ALN-APP-002
Research type
Research Study
Full 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)
IRAS ID
1009801
Contact name
Samuel Rigourd
Contact email
Sponsor organisation
Alnylam Pharmaceuticals Inc.
Research summary
Alnylam Pharmaceuticals, Inc., is sponsoring this multicentre, double blind, placebo-controlled research study to test the safety and efficacy of ALN-APP and find out what effects, if any, ALN-APP has on people with Cerebral Amyloid Angiopathy (CAA). CAA is a neurological condition in which amyloid proteins build up in the walls of blood vessels in the brain, causing problems such as bleeding into the brain. This study will be enrolling populations with two different types of CAA: Dutch-type CAA and Sporadic CAA.
If the screening tests confirm a participant meets the study requirements, they may enter the study, which will consist of 2 periods:
1) Double Blind Treatment Period: Participants will be randomly assigned 1:1 to receive 75mg ALN-APP or placebo intrathecally (injection into the spinal canal) every 6 months until month 18.2) Optional Open Label Extension: Participants will receive 75mg ALN-APP intrathecally every 6months starting at month 24 until month 36.
The duration of treatment with ALN-APP is up to 36 months. The estimated total time on study for each participant is up to 50 months, including up to 2 months of screening, followed by up to 36 months of treatment, and up to 12 months of safety follow-up.
This study is expected to include approximately 200 participants worldwide.
REC name
North of Scotland Research Ethics Committee 1
REC reference
24/NS/0060
Date of REC Opinion
19 Jun 2024
REC opinion
Further Information Favourable Opinion