cAPPricorn-1
Study Title: A Study to Evaluate the Efficacy, Safety and Tolerability of ALN-APP in Patients with CAA
Purpose
The cAPPricorn-1 clinical trial is a phase 2, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of mivelsiran, an investigational medication, in patients with cerebral amyloid angiopathy (CAA). Mivelsiran is an investigational synthetic small interfering RNA (siRNA) designed to target the amyloid precursor protein (APP) messenger RNA (mRNA). Early results have shown that mivelsiran can lower the levels of amyloid precursor protein, which may potentially help prevent the build-up of amyloid beta in the walls of blood vessels. cAPPricorn-1 will evaluate the potential effect of mivelsiran on the progression of CAA. cAPPricorn-1 will include a 24-month double-blind treatment period in which participants will be randomized 1: 1 to receive either mivelsiran or placebo, with an optional 18-month open-label extension period in which all participants will receive mivelsiran. UCLA is one of over 30 sites seeking to enroll a total of 150 participants.
- This study is looking to enroll people with CAA who are age 50 and older.
Participants will:
- Undergo screening with MRI of the brain and spine, a clinical assessment, a brief cognitive evaluation, bloodwork, and an ECG.
- If randomized, participants will receive intrathecal mivelsiran or placebo (via lumbar puncture) every 6 months, with additional evaluations to monitor safety with MRI, bloodwork, and evaluation of cerebrospinal fluid.
(PI: Kyle Kern, MD)
Contact: Isaias Gomez at (310) 825-1863 or send an email to IGomezSanchez@mednet.ucla.edu to participate in this study or for more information.
More information on the cAPPricorn-1 study is available at cappricorn1.com, or at clinicaltrials.gov ID#: NCT06393712.