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

a study on Cerebral Amyloid Angiopathy

Summary

Eligibility
for people ages 30 years and up (full criteria)
Location
at Los Angeles, California and other locations
Dates
study started
study ends around

Description

Summary

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.

Official Title

A Phase 2, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of Intrathecally Administered ALN-APP in Patients With Cerebral Amyloid Angiopathy (CAA)

Keywords

Cerebral Amyloid Angiopathy, CAA, ALN-APP

Eligibility

You can join if…

Open to people ages 30 years and up

(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

You CAN'T join if...

  • 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
  • Has recently received an investigational agent
  • Has had treatment with amyloid-targeting antibody

Note: other protocol defined inclusion / exclusion criteria apply

Locations

  • Clinical Trial Site accepting new patients
    Los Angeles California 90095 United States
  • Clinical Trial Site accepting new patients
    Orange California 92868 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Alnylam Pharmaceuticals
Links
Sign up for this study
ID
NCT06393712
Phase
Phase 2 Cerebral Amyloid Angiopathy Research Study
Study Type
Interventional
Participants
Expecting 200 study participants
Last Updated
Contact us about this trial