Introducing the cAPPricorn-1 clinical trial for people living with cerebral amyloid angiopathy (CAA).
cAPPricorn-1 is researching an investigational medication called mivelsiran (pronounced my-VEL-sur-an) to see if it is safe and effective. Participating in this trial is a chance to contribute to CAA research.
What is mivelsiran?
Mivelsiran is a type of medication called an RNAi therapeutic that is designed to lower the production of a specific protein called amyloid precursor protein (APP) in the brain. This may help reduce the buildup of harmful amyloid proteins in the brain's blood vessels and potentially slow down the worsening of CAA.
What are RNAi therapeutics?
RNAi therapeutics target specific disease-causing RNA proteins, such as APP. The RNAi therapeutic breaks up the RNA before the protein is made.
Through this process, RNAi therapeutics reduce the production of certain proteins, such as APP, that cause or worsen certain diseases.
Who can participate in cAPPricorn‑1?
We are looking for:
People with sporadic CAA who:
- Are 50 years of age or older
- Have received a diagnosis of CAA from their doctor
People with Dutch-type CAA who:
- Are 30 years of age or older
- Have a known gene mutation for Dutch-type CAA
Additional criteria will be assessed by the study team to determine your eligibility.
What to expect
Study participants will be randomly assigned to receive either the investigational medication (mivelsiran) or a placebo. The placebo looks like and is administered in the same way as mivelsiran but does not contain any medication. You will have an equal chance of being assigned to either group. This study is double-blind, meaning neither you nor the study doctor will know whether you receive mivelsiran or placebo.
The double-blind treatment period will last about 2 years, with an optional 18-month open-label extension period where all participants will receive mivelsiran (no placebo).
Overview of study timeline
Screening period
60 days | 1-3 visits
Double-blind treatment period
24 months | 12 visits
Optional open-label extension period
18 months | 5 visits
Follow-up period
1 year after last dose | 1 visit
The study medication will be given through a lumbar puncture, a routine medical procedure commonly used for diagnosis and treatment. During this procedure, cerebrospinal fluid (CSF) will also be collected for analysis and safety monitoring.
The lumbar puncture is done by a trained medical professional who will carefully insert a needle into your lower back, between the vertebrae (the bones of your spine), to collect CSF and/or administer the medication directly into the CSF. This method allows the medication to reach the brain and have its intended effect because the CSF is directly connected to the brain.
Learn more about cAPPricorn-1
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