diffuse intrinsic pontine gliomas
Conditions
Interventions
Treatment:ACT001+Radiation
Sponsors
Beijing Tiantan Hospital affiliated to Capital Medical University
Eligibility
Sex/Gender
All
Age
5 Years to 15 Years
Inclusion criteria
Inclusion criteria: 1. All patients and/or guardians must voluntarily sign a written informed consent form. 2. Newly diagnosed DIPG patients confirmed by histology (stereotactic biopsy) or imaging within 6 weeks before the first administration. 3. At the time of study enrollment, the age was = 5 years old and = 15 years old. 4. Expected life = 3 months. 5. The patient's LPS score is = 70 (excluding motor dysfunction caused by brain tumors involving the motor area or incomplete limbs). 6. There is at least one measurable DIPG lesion that meets RAPNO regulations (T2/FLAIR evaluation). If a measurable lesion has undergone pathological biopsy, it needs to be re evaluated 7 days after the biopsy and used as the baseline for imaging evaluation. 7. According to the following definition of laboratory examination results, there are enough Hematology and end organ functions, and the examination results should be completed within 7 days before the study drug administration: 1) Blood routine • Hematology functions are as follows, and the absolute neutrophil count (ANC) = 1.5 × 109/L; Platelet count = 75 × 109/L; Hemoglobin (Hb) = 80 g/L. 2) Coagulation function • Prothrombin time (PT) or partial Thromboplastin time (APTT) = 1.5 × Upper limit of normal value (ULN). 3) Liver function Total bilirubin = 1.5 × ULN and alanine aminotransferase (ALT) and Asparagus cochinchinensis aminotransferase (AST) = 2.5 × ULN. 4) Renal function Serum creatinine = 1.5 × ULN; Alternatively, based on serum creatinine at different ages, the creatinine clearance rate can be calculated as = 70 ml/min/1.73 m2, using the formula CrCl (ml/min/1.73 m2)=[height (cm) * k] * 88.4/Scr( µ Mol/L), the calculation parameter k for different ages can be found in Appendix 13.1. 8. The heart function meets the following conditions: The clinical significance of the electrocardiogram test results is assessed by the researcher as normal or abnormal without clinical significance; • The cardiac diastolic function is normal, and the echocardiography in the screening period shows that the left ventricular Ejection fraction (LVEF) is = 50%. 9. Female patients also need to meet the following criteria to be considered for inclusion: • Female patients with no Fertility (prepubertal women who received total Hysterectomy with bilateral oviduct oophorectomy or bilateral oophorectomy). • Adolescent female patients with Fertility must have negative serum Pregnancy test during screening, and must agree to use effective contraception methods from the screening period to 6 months after the last administration. 10. Based on medical history and examination, male patients in pre puberty or adolescence agree to use efficient contraceptive methods within 6 months from the screening period until the last administration.
Exclusion criteria
Exclusion criteria: 1. Previously received local treatment for the primary lesion (excluding stereotactic biopsy). 2. Previously underwent craniotomy biopsy. 3. Other malignant tumors other than primary tumors (except for fully treated cervical Carcinoma in situ, skin basal cell or squamous epithelial cell skin cancer, local prostate cancer and Ductal carcinoma in situ after radical surgery) within 5 years before screening. 4. Imaging examination indicates the presence of whole brain or spinal cord dissemination or extracranial metastasis lesions. 5. Imaging shows active cerebral hemorrhage. 6. Symptoms of the central nervous system are obvious, and long-term treatment with glucocorticoids is planned within 14 days before the first study drug administration and during the study period (equivalent to adults receiving 4 mg of dexamethasone daily and continuously taking hormone doses for more than three days). 7. Seizures and/or high intracranial pressure that are difficult to control with medication during screening. 8. There are uncontrollable active infections during screening. 9. HIV (HIV) antibody positive, syphilis antibody positive. 10. Clinically active hepatitis A, B, and C (for hepatitis B, if HBsAg or HBcAb is positive and HBV DNA is positive, it needs to be excluded; for hepatitis C, if HCVAb is positive, it needs to be excluded). 11. Inability to take medication orally or presence of gastrointestinal absorption dysfunction. 12. Unable to complete radiotherapy as planned. 13. Previous history of allogeneic organ transplantation or allogeneic peripheral blood stem cell or bone marrow transplantation treatment. 14. Known allergies to the study drug and its analogues. 15. Pregnant or lactating women. 16. Participated in other clinical studies and used the study drug or corresponding treatment before the study medication. 17. Other reasons determined by the researcher to be unsuitable for participation in this study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| ORR assessed by IRC(Exploratory period);AE during and after radiotherapy(Exploratory period);ORR assessed by IRC(Validation period);OS assessed by IRC(Validation period); | — |
Secondary
| Measure | Time frame |
|---|---|
| OS(Exploratory period);ORR assessed by investigator(Exploratory period);One year survival rate(Exploratory period);ORR assessed by investigators(Validation period);PFS assessed by IRC and investigators(Validation period);AE, SAE, etc(Validation period); | — |
Countries
China
Contacts
Public ContactQiu Xiaoguang
Beijing Tiantan Hospital affiliated to Capital Medical University
Outcome results
None listed