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Phase 2 Trial of ALN-APP for Cerebral Amyloid Angiopathy

This study is testing a drug called ALN-APP for people with Cerebral Amyloid Angiopathy (CAA), a condition where amyloid protein builds up in brain blood vessels. Researchers want to see if ALN-APP can slow down the disease and how safe it is. You might be able to join if you are 50 or older with sporadic CAA, or 30 or older with Dutch-type CAA (a specific genetic form). The study will compare ALN-APP to a placebo (an inactive substance) to see if ALN-APP reduces new brain bleeds, which are measured using MRI scans. The study is currently recruiting about 200 participants.

Study design
This is a Phase 2 interventional study involving approximately 200 participants. It includes a 24-month double-blind period where participants receive either ALN-APP or a placebo, followed by an optional 18-month open-label extension.
What's involved
Your participation, including screening, treatment, and safety follow-up, could last up to 50 months. Both ALN-APP and placebo will be given intrathecally (into the fluid surrounding the spinal cord).
Compensation
Not stated in the trial record.
Follow-up
The primary outcome of new brain bleeds will be measured for up to 24 months. Total study participation, including safety follow-up, can last up to 50 months.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06393712

A Phase 2 Trial of ALN-APP in Patients With Cerebral Amyloid Angiopathy

Active, Not Recruiting
PHASE2Ages 30+InterventionalTreatment
Alnylam Pharmaceuticals
~210 participants
Updated 2026-09-03 on ClinicalTrials.gov
What's tested:PlaceboALN-APP

At a glance

Recruiting sites
0 of 44 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Double-blind Treatment Period: Annualized Rate of New Lobar Cerebral Microbleeds (CMBs) Assessed on Magnetic Resonance Imaging (MRI) of Brain in Patients with Sporadic Cerebral Amyloid Angiopathy (sCAA)
Measured over Up to 24 months

Conditions

Cerebral Amyloid Angiopathy

Explore related trials

Cerebral Amyloid AngiopathyAmyloid-Beta Precursor Protein

Where it's being run

44 sites across 19 states
Canada7
California4
Florida4
Australia4
New York3
Pennsylvania3
Texas3
Netherlands3

Study leadership

  • Medical Director · STUDY_DIRECTOR · Alnylam Pharmaceuticals

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Is 50 years or older
Has probable CAA per the Boston Criteria Version 2.0
Is 30 years or older
Has known E693Q amyloid precursor protein (APP) gene mutation for Dutch-type CAA

Exclusion

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

What this trial measures

  • Double-blind Treatment Period: Annualized Rate of New Lobar Cerebral Microbleeds (CMBs) Assessed on Magnetic Resonance Imaging (MRI) of Brain in Patients with Sporadic Cerebral Amyloid Angiopathy (sCAA)Up to 24 months