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)

Launched by IONIS PHARMACEUTICALS, INC. · Nov 1, 2024

Keywords

ClinConnect Summary

The HERO study is looking at a new treatment called ION269 to see if it is safe and well-tolerated for adults with Down syndrome who are at risk for Alzheimer's disease. The main goal is to understand how the drug works in people who show signs of amyloid buildup in the brain, which is often linked to Alzheimer’s. This is an early-stage trial, and right now, they are recruiting participants aged between 18 and 35.

To join the study, participants need to have a diagnosis of Down syndrome, a stable cognitive state, and a supporting family member or caregiver who can help throughout the trial. They must also have had a brain scan that shows amyloid presence. Participants will be closely monitored for their safety and response to the treatment, and they will undergo some tests, including brain imaging. If you or someone you know meets these criteria and is interested, this trial could be an important opportunity to contribute to research that may help others in the future.

Gender

ALL

Eligibility criteria

• Inclusion criteria:
• • 1. Has a reliable study partner, that is, a parent, sibling, or caregiver ≥ 21 years of age, who has known the participant for \> 6 months (i.e., a reliable and competent individual with a close relationship with the participant) and is capable of providing accurate information about the participant's history, can attend all scheduled study visits and provide feedback regarding the participant's symptoms and performance as described in the protocol, and can comply with all study requirements and activities.
• • 2. Has a diagnosis of Down syndrome and has an intelligence quotient (IQ) ≥ 45.
• • 3. Has evidence of amyloid pathology on amyloid-positron emission tomography (PET) scan.
• • 4. Is assessed as being cognitively stable.
• • 5. Is in good health as evidenced by medical history, physical, and neurological examination, and with no diagnosis of dementia or mild cognitive impairment.
• Exclusion criteria:
• • 1. Has unstable psychiatric illness, including psychosis, or untreated major depression within 90 days before Screening, as determined by the Investigator.
• • 2. Has any unstable medical condition likely to hamper the evaluation of safety and/or efficacy of the Study Drug (e.g., moderate or severe untreated obstructive sleep apnea, medical history of clinically significant B12 or folate deficiency that is currently uncontrolled, clinically significant abnormalities of thyroid function, stroke, or other cerebrovascular conditions), as per Investigator's judgment.
• • 3. Is unable to complete MRI and amyloid/tau-PET procedures or has any contraindications to having a brain MRI (e.g., MRI-incompatible pacemaker, aneurysm clips, artificial heart valves, or other metal foreign body; claustrophobia that cannot be medically managed without requiring general anesthesia).
• • Note: Other protocol defined inclusion/exclusion criteria may apply.