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Evaluating ALN-APP for treating Cerebral Amyloid Angiopathy

A Phase 2, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of Intrathecally Administered ALN-APP in Patients With Cerebral Amyloid Angiopathy (CAA)

Phase 2 Interventional Alnylam Pharmaceuticals · NCT06393712

This study is testing a new treatment called ALN-APP to see if it can slow down the progression of Cerebral Amyloid Angiopathy in adults.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment200 (estimated)
Ages30 Years and up
SexAll
SponsorAlnylam Pharmaceuticals Industry-sponsored
Locations57 sites (Los Angeles, California and 56 other locations)
Trial IDNCT06393712 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to assess the effects of ALN-APP on the progression of Cerebral Amyloid Angiopathy (CAA) in adult patients. It includes a 24-month double-blind treatment phase followed by an optional 18-month open-label extension. The study will evaluate safety, tolerability, and pharmacodynamics of the treatment in patients with sporadic and Dutch-type CAA. Participants will be monitored for up to 50 months, including screening and follow-up.

Who should consider this trial

Good fit: Ideal candidates are adults aged 50 or older with sporadic CAA or those aged 30 or older with Dutch-type CAA and a specific genetic mutation.

Not a fit: Patients with moderate to severe Alzheimer's disease or significant cognitive impairment may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could slow the progression of Cerebral Amyloid Angiopathy and improve patient outcomes.

How similar studies have performed: Other studies targeting similar conditions have shown promise, but this specific approach with ALN-APP is novel.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria (sporadic CAA patients):

* Is 50 years or older
* Has probable CAA per the Boston Criteria Version 2.0

Inclusion Criteria (Dutch-type CAA patients):

* Is 30 years or older
* Has known E693Q amyloid precursor protein (APP) gene mutation for Dutch-type CAA

Exclusion Criteria:

* Moderate or severe stage Alzheimer's disease (AD) or significant cognitive impairment (CI)
* Has a history of previous clinical intracerebral hemorrhage (ICH) with onset less than 90 days prior to anticipated randomization in the study
* Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>3×upper limit of normal (ULN) at Screening
* Has estimated glomerular filtration rate (eGFR) \<30 mL/min/1.73m\^2 at Screening
* Has recently received an investigational agent
* Has had treatment with amyloid-targeting antibody

Note: other protocol defined inclusion / exclusion criteria apply

Where this trial is running

Los Angeles, California and 56 other locations

+7 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Cerebral Amyloid AngiopathyCAA
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.