What this study is
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.
What's being tested
- PrP-siRNA
Intervention · DRUG
Intrathecally administered divalent siRNA designed to target the PRNP mRNA. The structure has been published in PMID: 42033217
Study details
- Official title
- 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.
- Sponsor
- Broad Institute of MIT and Harvard
- Conditions
- Prion Disease
- Enrolling
- 45 people
- Starts
- 2026-05-15
- Est. completion
- 2029-08-14
- Design
- Open-label, non-randomized, sequential groups, Phase 1
- Record
- View on ClinicalTrials.gov ↗
Who can join
Age 18 Years and overSex ALLHealthy volunteers Not acceptedYou may qualify if
- 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
You can't join if
- Pregnancy or unwillingness to comply with the contraceptive requirements during the study period
- Contraindication to lumbar puncture
- Recent participation in a different prion disease clinical trial Additional inclusion and exclusion criteria apply and will be evaluated at screening.
Sign in and we'll check these against your records.Full criteria on ClinicalTrials.gov ↗
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Study locations (5)
5 openMassachusetts General Hospital
Boston, Massachusetts, United States
- MGH Prion Studies Coordinator · Contact617-643-2351MGHPrionStudy@partners.org
- Steven E Arnold, MD · Principal investigator
RecruitingMayo Clinic
Rochester, Minnesota, United States
- Sherry Klingerman, CCRP · Contact507-284-0451klingerman.sherry@mayo.edu
- Brenda Nelson · Contact507-293-9237nelson.brenda6@mayo.edu
- Vijay K Ramanan, MD PhD · Principal investigator
RecruitingColumbia University Medical Center
New York, New York, United States
- Katrina Cuasay, BA · Contact212-305-2077kc2305@cumc.columbia.edu
- Lambrini Whitney, RN · Contact212-342-5615lnw2109@cumc.columbia.edu
- Lawrence Honig, MD PhD · Principal investigator
RecruitingUniversity Hospitals Cleveland Medical Center
Cleveland, Ohio, United States
- Mary Andrews, BA, RRT, CCRP · Contact216-844-2386mary.andrews@uhhospitals.org
- Prerna Vatsa, BS, MS · Contact216-844-2319prerna.vatsa@uhhospitals.org
- Brian S Appleby, MD · Principal investigator
RecruitingVanderbilt University Medical Center
Nashville, Tennessee, United States
- Kelly Harper, BPS · Contact615-322-2538kelly.a.harper@vumc.org
- Leeza Kopaeva, BS · Contact615-421-4100leeza.kopaeva@vumc.org
- Amanda Peltier, MD · Principal investigator
Recruiting
Study team
Sponsor
Broad Institute of MIT and Harvard
Study ID NN112
Principal investigator
Eric V Minikel, PhD
Broad Institute of MIT and Harvard
Frequently asked questions
The sponsor hasn't published answers to common questions for this trial.