A Phase 1/2a Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION717 in Patients with Prion Disease
Active, not recruiting
- Conditions
- prion disease
- Registration Number
- jRCT2031230574
- Lead Sponsor
- Ionis Pharmaceuticals, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Not Recruiting
- Sex
- All
- Target Recruitment
- 3
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.
Study & Design
- Study Type
- Interventional
- Study Design
- parallel assignment
- Primary Outcome Measures
Name Time Method - Incidence of treatment-emergent adverse events (TEAEs) from baseline up to Week 29
- Secondary Outcome Measures
Name Time Method Maximum Observed Plasma Concentration (Cmax) of ION717 Day 1 and Week 9 Maximum observed plasma concentration (Cmax) of ION717
Area Under the Plasma Concentration-time Curve (AUC) of ION717 Day 1 and Week 9 Area under the plasma concentration-time curve (AUC) of ION717
Half-life of ION717 in Plasma Day 1 and Week 9 Half-life of ION717 in plasma
Cerebrospinal fluid (CSF) Concentration of ION717 Pre-dose and at multiple points post-dose up to Week 25 Cerebrospinal fluid (CSF) concentration of ION717
Percent Change from Baseline in Prion Protein (PrP) Concentration in CSF pre-dose and at multiple points post-dose up to Week 25 Percent change from baseline in prion protein (PrP) concentration in CSF
Amount of ION717 Excreted in Urine Day 1 Amount of ION717 excreted in urine post-dose