Project: Phase III clinical trials and registration of a new praziquantel orally disintegrating tablet formulation suitable for preschool-aged children with schistosomiasis

Acronym	PZQ4PSAC (Reference Number: RIA2016S-1641)
Duration	01/02/2018 - 31/01/2020
Project Topic	The Pediatric Praziquantel Consortium, founded in 2012, aims to develop and register a novel pediatric praziquantel orally disintegrating tablet (PZQ-ODT) and make it available for preschool-aged children in schistosomiasis-endemic countries. Such formulation represents an important advancement in addressing the huge disease burden and economic impact of schistosomiasis in sub-Saharan Africa. The goal of this project is to successfully implement the Phase III program that will allow the Consortium to submit the registration file to the European Medicinal Agency in Oct 2020. The key objectives of the Phase III program are: 1. To conduct an ICH GCP compliant Phase III clinical trial in Kenya and Cote d’Ivoire to demonstrate the efficacy and safety of a single dose of the PZQ ODTs in children age 3 months to 6 years infected with Schistosoma mansoni and Schistosoma haematobium. 2. To establish the final commercial process for both the Active Pharmaceutical Ingredient and the Drug Product at the respective final manufacturing sites. 3. To prepare the core registration dossier and have it ready for submission to the European Medicines Agency (EMA; Article 58). 4. To foster awareness of the project by communicating its results and achievements via existing communication channels at all stakeholder levels and to further refine the Product Delivery & Access plan and seek its endorsement by key stakeholders. 5. To provide the necessary project management and coordination activities to ensure the timely execution of the program. The EDCTP grant consortium consists of six beneficiaries: Université Félix Houphouët Boigny (UFHB), Kenya Medical Research Institute (KEMRI), Merck KGaA, Stichting Lygature, Swiss Tropical and Public Health Institute (TPH) and Imperial College of Science Technology and Medicine (ICL). Funding for the Phase III program has been secured through continuation of the cash contribution by Merck KgaA, in-kind contributions by four beneficiaries (Merck KGaA, Swiss TPH, Lygature and ICL), and a grant from the GHIT Fund. As part of the larger Pediatric Praziquantel Consortium, Astellas from Japan and Farmanguinhos (the technical-scientific unit institute of technology in drugs of the Fundação Oswaldo Cruz) in Brazil also provide in-kind contributions. The EDCTP contribution will allow EDCTP, the GHIT Fund and the Consortium partners to jointly support the fight against schistosomiasis as equal funding partners. In the first year, the Consortium has selected the PZQ-ODT formulation and dose with the best benefit/risk profile from the Phase II study. The selected formulation, dose and clinical Phase III trial design were confirmed by the EMA. Ethics confirmation from the Swiss IEC, and approvals from the Independent Ethics Committees in Cote d’Ivoire and Kenya have been received and clinical trial application submited to the regulatory authorities in both countries. Apart from Farmanguinhos (1st Drug Product manufacturer), the Consortium has scouted and selected a 2nd Drug Product manufacturing site in sub-Saharan Africa. The Access strategy was presented at a WHO Stakeholders meeting in Geneva, attended by representatives from potential procurement agencies and target countries. It has also submitted and secured another grant from the GHIT Fund (US$ 4,1 mio) to support among others the final regulatory submission stage and a supportive Phase III trial in Zimbabwe. More information on the project including our newsletter can be found on the website: www.pediatricpraziquantelconsortium.org.
Network	EDCTP2
Call	Strategic actions supporting large-scale clinical trials 2016

Project partner

Number	Name	Role	Country
1	Stichting Lygature	Coordinator	Netherlands
2	Kenya Medical Research Institute	Partner	Kenya
3	Merck KGaA	Partner	Germany
4	SCI Foundation	Partner	United Kingdom
5	Swiss Tropical and Public Health Institute	Partner	Switzerland
7	Université Félix Houphouët Boigny	Partner	Côte d'Ivoire