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

NCT06393712 · tracked via the Priya Life Science USA tracker

Sponsor

Alnylam Pharmaceuticals

Phase

Phase 2

Started

2024-05-17

Last updated

2026-09-18

Condition(s) studied

Cerebral Amyloid Angiopathy

Investigational drug(s) / intervention(s)

PlaceboALN-APP

Placebo: Placebo will be administered intrathecally

ALN-APP: ALN-APP will be administered intrathecally

Study summary

The purpose of the study is to evaluate the effect of ALN-APP on measures of CAA disease progression and to characterize the safety, tolerability, and pharmacodynamics (PD) of ALN-APP in adult patients with sporadic CAA (sCAA) and Dutch-type CAA (D-CAA). The study will be conducted over 2 periods: a 24-month double-blind treatment period and an optional 18-month open-label extension (OLE) period. The estimated duration of study participation, inclusive of screening, treatment, and additional safety follow-up, is up to 50 months.

Eligibility

Sex

ALL

Min age

30 Years

Max age

—

Healthy volunteers

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

Primary outcome measure(s)

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

Trial sites (57)

Facility	City	Region
Clinical Trial Site	Los Angeles	California
Clinical Trial Site	Orange	California
Clinical Trial Site	Palo Alto	California
Clinical Trial Site	Sacramento	California
Clinical Trial Site	San Francisco	California
Clinical Trial Site	Aurora	Colorado
Clinical Trial Site	New Haven	Connecticut
Clinical Trial Site	Gainesville	Florida
Clinical Trial Site	Jacksonville	Florida
Clinical Trial Site	Maitland	Florida
Clinical Trial Site	Naples	Florida
Clinical Trial Site	Chicago	Illinois
Clinical Trial Site	Lexington	Kentucky
Clinical Trial Site	New Orleans	Louisiana
Clinical Trial Site	Boston	Massachusetts
Clinical Trial Site	Plymouth	Massachusetts
Clinical Trial Site	Rochester	Minnesota
Clinical Trial Site	St Louis	Missouri
Clinical Trial Site	Hackensack	New Jersey
Clinical Trial Site	New York	New York
Clinical Trial Site	New York	New York
Clinical Trial Site	New York	New York
Clinical Trial Site	New York	New York
Clinical Trial Site	Durham	North Carolina
Clinical Trial Site	Canton	Ohio
Clinical Trial Site	Cleveland	Ohio
Clinical Trial Site	Abington	Pennsylvania
Clinical Trial Site	Philadelphia	Pennsylvania
Clinical Trial Site	Pittsburgh	Pennsylvania
Clinical Trial Site	Dallas	Texas
Clinical Trial Site	Dallas	Texas
Clinical Trial Site	Houston	Texas
Clinical Trial Site	San Antonio	Texas
Clinical Trial Site	Seattle	Washington
Clinical Trial Site	Adelaide	Australia
Clinical Trial Site	Brisbane	Australia
Clinical Trial Site	Heidelberg	Australia
Clinical Trial Site	Nedlands	Australia
Clinical Trial Site	Parkville	Australia
Clinical Trial Site	Calgary	Canada

+ 17 more sites — see the full list on the official registry below.

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Zilebesiran in Patients With Hypertension Not Adequately Controlled and With Either Established Cardiovascular Disease or High Risk for Cardiovascular Disease Recruiting

A Study to Evaluate ALN-6400 in Healthy Volunteers and Patients With Hereditary Hemorrhagic Telangiectasia (HHT) Active, not recruiting

DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World Setting Recruiting

A Study to Evaluate ALN-4324 on Insulin Sensitivity in Adults With Type 2 Diabetes Mellitus Recruiting

Other trials for the same condition

A Study of AMDX-2011P in Participants With CAA Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06393712 on ClinicalTrials.gov ↗ ← All trials in the USA