A Study Comparing the Blood Levels of Both Pegaspargase (S95014) Formulations (Liquid vs Lyophilized) in the Treatment of Paediatric Patients With Acute Lymphoblastic Leukemia (ALL)

A Multicentre, Phase II Randomized Study, Open-label, With 2-arm Parallel Group, Comparing the Pharmacokinetics of the Liquid and the Lyophilized Formulations of Pegaspargase (S95014) in Treatment of Paediatric Patients With Newly-Diagnosed Acute Lymphoblastic Leukemia (ALL)

The purpose of this study is to compare the pharmacokinetics (PK) of both lyophilized and liquid S95014 formulations during the induction phase after a single IV dose in newly diagnosed paediatric patients with ALL

Inclusion Criteria:

• Patients aged 1 to < 18 years
• Patients with cytologically confirmed and documented newly diagnosed ALL according to National Comprehensive Cancer Network guidelines 2020 (see Appendix 2), excluding B-cell Burkitt ALL
• Eastern Cooperative Oncology Group performance status (ECOG PS) 0-2 (see Appendix 3)
• Highly effective contraception method
• Signed informed consent and assent, when appropriate

Non-inclusion Criteria:

• Unlikely to cooperate in the study
• Pregnant and lactating women
• Participation in another interventional study at the same time; participation in non-interventional registries or epidemiological studies is allowed
• Participant already enrolled in the study (informed consent signed)
• Women of childbearing potential tested positive in a serum pregnancy test within 7 days prior to the treatment period
• Inadequate hepatic function (bilirubin > 1.5 times upper limit of normal (ULN), transaminases > 5x ULN)
• Inadequate renal function defined as serum creatinine > 1.5 x ULN
• Prior treatment with chemotherapy or radiotherapy (except steroids and intrathecal therapy)
• Prior surgery or bone marrow transplant related to the studied disease
• Down Syndrome
• Psychiatric illness/social situation that would limit compliance with study requirements
• Known history of pancreatitis
• Known history of significant liver disease
• Known carriers of HIV antibodies
• Significant laboratory abnormality likely to jeopardize the patients' safety or to interfere with the conduct of the study, in the investigator's opinion
• Pre-existing known coagulopathy (e.g. haemophilia and known protein S deficiency)
• History of previous or concurrent malignancy
• History of sensitivity to polyethylene glycol (PEG) or PEG-based drugs
• Severe or uncontrolled active acute or chronic infection
• Uncontrolled intercurrent illness including life-threatening acute tumor lysis syndrome (e.g. with renal failure), symptomatic congestive heart failure, cardiac arrhythmia