Central nervous system tumors [mainly diffuse intrinsic pontine glioma (DIPG)], non-central nervous system advanced solid tumors
Conditions
Interventions
376 mg/m2/day:The subjects will receive a single dose of ACT001, 188 mg/m2 on the morning of Day 1 of Cycle 1. After 24 hours of washout, the subjects begin to receive ACT001 administered twice a day
470 mg/m2/day:The subjects will receive a single dose of ACT001, 235 mg/m2 on the morning of Day 1 of Cycle 1. After 24 hours of washout, the subjects begin to receive ACT001 administered twice a day
590 mg/m2/day:The subjects will receive a single dose of ACT001, 295 mg/m2 on the morning of Day 1 of Cycle 1. After 24 hours of washout, the subjects begin to receive ACT001 administered twice a day
Sponsors
Tianjing Medical University Cancer Institute and Hospital
Eligibility
Sex/Gender
All
Age
5 Years to 14 Years
Inclusion criteria
Inclusion criteria: 1. All patients and / or their parents or legal guardians must voluntarily provide written informed consent. Where appropriate, communicate with the agency to confirm the requirements for obtaining informed consent; 2. Patients aged 5 to 15 years; 3. Patients with Zps score =1.0x10^9/L; platelet count >=75x10^9/L (platelet transfusion is acceptable, defined as no platelet transfusion within 7 days before admission); hemoglobin (RBC) >=80 G/L (RBC transfusion can be accepted, defined as no RBC transfusion within 7 days before admission); prothrombin time (PT) or partial prothrombin kinase time (PTT) =70 ml/min/1.73 m2 or the serum creatinine reference values of different ages are as follows: 5-8 years old, serum creatinine reference value is 40-60 umol/L; 9-15 years old, serum creatinine reference value is 50-80 umol/L; 11. The appropriate definition of liver function is: total bilirubin 50% 13. Female patients: female patients without fertility. (pre puberty women, who received hysterectomy with bilateral salpingoophorectomy or bilateral oophorectomy) or women with fertility must have negative urine pregnancy test at the time of study enrollment, and must be willing to use efficient contraceptive method from screening period to 6 months after the last administration; 14. Male patients: according to the history and examination of male patients before puberty or willing to use effective contraceptive methods from the screening period to 6 months after the last administration.
Exclusion criteria
Exclusion criteria: 1. Pregnant or lactating women; 2. Patients with uncontrollable active infection; 3. Patients with any major organ system disease that may affect their treatment tolerance; 4. Patients with pre-existing allergic reactions to act001 or related compounds; 5. Patients with known active infection: such as hepatitis B, hepatitis C, human immunodeficiency virus (HIV) infection. Patients with well controlled hepatitis B (negative for HBV DNA) can be included in this test, and can be treated with antiviral therapy at the same time. HBV DNA examination shall be carried out every 3 months (3 cycles) after being included in the group; 6. Patients who received anticoagulation treatment at the same time or in the past (within 7 days before admission), except those who used low molecular weight heparin or low-dose aspirin; 7. Patients who are currently receiving another trial drug, or who have obtained informed consent for this trial less than 4 weeks from the time of withdrawal from the previous clinical trial; 8. Patients currently receiving other antineoplastic drugs; 9. All herbal supplements, vitamins and nutritional supplements taken by the patient within 30 days prior to the first day of Administration (continued where appropriate) must be reviewed and approved by the investigator; 10. Patients who had been treated with MIBG within 6 weeks before screening; 11. Patients who received stem cell transplantation or rescue (including autotransplantation) or evidence of active graft-versus-host disease within 12 weeks before screening; 12. Patients who received myelosuppression chemotherapy: they received myelosuppression chemotherapy within 28 days (6 weeks if they received nitrosourea before enrollment) before the study (before randomization); 13. Hematopoietic growth factor: the last administration of long-acting growth factor (e.g., peg-cgsf) was less than 14 days, or the last administration of short-acting growth factor (e.g., non glitazidine, sagistine, EPO) was less than 7 days. In the event of a known adverse event, it is up to the investigator to determine whether a longer elution time is required; 14. Patients using biological agents (anti-tumor drugs): the last time biological agents were given was less than 4 weeks. In the event of a known adverse event, it is up to the investigator to determine whether a longer elution time is needed; 15. Monoclonal antibody: the distance from the last administration of monoclonal antibody to the group was less than 5 antibody half lives; 16. In the judgment of the investigator, the patient / parent / Guardian has any disease that may affect the written informed consent of the patient / parent / guardian and / or the completion of all research processes, as well as any disease of clinical significance that may affect the safety of the patient, the evaluation and completion of the study (not mentioned above); 17. According to the judgment of the researcher, there are other reasons that are not suitable to participate in this study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence and severity of adverse events (AE);DLT;RP2D; | — |
Secondary
| Measure | Time frame |
|---|---|
| Pharmacokinetic evaluation;DCR;ORR; | — |
Countries
China
Contacts
Public ContactZhao Qiang/ Shi Yehui
Tianjing Medical University Cancer Institute and Hospital
Outcome results
None listed