From 2019 to 2022, Oregon Health & Science University in Portland (USA) conducted a first clinical trial for PKAN patients in the USA with a new drug called "CoA-Z" for the treatment of PKAN.
This first part of the study has now been completed and the 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.
In the end, the duration of the study had to be shortened due to pandemic-related problems with the supply chain. However, despite the COVID-19 pandemic, this decentralised study was initially able to be carried out without significant adjustments, as central hospital visits were not planned and all necessary data and samples could be sent to OHSU from the families' respective hometowns, 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 participated 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.
The next steps are to review all data and analyze the information by OHSU's statisticians, including data on complications, subjects' adherence to study rules, and clinical information from the PKANready natural disease course 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 working group in the UK expects to start its study this year. These studies are different from each other, and the team at OHSU hopes to find helpful information from the different studies that can drive the development of CoA-Z.
Source: NBIA Disorders Association December 2022 Newsletter, p. 11: