pediatric patients with relapsed/refractory Acute Myeloid Leukemia
Conditions
Interventions
Sonrotoclax treatment group:Sonrotoclax treatment
Sponsors
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College.
Eligibility
Sex/Gender
All
Age
2 Years to 18 Years
Inclusion criteria
Inclusion criteria: 1. The patient's guardian understands the study procedures and voluntarily signs the Informed Consent Form (ICF). For patients >=8 years old, voluntary written assent is required; for patients =5%; or reappearance of blasts in the blood; or occurrence of extramedullary disease. Refractory disease is defined as no CR or CRi after 2 courses of intensive induction therapy; or no CR, CRi, or MLFS after 6 courses of non-intensive induction therapy. 4. Lansky score (=16 years old) >=50. 5. Life expectancy >3 months. 6. Adequate organ function: Creatinine clearance >=50mL/min; Adequate liver function: Aspartate aminotransferase (AST/SGOT) <=3×ULN; Alanine aminotransferase (ALT/SGPT) <=3×ULN; Total bilirubin level <=1.5×ULN. 7. Compliance with study requirements.
Exclusion criteria
Exclusion criteria: 1.Patients meeting any of the following criteria will be excluded from this study: Confirmed diagnosis of Acute Promyelocytic Leukemia (APL). 2.History of other malignancies within 2 years, except for MDS that has transformed to AML or other cured solid malignancies. 3.Known central nervous system involvement by leukemia (CNSL-2: CSF WBC count =0.005x10^9/L (5/ml) with visible blasts). 4.Prior allogeneic stem cell transplantation with active Graft-Versus-Host Disease (GVHD), or requiring immunosuppressive therapy. 5.Uncontrolled concurrent illnesses, including but not limited to: ongoing or active uncontrolled infections, cardiovascular disease, psychiatric illness, or social situations that would limit compliance with study requirements. 6.Known history of infection with human immunodeficiency virus (HIV). 7.Known positive for active Hepatitis B or Hepatitis C infection, unless the viral load is undetectable (Note: Patients who are HBcAb+ but HBsAg- are eligible only if Hepatitis B Virus (HBV) DNA is undetectable using an assay with a sensitivity of <=20 IU/mL. In such cases, patients should undergo regular monitoring of HBV DNA and receive prophylactic antiviral therapy according to standard institutional practice. Patients who are HCV antibody+ are eligible only if HCV RNA is undetectable and they are willing to monitor for HCV reactivation). 8.Receiving treatment with any moderate or strong CYP3A4 inhibitor (<= 7 days or 5 half-lives, whichever is longer) or moderate or strong CYP3A4 inducer (<= 14 days or 5 half-lives, whichever is longer) before the first dose of sonrotoclax; 9.History of hypersensitivity to an excipient or active ingredient of the sonrotoclax tablet. 10.Patients deemed unsuitable for enrollment by the investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Pharmacokinetic (PK);Dose-Limiting Toxicity,DLT; | — |
Secondary
| Measure | Time frame |
|---|---|
| Objective Response Rate (ORR), Composite Complete Remission (CR) rate, Measurable Residual Disease (MRD) negativity rate among evaluable patients, and 1-year Relapse-Free Survival (RFS) rate;Adverse Event; | — |
Countries
China
Contacts
Public ContactXiaofan Zhu
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College.
Outcome results
None listed