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3 recruiting studies matching your filters
Phase 1
An Open-label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered PrP-siRNA in Adult Patients Diagnosed With Symptomatic Prion Disease.
The purpose of this trial is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic impact of PrP-siRNA in symptomatic prion disease patients.
Prion Disease
Estimated
~$10 – $7,500
Broad Institute of MIT and HarvardNCT07444580
Biomarker Profiling in Individuals at Risk for Prion Disease
We are doing this research to identify biomarkers in individuals who are at-risk for familial prion disease. We hope to use these biomarkers to predict timing …
CJD (Creutzfeldt Jakob Disease)Prion DiseasesGSS+2 more
Estimated
~$10 – $750
Massachusetts General HospitalNCT05124392
Phase 1
A Phase 1/2a Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION717 in Patients With Prion Disease
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intrathecal (IT) delivery of ION717.
Prion Disease
Estimated
~$10 – $8,750
Ionis Pharmaceuticals, Inc.NCT06153966