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

NCT ID NCT05231785

First seen Jun 27, 2026 · Last updated Jun 27, 2026

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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for EARLY-ONSET ALZHEIMER DISEASE are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

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.

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

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