The eWHORM partnership started a Phase II proof-of-concept adaptive basket trial of oxfendazole following a favourable review of the trial protocol from the European Medicines Agency (EMA). The study is examining the safety and efficacy of oxfendazole in treating multiple helminthic diseases, including onchocerciasis, loiasis, mansonellosis, and soil-transmitted helminthic infections. The innovative design of the trial aims to expedite drug development, improve trial efficiency, optimize resource utilization, and enable swifter access to improved treatments for patients with helminthic infections.

The HELP Consortium’s successful efforts have demonstrated the safety of our field-adapted oxfendazole formulation in a Phase I trial performed by partners Swiss TPH and Ifakara Health Institute in Tanzania, opening the path to advance oxfendazole to the next stage of clinical evaluation. The newly established eWHORM partnership is now designing a Phase II proof-of-concept adaptive basket trial  for oxfendazole, targeting river blindness and several other helminth infections. This innovative trial design aims to expedite drug development, improve trial efficiency, optimize resource utilization, and ultimately enable swifter access to improved treatments for patients.

All regulatory approvals were obtained for the Phase I study in Tanzania, sponsored by Swiss Tropical and Public Health Institute (Swiss TPH) and conducted by the Ifakara Health Institute in Tanzania. The Phase I clinical trial assessing the bioavailability of oxfendazole was initiated and final follow-up visits were completed for the first two patient cohorts. Two safety review committee meetings were held, and no safety concerns were noted.

In 2021, we began preparing for a Phase I trial to test the bioavailability of an oxfendazole tablet that is field adapted and easy to use. The trial will be sponsored by the Swiss Tropical and Public Health Institute and conducted by the Ifakara Health Institute in Tanzania. 

No serious adverse events occurred in the single ascending dose or multiple ascending dose studies (NCT02234570 and NCT03035760, clinicaltrial.gov) conducted by the Oxfendazole Development Group under the auspices of the US National Institute of Allergy and Infectious Diseases. Based on the data in healthy volunteers, oxfendazole applied orally in a liquid formulation was safe and well tolerated at doses up to 60 mg/kg and showed acceptable safety and tolerability profiles after five repeated daily doses of up to 15 mg/kg, with a few reports of non-serious adverse events in the two studies. There was no increase in adverse events with an increased dose.

Therefore, the available data suggest that oxfendazole is safe for use in humans and a Phase I bioavailability study is planned using a solid, field-adapted tablet. With recently obtained funding from the European Union’s Horizon 2020 research and innovation programme, the HELP Consortium will conduct a bioavailability Phase I trial in Tanzania to evaluate the safety, tolerability, and pharmacokinetics of the tablet.

DNDi developed a field-adapted tablet.