Diffuse midline glioma
Conditions
Sponsors
Shunyi Women's & Children's Hospital of Beijing Children's Hospital
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Inclusion criteria
Inclusion criteria: 1. Age: Age >= 18 years, any gender; 2. Diagnosis: Patients with primary, recurrent, or progressive diffuse midline glioma (DMG), H3 K27-altered, confirmed as follows: Brainstem primary DMG: Radiologically confirmed. Other midline CNS locations (e.g., thalamus, spinal cord): Histopathologically confirmed (via surgical resection or biopsy) as DMG, H3 K27-altered; For brainstem or other midline tumors (e.g., thalamus, spinal cord), pathological confirmation of high-grade glioma, IDH-mutant, H3K27-wildtype. 3. Informed Consent: Written informed consent must be provided by the participant and parent(s) or legal guardian(s); 4. Prior Therapy: No prior systemic anti-tumor therapy. Radiotherapy: Not received, currently receiving, or completed; 5. Performance Status: Karnofsky Performance Status (KPS) >= 40 (for participants >= 16 years). Eastern Cooperative Oncology Group (ECOG) Performance Status = 1.0 × 10^9/L. b) Platelet count >= 100 × 10^9/L. c) Hemoglobin >= 80 g/L. d) Adequate renal, hepatic, and cardiopulmonary function: Renal: Serum creatinine = 70 mL/min/1.73m^2. Hepatic: Serum Alanine Aminotransferase (ALT) and Aspartate Aminotransferase (AST) = 94% on room air; 7. Stable Neurological Function: Participants must be on a stable regimen of = 1 week prior to enrollment.
Exclusion criteria
Exclusion criteria: 1. Prior Malignancy: History of other definitively diagnosed malignant diseases within the past 3 years; 2. Prior Antitumor Therapy: Participation in other clinical trials involving any prior antitumor therapy beyond surgery and dexamethasone. Subjects who have received other antitumor agents may be excluded based on investigator assessment; 3. Unresolved Conditions: Any of the following unresolved conditions: Significant post-operative complications; Active autoimmune disease; Severe immunodeficiency; Uncontrolled active infection; 4. Active Viral Infections: Active infection with HIV, Hepatitis B Virus (HBV), or Hepatitis C Virus (HCV); 5. Immunosuppressants/Corticosteroids: Chronic use of immunosuppressive medications or systemic corticosteroid therapy; 6. Dysphagia: Clinically assessed significant dysphagia compromising safe oral/enteral intake of study agents; 7. Significant Comorbidities: Any clinically significant systemic disease or medical condition (e.g., severe cardiac, pulmonary, hepatic, or other organ dysfunction) that, in the judgment of the Principal Investigator, could jeopardize subject safety, interfere with the absorption/metabolism of study agents, or compromise the assessment of study outcomes; 8. Protocol Compliance: Inability or unwillingness, in the investigator's judgment, to comply with the scheduled visits, treatment plan, laboratory tests, or other study procedures; 9. Hypersensitivity: Known hypersensitivity or severe intolerance to any component of the investigational product(s)/study drug(s); 10. Pregnancy/Lactation: Pregnancy or lactation.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Proportion of subjects achieving Overall Survival at 12 months; | — |
Secondary
| Measure | Time frame |
|---|---|
| Overall survival;Progression free survival;Change in tumor volume after completion of 2-3 treatment cycles compared to baseline;QLQ-C30;QLQ-BN20; | — |
Countries
China
Contacts
Public ContactZhang Wei
Beijing Children's Hospital, Capital Medical University, National Center for Children's Health,
Outcome results
None listed