Prospective, Single-arm, Multicentre Study, Using a Historical Control, to Evaluate the Efficacy/Safety and Population Pharmacokinetics of Benznidazole in Children With Chronic Indeterminate Chagas Disease

LPRI747-301

This study evaluate the efficacy and safety of benznidazole in the treatment of chronic indeterminate Chagas disease in children. All subjects will receive benznidazole and results will be compared to historically to a placebo arm.

2019-09-19

2027-08-30

2028-03-30

Active (not recruiting)

Early phase I

178

Inclusion Criteria: * Age between 2 years and 18 years (age limits inclusive) * Diagnosis of Trypanosoma cruzi (T. cruzi) infection by conventional serology based on positive ELISA and at least 1 other positive conventional serology test (recombinant ELISA or IIF) * Written informed consent by parent/legal representative and informed assent from patients if \>7 years old when applicable (as requirements may vary by country and by site) * Females of childbearing potential (ie, female patients who have experienced menarche) and male patients must agree to use highly effective contraception if sexually active from the time of signing of the informed consent/assent form until ≥5 days after the last dose of study treatment Exclusion Criteria: * Pregnant or intending to become pregnant during treatment and within 5 days after the last dose of study treatment * Patient presenting any other acute or chronic health conditions, which in the opinion of the Principal Investigator (PI), may interfere with the pharmacokinetic (PK), efficacy, and/or safety evaluation of the study treatment * Signs and/or symptoms of acute Chagas Disease * Known history of hypersensitivity or serious adverse reactions to nitroimidazoles * History of Chagas Disease treatment with Benznidazole (BZN) or nifurtimox * Immunocompromised (clinical history compatible with human immunodeficiency virus (HIV) infection, primary immunodeficiency, or prolonged treatment with corticosteroids or other immunosuppressive drugs) * Abnormal laboratory test values (as per protocol-specified ranges) at Screening for the following parameters: total white blood cell (WBC) count, platelet count, alanine aminotransferase (ALT), aspartate aminotransferase (AST), total bilirubin, and creatinine * Abnormal ECG (as per protocol-specified ranges) and/or any Chagas disease associated findings * Any condition that prevents the patient from taking oral medication * Patient is known to or suspected of not being able to comply with the study protocol and the use of the investigational medicinal product (IMP) * Evidence or history of alcohol or drug abuse (within the last 12 months) * Any planned procedure that may interfere with highly effective contraception during treatment and within 5 days after the last dose of study treatment * Employee of the Investigator or trial centre, or family member of the employees or the Investigator * Any condition that, in the opinion of the Investigator, may jeopardise the trial conduct according to the protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'Historical control-based comparison, single-arm assignment, multicentre study', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 178, 'type': 'ACTUAL'}}

2023-08-14