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

Protocol # :
Low-grade Glioma
Advanced Solid Tumor
Disease Sites
Brain and Nervous System
Melanoma, Skin
Principal Investigator
Chi, Susan, N
Site Research Nurses
Gouthro, Michelle, S.
Strachan, Marylynne

Trial Description

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.

Eligibility Requirements

Inclusion Criteria:

- Age 6 months to 25 years with:

1. Arms 1 & 2: a relapsed or progressive LGG with documented known activating BRAF

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

- Known or suspected diagnosis of neurofibromatosis type 1 (NF-1)

- Other inclusion/exclusion criteria as stipulated by protocol may apply