Status as of 2026-10-16. Please check the study registry for the latest position.
Key facts
- Status: Completed per EU register; results not located.
- Design correction: participants chose not to learn their own FFI genotype, so the trial treats 10 carriers with doxycycline and gives placebo to 15 non-carriers from the same family; it is funded by Fondazione Telethon.
- Dose 100 mg per day (200 mg since June 2019) with assessments every six months; results are not yet published in the sources found (the EU register lists the trial as completed).
- Mice: doxycycline improved memory and circadian rhythm but did not delay onset or prolong survival.
- As of 2013 no drug, supplement or lifestyle measure had been shown to delay genetic prion disease.
- Where: Italy
- Dates: Started 2011; planned ten years
- In April 2015 an independent review listed it as the only ongoing prion trial, in a single Italian fatal familial insomnia family.
- Dose was 100 mg a day, raised to 200 mg from June 2019, with assessments every six months.
- The mouse evidence was modest: doxycycline improved memory and circadian rhythm but did not delay onset or prolong survival.
Timeline
- 2011Trial starts with a planned ten-year duration.
- 2015Listed as the only ongoing prion trial.
- 2019Dose raised to 200 mg a day.
Sources for this page
- EU Clinical Trials Register: Fatal familial insomnia, preventive treatment with doxycycline of at-risk individuals (2010-022233-28)
- Preventive study in subjects at risk of fatal familial insomnia (Prion 2015)
- Preventive pharmacological treatment in subjects at risk for FFI: science and public engagement (Prion 2022; participants and funding)
- Doxycycline in FFI mice (Neurobiology of Disease 2021; PMC full text)
- cureffi: Current status of efforts to delay genetic prion disease in humans (2013)
- cureffi.org: Completed clinical trials in prion disease (15 April 2015)