Status:
ACTIVE_NOT_RECRUITING
PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717
Lead Sponsor:
Ionis Pharmaceuticals, Inc.
Conditions:
Prion Disease
Eligibility:
All Genders
18+ years
Phase:
PHASE1
PHASE2
Brief Summary
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intrathecal (IT) delivery of ION717.
Detailed Description
This is a first-in-human, randomized, multi-center study in participants with prion disease. Eligible participants will receive ION717 and placebo. The study will consist of a screening period of up t...
Eligibility Criteria
Inclusion
- Key Inclusion Criteria
- 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.
- Key Exclusion Criteria
- Clinically significant abnormalities in medical history, laboratory tests or physical examination that would render a patient unsuitable for inclusion.
- 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.
- Have any other condition, which, in the opinion of the Investigator would make the patient unsuitable for inclusion or could interfere with the patient participating in or completing the study.
Exclusion
Key Trial Info
Start Date :
January 4 2024
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 1 2027
Estimated Enrollment :
56 Patients enrolled
Trial Details
Trial ID
NCT06153966
Start Date
January 4 2024
End Date
July 1 2027
Last Update
December 27 2024
Active Locations (16)
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1
UCLA Neurology Clinic
Los Angeles, California, United States, 90095
2
University of Colorado Hospital
Denver, Colorado, United States, 80204
3
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
4
NYU Langone Health
New York, New York, United States, 10016