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CoA-Z Clinical Trial

We will share information about the CoA-Z phase II clinical trial on this page so check back often for more details!
Last updated: 11/15/2019

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This will be the first in a series of updates on our work on CoA-Z in preparation for the trial you’ve all been waiting for. We want to share information about the plans for the US and Canadian trial, since we are now close to launch. In addition to the incredible support received through local small grants and your donations to the Spoonbill Foundation, Stichting Lepelaar, and “ZZF”, the Dutch Foundation for rare diseases, all of which have truly kept the trial plans moving forward, our team at OHSU has now also been awarded a large grant from the National Institutes of Health. Specifically, the Eunice Kennedy Shriver National Institute of Child Health and Human Development has awarded us funding to support running the CoA-Z clinical trial over several years in the US and Canada. Spoonbill dollars will continue to be instrumental to fund the ongoing manufacturing and formulation of CoA-Z, database development, and other costs not covered by the grant.

Foundational Study

In order to prepare for the CoA-Z clinical trial, we are currently completing a small, foundational study of CoA-Z in a small number of adults and children with PKAN. These research participants were the first people to ever be given CoA-Z. This was done under our supervision at OHSU, and they received the compound for a very short predetermined period. The information gained from this short study has helped us refine the dosing plan and schedule of activities for the clinical trial, such as when blood samples will be collected. While this study was not a strict requirement by the FDA, we and our reviewers at the NIH felt it was important to help us confirm that CoA-Z is safe, at least over the short period of this study, and to test some of the processes and parameters we have set for the phase 2 clinical trial before it is officially launched. We are very grateful to the individuals and families who were willing to travel to OHSU for an extended stay in Portland to help us get this critical data. Their contributions will benefit everyone who participates in the upcoming trials.

CoA-Z Paper Publication

We also want to provide everyone with information about an important new scientific paper authored by our OHSU team in partnership with our Dutch colleagues. The paper was released online in the journal EMBO Molecular Medicine. This peer-reviewed paper helps explain the rationale behind our proposed CoA-Z trial in humans. In addition to providing a link to the paper once it went live, we also developed some Frequently Asked Questions that summarize key points and their relevance for PKAN families. Dr. Sibon’s group in the Netherlands also published a separate paper in the same journal issue that reveals important insights into the biochemical changes in PKAN and related disorders. Together, these two publications provide a solid foundation for launching studies of CoA-Z in people.

You can read both papers and the FAQs at the links below:
CoA-Z Paper FAQs
CoA‐mtACP‐PDH Pathway Paper FAQs

Acknowledgements

We would like to thank the following groups and organizations who have supported this trial:

  • Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
  • National Institute of Neurological Disorders and Stroke (NINDS)
  • Food Innovation Center at Oregon State University (OSU)
  • Collins Foundation
  • Friends of Doernbecher
  • Spoonbill Foundation / Lepelaar Stichting
  • NBIA Disorders Association (NBIADA)
  • Oregon Clinical and Translational Research Institute (OCTRI)