Consortium Pediatric Praziquantel Program successfully completed Phase II in Ivory Coast
19 DEC 2018
The important milestone of Last Patient Last Visit (LPLV) in the clinical Phase II trial was achieved on November 17, 2018
The Pediatric Praziquantel (PZQ) Formulation program led by Merck KGaA, Darmstadt, Germany, within a Consortium of partners from private and public sectors, reached an important milestone: the Last Patient Last Visit (LPLV) in the clinical Phase II trial was recorded on November 17, 2018.
The investigators responsible for implementing the Phase II trial were provided by the Université Félix Houphouët-Boigny (UFHB), one of the eight Consortium partners, in Ivory Coast. The study aimed at assessing the efficacy and safety of two different orodispersible tablets (ODTs) formulations of PZQ in schistosoma-infected children under the age of six. Current results indicate that both ODT formulations are well tolerated at all doses tested; one formulation, levopraziquantel (L-PZQ) ODT, is confirmed for further development.
At present, the group of schistosoma-infected preschool-age children (over 20 million according to current estimation) is left untreated in public health programs due to lack of clinical data and a missing appropriate child-friendly formulation of the drug praziquantel (the current standard of care). This program wants to fill this treatment gap and the ODT which can be dissolved on the tongue or in a small quantity of water represents the solution for treating schistosomiasis in very young patients.
Implementing a trial involving vulnerable children living in remote rural settings in a lower middle income endemic country has not been an easy task; over 7900 screening visits have been conducted and 444 patients were enrolled in the study. The trial is an example of how a public-private collaborative partnership can promote capacity-building and high-quality pediatric clinical research on neglected tropical diseases in sub-Saharan Africa.
The success of this trial has been built up through a solid team spirit, and experienced and very motivated people who continue to be extremely committed towards making a pediatric PZQ formulation available to the very young patients infected with schistosomiasis.
With this objective in mind, the Pediatric PZQ team is currently preparing for the confirmatory Phase III trial in schistosoma-infected preschool-age children. Co-funded by the Consortium, the European & Developing Countries Clinical Trials Partnership (EDCTP) and the Global Health Innovative Technology (GHIT) Fund, this clinical study will be implemented in Ivory Coast and Kenya and is planned to start in the second quarter of 2019.
Schistosomiasis, also known as bilharzia, is one of the most prevalent parasitic diseases in Africa, and a very important one in terms of public health burden and economic impact. The disease affects over 200 million people worldwide with over 90% of cases occurring in sub-Saharan Africa; more than 10% are preschool-age children. An estimated 200,000 people die every year from the effects of schistosomiasis such as liver and kidney infections, bladder cancer, and blood loss.
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. Its mission is to develop, register and provide access to a suitable pediatric praziquantel formulation for treating schistosomiasis in this age group. The Consortium operates on a not-for-profit basis.
For more information: https://www.pediatricpraziquantelconsortium.org/