Early safety trial of ION269 for Alzheimer's in down syndrome ends after just one participant
NCT ID NCT06673069
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-stage study tested a drug called ION269 in adults with Down syndrome who also showed signs of Alzheimer's disease. The goal was to check if the drug was safe and tolerable when injected into the spinal fluid. Only one person took part, and the study was stopped early, so we have very little information about how well it works or what the risks might be.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- ION269 (given as an injection into the spinal fluid)
- What this could lead to
- If successful, this could point toward a treatment to slow Alzheimer's disease in people with Down syndrome.
- What could go wrong
- This was a very early, tiny Phase 1 safety study with only 1 participant, and it was terminated early. It is far too small to know if the drug works or is safe.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
-
1 person
The number who actually took part.
- Started
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Dec 2024
- Finished
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Dec 2025
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
35 to 55 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Ionis Investigative Site
Indianapolis, Indiana, 46202, United States
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Ionis Investigative Site
Kansas City, Kansas, 66205, United States
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Ionis Investigative Site
Lexington, Kentucky, 40536, United States
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Ionis Investigative Site
St Louis, Missouri, 63110, United States
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Ionis Investigative Site
Madison, Wisconsin, 53705, United States
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Ionis Investigative Site
Barcelona, 08041, Spain
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