Phase 2
N=444
An Open Label Dose Finding Safety and Efficacy in Children and Infants Infected With Schistosomiasis (S.Mansoni)
Schistosomiasis
Bottom Line
View on ClinicalTrials.gov: NCT02806232 ↗Enrolled (actual)
444
Serious AEs
0.4%
Results posted
Nov 2019
Primary outcome: Primary: Number of Participants With Clinical Cure Determined by Kato-Katz Method — 51; 43; 47; 46 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Biltricide (racemate praziquantel) oral tablets (Drug); Racemate Praziquantel ODT (Drug); Levo Praziquantel ODT (Drug)
- Age
- Pediatric · 0+ yrs
- Sex
- All
- Sponsor
- Merck KGaA, Darmstadt, Germany
- Primary completion
- Oct 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Clinical Cure Determined by Kato-Katz Method |
51; 43; 47; 46; 44; 49 | — |
| SECONDARY Egg Reduction Rate (Percent) |
94.6; 83.3; 88.6; 88.6; 88.3; 92.3 | — |
| SECONDARY Number of Participants With Clinical Cure Determined by Point-of-Care Circulating Cathodic Antigen (POC-CCA) Test |
8; 3; 3; 8; 4; 6 | — |
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
Data sourced from ClinicalTrials.gov (NCT02806232). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.