Development program

The Pediatric Praziquantel Consortium aims to reduce the global disease burden of schistosomiasis by addressing the medical need of infected preschool-age children. Our mission is to develop, register and provide access to a suitable pediatric praziquantel formulation for treating schistosomiasis in this age group. After having successfully completed the pre-clinical phase, the program has progressed into clinical development.

Soon after its formation, the consortium established a pediatric drug development program, divided into two major steps: preclinical development, and clinical development.

Preclinical development

Our preclinical program (completed in 2014) focused on the development of novel child-appropriate praziquantel formulations. For more information about these orally dispersible (ODT) tablets and the formulation technology, please visit our pediatric formulation section

The preclinical development program consisted of six major activities: 

  • Manufacture of the L-Praziquantel active pharmaceutical ingredient (API)
  • Development of novel orally dispersible tablet (ODT) formulation candidates
  • Development and validation of analytical and bioanalytical methods 
  • Metabolism and pharmacokinetics
  • Toxicology, including safety pharmacology
  • Good Manufacturing Practice (GMP): manufacture and documentation of the new ODT for use in clinical trials.

Clinical development

The clinical program is set up in line with FDA recommendations for pediatric development. It has been designed with the support of regulatory authorities and a panel of international experts, including clinicians from endemic countries. PZQ is an existing drug, but the PZQ-ODT formulations are new and require a full phase I-III clinical development program:

  • Two phase I bioavailability studies in healthy adult volunteers were performed in South Africa with the aim to determine the pharmacokinetic properties of the Racemate (Rac)-PZQ and L-PZQ formulation candidates in comparison to the current Cesol 600 mg PZQ commercial racemate tablet formulation. The pharmacokinetics of the two formulations candidates was characterized and both formulations showed a good safety profile and acceptable palatability.  The studies were completed in 2015.
  • A swill-and-spit taste study was performed in 2015 in Tanzanian children (age 6-11 years) to compare the “overall palatability” of the new L-PZQ ODT and Racemate PZQ ODT formulations with the current Cesol 600 mg PZQ commercial racemate tablet formulation. This study was completed in 2015.
  • A phase II safety and efficacy study is ongoing in Ivory Coast in S. mansoni-infected children of different ages (range 3 months - 6 years). The study outcome will hopefully allow the selection of the formulation (L-PZQ or Rac-PZQ) and the dose that will be included in the Phase III program.
  • Phase III single-arm confirmatory clinical trials in S. mansoni and S. haematobium-infected children (age 3 months - 6 years) are planned.

All of our clinical trials are conducted according to Good Clinical Practice and applicable ethical guidance and regulations, in order to protect the wellbeing and rights of adults and children enrolled in the trial. The trials are designed to reflect public health needs and priorities of the countries in which the trials are carried out.

For more information about where we stand, please visit our timeline.