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

Launched by ALNYLAM PHARMACEUTICALS · Apr 26, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a treatment called ALN-APP for patients with a condition known as Cerebral Amyloid Angiopathy (CAA), which affects the blood vessels in the brain and can lead to problems like strokes or bleeding. The main goal of the study is to see how well ALN-APP works in slowing down the progression of CAA and to learn more about its safety and how the body responds to it. The study will last for about 50 months and includes a 24-month period where participants receive either the treatment or a placebo (an inactive substance) without knowing which one they are getting, followed by an optional 18-month period where everyone can receive the treatment.

To be eligible for this study, participants need to be at least 50 years old if they have sporadic CAA, or at least 30 years old if they have a specific genetic form of CAA known as Dutch-type CAA. They must also meet certain health criteria, such as not having severe Alzheimer’s disease or recent brain bleeding. Participants can expect regular check-ups and monitoring throughout the trial, and their involvement will help researchers better understand CAA and develop potential new treatments.

Gender

ALL

Eligibility 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