Trial design
FIREFLY-1 (PNOC026; Sex and/or gender were not considered in the trial design as no sex differences have been seen in previous clinical trials in pLGG, although, in line with rates generally seen in childhood cancerTovorafenib was administered at the recommended phase 2 dose of 420 mg mAn independent data safety monitoring board (DSMB) was established before initiation of the trial and was in place for the duration of the trial. Medical monitoring of the trial was provided by the sponsor and used a safety review committee that included the principal investigator (or their designee) from each active clinical site.The protocol, protocol amendments, informed consent form, pediatric assent form, investigator brochure and other relevant documents were approved by an institutional review board/independent ethics committee at each trial site. As applicable according to local regulations, the protocol and all protocol amendments were reviewed and approved by each pertinent competent authority.This trial was conducted in accordance with the protocol and consensus ethical principles derived from international guidelines, including the Declaration of Helsinki, Council for International Organizations of Medical Sciences (CIOMS) International Ethical Guidelines, applicable International Council for Harmonization Good Clinical Practice (GCP) guidelines and other applicable laws and regulations. All patients and/or their legally authorized representative provided written informed consent and pediatric assent before enrollment in the trial, according to local regulations. No direct compensation was provided to patients or families for participating in the trial.