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

NCT ID NCT05231785

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

First seen Jun 27, 2026 · Last updated Aug 13, 2026 · Updated 4 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.

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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

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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