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: Active_not_recruiting
Location: See all (44) 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
California
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Los Angeles
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Palo Alto
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Sacramento
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San Francisco
Florida
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Gainesville
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Jacksonville
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Maitland
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Naples
Illinois
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Chicago
Kentucky
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Lexington
Louisiana
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New Orleans
Massachusetts
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Boston
Minnesota
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Rochester
Missouri
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St Louis
North Carolina
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Durham
New York
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New York
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New York
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New York
Ohio
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Cleveland
Pennsylvania
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Abington
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Philadelphia
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Pittsburgh
Texas
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Dallas
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Dallas
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Houston
Washington
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Seattle
Other Locations
Australia
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Adelaide
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Brisbane
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Heidelberg
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Nedlands
Canada
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Calgary
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Kelowna
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Moncton
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Montreal
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Ottawa
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Toronto
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Victoria
Netherlands
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Amsterdam
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Leiden
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Nijmegen
Switzerland
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Bern
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Geneva
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Sankt Gallen
United Kingdom
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London
Time Frame
Start Date: 2024-05-17
Completion Date: 2029-12-14
Participants
Target number of participants: 210
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