Pediatric low-grade glioma harboring an activating RAF alteration requiring first-line systemic therapy MedDRA version: 21.1 Level: PT Classification code 10065443 Term: Malignant glioma System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Conditions
Interventions
Product Name: Tovorafenib
Product Code: DAY101
Pharmaceutical Form: Tablet
INN or Proposed INN: tovorafenib
CAS Number: 1096708-71-2
Current Sponsor code: DAY101
Other descriptive name: QHA01, C200601
Sponsors
Day One Biopharmaceuticals, Inc. (Day One)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Less than 25 years of age with LGG with known activating RAF alteration - Histopathologic diagnosis of glioma or glioneuronal tumor - At least one measurable lesion as defined by RANO criteria - Meet indication for first-line systemic therapy Are the trial subjects under 18? yes Number of subjects for this age range: 400 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 50 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: - Patient has any of the following tumor-histological findings: a) Schwannoma b) Subependymal giant cell astrocytoma (Tuberous Sclerosis) c) Diffuse intrinsic pontine glioma, even if histologically diagnosed as WHO Grade I-II - Patient’s tumor has additional pathogenic activating molecular alterations - Known or suspected diagnosis of neurofibromatosis Type 1 or 2 (NF-1/NF-2) - Prior or ongoing nonsurgical anticancer therapy for this indication (eg, chemotherapy, oral/IV targeted therapy) including radiation.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To compare the ORR assessed per RANO-LGG criteria by independent review committee (IRC) of tovorafenib monotherapy versus SoC chemotherapy in patients with pediatric low-grade glioma harboring an activating RAF alteration requiring first-line systemic therapy.;Secondary Objective: KEY SECONDARY OBJECTIVES: - To compare the PFS assessed by IRC of tovorafenib monotherapy versus SoC chemotherapy per RANO-LGG criteria. - To compare the duration of response (DOR) assessed by IRC of tovorafenib monotherapy versus SoC chemotherapy per RANO-LGG criteria - To compare the OS of tovorafenib monotherapy versus SoC chemotherapy. ;Primary end point(s): ORR, per RANO-LGG criteria, defined as the proportion of patients with overall confirmed response of complete response (CR) or partial response (PR).;Timepoint(s) of evaluation of this end point: After the last patient has been followed up for 12 months (anticipated to occur at approximately 36 months after first patient enrolled). | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): KEY SECONDARY ENDPOINTS: - PFS per RANO-LGG criteria, defined as time from randomization to PD or death from any cause, whichever comes first. - DOR per RANO-LGG criteria, defined as time from the first imaging of tumor response (CR or PR) that was subsequently confirmed to PD or death from any cause, whichever comes first. - OS, defined as time from randomization up to death from any cause.;Timepoint(s) of evaluation of this end point: Up to 60 months | — |
Countries
Australia, Austria, Belgium, Canada, Czechia, Czech Republic, Denmark, Finland, France, Germany, Greece, Hungary, Ireland, Israel, Italy, Korea, Republic of, Netherlands, New Zealand, Norway, Singapore, Slovenia, Spain, Sweden, Switzerland, United Kingdom, United States
Contacts
Public ContactFIREFLY-2 Clinical Trial Team
Day One Biopharmaceuticals, Inc. (Day One)
Outcome results
None listed