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Ivy Brain Tumor Center Announces Completion of Enrollment in Phase 3 Gliofocus Study for Patients with Newly-Diagnosed Glioblastoma
● Gliofocus is a global, randomized, controlled Phase 3 clinical trial comparing niraparib vs temozolomide in patients with newly-diagnosed, MGMT-unmethylated glioblastoma (GBM)
● The first patient in the Gliofocus study was dosed at the Ivy Brain Tumor Center in June 2024.
● The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to niraparib for the treatment of malignant glioma, including GBM in November 2025.
The Ivy Brain Tumor Center at Barrow Neurological Institute today announced completion of randomization in the landmark Phase 3 Gliofocus study, a global trial evaluating niraparib versus standard-of-care temozolomide in adults with newly diagnosed, MGMT-unmethylated glioblastoma (GBM).
This multi-center, open-label, randomized study launched in June 2024 following encouraging results from early-phase trials conducted at the Ivy Center. In collaboration with GSK, the Gliofocus study enrolled 450 participants across more than 90 clinical sites in 11 countries.
Niraparib received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration in November 2025 for the treatment of malignant glioma, including GBM. The ODD is a special status granted by the U.S. Food and Drug Administration (FDA) to drugs and biologics intended to treat, diagnose, or prevent rare diseases or conditions. It provides development incentive program for therapies targeting rare diseases, offering regulatory and financial benefits to encourage innovation in areas of unmet medical need.
“Our program is focused on accelerating the pace of CNS oncology drug development. Using a hybrid Phase 0 study approach, we advanced this therapy from a proof-of-concept, tissue-based trial to a global Phase 3 study in under three years.”
Nader Sanai, MD, Director of the Ivy Brain Tumor Center
Dr. Sanai added, “These findings reinforce our central thesis that hybrid Phase 0 trials can meaningfully compress development timelines and expand access to promising therapies for patients with brain tumors.”
With over 250,000 new cases annually, GBM is the most common and the most lethal brain cancer in adults with less than 5% chance to survive over 5 years. Approximately 60% of these patients have tumors with an unmethylated MGMT promoter, a biomarker associated with limited benefit from standard chemotherapy with temozolomide. PARP inhibitors may be a relevant therapeutic strategy in MGMT-unmethylated GBM because these tumors rely on alternative DNA repair pathways that PARP inhibition can block, increasing sensitivity to radiotherapy. Among all the DNA repair drugs evaluated by the Ivy Center, niraparib demonstrates superior brain penetration and target engagement. The Phase 0/2 study, preliminary results directly led to the initiation of the Gliofocus Study, and if successful, this research could yield the first -approval in over twenty years for life-prolonging treatment for glioblastoma, addressing a major unmet need as the first therapy specifically developed for this underserved patient group.
Patients and care partners can learn more about Gliofocus by visiting Gliofocus.org and NCT06388733.
The Ivy Center is home to the largest Phase 0 drug development program for brain cancer worldwide. Click here for a complete list of Ivy Center clinical trials.
About Niraparib
Niraparib is an oral, once-daily PARP inhibitor with current indications in first-line and recurrent maintenance for advanced ovarian cancer. It is currently approved under the brand name Zejula for the treatment of ovarian cancer. The Gliofocus study is sponsored by the Ivy Brain Tumor Center with funding and collaboration from GSK.
About Ivy Brain Tumor Center
Ivy Brain Tumor Center at Barrow Neurological Institute in Phoenix, AZ, is a tertiary care and nonprofit translational research program that employs bold, early-phase clinical trial strategies to identify new treatments for aggressive brain tumors, including GBM. Our leading experts in neurosurgical oncology, neuro-oncology, radiation oncology, neuroradiology, neuropathology and neuroscience nursing treat more patients annually than any other brain tumor center in the United States. The Ivy Center’s Phase 0 clinical trials program is the largest in the world and enables personalized care in a fraction of the time and cost associated with traditional drug development. In addition, unlike conventional clinical trials focusing on single drugs, the Ivy Center’s accelerated program tests therapeutic combinations matched to individual patients. We leave no stone unturned in the pursuit of hope and healing. Learn more at IvyBrainTumorCenter.org. Follow the Ivy Brain Tumor Center on Facebook, Instagram, X, and LinkedIn.