PNOC029_Tovorafenib for the treatment of newly diagnosed or recurrent craniopharyngioma in children and young adults

You may be able to join if

• 3.3.1. Newly Diagnosed Participants • Newly diagnosed craniopharyngioma, as based on imaging characteristics and central radiology review. Participants will initially be screened within confines of a screening consent and only those participants with findings consistent with craniopharyngioma and without findings suggesting an indeterminate lesion or lesion of an alternative diagnosis (including abnormal tumor markers found in blood or CSF, if completed as part of SOC work-up or if lesion concerning for alternate diagnosis) will move ahead with enrollment on the treatment protocol. Additionally, for patients that have undergone initial biopsy to confirm diagnosis, are within 6 weeks of radiographic diagnosis, and are planned to undergo follow up second surgery for additional tumor resection as per standard of care recommendations, these patients will also be considered eligible. Patients beyond 6 weeks of radiographic diagnosis should be discussed with study chairs.
• 3.3.3. All Participants: Performance Score: Karnofsky ≥ 50 for participants > 16 years of age and Lansky ≥ 50 for participants ≤ 16 years of age (See Appendix A). Participants who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
• 3.3.3. All Participants: Corticosteroids: Participants who are receiving dexamethasone must be on a stable or decreasing dose for at least 1 week prior to registration. The patient steroid dose should be no more than a steroid-equivalent of dexamethasone 0.1 mg/kg/day (or maximum 4mg/day; whichever is the lower dose) at time of enrollment. Participants that have been stable on physiologichormone replacement for hypopituitarism are allowed.
• 3.3.3. All Participants: Organ Function Requirements • Adequate Bone Marrow Function Defined as: - Peripheral absolute neutrophil count (ANC) ≥ 1000/mm3 - Platelet count ≥ 100,000/mm3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment). • Adequate Renal Function Defined as: - A serum creatinine < 1.5 Upper Limit normal (ULN) based on age and gender • Adequate Liver Function Defined as: - Bilirubin (sum of conjugated + unconjugated) ≤ 1.5 x upper limit of normal (ULN) for age (except in patients with documented Gilber syndrome). - SGPT ((ALT) ≤ 3 x ULN. - Serum albumin ≥ 2 g/dL (20g/L). • Adequate Neurologic Function Defined as: - Participants with seizure disorder may be enrolled if well controlled. Participants on non-enzyme inducing anticonvulsants may be excluded pending interaction(s) with study drug. See Appendix B for a list of recommended non-enzyme inducing anticonvulsants. • Adequate Pulmonary Function Defined as: - No evidence of dyspnea at rest, no exercise intolerance due to pulmonary insufficiency, and a pulse oximetry of > 92% while breathing room air. - PT/PTT/INR within institutional normal limits or deemed appropriate for surgical intervention by the treating team for patients undergoing surgery biopsy/resection
• 3.3.3. All Participants: The effects of tovorafenib on the developing human fetus are unknown. For this reason, women of child-bearing potential and men must agree to use adequate contraception (non-hormonal contraception; barrier method of birth control; abstinence – note, tovorafenib can make hormonal contraceptives ineffective) prior to study entry, for the duration of study participation and 28 days after completion of tovorafenib administration, whichever is later. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately.
• 3.3.3. All Participants: A legal parent/guardian or patient must be able to understand, and willing to sign, a written informed consent and assent document, as appropriate.