Clinical Research Directory

Inclusion Criteria: 1. Histologically confirmed SHH-activated medulloblastoma meeting at least one of the following conditions: Tumor recurrence demonstrated by imaging or cerebrospinal fluid examination after standard treatment consisting of surgery, radiotherapy, and chemotherapy; or Refractory medulloblastoma, defined as an increase in tumor size of more than 25% or the appearance of new lesions after two cycles of standard first-line chemotherapy. 2. Medulloblastoma with a TP53 mutation identified by genetic testing of tumor tissue, including at least one of the following: A TP53 mutation present at initial diagnosis; A newly detected TP53 mutation during subsequent treatment in a patient with relapsed medulloblastoma; or A newly detected TP53 mutation during subsequent treatment in a patient with refractory medulloblastoma. 3. Age ≥18 months and ≤18 years. 4. Written informed consent obtained from the participant's legal guardian. Participants must meet either Inclusion Criterion 1 or Inclusion Criterion 2, in addition to Inclusion Criteria 3 and 4. Exclusion Criteria: 1. History of another malignancy for which the participant received chemotherapy and abdominal radiotherapy. 2. Severe progressive or persistent heart failure, defined as New York Heart Association (NYHA) class III or IV heart failure or a left ventricular ejection fraction (LVEF) \<50%. 3. Severe progressive or persistent renal failure, defined as a glomerular filtration rate (GFR) \<30 mL/min/1.73 m² or serum creatinine \>5 mg/dL (442 μmol/L). 4. Severe hepatic dysfunction, defined as aspartate aminotransferase (AST) or alanine aminotransferase (ALT) ≥5 times the age-appropriate upper limit of normal (ULN), or total serum bilirubin ≥3 times the age-appropriate ULN.