- Laboratory research
- Phase 1Is it safe? What dose?
- Phase 2Does it seem to work?
- Phase 3Does it work in more people?
- Regulator review
This section lists trials and studies in prion disease. Completed trials of flupirtine, pentosan polysulfate, quinacrine and doxycycline are described in a 2015 review (see flupirtine and doxycycline). They enrolled people with symptoms, mostly with sporadic CJD, and none of the randomised trials showed a survival benefit. Pages for current and past trials and for observational studies are linked below, and approaches still in the laboratory are under therapies.
Two PrP-lowering trials are active as of the CJD Foundation page checked on 31 October 2026: the Ionis antisense drug trial (PrProfile) and the siRNA trial PRiSM. A Regeneron siRNA trial, ALN-6457, is also listed. Who may join is decided by each sponsor and trial team, and entry criteria change over time, so ask the trial contact or a prion specialist. Joining a trial, or not joining, is a personal choice.
Explore
- ALN-6457 (NCT07832370)Registry record NCT07832370: Regeneron's Phase 1/2 open-label trial of ALN-6457 in adults with symptomatic prion disease, not yet recruiting, estimate
- Current trials
- Doxycycline prevention in FFI carriers (EudraCT 2010-022233-28)Italian trial of doxycycline in one fatal familial insomnia family: carriers over 42 years receive doxycycline and non-carrier relatives receive place
- Doxycycline versus placebo in CJD (Italy and France, 121 patients)French and Italian phase 2 randomised placebo-controlled trial of doxycycline 100 mg daily in CJD, published in January 2014.
- Flupirtine in CJD (German phase 2)German randomised double-blind trial of the painkiller flupirtine in 28 CJD patients, published in 2004: slower decline on a cognitive scale but no su
- Past trials and what they found
- PRiSM (NN112)PRiSM (NN112, NCT07444580) is a Phase 1 first-in-human trial of the divalent siRNA PrP-siRNA given as one lumbar-puncture dose to people with symptoma
- PrProfile (NCT06153966): ION717 in prion diseaseActive, not recruiting (enrolment completed December 2024).
- Quinacrine in CJD (UK PRION-1 and UCSF placebo-controlled trial)Quinacrine studies in prion disease: the UK PRION-1 patient-preference study (107 volunteers, 300 mg a day, reported March 2009), a French compassiona
- Observational studies and registriesObservational studies and registries in prion disease, such as the UK National Prion Monitoring Cohort, which follow patients without giving a treatment.