drugset / Trial / NCT07444580
PrP-targeting siRNA Safety & Mechanism Study
Phase 1
Recruiting
30 enrolled
Broad Institute of MIT and Harvard
National Institute of Neurological Disorders and Stroke (NINDS) · collab
Non-randomizedSequentialOpen-labelTreatment
Summary
The purpose of this trial is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic impact of PrP-siRNA in symptomatic prion disease patients.
Timeline
- Start
- 2026-05-15
- Primary completion
- 2029-08-14
- Completion
- 2029-08-14
Publications
- Background Gentile JE, Corridon TL, Serack FE, Echeverria D, Kennedy ZE, Gallant-Behm CL, Hassler MR, Kinberger G, Kamath NG, Lian Y, Gross KY, Miller R, DeSouza-Lenz K, Howard M, Guzman K, Chan N, Curtis DT, Fettes K, Lemaitre M, Cappon G, Jackson AL, Yamada K, Alterman JF, Coffey AA, Minikel EV, Khvorova A, Vallabh SM. Divalent siRNA for prion disease. bioRxiv. 2024 Dec 5;2024.12.05.627039. https://doi.org/10.1101/2024.12.05.627039
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | PrP-siRNA | Small interfering RNA (siRNA) | 50 mg | Intrathecal |
| Subject | PrP-siRNA | Small interfering RNA (siRNA) | 100 mg | Intrathecal |
| Subject | PrP-siRNA | Small interfering RNA (siRNA) | 200 mg | Intrathecal |