NCT06393712

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

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)

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 200 (estimated)
• Sponsor: Alnylam Pharmaceuticals · Industry
• Sex: All
• Age: 30 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Cerebral Amyloid Angiopathy

Interventions

Type	Name	Description
DRUG	Placebo	Placebo will be administered intrathecally
DRUG	ALN-APP	ALN-APP will be administered intrathecally

Timeline

• Start date: 2024-05-17
• Primary completion: 2027-08-09
• Completion: 2029-12-14
• First posted: 2024-05-01
• Last updated: 2026-03-31

Locations

57 sites across 6 countries: United States, Australia, Canada, Netherlands, Switzerland, United Kingdom

Regulatory

• FDA-regulated drug study