PrP-targeting siRNA Safety & Mechanism Study

Conditions

• Prion Disease

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• PrP-siRNA — DRUG
  Intrathecally administered divalent siRNA designed to target the PRNP mRNA. The structure has been published in DOI: 10.1101/2024.12.05.627039

Study Details

The purpose of this trial is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic impact of PrP-siRNA in symptomatic prion disease patients.

Key Dates

Start date

May 15, 2026

Status verified

Jun 2026

Primary completion

Aug 14, 2029

Completion

Aug 14, 2029

Study Design

Enrollment

30 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• No Intervention: Arm 1: Observational
  In Arm 1, participants will undergo lumbar punctures and other study activities at baseline (Week 0) and at Week 4 and Week 8. Investigational drug will not be administered. We will prioritize enrollment in Arm 2; Arm 1 will be open to enrollment whenever Arm 2 is not open to enrollment.
• Experimental: Arm 2: Single ascending dose
  In Arm 2, participants will be admitted to the clinical trial center and receive a single intrathecal dose of PrP-siRNA. Dose levels to be sequentially evaluated are 50, 100, and 200 mg. Patients will be discharged on Day 2 and then periodically return to the study center on an outpatient basis at Week 1, 2, 4, 8, 12 and 24 for safety monitoring and study activities through the 24 week follow up period.

Primary Outcome Measure

Frequency of adverse events [ Time Frame: Baseline to week 24 ]

Central Contacts

• Broad Institute
  617 714 7000
  [email protected]

Locations (5)

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