Overview
The purpose of this trial is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic impact of PrP-siRNA in symptomatic prion disease patients.
Description
This is a first-in-human, open label, single ascending dose study in participants with prion disease. The study will consist of a screening period of up to 2 weeks, administration of a single intrathecal dose of PrP-siRNA, and a 24-week follow-up period. Multiple dose levels will be tested. This trial also includes an observational arm in which participants will not receive investigational drug, and will be followed for an 8-week period after baseline.
Eligibility
Key inclusion criteria:
- clinically manifested symptoms of prion disease, in the opinion of the investigator;
- a diagnosis of probable prion disease according to CDC criteria;
- a positive CSF RT-QuIC or PRNP genetic test;
- no more than moderate functional impairment as quantified by an MRC-PDRS score ≥15; and
- availability of a study partner to assist with study procedures.
Key exclusion criteria:
- pregnancy;
- contraindication to LP; or
- recent participation in a different prion disease clinical trial.
Additional inclusion and exclusion criteria apply and will be evaluated at screening.
