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

DAY101-001/PNOC026

FIREFLY-1 is an ongoing, Phase 2, multi center, open-label study to evaluate the safety and efficacy of oral pan-RAF inhibitor DAY101 in pediatric, adolescent, and young adult patients with recurrent or progressive low-grade glioma or an advanced solid tumor harboring a known RAF alteration.

2021-04-22

2022-12-22

2024-06-10

Recruiting

Early phase I

140

Inclusion Criteria: * Age 6 months to 25 years with: 1. Arms 1 \& 2: a relapsed or progressive LGG with documented known activating BRAF alteration 2. Arm 3: locally advanced or metastatic solid tumor with documented known or expected to be activating RAF fusion * Confirmation of histopathologic diagnosis of LGG and molecular diagnosis of activating BRAF alteration * Must have received at least one line of systemic therapy and have 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: * Patient's tumor has additional previously-known activating molecular alterations * Patient has symptoms of clinical progression in the absence of radiographic progression * Known or suspected diagnosis of neurofibromatosis type 1 (NF-1) * Other inclusion/exclusion criteria as stipulated by protocol may apply

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'Single intervention across 3 arms - - no comparator', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 140, 'type': 'ESTIMATED'}}

2023-12-27