A Phase 2 Trial of ALN-APP in Patients with Cerebral Amyloid Angiopathy (cAPPricorn-1)

The purpose of the study is to evaluate the effect of ALN-APP on measures of cerebral amyloid angiopathy (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.

In order to participate you must meet the following criteria:

• Are 50 years of age or older.
• Have probable CAA per the Boston Criteria Version 2.0.
• For Dutch-type CAA patients:
  • Are 30 years of age or older.
  • Have known E693Q amyloid precursor protein (APP) gene mutation for Dutch-type CAA.

You will be excluded from the study if any of the following criteria apply to you:

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

This is a partial list of eligibility requirements. To inquire about your eligibility, please call the contact number provided. If you wish to inquire via email, please include the title of the study in your message.