Clinical trial NCT02806232: An Open Label Dose Finding Safety and Efficacy in Children and Infants Infected With Schistosomiasis (S.Mansoni)
Official title: Open-label, Dose-finding, 2-parts, Efficacy Phase II Study With Three Formulations (Racemate Raziquantel Commercial Oral Tablets, New Oral Disintegrating Tablets of Racemate Praziquantel and L-praziquantel) in Schistosomiasis (S. Mansoni) Infected Children Aged 2-6 Years (Part 1), Followed by an Assessment of Efficacy and Safety With the Selected Formulation and Dosage in S. Mansoni Infected Infants Aged 3-24 Months (Part 2)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Schistosomiasis
Keywords: Praziquantel ODT formulation
Biltricide
Lead sponsor: Merck KGaA, Darmstadt, Germany
Sponsor class: INDUSTRY
First posted: 2016-06-20
Enrollment: 444
Sex: ALL
Minimum age (years): 3
Maximum age (years): 6
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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

Primary outcomes:
measure: Number of Participants With Clinical Cure Determined by Kato-Katz Method
timeFrame: 14-21 days post treatment
Secondary outcomes:
measure: Egg Reduction Rate (Percent)
timeFrame: Baseline, 14-21 days post treatment
measure: Number of Participants With Clinical Cure Determined by Point-of-Care Circulating Cathodic Antigen (POC-CCA) Test
timeFrame: Day 2, Day 8 and 14-21 days post treatment
Other outcomes:
Locations:
city: Darmstadt
country: Germany
facility: Please Contact the Communication Center
Study officials:
affiliation: Merck KGaA, Darmstadt, Germany
name: Medical Responsible
role: STUDY_DIRECTOR
References: