Go to family friendly version
| Diagnosis | Low-grade glioma or solid tumors with RAF alterations | Study Status | Open |
| Phase | II |
| Age | Child, Adult - (6 Months to 25 Years) | Randomisation | NO |
| Line of treatment | Disease relapse or progression |
| Routes of Treatment Administration | Day101: oral (tablet formulation) |
| Last Posted Update | 2023-09-28 |
| ClinicalTrials.gov # | NCT04775485 |
International Sponsor
Day One Biopharmaceuticals, Inc.Principal Investigators for Canadian Sites
Montreal Children's Hospital – Dr. Nada Jabado
CHU Ste-Justine – Dr. Sebastien Perreault
CHU de Quebec – Dr. Valerie Larouche
Centres
Medical contact
Clinical Research Unit
Social worker/patient navigator contact
Clinical Research Unit
Clinical research contact
Stephanie Badour
Medical contact
Dr. Henrique Bittencourt
Dr. Monia Marzouki
Dr. Sebastien Perreault (neuro-onc)
Social worker/patient navigator contact
Marie-Claude Charrette
Clinical research contact
Marie Saint-Jacques
Medical contact
Raoul Santiago
Social worker/patient navigator contact
Isabelle Audet
Clinical research contact
Barbara Desbiens
Study Description
Brief Summary:
FIREFLY-1 is a 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 harboring a known BRAF alteration.
Detailed Description:
Approximately 60 pediatric patients will be treated with DAY101, an oral pan-RAF inhibitor, for a planned period of 26 cycles will be treated with DAY101 for a planned period of 26 cycles (approximately 24 months).
DAY101 will be administered at the recommended Phase 2 dose (RP2D) of 420 mg/m2 (not to exceed 600 mg) orally once weekly (QW) for each 28-day treatment cycle.
Treatment cycles will repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients will undergo radiographic evaluation of their disease at the end of every third cycle. Patients will continue on DAY101 until radiographic evidence of disease progression by RANO criteria as determined by treating investigator, unacceptable toxicity, patient withdrawal of consent, or death.
Patients who have radiographic evidence of disease progression may be allowed to continue DAY101 if, in the opinion of the investigator and approval by the Sponsor, the patient is deriving clinical benefit from continuing study treatment. Disease assessments for patients being treated beyond progression should continue as per regular schedule.
DAY101 is an oral pan-RAF inhibitor administered as an oral tablet at 420 mg/m2 (not to exceed 600 mg).
Inclusion Criteria
- Age 6 months to 25 years with a relapsed or progressive LGG or solid tumor with known activating RAF alteration (BRAF or CRAF/RAF1 fusion or BRAF V600 mutations (LGG only))
- Confirmation of histopathologic diagnosis of LGG or solid tumor and molecular diagnosis of activating RAF alteration (BRAF or CRAF/RAF1 fusion or BRAF V600 mutations (LGG only))
- 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 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
