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 (RAISE)

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

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

Study Overview

• Status: Completed
• Conditions: Lymphoma
• Intervention / Treatment: Drug: RASBURICASE SR29142

Detailed Description

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

• Study Type: Interventional
• Enrollment (Actual): 50
• Phase: Phase 4

Contacts and Locations

Study Locations

• China
  • China, China
    • investigational site CHINA

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 18 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: rasburicase; rasburicase 0.20 mg/kg/day by intravenous (IV) over 30 minutes for 1 to 5 days according to the level of plasma uric acid or Investigator's clinical judgement	Drug: RASBURICASE SR29142; Pharmaceutical form:solution for infusion Route of administration: intravenous

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of AEs and SAEs	Incidence of AE or SAE will be summarized as the number and percentage of subjects who experienced any AE or SAE during the treatment period.	Day 1 to Day 7

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of responders after completion of rasburicase treatment under chemotherapy	Response will be defined as achievement of normal uric acid levels (≤ 8.0 mg/dL) in those patients whose uric acid levels are >8.0 mg/dL.	Day 1 to Day 7
Proportion of patients who can maintain the normal uric acid levels throughout the study	In those patients whose baseline plasma uric acid levels are ≤ 8 mg/dL but with a high risk of TLS.	Day 1 to Day 7
Percentage of the maximum decreasing degree of plasma uric acid level from baseline		Day 1 to Day 7

Collaborators and Investigators

• Sponsor: Sanofi

Study record dates

Study Major Dates

• Study Start (Actual): May 14, 2020
• Primary Completion (Actual): March 12, 2021
• Study Completion (Actual): March 12, 2021

Study Registration Dates

• First Submitted: April 13, 2020
• First Submitted That Met QC Criteria: April 13, 2020
• First Posted (Actual): April 16, 2020

Study Record Updates

• Last Update Posted (Actual): April 25, 2022
• Last Update Submitted That Met QC Criteria: April 22, 2022
• Last Verified: April 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Lymphoma; Hyperuricemia; Antirheumatic Agents; Gout Suppressants; Rasburicase
• Other Study ID Numbers: RASBUL09107; U1111-1233-0737 (Other Identifier: UTN)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Yes
• IPD Plan Description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No