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

JRCT ID: jRCT2031230574

Registered date:18/01/2024

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: prion disease
• Date of first enrollment: 18/03/2024
• Target sample size: 3
• Countries of recruitment: United States,Japan,Canada,Japan,Germany,Japan,Italy,Japan,France,Japan,Spain,Japan,Israel,Japan,Australia,Japan
• Study type: Interventional

Intervention(s)

Regimen 1: ION717 + Placebo
Regimen 2: ION717 + Placebo

Outcome(s)

• Primary Outcome: Incidence of treatment-emergent adverse events (TEAEs) from baseline up to Week 29

Secondary Outcome

- Maximum Observed Plasma Concentration (Cmax) of ION717 on Day 1 and Week 9
- Area Under the Plasma Concentration-time Curve (AUC) of ION717 on Day 1 and Week 9
- Half-life of ION717 in Plasma on Day 1 and Week 9
- Cerebrospinal fluid (CSF) Concentration of ION717 Pre-dose and at multiple points post-dose up to Week 25
- Amount of ION717 Excreted in Urine post-dose on Day 1
- Percent Change from Baseline in Prion Protein (PrP) Concentration in CSF pre-dose and at multiple points post-dose up to Week 25

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include 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.

Exclude 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 Information

• Primary Sponsor: Deurell Erik
• Secondary ID(s): NCT06153966,2023-503355-98