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NCT06153966 | RECRUITING | Prion Disease

PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717
Sponsor:

Ionis Pharmaceuticals, Inc.

Brief Summary:

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intrathecal (IT) delivery of ION717.

Condition or disease

Prion Disease

Intervention/treatment

ION717

Placebo

Phase

PHASE1

PHASE2

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.

Study Type : INTERVENTIONAL
Estimated Enrollment : 76 participants
Masking : DOUBLE
Primary Purpose : TREATMENT
Official Title : A Phase 1/2a Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION717 in Patients With Prion Disease
Actual Study Start Date : 2024-01-04
Estimated Primary Completion Date : 2027-02
Estimated Study Completion Date : 2030-06

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 18 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers:
Criteria
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.
    • PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717

    Location Details

    NCT06153966

    Please Choose a site

    How to Participate

    Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.

    Locations

    RECRUITING

    United States, Massachusetts

    Massachusetts General Hospital

    Boston, Massachusetts, United States, 02114

    RECRUITING

    United States, New York

    NYU Langone Health

    New York, New York, United States, 10016

    RECRUITING

    United States, Ohio

    University Hospitals Cleveland Medical Center

    Cleveland, Ohio, United States, 44106

    RECRUITING

    Australia,

    Royal Melbourne Hospital

    Parkville, Australia, 3050

    ACTIVE NOT RECRUITING

    Canada, Quebec

    McGill University Health Centre

    Montreal, Quebec, Canada, H3A 2B4

    ACTIVE NOT RECRUITING

    France,

    Pitié Salpêtrière University Hospital

    Paris, France, 75013

    ACTIVE NOT RECRUITING

    Germany,

    University Medical Center Göttingen

    Göttingen, Germany, 37075

    RECRUITING

    Israel,

    Tel Aviv Sourasky Medical Center

    Tel Aviv, Israel, 6423906

    ACTIVE NOT RECRUITING

    Italy,

    I.R.C.C.S. Foundation Carlo Besta Neurological Institute

    Milan, Italy, 20133

    ACTIVE NOT RECRUITING

    Japan, Isesaki-shi

    Mihara Memorial Hospital

    Gunma, Isesaki-shi, Japan, 372-0006

    RECRUITING

    Japan, Mr. Kodaira

    National Center of Neurology and Psychiatry

    Tokyo, Kodaira, Japan, 187-8551

    RECRUITING

    Japan, Mr. Shimonoseki

    Neuromuscular Center Yoshimizu Hospital

    Yamaguchi, Shimonoseki, Japan, 751-0826

    ACTIVE NOT RECRUITING

    Spain,

    Barcelona Clinic Hospital

    Barcelona, Spain, 08036