PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: ION717, Placebo.
- Who it may be relevant to
- Registry conditions: Prion Disease. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Australia, Canada, France, Germany +4
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Phase 1/2a Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION717 in Patients With Prion Disease
Overview
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, multi-center study in participants with prion disease. The study will consist of a screening period of up to 6 weeks, a 30-week treatment period, a 142-week open-label extension period and a 32-week post-treatment period. Multiple dose levels will be tested.
The trial consists of three Regimens. Regimens 1 and 2 are fully enrolled. Participants in Regimens 1 and 2 received multiple doses of study drug (ION717 and placebo) during the 30-week double-blind treatment period; the order of doses (i.e. whether a given dose was ION717 or placebo) was blinded. The trial sites listed below are actively recruiting eligible participants for Regimen 3. Regimen 3 is open label.
Interventions
- Drug ION717
ION717 will be administered by IT injection. - Drug Placebo
Placebo-matching ION717 will be administered by IT injection.
Primary outcome measures
- Incidence of treatment-emergent adverse events. [Time frame: Baseline up to Week 33]
Secondary outcome measures (6)
- Maximum Observed Plasma Concentration (Cmax) of ION717 [Time frame: on Day 1 and Week 9]
- Area Under the Plasma Concentration-time Curve (AUC) of ION717 [Time frame: on Day 1 and Week 9]
- Half-life (t1/2λz) of ION717 in Plasma [Time frame: on Day 1 and Week 9]
- Cerebrospinal fluid (CSF) Concentration of ION717 [Time frame: Pre-dose and at multiple points post-dose up to Week 33]
- Amount of ION717 Excreted in Urine [Time frame: Post-dose on Day 1]
- Percent Change from Baseline in Prion Protein (PrP) Concentration in CSF [Time frame: Pre-dose and at multiple points post-dose up to Week 33]
Eligibility criteria
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.
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Double blind
- Primary purpose
- Treatment
Study locations
United States · 3 centers
- Massachusetts General Hospital — Boston
- NYU Langone Health — New York
- University Hospitals Cleveland Medical Center — Cleveland
Japan · 3 centers
- Mihara Memorial Hospital — Gunma
- National Center of Neurology and Psychiatry — Tokyo
- Neuromuscular Center Yoshimizu Hospital — Yamaguchi
Australia · 1 center
- Royal Melbourne Hospital — Parkville
Canada · 1 center
- McGill University Health Centre — Montreal
France · 1 center
- Hôpital Universitaire Pitié Salpêtrière — Paris
Germany · 1 center
- University Medical Center Göttingen — Göttingen
Israel · 1 center
- Tel Aviv Sourasky Medical Center — Tel Aviv
Italy · 1 center
- Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta — Milan
Spain · 1 center
- Hospital Clinic De Barcelona — Barcelona
Identifiers
NCT: NCT06153966 · ION717-CS2 · 2023-503355-98