A Phase 1b Study to Assess the Safety, Tolerability, and Pharmacokinetics of ION269 in Adults With Down Syndrome (Hero Study)
NCT: NCT06673069 ·
Status: TERMINATED ·
Phase: Phase 1
· Sponsor: Ionis Pharmaceuticals, Inc.
· Started: 2024-12-20
· Est. Completion: 2025-12-03
Official Summary
The primary purpose of this study is to evaluate the safety and tolerability of ION269 in adults with Down syndrome with evidence of brain amyloid positivity.
Study Design
- Study Type: INTERVENTIONAL
- Allocation: RANDOMIZED
- Model: SINGLE_GROUP
- Masking: QUADRUPLE
- Enrollment: 1 participants
Interventions
- DRUG: ION269 — Administered as intrathecal (IT) injection.
Primary Outcomes
- Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) (Up to approximately Week 40)
- Number of Participants With Change from Baseline in Laboratory Assessments (Up to approximately Week 40)
- Number of Participants With Change from Baseline in Cerebrospinal fluid (CSF) Safety Laboratory Assessments (Up to approximately Week 40)
- Number of Participants With Change From Baseline in Vital Signs (Up to approximately Week 40)
- Number of Participants With Change From Baseline in Weight (Up to approximately Week 40)
Secondary Outcomes
- CSF Concentrations of ION269 (Pre-dose and post-dose at multiple timepoints up to Week 40)
- Area Under the Plasma Concentration-time Curve (AUC) of ION269 From Time 0 to Time of Last Measurable Concentration (Pre-dose and post-dose at multiple timepoints up to Week 40)
- Maximum Observed Plasma Concentration (Cmax) of ION269 (Pre-dose and post-dose at multiple timepoints up to Week 40)
- Time to reach Cmax (Tmax) of ION269 (Pre-dose and post-dose at multiple timepoints up to Week 40)
- Change From Baseline in Concentration of CSF Soluble Amyloid Precursor Protein Alpha (sAPPα) (Baseline (Day 1) and Week 36)
Trial Locations
- Ionis Investigative Site, Indianapolis, Indiana, United States
- Ionis Investigative Site, Kansas City, Kansas, United States
- Ionis Investigative Site, Lexington, Kentucky, United States
- Ionis Investigative Site, St Louis, Missouri, United States
- Ionis Investigative Site, Madison, Wisconsin, United States
- Ionis Investigative Site, Barcelona, Spain
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