Clinical trial NCT04349306: Evaluate the Efficacy and Safety of Rasburicase (Fasturtec®) in the Prevention and Treatment of Hyperuricemia in Pediatric Patients With Non-Hodgkin's Lymphoma and Acute Leukemia
Official title: An Open-label, Single-arm, Multi-center Study to Evaluate the Efficacy and Safety of Rasburicase (Fasturtec) in the Prevention and Treatment of Hyperuricemia in Pediatric Patients With Non-Hodgkin¿s Lymphoma and Acute Leukemia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Lymphoma
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2020-04-16
Enrollment: 50
Sex: ALL
Minimum age (years): 2
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Primary Objective:

To evaluate safety of rasburicase in pediatric patients with NHL and AL

Secondary Objective:

To assess efficacy of rasburicase for prevention and treatment of hyperuricemia

Detailed description: Study duration per participants is approximatively 14 days including a 5-day treatment period.

Eligibility criteria: Inclusion criteria :

* Patient or parent/legal guardian is willing and able to provide signed informed consent, and if required, the patient is willing to provide assent.
* Children or adolescent aged 2 to 18 years old (inclusive) at time of signing of informed consent.
* At screening, the patient is expected to have a minimum life expectancy of 45 days and has a performance status (PS) no greater than 3 on the Eastern Cooperative Oncology Group (ECOG) scale, or a PS no less than 30 on the Lansky score as per the Investigator's preference (see Appendix D for ECOG and Lansky scale).
* Newly diagnosed NHL or AL who is at the initiation of or during the first cycle of chemotherapy,baseline blood uric acid greater than 8 mg/dL (473 mol/L) at screening.
* If newly diagnosed NHL patient with blood uric acid no greater than 8 mg/dL at screening, the patient must be diagnosed with Stage III or IV non-Hodgkin's lymphoma with high tumor burden which will be high risk of TLS defined, with one or more of following below: A. Burkitt lymphoma/leukemia or -lymphoblastic lymphoma, and/or B. Has at least one of lymph node or tumor, the diameter \>5 cm, and/or C. Lactate dehydrogenase (LDH) no less than 2 times the upper limit of normal (ULN).
* If newly diagnosed AL patient is with blood uric acid no greater than 8 mg/dL at the screening but with a high risk of TLS defined with one of the following below criteria: A. White blood cell (WBC) no less than 100.0 10-9/L, or B. WBC \< 100.0 10-9/L with LDH no less than 2 ULN.
* The patient will receive the chemotherapy, and will be confined in hospital for at least 14 days after first dose of rasburicase.

Exclusion criteria:

* Acute promyelocytic leukemia
* Patient who has been treated or planned to receive allopurinol within 72 hours of rasburicase administration.
* Patients with abnormal liver or renal function: alanine aminotransferase (ALT) \>5 ULN, total bilirubin \>3 ULN, serum creatinine \>3 ULN.
* Documented history of hereditary allergy or asthma.
* Patients with known deficiency of glucose-6-phosphate dehydrogenase (G6PD), or a history of hemolytic disease or methemoglobinemia.
* Patients with severe infection or active bleeding.
* Previous therapy with urate oxidase.
* Hypersensitive reaction against rasburicase or any of the other ingredients of the study drug.
* Patient is not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or patients potentially at risk of noncompliance to study procedures.
* Pregnant or breastfeeding woman.
* Woman of childbearing potential (WOCBP) not protected by highly-effective method(s) of birth control and/or who are unwilling or unable to be tested for pregnancy (see contraceptive guidance in Appendix A).
* Male participant with a female partner of childbearing potential not protected by highly-effective method(s) of birth control

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Primary outcomes:
measure: Incidence of AEs and SAEs
timeFrame: Day 1 to Day 7
Secondary outcomes:
measure: Number of responders after completion of rasburicase treatment under chemotherapy
timeFrame: Day 1 to Day 7
measure: Proportion of patients who can maintain the normal uric acid levels throughout the study
timeFrame: Day 1 to Day 7
measure: Percentage of the maximum decreasing degree of plasma uric acid level from baseline
timeFrame: Day 1 to Day 7
Other outcomes:
Locations:
city: China
country: China
facility: investigational site CHINA
Study officials:
affiliation: Sanofi
name: Clinical Sciences & Operations
role: STUDY_DIRECTOR
References: