TrialBridgeNCT07856069
Arsenic Trioxide Combined With Chemotherapy for Relapsed SHH-Activated and/or TP53-Mutant Medulloblastoma
Not Yet Recruiting · Phase 2 · Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University · registry updated 2026-10-02
Inclusion and exclusion lines below are quoted from ClinicalTrials.gov. No match score is shown, because a score needs a person's age, biomarkers, and treatment dates. Confirm the record with the study team.
This prospective, open-label, single-arm, multicenter study will evaluate the efficacy and safety of arsenic trioxide (ATO) combined with chemotherapy in pediatric patients with relapsed or refractory SHH-activated medulloblastoma and/or TP53-mutant medulloblastoma. Eligible participants will be 18 months to 18 years of age. Participants will receive six cycles of induction chemotherapy containing ATO in combination with etoposide and either cisplatin or cyclophosphamide. The primary outcome is the objective response rate (ORR), defined as the proportion of participants achieving a complete response or partial response, assessed according to the Response Assessment in Pediatric Neuro-Oncology (RAPNO) criteria by an independent review committee. Secondary outcomes include overall survival, progression-free survival, and treatment-related safety, including adverse events and serious adverse events. A total of 78 participants are planned for enrollment.
Inclusion
- 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.
- 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.
- Age ≥18 months and ≤18 years.
- 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
- History of another malignancy for which the participant received chemotherapy and abdominal radiotherapy.
- 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%.
- 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).
- 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.