Evaluating ION269 for safety in adults with Down syndrome at risk for Alzheimer's disease
A Phase 1b Study to Assess the Safety, Tolerability, and Pharmacokinetics of ION269 in Adults With Down Syndrome (Hero Study)
This study is testing a new drug called ION269 to see if it's safe for adults with Down syndrome who are at risk for Alzheimer's disease.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 30 (estimated) |
| Ages | 35 Years to 55 Years |
| Sex | All |
| Sponsor | Ionis Pharmaceuticals, Inc. Industry-sponsored |
| Locations | 6 sites (Indianapolis, Indiana and 5 other locations) |
| Trial ID | NCT06673069 on ClinicalTrials.gov |
What this trial studies
This phase 1b study aims to assess the safety, tolerability, and pharmacokinetics of ION269 in adults with Down syndrome who show signs of brain amyloid positivity. Participants will be grouped into three cohorts and will receive a single ascending dose of the drug over a 36-week treatment period, followed by a 4-week follow-up. The study is open-label and multi-center, focusing on individuals who are cognitively stable and in good health.
Who should consider this trial
Good fit: Ideal candidates are adults with Down syndrome, an IQ of 45 or higher, and evidence of amyloid pathology.
Not a fit: Patients with unstable psychiatric illnesses or those diagnosed with dementia or mild cognitive impairment may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to a new treatment option for adults with Down syndrome at risk for Alzheimer's disease.
How similar studies have performed: While this approach is novel, similar studies targeting amyloid positivity in Alzheimer's disease have shown promise in other populations.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Indianapolis, Indiana and 5 other locations
- Ionis Investigative Site — Indianapolis, Indiana, United States (Recruiting)
- Ionis Investigative Site — Kansas City, Kansas, United States (Recruiting)
- Ionis Investigative Site — Lexington, Kentucky, United States (Recruiting)
- Ionis Investigative Site — Saint Louis, Missouri, United States (Recruiting)
- Ionis Investigative Site — Madison, Wisconsin, United States (Recruiting)
- Ionis Investigative Site — Barcelona, Spain (Recruiting)
Study contacts
- Study coordinator: Ionis Pharmaceuticals
- Email: IonisHEROStudy@clinicaltrialmedia.com
- Phone: (844) 599-7690
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.