This is a single-arm, open-label, multicenter, phase III clinical study that aims to evaluate the efficacy and safety of Nelarabine injection in the treatment of refractory or recurrent T-lymphoblastic leukemia (T-ALL) and T-lymphoblastic lymphoma (T-LBL) in both children and adults. The trial includes 83 subjects, consisting of 35 adults and 48 children, and aims to evaluate the composite complete response rate (CCR) within 2 cycles, assessed by the Independent Review Committee (IRC), following treatment with Nelarabine injection for children and adults with refractory or recurrent T-ALL and T-LBL. The sample size of this study is estimated according to the treatment period of 4 cycles.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
83
Nelarabine is a prodrug of the nucleotide metabolism inhibitor deoxyguanosine analogue 9-β-arabinoguanine (ARA-G). Nelarabine undergoes catalytic transformation by adenosine deaminase (ADA), resulting in the removal of its methoxy group and conversion into ARA-G, Subsequently, ARA-G undergoes sequential monophosphorylation by deoxyguanosine kinase and deoxycytosine nucleoside kinase, yielding the active compound 5'-Guanosine triphosphate (GTP), ARA-GTP. This active compound accumulates within leukemic blast cells and binds to deoxyribonucleic acid (DNA), effectively inhibiting DNA synthesis and ultimately leading to cell death.
Anhui Provincial Hospital
Hefei, Anhui, China
Aerospace Medical Center
Beijing, Beijing Municipality, China
Beijing Tongren Hospital,CMU
Beijing, Beijing Municipality, China
Children's Hospital of Chongqing Medical University
Chongqing, Chongqing Municipality, China
The Second Affiliated Hospital of Army Military Medical University
Chongqing, Chongqing Municipality, China
Composite complete response rate (CCR) assessed by the Independent Review Committee (IRC)
Proportion of complete responses (CCR) after 2 cycles of antineoplastic therapy as determined by independent review committee (IRC) assessment
Time frame: During the study period (Baseline up to two months)
Composite complete response rate (CCR) assessed by the investigator
Proportion of complete responses (CCR) after 2 cycles of antineoplastic therapy as determined by the investigator.
Time frame: During the study period (Baseline up to two months)
Objective Remission Rate (ORR)
The best Objective Remission Rate (ORR) for both complete response (CR) and partial response (PR) cases within 2 cycles.
Time frame: During the study period (Baseline up to two months)
Duration of complete remission (DOCR)
Proportion of patients whose tumor shrinks or stabilizes for some time, including cases of complete response (CR), partial response (PR), and stable disease (SD).
Time frame: During the study period (Baseline up to two years)
Disease-free survival (DFS)
It refers to the time from the first day of absence of disease to the recurrence of disease.
Time frame: During the study period (Baseline up to two years)
Overall survival (OS)
It indicates the time interval from randomization to death from any cause.
Time frame: During the study period (Baseline up to two years)
Incidence of adverse events (AEs)
Adverse events refer to all adverse medical events that occur after a patient or clinical trial subject receives an experimental drug, which can be expressed as symptoms, signs, diseases, or abnormalities in laboratory tests, but are not necessarily related to the treatment of the experimental drug.
Time frame: During the study period (Baseline up to two years)
Incidence of serious adverse events (SAEs)
Serious adverse events include death, life-threatening, permanent or serious disability or loss of function, hospitalization or prolonged hospitalization, and adverse medical conditions such as congenital abnormalities or birth defects.
Time frame: During the study period (Baseline up to two years)
Peak time (Tmax)
Time to maximum plasma concentration.
Time frame: During the study period (Baseline up to two years)
Peak concentration(Cmax)
Pharmacokinetic parameters, including but not limited to: Peak concentration(Cmax)
Time frame: During the study period (Baseline up to two years)
Half-life(t1/2)
It is the time required for half of the drug to be eliminated from the plasma
Time frame: During the study period (Baseline up to two years)
Negative rate of minimal residual disease in bone marrow (MRD)
The proportion of trace leukemic cells negative in patients with hematologic tumors after treatment.
Time frame: During the study period (Baseline up to two years)
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