A Phase 1b Study - ION269 in Adults with Down Syndrome
Research type
Research Study
Full title
A Phase 1b Study to Assess the Safety, Tolerability, and Pharmacokinetics of ION269 in Adults with Down Syndrome and Adults with Early Symptomatic Alzheimer’s Disease (HERO Study)
IRAS ID
1009489
Contact name
Tiffany Baumann
Contact email
Sponsor organisation
Ionis Pharmaceuticals, Inc.
Eudract number
2023-509257-31
Research summary
This is an early phase (Phase 1) first-in human study. It will be multi-center, open-label (not blinded), single-ascending-dose study in approximately 30 adults with Down syndrome who are in the pre-clinical phase of Alzheimer’s disease (meaning they will not yet be diagnosed with dementia but are at risk of developing it). The study drug is intrathecally-administered (injected into the spinal fluid). The study will test three dose-levels of ION269 (10 adults in each cohort). ION269 is an investigational antisense oligonucleotide that aims to reduce amyloid precursor protein, which is believed to be responsible for Alzheimer’s disease in people with Down syndrome. The primary objective of the study is to evaluate the safety and tolerability of ION269. The study includes a 12-week Screening Period, a 36-week Treatment Period, and a 4-week Follow-up Period. Each participant will be participating in the study for up to approximately 364 days. The first participant in each cohort will be dosed as a sentinel, that is, no other participant within the cohort will be dosed until safety data is reviewed through Day 28 of the first participant. After the sentinel, remaining participants must be staggered by at least 3 days between dosing to ensure safety monitoring. During the study, an Independent Safety Monitoring Committee (ISMC) will evaluate study data on a routine basis and provide recommendations to the Sponsor.
REC name
London - Surrey Borders Research Ethics Committee
REC reference
24/LO/0789
Date of REC Opinion
23 Dec 2024
REC opinion
Further Information Favourable Opinion