PrP-targeting siRNA Safety & Mechanism Study
ConditionPrion Disease
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18+
SponsorBroad Institute of MIT and Harvard
About this trial
The purpose of this trial is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic impact of PrP-siRNA in symptomatic prion disease patients.
Eligibility criteria
Qualifiers
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
Disqualifiers
pregnancy;
contraindication to LP; or
recent participation in a different prion disease clinical trial.
Trial design
Treatments tested in this trial
- PrP-siRNA
Treatment groups
30 Participants
are divided into 2 treatment groupsSponsors and collaborators
Broad Institute of MIT and Harvard
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
Collaborator
Source ClinicalTrials.gov