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New hope for Alzheimer's in down syndrome: drug trial launches

NCT ID NCT07636811

Disease control Sponsor: Alnylam Pharmaceuticals Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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 Results may not be published yet.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
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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Sep 04, 2026 · Updated 5 times

Summary

This study tests a drug called mivelsiran in 58 adults with Down syndrome who have early-stage Alzheimer's. Participants receive either the drug or a placebo via injection into the spine every few months for two years. The goal is to see if the drug can reduce brain changes linked to Alzheimer's and slow the disease.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
mivelsiran (ALN-APP)
What this could lead to
If successful, this could point toward a treatment that slows or stops Alzheimer's progression in people with Down syndrome.
What could go wrong
This is an early Phase 2 trial with only 58 participants, so results may not apply to everyone. The drug is injected into the spine, which carries risks like infection or headache.

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 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 58 people

The number the study aims to enrol. It can still change while the study runs.

Started

Aug 2026

Expected to finish

Jul 2031

An estimate. End dates often move.

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

40 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: * Clinical diagnosis of Down-Syndrome (DS) associated with trisomy 21 * Positive amyloid PET scan * Cognitively stable in the opinion of the investigator * Seizures must be well controlled with no occurrence of seizures in the 6 months prior to screening Exclusion Criteria: * Has severe intellectual disability (ID) * Has a history of DS regression disorder * Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>2×upper limit of normal (ULN) at Screening * Has estimated glomerular filtration rate (eGFR) \<45 mL/min/1.73m\^2 at Screening * Has recently received an investigational agent * Has had treatment with amyloid-targeting antibody * Comorbidities such as obstructive sleep apnea and hypothyroidism that are not well controlled Note: other protocol defined inclusion / exclusion criteria apply

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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.

Down syndrome-associated Alzheimer's disease (DS-ad)

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    3 sites in 2 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Clinical Study Site

    RECRUITING

    Maitland, Florida, 32751, United States

  • Clinical Study Site

    RECRUITING

    Naples, Florida, 34105, United States

  • Clinical Trial Site

    RECRUITING

    Seville, Spain