PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717
NCT ID: NCT06153966
Last Updated: 2024-12-27
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE1/PHASE2
56 participants
INTERVENTIONAL
2024-01-04
2027-07-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
DOUBLE
Study Groups
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ION717 + Placebo, Regimen 1
Participants will receive multiple doses of study drug (ION717 and placebo) during the 30-week double-blind treatment period; the order of doses is blinded. Participants will then receive multiple doses of ION717 during the 70-week open-label extension period.
ION717
ION717 will be administered by IT injection.
Placebo
Placebo-matching ION717 will be administered by IT injection.
ION717 + Placebo, Regimen 2
Participants will receive multiple doses of study drug (ION717 and placebo) during the 30-week double-blind treatment period; the order of doses is blinded. Participants will then receive multiple doses of ION717 during the 70-week open-label extension period.
ION717
ION717 will be administered by IT injection.
Placebo
Placebo-matching ION717 will be administered by IT injection.
Interventions
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ION717
ION717 will be administered by IT injection.
Placebo
Placebo-matching ION717 will be administered by IT injection.
Eligibility Criteria
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Inclusion Criteria
* 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.
Exclusion Criteria
* 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.
18 Years
ALL
No
Sponsors
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Ionis Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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UCLA Neurology Clinic
Los Angeles, California, United States
University of Colorado Hospital
Denver, Colorado, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
NYU Langone Health
New York, New York, United States
University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States
Royal Melbourne Hospital
Parkville, , Australia
University of Alberta
Edmonton, Alberta, Canada
McGill University Health Centre
Montreal, Quebec, Canada
Hôpital Universitaire Pitié Salpêtrière
Paris, , France
University Medical Center Göttingen
Göttingen, , Germany
Tel Aviv Sourasky Medical Center
Tel Aviv, , Israel
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
Milan, , Italy
Mihara Memorial Hospital
Gunma, Isesaki-shi, Japan
National Center of Neurology and Psychiatry
Tokyo, Kodaira-shi, Japan
Neuromuscular Center Yoshimizu Hospital
Yamaguchi, Shimonoseki-shi, Japan
Hospital Clinic De Barcelona
Barcelona, , Spain
Countries
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Other Identifiers
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2023-503355-98
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
ION717-CS2
Identifier Type: -
Identifier Source: org_study_id