Clinical Trials

FIREFLY-1 is a pivotal Phase 2, multicenter open-label study designed to evaluate the safety and efficacy of tovorafenib in patients aged 6 months to 25 years with relapsed or progressive low-grade glioma or solid tumors harboring a known activating BRAF alteration. The low-grade glioma arms have completed enrollment. Results from the low-grade glioma registrational arm 1 ‘The type II RAF inhibitor tovorafenib in relapsed/refractory pediatric low-grade glioma: the phase 2 FIREFLY-1 trial’ were published in Nature Medicine in 2024.

Additional information about FIREFLY-1 may be found at ClinicalTrials.gov, using identifier NCT04775485.

FIREFLY-2 is a pivotal Phase 3, randomized, multicenter open-label trial to evaluate the efficacy, safety, and tolerability of tovorafenib monotherapy, an investigational, oral, RAF inhibitor versus standard of care (SoC) chemotherapy in patients with RAF-altered pediatric low-grade glioma requiring front-line systemic therapy. The primary endpoint will be overall response rate (ORR) based upon Response Assessment for Neuro-Oncology (RANO) criteria. Secondary endpoints include progression-free survival (PFS) by independent review committee (IRC) per RANO-LGG, duration of response (DOR) assessed by IRC per RANO-LGG, and overall survival.

Additional information about the FIREFLY-2 trial may be found at ClinicalTrials.gov, using identifier NCT05566795.