PrP-targeting siRNA Safety & Mechanism Study
A legfontosabb adatok
- Hol
- Boston, Cleveland, Nashville, New York és Rochester (Egyesült Államok)Mind a 5 helyszín megnézése
- Fázis
- 1. fázis
Helyszínek itt: Egyesült Államok
- Massachusetts General HospitalBoston, Egyesült ÁllamokContact: MGH Prion Studies CoordinatorRésztvevőt keres
- University Hospitals Cleveland Medical Center*Cleveland, Egyesült ÁllamokContact: Mary Andrews, BA, RRT, CCRPRésztvevőt keres
- Vanderbilt University Medical CenterNashville, Egyesült ÁllamokContact: Kelly Harper, BPSRésztvevőt keres
- Columbia University Medical CenterNew York, Egyesült ÁllamokContact: Katrina Cuasay, BARésztvevőt keres
- Mayo ClinicRochester, Egyesült ÁllamokContact: Sherry Klingerman, CCRPRésztvevőt keres
Kérdések a vizsgálat csapatának
- Milyen gyakran kell bejárnom, és egy-egy alkalom mennyi ideig tart?
- Megtérítik az utazási költséget?
- Megtarthatom a saját orvosomat?
- Mi történik, ha ki akarok lépni?
Contact the study team: see the registry record → Contacts and Locations
Oldal nyomtatása az orvosomnakA vizsgálat adatai
- Ki végzi
- Broad Institute of MIT and Harvard
- Azonosító
- NCT07444580
- A regiszteradat frissítve
- 2026. október 16.
- Eredeti leírás
The purpose of this trial is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic impact of PrP-siRNA in symptomatic prion disease patients and in pre-symptomatic adults at genetic risk for prion disease.A teljes eredeti leírásKevesebb
An Open-label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered PrP-siRNA in Adults Diagnosed With Symptomatic Prion Disease and in Pre-symptomatic Adults at Genetic Risk for Prion Disease.
The purpose of this trial is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic impact of PrP-siRNA in symptomatic prion disease patients and in pre-symptomatic adults at genetic risk for prion disease.
Bevonási feltételek: 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; availability of a caregiver to assist with study procedures; lack of clinically manifested symptoms of prion disease, as demonstrated by an MRC Score of 20, a MoCA score between 24-30 inclusive, and a neurological exam with no clinically significant findings, in the opinion of the investigator; a PRNP genetic test indicating the presence of a highly penetrant genetic variant causal for prion disease.
Kizáró feltételek: recent participation in a different prion disease clinical trial; Additional inclusion and exclusion criteria apply and will be evaluated at screening; pregnancy or unwillingness to comply with the contraceptive requirements during the study period; contraindication to lumbar puncture.