A Phase 2, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of Intrathecally Administered ALN-APP in Patients With Cerebral Amyloid Angiopathy (CAA)

What is this Study about?

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.

What Will Happen in This Study?

Study drug or placebo will be administered intrathecally.
Planned duration of treatment is 24 months with an optional 18-month open-label extension period.

Who is Eligible to Participate in this Study?

Male or female participants aged 50 years of age
Has a history of previous clinical intracerebral hemorrhage.
Has probable CAA.

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