PKAN study with CoA-Z completed in the USA

From 2019 to 2022, Oregon Health & Science University in Portland (USA) conducted the first clinical trial for PKAN patients in the USA with a new preparation called "CoA-Z" for the treatment of PKAN.

This first part of the study has now been completed and analysis of the data has begun with the aim of determining the safety and tolerability of CoA-Z and whether it could be suitable as a therapy for PKAN. To do this, hundreds of biomarker blood samples collected and frozen over the course of three years must now be analyzed in the laboratory.

The duration of the study had to be shortened in the end due to pandemic-related problems with the supply chain. However, despite the COVID-19 pandemic, this decentralized study could initially be carried out without significant adjustments, as central clinic visits were not planned and all necessary data and samples could be sent to the OHSU from the families' respective homes, which was in line with the restrictions imposed by the pandemic anyway.

From December 2019 to September 2021, 77 children and adults with PKAN were enrolled in the study. The majority of the study participants took part as subjects until the end of their two-year study period or until the end of the active part of the study in summer 2022.

Next steps are for OHSU statisticians to review all data and analyze the information, including data on complications, subject adherence to study rules, and clinical information from the PKANready natural history study, which took place in parallel with the CoA-Z study.

Meanwhile, Dutch and British PKAN research teams are working on similar clinical trials. The study in the Netherlands is already well advanced and the UK team expects to start their study this year. These trials are different from each other and the team at OHSU is hoping to gain useful information from the different trials that can help drive the development of CoA-Z.

Source: December 2022 Newsletter of the NBIA Disorders Association, p. 11:
https://www.nbiadisorders.org/news-events/nbia-newsletters/62-2022-newsletters/477-2022-december-newsletter/