HELP ADVANCE CAA
RESEARCH

For healthcare providers

The cAPPricorn-1 clinical trial for patients with cerebral amyloid angiopathy (CAA)

Who: We’re looking for participants who might qualify for cAPPricorn-1, a phase 2, randomized, double-blind, placebo-controlled study.

What: To evaluate the efficacy and safety of mivelsiran, an intrathecally administered investigational medication, in patients with CAA.

Why: CAA is a major cause of spontaneous intracerebral hemorrhage (ICH) and is linked to cognitive decline and other neurological symptoms.

• No treatments are currently approved for CAA, leaving a significant unmet need for novel therapies that can potentially slow disease progression.

Mivelsiran

Mivelsiran (pronounced my-VEL-sur-an) is an investigational synthetic small interfering RNA (siRNA) designed to target amyloid precursor protein (APP) messenger RNA. It comprises a synthetic siRNA conjugated to a hexadecyl (C16) group for enhanced central nervous system delivery. Mivelsiran is being developed for patients with CAA.

Mivelsiran is designed to reduce APP production and therefore lower downstream amyloid-beta protein species, aiming to prevent or slow further amyloid deposition, while allowing natural clearance processes to facilitate the removal of vascular amyloid deposits, with the net effect of potentially slowing the progression of CAA.

What is RNAi?

RNA interference (RNAi) is a natural cellular process that regulates gene expression. It involves the silencing of specific genes by small RNA molecules, such as siRNA or microRNA (miRNA). These small RNA molecules bind to target messenger RNA (mRNA), leading to its degradation, which prevents translation into proteins.

Eligibility

cAPPricorn-1 is looking to recruit people with sporadic cerebral amyloid angiopathy (sCAA) and people with Dutch-type cerebral amyloid angiopathy (D-CAA).

For sCAA, participants must:

• Be 50 years of age or older
• Have received a diagnosis of CAA from their doctor

For D-CAA, participants must:

• Be 30 years of age or older
• Have a known gene mutation for D-CAA

Participants must have a study partner to qualify. Other criteria may apply.

Study design

The double-blind treatment period will last 2 years, with an optional 18-month open-label extension (OLE). During the double-blind treatment period, participants will be randomized 1:1 to receive mivelsiran or placebo. In the OLE period, all participants will receive mivelsiran.