A Study to Evaluate DAY101 in Pediatric and Young Adult Patients With Relapsed or Progressive Low-Grade Glioma

Official Title

FIREFLY-1: A Phase 2, Open-Label, Multicentre 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

Summary:

FIREFLY-1 is a Phase 2, multi centre, 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.

Trial Description

Primary Outcome:

  • Overall response rate (ORR) by independent radiology review committee (IRC) based on RANO criteria
Secondary Outcome:
  • Assess the safety and tolerability of DAY101
  • Relationship between pharmacokinetics (PK) and drug effects
  • Effect on electrocardiogram (ECG) and QT interval corrected for heart rate by Fridericia's formula (QTcF) prolongation
  • ORR by Investigator using RANO criteria
  • ORR by IRC and Investigator using RAPNO criteria
  • Progression free survival (PFS) by IRC and Investigator using RANO and RAPNO criteria
  • Duration of response (DOR) with best overall response of CR or PR using RANO and RAPNO criteria
  • Time to response following initiation of DAY101
  • Clinical benefit rate based on the proportion of patients with best overall response
  • Evaluate visual acuity (VA) outcomes compared with baseline
  • Evaluate the concordance of prior local laboratory BRAF molecular profiling with a central BRAF alteration assay being evaluated by the Sponsor
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).

View this trial on ClinicalTrials.gov

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Resources

Canadian Cancer Society

These resources are provided in partnership with the Canadian Cancer Society