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Clinical Trials in the Netherlands / NCT06393712
Active, not recruiting Phase 2

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

NCT06393712 · tracked via the Priya Life Science Netherlands 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
No
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)

FacilityCityRegionStatus
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.

More Alnylam Pharmaceuticals trials in the Netherlands

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-5288 in Patients With Alzheimer's Disease Recruiting
A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy Enrolling by invitation
HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB Recruiting
ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis) Recruiting

Other trials for the same condition

Stimulating Amyloid Clearance in Cerebral Amyloid Angiopathy Enrolling by invitation
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 Netherlands