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
Location: See all (13) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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
Participation Requirements
Sex: All
Minimum Age: 30
Healthy Volunteers: f
View:
• Is 50 years or older
• Has probable CAA per the Boston Criteria Version 2.0
• Is 30 years or older
• Has known E693Q amyloid precursor protein (APP) gene mutation for Dutch-type CAA
Locations
United States
Florida
Clinical Trial Site
RECRUITING
Naples
Louisiana
Clinical Trial Site
RECRUITING
New Orleans
Massachusetts
Clinical Trial Site
RECRUITING
Plymouth
Missouri
Clinical Trial Site
RECRUITING
Saint Louis
New York
Clinical Trial Site
RECRUITING
New York
Clinical Trial Site
RECRUITING
New York
Pennsylvania
Clinical Trial Site
RECRUITING
Abington
Texas
Clinical Trial Site
RECRUITING
Dallas
Clinical Trial Site
RECRUITING
Houston
Other Locations
Canada
Clinical Trial Site
RECRUITING
Calgary
Clinical Trial Site
RECRUITING
Ottawa
Clinical Trial Site
RECRUITING
Richmond
Clinical Trial Site
RECRUITING
Toronto
Contact Information
Primary
Alnylam Clinical Trial Information Line
clinicaltrials@alnylam.com
1-877-ALNYLAM
Backup
Alnylam Clinical Trial Information Line
clinicaltrials@alnylam.com
1-877-256-9526
Time Frame
Start Date: 2024-05-17
Estimated Completion Date: 2029-11-01
Participants
Target number of participants: 200
Treatments
Experimental: ALN-APP
Participants will be administered multiple doses of ALN-APP during the double-blind treatment period and optional open-label extension period.
Placebo_comparator: Placebo/ALN-APP
Participants will be administered multiple doses of placebo during the double-blind treatment period. Participants who continue into the optional open-label extension period will be administered multiple doses of ALN-APP.
Related Therapeutic Areas
Sponsors
Leads: Alnylam Pharmaceuticals