PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717
A Phase 1/2a Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION717 in Patients With Prion Disease
2 other identifiers
interventional
76
9 countries
13
Brief Summary
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intrathecal (IT) delivery of ION717.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_1
Started Jan 2024
Longer than P75 for phase_1
13 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
November 21, 2023
CompletedFirst Posted
Study publicly available on registry
December 1, 2023
CompletedStudy Start
First participant enrolled
January 4, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 1, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
June 1, 2030
April 24, 2026
April 1, 2026
3.1 years
November 21, 2023
April 23, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
Incidence of treatment-emergent adverse events.
Baseline up to Week 33
Secondary Outcomes (6)
Maximum Observed Plasma Concentration (Cmax) of ION717
on Day 1 and Week 9
Area Under the Plasma Concentration-time Curve (AUC) of ION717
on Day 1 and Week 9
Half-life (t1/2λz) of ION717 in Plasma
on Day 1 and Week 9
Cerebrospinal fluid (CSF) Concentration of ION717
Pre-dose and at multiple points post-dose up to Week 33
Amount of ION717 Excreted in Urine
Post-dose on Day 1
- +1 more secondary outcomes
Study Arms (3)
ION717 + Placebo, Regimen 1
EXPERIMENTALParticipants will receive multiple doses of study drug (ION717 and placebo) during the 30-week treatment period; the order of doses is blinded. Participants will then receive multiple doses of ION717 during the 142-week open-label extension period.
ION717 + Placebo, Regimen 2
EXPERIMENTALParticipants will receive multiple doses of study drug (ION717 and placebo) during the 30-week treatment period; the order of doses is blinded. Participants will then receive multiple doses of ION717 during the 142-week open-label extension period.
ION717, Regimen 3
EXPERIMENTALParticipants will receive multiple doses of ION717 during the 30-week treatment period and the 142-week open-label extension period.
Interventions
Eligibility Criteria
You may qualify if:
- A confirmed diagnosis of probable or definite prion disease.
- Early-stage prion disease at the time of Screening.
- Willing to meet all study requirements, including travel to Study Center, procedures, measurements and visits.
- Patients must have a caregiver who is ≥ 18 years old and who is able and willing to facilitate the patient's involvement, to the best of their ability, for the duration of the trial; caregivers must also be able and willing to provide information about themselves and the patient for the duration of the trial.
- Aged ≥ 18 at the time of informed consent.
You may not qualify if:
- Any contraindication or unwillingness to undergo an MRI.
- Obstructive hydrocephalus, presence of a functional ventriculoperitoneal shunt for the drainage of cerebrospinal fluid (CSF) or an implanted central nervous system (CNS) catheter.
- Known brain or spinal disease that would interfere with the LP process, CSF circulation or safety assessment.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (13)
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
NYU Langone Health
New York, New York, 10016, United States
University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44106, United States
Royal Melbourne Hospital
Parkville, 3050, Australia
McGill University Health Centre
Montreal, Quebec, H3A 2B4, Canada
Hôpital Universitaire Pitié Salpêtrière
Paris, 75013, France
University Medical Center Göttingen
Göttingen, 37075, Germany
Tel Aviv Sourasky Medical Center
Tel Aviv, 6423906, Israel
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
Milan, 20133, Italy
Mihara Memorial Hospital
Gunma, Isesaki-shi, 372-0006, Japan
National Center of Neurology and Psychiatry
Tokyo, Kodaira-shi, 187-8551, Japan
Neuromuscular Center Yoshimizu Hospital
Yamaguchi, Shimonoseki-shi, 751-0826, Japan
Hospital Clinic De Barcelona
Barcelona, 08036, Spain
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Who Masked
- PARTICIPANT, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 21, 2023
First Posted
December 1, 2023
Study Start
January 4, 2024
Primary Completion (Estimated)
February 1, 2027
Study Completion (Estimated)
June 1, 2030
Last Updated
April 24, 2026
Record last verified: 2026-04
Data Sharing
- IPD Sharing
- Will share
Ionis may share anonymized individual participant data, aggregated clinical data, and other types of data that support the results in this study. Data requests from qualified researchers will be considered once all three of the following criteria are met: (1) 12 months from marketing approval of the study drug in both the United States and European Union; (2) 18 months from conclusion of the study; and (3) 6 months from publication of study article. Access would be via a secure environment and is contingent upon approval of a research proposal and entry into an appropriate data use agreement. Requests to access data can be submitted via the website https://vivli.org/ourmember/ionis/.