The Gliofocus Study, a global Phase 3 clinical trial evaluating niraparib against temozolomide in patients with newly diagnosed MGMT-unmethylated glioblastoma, has expanded to include new sites in Europe and the United States. This expansion aims to accelerate the assessment of niraparib as a potential treatment for this aggressive form of brain cancer.
The newly activated sites include Groupe Hospitalier Pitié-Salpêtrière in Paris, France; Hospital Universitario 12 de Octubre in Madrid, Spain; The NeuroMedical Center Clinic in Baton Rouge, Louisiana; and UC San Diego Moores Cancer Center in La Jolla, California. The Gliofocus Study (NCT06388733) is an open-label, randomized trial comparing niraparib, a PARP1/2 selective inhibitor, to temozolomide in adult patients with newly diagnosed MGMT-unmethylated glioblastoma.
Rationale for Niraparib in Glioblastoma
Glioblastoma is one of the deadliest human cancers, with approximately 250,000 new cases diagnosed worldwide each year. In about 60% of glioblastoma tumors, the MGMT promoter is unmethylated, rendering temozolomide, the standard first-line chemotherapy, less effective. The historical median overall survival for these patients is only 12.7 months. Niraparib, an oral, once-daily PARP inhibitor, has shown promise in early-phase trials.
Dr. Juan M. Sepúlveda, study investigator at Hospital Universitario 12 de Octubre, noted, "Patients with MGMT-unmethylated glioblastoma have little benefit from chemotherapy such as temozolomide, and there is an urgent need to find new drugs that improve the efficacy of radiotherapy. PARP inhibition has a strong rationale, and niraparib is a safe drug that can be easily combined with radiotherapy in brain tumours."
Promising Early-Phase Data
Positive interim data from the Ivy Center’s Phase 0/2 clinical trial of niraparib in patients with MGMT-unmethylated glioblastoma prompted its advancement to the Phase 3 setting. In the ongoing early-phase trial, niraparib achieved pharmacologically relevant concentrations in glioblastoma tumor tissue, exceeding those of other PARP inhibitors. This led to a median overall survival of 20.3 months, compared to the historical 14 months observed with standard of care. The safety profile of niraparib was consistent with previous findings, with no new concerns identified.
Trial Design and Objectives
The Gliofocus Study, supported by GSK, aims to enroll 450 participants across more than 100 clinical sites in 13 countries. Along with the newly activated U.S. sites, additional locations in Texas, Minnesota, Maine, and Vermont are expected to open soon. The primary objective of the study is to evaluate the efficacy of niraparib compared to temozolomide in this patient population. Final results are intended to be submitted to regulatory authorities to support the potential approval of niraparib for newly diagnosed MGMT-unmethylated glioblastoma.
Dr. David Piccioni, study investigator at UC San Diego Moores Cancer Center, stated, "The Gliofocus Study provides an opportunity for patients to access cutting-edge research that has the potential to change glioblastoma treatment in the future. Our teams are dedicated to bringing the latest scientific advancements to our community and providing new treatment options to patients and families."
