NCT04775485 · Day One Biopharmaceuticals, Inc.
A Study to Evaluate Tovorafenib in Pediatric and Young Adult Participants With Relapsed or Progressive Low-Grade Glioma and Advance Solid Tumors
(FIREFLY-1)
What this study is about
This is a Phase 2, multi center, where both patients and doctors know the treatment given study to evaluate the safety and effectiveness of Type II RAF (tovorafenib) in pediatric participants with low-grade glioma or advanced solid tumors.
View original scientific description
This is a Phase 2, multi center, open-label study to evaluate the safety and efficacy of Type II RAF (tovorafenib) in pediatric participants with low-grade glioma or advanced solid tumors. Qualifying genomic alterations will be identified through molecular assays as routinely performed at Clinical Laboratory Improvement Amendments (CLIA) of 1988 or other similarly certified laboratories prior to enrollment into any of the arms.
Interventions
DRUG
Tovorafenib
Tovorafenib is an oral Type II RAF kinase inhibitor available in 100 mg immediate-release tablet or 25 mg/milliliter (mL) powder for reconstitution.
Primary outcome measures
Arm 1: Overall response rate
Time frame: Up to 48 months
ORR is defined as percentage of participants with best overall confirmed response of complete response (CR) or partial response (PR) by the Response Assessment in Neuro-Oncology - high-grade glioma (RANO-HGG) criteria.
Arm 2: Number of participants reporting adverse events
Time frame: Up to 48 months
An adverse event (AE) is any untoward medical occurrence in a participant or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention.
Arm 2: Number of participants with clinically significant changes in clinical chemistry parameters
Time frame: Up to 48 months
Arm 2: Number of participants with clinically significant changes in hematology parameters
Time frame: Up to 48 months
Arm 3: Overall response rate
Time frame: Up to 48 months
Determined by the treating investigator and measured by Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 or RANO-HGG criteria, as appropriate.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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 stipulat
Where
- San Francisco, California
- Washington D.C., District of Columbia
- Chicago, Illinois
- Baltimore, Maryland
- Boston, Massachusetts
- Ann Arbor, Michigan
- St Louis, Missouri
- New York, New York
- Durham, North Carolina
- Portland, Oregon
- Philadelphia, Pennsylvania
- Houston, Texas
And 2 more locations — see the full list below.
Collaborators
Pacific Pediatric Neuro-Oncology Consortium
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Apr 10, 2025 · Source of record for eligibility and locations