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PLANready Natural History Study

Help Advance Research by Participating in the PLANready Natural History Study

For the past two years the INADcure Foundation has been partnering with the NBIAcure Team at Oregon Health and Science University. The Foundation is funding a 5-year Natural History Study for PLAN/INAD.

The OHSU Natural History Study is Recruiting Participants!

The purpose of this study is to help researchers better understand the natural history of PLAN/INAD, meaning how symptoms appear and change over time. By studying individuals with INAD the NBIA Research Group at OHSU hopes to identify disease markers that can be used in future clinical trials. A disease marker is any symptom or measurement that happens reliably in a disease, changes predictably with disease progression, and becomes “better” with successful treatment. Natural history studies provide data that serve as the foundation for future drug trial, are required by the FDA, and can hold up a study from starting if natural history data is not sufficient. Supporting this work is essential in order to ensure the rapid advancement through the FDA’s clinical trial application process when potential new drugs or treatments (like gene therapy) are ready for clinical trials.

What is a Natural History Study and Why is it Important?

Based on the results of a recent feasibility study, the OHSU team has set a goal to enroll 8-10 more subjects with infantile PLAN (INAD). Currently, there are 28 participants. Enrollment has been their greatest challenge to date, and we want to help because we know how important this data will be to ensure the rapid advancement through the FDA’s clinical trial application process when potential new drugs are ready for clinical trials.

  • Participation in the study takes about 20 minutes every 3 to 6 months.
  • The study is completed online through a secure portal.
  • Fluency in English is required
  • Those who have lost children can also participate with a one-time entry.

To learn more please reach out to Allison Gregory, M.S., C.G.C. at gregorya@ohsu.edu.

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