PrP-targeting siRNA Safety & Mechanism Study
Recruiting
Phase 1
Interventional Study
Prion Disease
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
At a Glance
- Age
- 18 and older
- Sex
- Any
- Trial phase
- Phase 1
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 30 (estimated)
- Sponsor
- Broad Institute of MIT and Harvard · Other
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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.
Trial Locations
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Eligibility Criteria
Key inclusion criteria:
1. clinically manifested symptoms of prion disease, in the opinion of the investigator;
2. a diagnosis of probable prion disease according to CDC criteria;
3. a positive CSF RT-QuIC or PRNP genetic test;
4. no more than moderate functional impairment as quantified by an MRC-…
Contacts