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
| Arm | Description | Assigned 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. |
|
|
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. |
|
Recruiting Locations
More Details
- NCT ID
- NCT06393712
- Status
- Recruiting
- Sponsor
- Alnylam Pharmaceuticals