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New drug targets Alzheimer's proteins in Early-Onset patients

NCT ID NCT05231785

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 15, 2026 · Updated 6 times

Summary

This early-stage trial tests a drug called ALN-APP (mivelsiran) in 60 adults with mild cognitive impairment or mild dementia due to early-onset Alzheimer's disease. The drug is given via a spinal injection to see if it safely lowers certain proteins linked to Alzheimer's in the brain. The study will first test a single dose, then multiple doses over 12 months.

What this could mean

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

Active substance
ALN-APP (mivelsiran)
What this could lead to
If successful, this could point toward a treatment that slows or modifies early-onset Alzheimer's disease progression.
What could go wrong
This is a very early Phase 1 trial with only 60 participants, focused on safety and dosing. It may not show any benefit, and side effects are unknown.

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

About 60 people

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

Started

Feb 2022

Expected to finish

Apr 2029

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

18 years and older

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: * Has mild cognitive impairment or mild dementia due to EOAD * Has Clinical Dementia Rating (CDR) global score 0.5 or 1.0 and Mini Mental State Examination (MMSE) \>20 Exclusion Criteria: * Has Non-Alzheimer's disease dementia * Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>2×upper limit of normal (ULN) * Has estimated glomerular filtration rate (eGFR) \<45 mL/min/1.73m\^2 at Screening * Has recently received an investigational agent * Has recent treatment with amyloid-targeting antibody Note: other protocol defined inclusion / exclusion criteria apply

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Conditions

The condition(s) this trial relates to.

Alzheimer disease early-onset autosomal dominant Alzheimer disease

As listed by the trial registrant

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

Early-onset Alzheimer disease

How to take part

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

  1. The places running it

    6 sites in 4 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 Trial Site

    COMPLETED

    La Jolla, California, 92037, United States

  • Clinical Trial Site

    RECRUITING

    Indianapolis, Indiana, 46202, United States

  • Clinical Trial Site

    RECRUITING

    Montreal, H3A 2B4, Canada

  • Clinical Trial Site

    RECRUITING

    Toronto, M3B 2S7, Canada

  • Clinical Trial Site

    RECRUITING

    Amsterdam, 1081 BT, Netherlands

  • Clinical Trial Site

    COMPLETED

    Groningen, 9713 GG, Netherlands

  • Clinical Trial Site

    RECRUITING

    Huntley Street, WC1E 6AG, United Kingdom

  • Clinical Trial Site

    RECRUITING

    Sheffield, S10 2JF, United Kingdom

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