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Ongoing Clinical Research Studies for Prion Disease

Prion disease is currently an invariably fatal condition without treatment or cure. New treatments are currently being investigated, and observational studies (clinical research with no drug treatment) are also being done to better understand the disease. This site will be regularly updated with available research study information for prion disease. 


Below is a list of clinical research studies for prion diseases. 

For additional information about each study, including eligibility criteria, study locations, and enrollment details, please visit the linked ClinicalTrials.gov pages.


Clinical Trials

Clinical Trial

ClinicalTrials.gov Page

Status 
(U.S. Sites)

Status
(International Sites)

Community Statement

PrP-targeting siRNA Safety & Mechanism Study (PRiSM)

PRiSM

Recruiting

N/A

4/22/2026

A Trial of ALN-6457 in Adult Participants With Symptomatic Prion Disease (PrD)

ALN-6457

Not Recruiting

N/A

Pending

ION717-CS2: A Phase 1/2a Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION717 in Patients with Prion Disease

ION717-CS2

Not Recruiting

Not Recruiting

3/17/2026


Observational Studies

Clinical Trial

ClinicalTrials.gov Page

Status 
(U.S. Sites)

Status
(International Sites)

Teleneurology Assessment Program for Creutzfeldt-Jakob Disease (TAPCJD)

TAPCJD

Recruiting

N/A

PROSPECT Registry for Individuals at Risk for Genetic Prion Disease (PROSPECT)

PROSPECT

Recruiting

N/A