A Phase 2 Trial of ALN-APP in Patients With Cerebral Amyloid Angiopathy
ConditionCerebral Amyloid Angiopathy
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age30+
SponsorAlnylam Pharmaceuticals
About this trial
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 criteria
Qualifiers
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
Disqualifiers
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/1.73m² at Screening
Trial design
Treatments tested in this trial
- Placebo
- ALN-APP
Treatment groups
200 Participants
are divided into 2 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov