Title: FIREFLY-1: A Phase 2, Open-Label, Multicenter Study to Evaluate the Safety and Efficacy of the Oral Pan-RAF Inhibitor DAY101 in Pediatric Patients With BRAF-Altered, Recurrent or Progressive Low-Grade Glioma

Responsible Party: DOT Therapeutics-1 Inc.

Collaborator: Pacific Pediatric Neuro-Oncology Consortium

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Description: Researchers are conducting a phase 2, multicenter study to determine the safety and efficacy of an oral pan-RAF inhibitor, DAY101, in pediatric, adolescent, and young adult patients with recurrent or progressive low-grade glioma harboring a BRAF alteration. 

The trial is seeking to enroll roughly 60 patients. Patients will receive DAY101 for up to 26 cycles over 24 months. Patients will receive treatment until radiographic evidence of disease progression, unacceptable toxicity, patient withdrawal of consent, or death. 

The primary outcome measure of the trial is overall response rate by independent radiology review committee based on RANO criteria. Secondary outcome measures include an assessment of the safety and tolerability of DAY101 and the relationship between pharmacokinetics and drug effects.

Patients must be 6 months to 25 years of age. They must have relapsed or progressive low-grade glioma with a known activating BRAF alteration and must have received at least 1 line of systemic therapy with evidence of radiographic progression. For a complete list of eligibility criteria, please see the reference.

Status: Recruiting

This study is sponsored by Day One Biopharmaceuticals, Inc.


Clinicaltrials.gov. A study to evaluate DAY101 in pediatric and young adult patients with relapsed or progressive low-grade glioma (FIREFLY-1). NCT04775485. Accessed May 23, 2022.