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

Purpose

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.

Condition

  • Cerebral Amyloid Angiopathy

Eligibility

Eligible Ages
Over 30 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

(sporadic CAA patients): - Is 50 years or older - Has probable CAA per the Boston Criteria Version 2.0 Inclusion Criteria (Dutch-type CAA patients): - Is 30 years or older - Has known E693Q amyloid precursor protein (APP) gene mutation for Dutch-type CAA

Exclusion Criteria

  • Moderate or severe stage Alzheimer's disease (AD) or significant cognitive impairment (CI) - 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 Note: other protocol defined inclusion / exclusion criteria apply

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
ALN-APP 		Participants will be administered multiple doses of ALN-APP during the double-blind treatment period and optional open-label extension period.
  • Drug: Placebo
    Placebo will be administered intrathecally
  • Drug: ALN-APP
    ALN-APP will be administered intrathecally
    Other names:
    • mivelsiran
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.
  • Drug: Placebo
    Placebo will be administered intrathecally
  • Drug: ALN-APP
    ALN-APP will be administered intrathecally
    Other names:
    • mivelsiran

Recruiting Locations

Clinical Trial Site
Los Angeles, California 90095

Clinical Trial Site
Orange, California 92868

Clinical Trial Site
Palo Alto, California 94304

Clinical Trial Site
Sacramento, California 95816

Clinical Trial Site
San Francisco, California 94114

Clinical Trial Site
Aurora, Colorado 80045

Clinical Trial Site
New Haven, Connecticut 06510

Clinical Trial Site
Gainesville, Florida 32608

Clinical Trial Site
Jacksonville, Florida 32224

Clinical Trial Site
Maitland, Florida 32751

Clinical Trial Site
Naples, Florida 34105

Clinical Trial Site
Chicago, Illinois 60612

Clinical Trial Site
Lexington, Kentucky 40504

Clinical Trial Site
New Orleans, Louisiana 70121

Clinical Trial Site
Boston, Massachusetts 02114

Clinical Trial Site
Plymouth, Massachusetts 02360

Clinical Trial Site
Rochester, Minnesota 55905

Clinical Trial Site
St Louis, Missouri 63110

Clinical Trial Site
Hackensack, New Jersey 07601

Clinical Trial Site
New York, New York 10016

Clinical Trial Site
New York, New York 10029

Clinical Trial Site
New York, New York 10032

Clinical Trial Site
New York, New York 10065

Clinical Trial Site
Durham, North Carolina 27705

Clinical Trial Site
Canton, Ohio 44718

Clinical Trial Site
Cleveland, Ohio 44195

Clinical Trial Site
Abington, Pennsylvania 19001

Clinical Trial Site
Philadelphia, Pennsylvania 19104

Clinical Trial Site
Pittsburgh, Pennsylvania 15213

Clinical Trial Site
Dallas, Texas 75243

Clinical Trial Site
Dallas, Texas 75390

Clinical Trial Site
Houston, Texas 77030

Clinical Trial Site
San Antonio, Texas 78229

Clinical Trial Site
Seattle, Washington 98104

More Details

NCT ID
NCT06393712
Status
Recruiting
Sponsor
Alnylam Pharmaceuticals

Study Contact

Alnylam Clinical Trial Information Line
1-877-ALNYLAM
clinicaltrials@alnylam.com

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Vanderbilt University Medical Center