An Open Label Dose Finding Safety and Efficacy in Children and Infants Infected With Schistosomiasis (S.Mansoni)
Condition: Schistosomiasis
MeSH terms: Schistosomiasis
Intervention: Biltricide (racemate praziquantel) oral tablets; Racemate Praziquantel ODT; Levo Praziquantel ODT
Intervention description: Biltricide (600 mg tablet) was administered to participants at a dose of 20 mg/kg in Part 1, Cohort 1 and at a dose of 40mg/kg in Part 1, Cohort 2.; Racemate Praziquantel (PZQ) (150) mg was administered at a dose of 40 mg/kg in Part 1, Cohort 3 and at a dose of 60 mg/kg in Part 1, Cohort 4.; Levo PZQ (150 mg) was administered at a dose of 30 mg/kg in Part 1 Cohort 5, 45 mg/kg Part 1 Cohort 6, 60 mg/kg Part 1 Cohort 7, 50 mg/kg Part 2 Cohort 8, and 50 mg/kg Part 2 Cohort 9.
Intervention MeSH terms: Praziquantel
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 444.0
Sex: All
Minimum age: 3 Months
Maximum age: 6 Years
Healthy volunteers: No
Keywords: Praziquantel ODT formulation; Biltricide

Summary:

      The Phase II study consisted of two parts, part 1 is open label, randomized, controlled and
      exploratory dose finding in children aged between 2 and 6 years infected with S. mansoni.
      Part 2 investigated efficacy and safety with the selected formulation and dosage in S.
      mansoni infected children aged between 3 months - 2 years.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Male and female children aged 2 to 6 years (Part 1) and 3 to 24 months (Part 2)

          -  S. mansoni positive diagnosis defined as positive egg counts in stool (greater than
             [>]1 egg/1 occasion) according to World Health Organization (WHO) classification :
             light (1-99 eggs per gram of faeces), moderate (100-399 eggs per gram of faeces) and
             heavy (greater than or equal to [>=]400 eggs per gram of faeces) infections

          -  Minimum weight of 8.0 kg in 2- to 6-year-old children and of 4.0 kg in 3- to 24-month
             infants

             • Parents/legal representative ability to communicate well with the Investigator, to
             understand the protocol requirements and restrictions, and willing their children to
             comply with the requirements of the entire trial, i.e.

          -  To be examined by a study physician at screening and 14-21 days after treatment

          -  To provide stool and urine samples at screening, 24 hours and 8 days after treatment,
             as well as 14-21 days after treatment

          -  To provide finger prick blood samples for Pharmacokinetics (PK) studies and blood
             samples for safety assessments

        Exclusion Criteria:

          -  Treatment in the 4 weeks prior to study screening with Praziquantel (PZQ) , other
             anti-helminthic, antimalarial or anti-retroviral compounds or any other medication
             that might affect the PK of PZQ such as certain antiepileptics (e.g., carbamazepine or
             phenytoin), glucocorticosteroids (e.g., dexamethasone), chloroquine, rifampicin or
             cimetidine

          -  For children being breast fed, treatment of the mothers/wet nurses with PZQ in the 3
             days prior to administration of Investigational medicinal product

          -  Previous history of adverse reactions associated with PZQ treatment

          -  Marked increases of the liver transaminases (alanine aminotransferase and/or aspartate
             aminotransferase) above 3x Upper Limit of Normal (ULN)

          -  History of acute or severe chronic disease including hepato-splenic schistosomiasis

          -  Fever defined as temperature above 38.0 degree centigrade

          -  Debilitating illnesses such as tuberculosis, malnutrition, etc. as well as a medical
             history of seizures

          -  Mixed S. haematobium and S. mansoni infections

          -  Findings in the clinical examination of schistosome-infected children participating in
             the study as performed by the study clinician on the treatment day, that in the
             opinion of the Investigator constitutes a risk or a contraindication for the
             participation of the subject in the study or that could interfere with the study
             objectives, conduct or evaluation

          -  Unlikelihood to comply with the protocol requirements, instructions and trial-related
             restrictions, e.g., uncooperative attitude, inability to return for follow-up visits,
             and improbability of completing the trial