The Pediatric Praziquantel Consortium is an international partnership that 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, with proven safety and efficacy 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.
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.
Our clinical development program is in line with FDA recommendations for pediatric development. It has been designed with the help of clinicians from endemic countries and has been discussed with representatives from endemic country regulatory authorities.
The program comprises several studies:
- Phase I bioavailability studies in healthy adult volunteers in South Africa, to determine the pharmacokinetic properties of the Racemate (Rac)-PZQ and L-PZQ formulation candidates in comparison to the current 600 mg PZQ commercial racemate tablet formulation (Cesol). These studies were completed in 2015.
- A swill-and-spit taste study in Tanzanian school-age children, with infected and healthy volunteers, to assess overall palatability of the new candidate pediatric ODT formulations. This study was completed in 2015.
- A phase II safety and efficacy study in S. mansoni-infected children of different ages (range 3 months - 6 years) in Ivory Coast. This study, which is currently ongoing, will assess the two formulation candidates L-PZQ and Rac-PZQ ODTs.
- A phase III pediatric clinical trial in Africa is planned after successful completion of the current phase II study, expected in 2017.
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.