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

• Status: Active, not recruiting
• Trial end date: 2025-10-01
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Ionis Pharmaceuticals, Inc.

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.