Pan-RAF Inhibitor DAY101 in Patients with BRAF Altered, Recurrent or Progressive LGG (FIREFLY-1)

Study title

“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 BRAF-Altered, Recurrent or Progressive Low-Grade Glioma”

 

The aim of the study is to find out whether treatment with DAY101 is safe and effective in pediatric patients with recurrent or progressive Low-Grade Glioma (LGG) with a change (alteration) in the BRAF gene. DAY101 is a so-called Pan-RAF inhibitor, which blocks the activity of an important growth signaling pathway (MAPK / ERK) in the cancer cells and thus leads to the death of the cancer cells. The study therefore only treats patients whose tumor tissue has certain mutations in the BRAF gene, which lead to an excessive activation of the MAPK / ERK signaling pathway.

Patients between 6 and 25 years of age with relapsing / refractory or progressive LGG can participate in this study. A BRAF change in the tumors must be proven by molecular assays.

DAY101 is taken in tablet form once a week in 28-day cycles.

Inclusion of patients in Heidelberg has been possible since 26 August 2021.

  • Children, adolescents and young adults with a relapsed or progressive LGG
  • Age ≥ 6 and ≤ 25 years
  • a documented known activating BRAF alteration, as identified through molecular assays
  • Karnofsky-/Lansky-Score > 50
  • At least one measurable lesions as defined by RANA criteria

For more information, see the Study Registry: The study is registered with the U.S. National Institutes of Health (ClinicalTrials.gov).
See NCT04775485

EudraCT-Number: 2020-003657-30 (Clinical Trials Register)