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Evaluating Tovorafenib for Pediatric Low-Grade Glioma and Advanced Solid Tumors

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 RAF-Altered, Recurrent or Progressive Low-Grade Glioma and Advanced Solid Tumors

Phase 2 Interventional Day One Biopharmaceuticals, Inc. · NCT04775485

This study is testing a new drug called Tovorafenib to see if it can help kids and young adults with certain brain tumors and advanced solid tumors feel better.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment141 (estimated)
Ages6 Months to 25 Years
SexAll
SponsorDay One Biopharmaceuticals, Inc. Industry-sponsored
Locations35 sites (San Francisco, California and 34 other locations)
Trial IDNCT04775485 on ClinicalTrials.gov

What this trial studies

This Phase 2, multi-center, open-label study aims to assess the safety and efficacy of Tovorafenib in pediatric and young adult participants with relapsed or progressive low-grade glioma and advanced solid tumors. Participants will undergo genomic testing to identify qualifying alterations before enrollment. The study includes a screening period, treatment phase, long-term extension, and follow-up assessments to monitor safety and efficacy outcomes.

Who should consider this trial

Good fit: Ideal candidates are pediatric and young adult patients with relapsed or progressive low-grade glioma or advanced solid tumors with specific genomic alterations.

Not a fit: Patients with tumors that have additional known activating molecular alterations or those without radiographically recurrent disease may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for children and young adults with challenging low-grade gliomas and advanced solid tumors.

How similar studies have performed: Other studies have shown promise with targeted therapies in similar patient populations, suggesting potential for success with this approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Low Grade Glioma \& Low-Grade Glioma Extension: a relapsed or progressive LGG with documented known activating BRAF alteration.
* Advanced Solid Tumor: locally advanced or metastatic solid tumor with documented known or expected to be activating RAF fusion.
* Participants must have histopathologic verification of malignancy at either original diagnosis or relapse.
* Must have received at least one line of prior systemic therapy and have documented evidence of radiographic progression.
* Must have at least 1 measurable lesion as defined by RANO (Arms 1 \& 2) or RECIST v1.1 (Arm 3) criteria

Exclusion Criteria:

* Participant's tumor has additional previously-known activating molecular alterations.
* Participant has symptoms of without radiographically recurrent or radiographically progressive disease.
* Known or suspected diagnosis of neurofibromatosis type 1 (NF-1) via genetic testing or current diagnostic criteria.

Other inclusion/exclusion criteria as stipulated by protocol may apply

Where this trial is running

San Francisco, California and 34 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Low-grade GliomaAdvanced Solid TumorFIREFLY-1DAY101Tovorafenib
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.