A Phase 2 Trial of ALN-APP in Patients With Cerebral Amyloid Angiopathy
NCT ID: NCT06393712
Last Updated: 2025-11-13
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2
200 participants
INTERVENTIONAL
2024-05-17
2029-11-01
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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ALN-APP
Participants will be administered multiple doses of ALN-APP during the double-blind treatment period and optional open-label extension period.
ALN-APP
ALN-APP will be administered intrathecally
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.
Placebo
Placebo will be administered intrathecally
ALN-APP
ALN-APP will be administered intrathecally
Interventions
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Placebo
Placebo will be administered intrathecally
ALN-APP
ALN-APP will be administered intrathecally
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
Exclusion Criteria
* Has a history of previous clinical intracerebral hemorrhage (ICH) with onset less than 90 days prior to anticipated randomization in the study
* Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>3×upper limit of normal (ULN) at Screening
* Has estimated glomerular filtration rate (eGFR) \<30 mL/min/1.73m\^2 at Screening
* Has recently received an investigational agent
* Has had treatment with amyloid-targeting antibody
30 Years
ALL
No
Sponsors
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Alnylam Pharmaceuticals
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Alnylam Pharmaceuticals
Locations
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Clinical Trial Site
Palo Alto, California, United States
Clinical Trial Site
Sacramento, California, United States
Clinical Trial Site
San Francisco, California, United States
Clinical Trial Site
Aurora, Colorado, United States
Clinical Trial Site
Gainesville, Florida, United States
Clinical Trial Site
Jacksonville, Florida, United States
Clinical Trial Site
Maitland, Florida, United States
Clinical Trial Site
Naples, Florida, United States
Clinical Trial Site
Chicago, Illinois, United States
Clinical Trial Site
Lexington, Kentucky, United States
Clinical Trial Site
New Orleans, Louisiana, United States
Clinical Trial Site
Boston, Massachusetts, United States
Clinical Trial Site
Plymouth, Massachusetts, United States
Clinical Trial Site
Rochester, Minnesota, United States
Clinical Trial Site
St Louis, Missouri, United States
Clinical Trial Site
Hackensack, New Jersey, United States
Clinical Trial Site
New York, New York, United States
Clinical Trial Site
New York, New York, United States
Clinical Trial Site
New York, New York, United States
Clinical Trial Site
Durham, North Carolina, United States
Clinical Trial Site
Canton, Ohio, United States
Clinical Trial Site
Cleveland, Ohio, United States
Clinical Trial Site
Abington, Pennsylvania, United States
Clinical Trial Site
Philadelphia, Pennsylvania, United States
Clinical Trial Site
Dallas, Texas, United States
Clinical Trial Site
Dallas, Texas, United States
Clinical Trial Site
Houston, Texas, United States
Clinical Trial Site
San Antonio, Texas, United States
Clinical Trial Site
Seattle, Washington, United States
Clinical Trial Site
Heidelberg, , Australia
Clinical Trial Site
Herston, , Australia
Clinical Trial Site
Nedlands, , Australia
Clinical Trial Site
Parkville, , Australia
Clinical Trial Site
Calgary, Alberta, Canada
Clinical Trial Site
Richmond, British Columbia, Canada
Clinical Trial Site
Victoria, British Columbia, Canada
Clinical Trial Site
Moncton, New Brunswick, Canada
Clinical Trial Site
Hamilton, Ontario, Canada
Clinical Trial Site
Ottawa, Ontario, Canada
Clinical Trial Site
Toronto, Ontario, Canada
Clinical Trial Site
Montreal, Quebec, Canada
Clinical Trial Site
Montreal, , Canada
Clinical Trial Site
Richmond, , Canada
Clinical Trial Site
Amsterdam, , Netherlands
Clinical Trial Site
Leiden, , Netherlands
Clinical Trial Site
Nijmegen, , Netherlands
Clinical Trial Site
Bern, , Switzerland
Clinical Trial Site
Geneva, , Switzerland
Clinical Trial Site
Denmark Hill, , United Kingdom
Clinical Trial Site
Glasgow, , United Kingdom
Clinical Trial Site
London, , United Kingdom
Countries
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Central Contacts
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Alnylam Clinical Trial Information Line
Role: CONTACT
Phone: 1-877-256-9526
Email: [email protected]
Other Identifiers
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2023-510137-29-00
Identifier Type: CTIS
Identifier Source: secondary_id
ALN-APP-002
Identifier Type: -
Identifier Source: org_study_id