18 September 2019

From the Program Lead: The Consortium has started the Phase III trial and is preparing for access

The pivotal phase III study with the L-praziquantel pediatric oral dispersible tablets (ODTs) has started, shortly after establishing the recommended dose and encouraging results of the phase II study in pediatric schistosomiasis.

Dear reader,

I am pleased to announce the start of the pivotal phase III study with the L-praziquantel pediatric oral dispersible tablets (ODTs), shortly after establishing the recommended dose and encouraging results of the phase II study in pediatric schistosomiasis. This study is designed to evaluate the efficacy and safety of the L-PZQ ODTs in children 3 months to 6 years of age infected with schistosomiasis. It is the last step of the clinical development program which, in case of positive outcome, will allow us to complete the clinical data package needed for the registration file.

Last March, the Consortium partners had the opportunity to visit one of the clinical sites of this study based in Homabay (Kenya).This event was followed by a meeting at the Kenya Medical Research Institute (KEMRI) in Kisumu, where the partners acknowledged their strong bond and continued commitment to the program and learned from local stakeholders about some of the issues for ensuring access to the pediatric praziquantel formulation in the future. Please refer to the short articles in this newsletter on our visit to Kenya and the phase III trial currently ongoing.

In addition, the Global Health Innovative Technology (GHIT) Fund announced it will invest another US$ 4,1 million to support the development, registration and access of our novel child-friendly praziquantel ODT formulation. I am grateful that the GHIT Fund has once more reaffirmed its confidence in our goal to close the current treatment gap for preschool-age children suffering from schistosomiasis.

The team has also continued working to shape potential access paths for the pediatric product once it will be registered.  Since the start of the Consortium in 2012, we have continuously sought and received guidance and feedback from the neglected tropical diseases (NTD) department of the World Health Organization (WHO-NTD). Last December, WHO-NTD held a Stakeholders’ Meeting on access to pediatric praziquantel for preventive chemotherapy. The meeting allowed us to share the current status of the project to prospective funders and endemic country representatives, and to discuss future procurement mechanisms.

I hope you will enjoy this newsletter, and please feel free to reach out to us if you would like to learn more about the Consortium and our work.