Health TrialsbyHealthPanel

PrP-targeting siRNA Safety & Mechanism Study

ConditionPrion Disease
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18+
SponsorBroad Institute of MIT and Harvard

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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; and

Disqualifiers

pregnancy;

contraindication to LP; or

recent participation in a different prion disease clinical trial.

Trial design

Design model

Sequential

Treatments tested in this trial

  • 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

Treatment groups

30 Participants
are divided into 2 treatment groups
Group A: Arm 1: ObservationalNo intervention 0 interventions
Group B: Arm 2: Single ascending doseExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Frequency of adverse events

Time frame
Baseline to week 24

Secondary outcomes

1

CSF PrP concentration

Prion protein (PrP) concentration in cerebrospinal fluid (CSF), a pharmacodynamic (PD) biomarker for PrP-siRNA activity

Time frame
4 weeks post-dose
2

CSF PrP concentration

Prion protein (PrP) concentration in cerebrospinal fluid (CSF), a pharmacodynamic (PD) biomarker for PrP-siRNA activity

Time frame
8 weeks post-dose
3

CSF PrP concentration

Prion protein (PrP) concentration in cerebrospinal fluid (CSF), a pharmacodynamic (PD) biomarker for PrP-siRNA activity

Time frame
12 weeks post-dose
4

CSF PrP concentration

Prion protein (PrP) concentration in cerebrospinal fluid (CSF), a pharmacodynamic (PD) biomarker for PrP-siRNA activity

Time frame
24 weeks post-dose

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Broad Institute of MIT and Harvard

Lead sponsor

National Institute of Neurological Disorders and Stroke (NINDS)

Collaborator

Source ClinicalTrials.gov
Apply now