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

Prion Disease Clinical Trial

Official title:

A Phase 1/2a Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION717 in Patients With Prion Disease

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT06153966
• Other study ID #: ION717-CS2
• Secondary ID: 2023-503355-98
• Status: Active, not recruiting
• Phase: Phase 1/Phase 2
• Start date: January 4, 2024
• Est. completion date: October 2025

Study information

• Verified date: April 2024
• Source: Ionis Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Description:

This is a first-in-human, randomized, multi-center study in participants with prion disease. Eligible participants will receive ION717 and placebo. The order of doses will be randomized and blinded to participants, study sites and the Sponsor. The study will consist of a screening period of up to 6 weeks, a 24-week double-blind treatment period and a 36-week post-treatment period. Multiple dose levels will be tested. As of April 2024, study recruitment is paused because capacity has been reached for the current stage of the study. Recruitment is anticipated to resume later in 2024. Central Contact: Ionis Pharmaceuticals Telephone: (844) 221-3587 Email: PrionDisease@clinicaltrialmedia.com

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 56
• Est. completion date: October 2025
• Est. primary completion date: March 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Prion Disease
• Prion Diseases

Intervention

Drug:
• ION717
ION717 will be administered by IT injection.
• Placebo
Placebo-matching ION717 will be administered by IT injection.

Locations

Country	Name	City	State
Canada	University of Alberta	Edmonton	Alberta
Canada	McGill University Health Centre	Montréal	Quebec
Germany	University Medical Center Göttingen	Göttingen
Israel	Tel Aviv Sourasky Medical Center	Tel Aviv
Japan	Mihara Memorial Hospital	Gunma	Isesaki-shi
Japan	National Center of Neurology and Psychiatry	Tokyo	Kodaira-shi
Japan	Neuromuscular Center Yoshimizu Hospital	Yamaguchi	Shimonoseki-shi
Spain	Hospital Clinic De Barcelona	Barcelona
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	University Hospitals Cleveland Medical Center	Cleveland	Ohio
United States	UCLA Neurology Clinic	Los Angeles	California

Sponsors (1)

Lead Sponsor	Collaborator
Ionis Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Canada,  Germany,  Israel,  Japan,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of treatment-emergent adverse events.		Baseline up to Week 29
Secondary	Maximum Observed Plasma Concentration (Cmax) of ION717		on Day 1 and Week 9
Secondary	Area Under the Plasma Concentration-time Curve (AUC) of ION717		on Day 1 and Week 9
Secondary	Half-life (t1/2?z) of ION717 in Plasma		on Day 1 and Week 9
Secondary	Cerebrospinal fluid (CSF) Concentration of ION717		Pre-dose and at multiple points post-dose up to Week 25
Secondary	Amount of ION717 Excreted in Urine		Post-dose on Day 1
Secondary	Percent Change from Baseline in Prion Protein (PrP) Concentration in CSF		Pre-dose and at multiple points post-dose up to Week 25