NCT06673069

Hero: A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics (PK) and Pharmacodynamics (PD) of ION269 in Participants With Down Syndrome (DS) at Risk for Alzheimer's Disease (AD)

A Phase 1b Study to Assess the Safety, Tolerability, and Pharmacokinetics of ION269 in Adults With Down Syndrome (Hero Study)

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: Ionis Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 35 Years – 55 Years
• Healthy volunteers: Not accepted

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.

Detailed description

This is a phase 1b multi-center, open-label, single ascending dose (SAD) study in adult participants with Down syndrome with evidence of brain amyloid positivity. Participants will be examined in 3 separate cohorts and will receive a single dose of study drug during the 36-week treatment period, followed by a 4-week follow-up period.

Conditions

• Alzheimer Disease
• Down Syndrome

Interventions

Type	Name	Description
DRUG	ION269	Administered as intrathecal (IT) injection.

Timeline

• Start date: 2024-12-20
• Primary completion: 2025-12-03
• Completion: 2025-12-03
• First posted: 2024-11-04
• Last updated: 2026-02-20

Locations

6 sites across 2 countries: United States, Spain

Regulatory

• FDA-regulated drug study