A Phase I, Open-label, Dose-escalation and Expansion Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of HSK42360 in Pediatric Patients With BRAF V600-Mutant Malignant Brain Tumors
This is a phase I, open-label, dose-escalation and expansion study to evaluate the safety, tolerability, PK of HSK42360 when given orally in pediatric patients with active BRAF V600 mutation recurrent malignant brain tumors.
• Age ≥6 and \<18 years.
• Karnofsky/Lansky Performance Status \>60.
• Life expectancy ≥ 3 months.
• Patients with recurrent malignant brain tumors confirmed by histology or cytology, who have failed standard treatment (disease progression after treatment or intolerable treatment); patients who have previously received BRAF and/or MEK inhibitor therapy are allowed to be included in this study.
• Positive BRAF V600 mutation result confirmed prior to the administration of HSK42360.
• Patients will provide blood or tumor sample according to their own willingness.
• Measurable disease by RANO criteria.
• Patients with inactive CNS lesions, or patients treated with ≤5mg/day corticosteroid and without convulsion for ≥2 weeks.
• Adequate hematologic, hepatic, and renal function.
⁃ Women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation, and for 90 days after the last dose.