance with the study requirements and/or follow-up procedures.
* 11\. Inability to undergo MRI brain with IV contrast.
* 12\. Biopsy and/or resection (whichever is later) occurring \>6 weeks prior to planned RT start date.
* 13\. Surgical wound complication recovery at the time of enrollment.
* 14\. Known hypersensitivity to the components of niraparib, TMZ, or their formulation excipients.
* 15\. Known hypersensitivity to dacarbazine (DTIC).
* 16\. Prior therapy with PARP inhibitors for systemic cancer.
* 17\. Received a live vaccine within 30 days before the planned start of study intervention. Coronavirus disease 2019 (COVID-19) vaccines that do not contain live viruses are allowed. Note: mRNA and adenoviral-based COVID-19 vaccines are considered non-live.
* 18\. Received a transfusion (platelets or red blood cells) or colony-stimulating factors (e.g., granulocyte macrophage colony-stimulating factor or recombinant erythropoietin) within 4 weeks of the planned start of study intervention.
* 19\. Treatment with another investigational drug or other intervention within 5 half-lives of the investigational product.
* 20\. Treatment with tumor treating fields (e.g., Optune) for GBM.
* 21\. Presence of known isocitrate dehydrogenase (IDH) mutation.
* 22\. Presence of known H3 mutation.
* 23\. Previous diagnosis of WHO Grade 2 or 3 glioma.
Primary outcome measure(s)
Overall survival — 24 months Overall survival, defined as the time from the date of randomization to the date of death due to any cause.
Trial sites (95)
Facility
City
Region
Status
University of Alabama at Birmingham
Birmingham
Alabama
Ivy Brain Tumor Center
Phoenix
Arizona
Scripps Cancer Center
La Jolla
California
Moores UCSD Cancer Center
La Jolla
California
Smilow Cancer Hospital at Yale New Haven
Guilford
Connecticut
Indiana University
Indianapolis
Indiana
The NeuroMedical Center
Baton Rouge
Louisiana
MaineHealth Maine Medical Center Care
South Portland
Maine
Tufts Medical Center
Boston
Massachusetts
University of Michigan Rogel Cancer Center
Ann Arbor
Michigan
Allina Health
Minneapolis
Minnesota
University of Minnesota Health Clinics and Surgery Center, Minneapolis
Minneapolis
Minnesota
Saint Lukes Neuro Oncology
Kansas City
Missouri
Washington University, School of Medicine
St Louis
Missouri
Jersey Shore University Medical Center
Neptune City
New Jersey
Atlantic Health System
Summit
New Jersey
Northwell Health
New Hyde Park
New York
New York University Ambulatory Care Center
New York
New York
Montefiore Medical Center
The Bronx
New York
Duke Cancer Center Brain Tumor Clinic
Durham
North Carolina
Wake Forest Baptist Health
Winston-Salem
North Carolina
University of Cincinnati Cancer Institute
Cincinnati
Ohio
The Cleveland Clinic Foundation
Cleveland
Ohio
The Ohio State University
Columbus
Ohio
Providence Portland Medical Center
Portland
Oregon
Thomas Jefferson University
Philadelphia
Pennsylvania
University of Pittsburgh Medical Center Health System
Pittsburgh
Pennsylvania
Medical University of South Carolina - Department of Neurosurgery
Charleston
South Carolina
Baylor Scott & White Health
Temple
Texas
The University of Vermont Medical Center
Burlington
Vermont
University of Washington Medical Center
Seattle
Washington
University of Wisconsin Cancer Center
Madison
Wisconsin
St Vincent's Hospital Melbourne
Fitzroy
Victoria
Austin Health
Heidelberg
Victoria
Peter MacCallum Cancer Centre
Melbourne
Victoria
Bayside Health (formerly The Alfred Hospital)
Melbourne
Victoria
BC Cancer - Vancouver
Vancouver
British Columbia
Sunnybrook Health Sciences Centre
Toronto
Ontario
University Health Network - Princess Margaret Cancer Centre
Toronto
Ontario
CHUM (Centre hospitalier de l'Université de Montréal)
Montreal
Quebec
+ 55 more sites — see the full list on the official registry below.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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