CoA-Z Trial Update

Update: OHSU CoA-Z in PKAN trial concludes and analysis begins

By Allison Gregory

 December 2022

 Allison Gregory
 Allison Gregory, a genetic counselor from the NBIA research group at OHSU, helped manage the CoA-Z trial.

Our NBIA research team at Oregon Health & Science University has concluded the first part of its study of a compound to treat PKAN, called CoA-Z, and will begin analyzing the data to determine its safety, how well it was tolerated, and whether it holds potential as a therapy for PKAN.

Although supply-chain issues brought on by the COVID-19 pandemic caused us to limit the trial’s duration, its home-based, remote design allowed the study to continue without needing to be changed significantly to adapt to COVID restrictions.

The first subjects were enrolled in December of 2019, and enrollment continued through September of 2021. During that time, 77 children and adults with PKAN joined. The study had a high retention rate, meaning most participants were able to continue until their two-year study time had ended, or until the active part of the study ended in summer 2022.

While completing everyone’s participation was a huge milestone, there is still much work to be done. Hundreds of biomarker blood samples collected and frozen over nearly three years are being processed and analyzed in the lab.

Next steps are to check all of the data and collaborate with OHSU statisticians to analyze the information, including data on complications and compliance with the study, and clinical information from the PKANready natural history study that ran in parallel with the trial.

Meanwhile, teams in The Netherlands and the U.K. are moving forward with similar trials. The Dutch team is well along with its trial, and the U.K. team expects to launch in 2023. The trials differ in valuable ways that we hope will provide more information to advance CoA-Z development.

To everyone who participated and has supported the trial, we send our thanks.


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