Oregon Health & Science University in Portland (USA) has conducted a first-in-class clinical trial for PKAN patients in the U.S. from 2019 to 2022 with a new compound 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 stored frozen over the course of three years now need to be analyzed in the laboratory.
The study duration had to be shortened in the end due to pandemic-related supply chain problems. But this decentralized study could initially be conducted without major adjustments despite the COVID-19 pandemic, because central clinic visits were not planned and all necessary data and samples could be sent to OHSU from the families' respective homes, which was in line with the pandemic restrictions anyway.
From December 2019 to September 2021, 77 children and adults with PKAN were enrolled in the study. A majority of the study participants participated as subjects until the end of their respective two-year study periods or until the end of the active portion of the study in the summer of 2022.
Next steps include review of all data and analysis of information by OHSU statisticians, including data on complications, subject compliance with study rules, and clinical information from the PKANready natural history study, which ran concurrently with the CoA-Z study.
Meanwhile, Dutch and British PKAN research teams are working on similar clinical trials. The study in the Netherlands is well advanced and the UK team expects to start their study this year. These studies differ from each other, and the team at OHSU hopes to gain helpful information from the differently conducted studies that can advance the development of CoA-Z.
Source: December 2022 Newsletter of the NBIA Disorders Association, p. 11: