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

NCT ID NCT06673069

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

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

Dec 2024

Finished

Dec 2025

Lead sponsor

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

Anyone

Healthy volunteers

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

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

  • 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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