Early safety trial of ION269 for Alzheimer's in down syndrome ends after just one participant

NCT ID NCT06673069

Terminated Disease control Sponsor: Ionis Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

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.

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Conditions

The condition(s) this trial relates to.

Alzheimer disease Down syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

ALZHEIMER DISEASE DOWN SYNDROME

Contacts and locations

Locations

Ionis Investigative Site

Indianapolis, Indiana, 46202, United States
Ionis Investigative Site

Kansas City, Kansas, 66205, United States
Ionis Investigative Site

Lexington, Kentucky, 40536, United States
Ionis Investigative Site

St Louis, Missouri, 63110, United States
Ionis Investigative Site

Madison, Wisconsin, 53705, United States
Ionis Investigative Site

Barcelona, 08041, Spain

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