A Phase 2 Trial of ALN-APP in Patients With Cerebral Amyloid Angiopathy
Phase 2 – studying effectiveness and dosage
Conditions:
Cerebral Amyloid Angiopathy
Sponsor: Alnylam Pharmaceuticals
trial.available_in:
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Overview
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.
Who can participate
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
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