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NOT YET RECRUITING
NCT07832370
PHASE1/PHASE2

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

Sponsor: Regeneron Pharmaceuticals

View on ClinicalTrials.gov

Summary

This study will test a Regeneron drug called ALN-6457 ("study drug") to see if it can help treat Prion Disease (PrD). In PrD, the prion protein folds into an abnormal shape. These abnormal proteins build up in the brain and cause damage to brain cells, leading to problems with thinking, memory, and movement. ALN-6457 is designed to reduce the amount of prion protein made in the brain. ALN-6457 works by targeting the genetic "instructions" that cells use to produce this protein, with the goal of slowing or stopping disease progression. This study is looking at: * What side effects ALN-6457 might cause * How well ALN-6457 works * What is the best dose of ALN-6457 for treating prion disease * How much ALN-6457 is in the blood and Cerebrospinal Fluid (CSF, fluid that surrounds the brain and spinal cord) at different times

Official title: Phase 1/2 Open-Label Trial With Extended Follow-Up to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Multiple Doses of ALN-6457 in Participants With Symptomatic Prion Disease

Key Details

Gender

All

Age Range

18 Years - Any

Study Type

INTERVENTIONAL

Enrollment

60

Start Date

2026-10-23

Completion Date

2031-02-27

Last Updated

2026-09-22

Healthy Volunteers

No

Conditions

Interventions

DRUG

ALN-6457

Administered per the protocol