Atorvastatin Trial

UPDATE (July 22, 2022): THE ATORVASTATIN TRIAL IS NOW FULLY ENROLLED. THANK YOU TO OUR MEMBERS WHO ARE PARTICIPATING IN THIS IMPORTANT EFFORT! The atorvastatin experimental proof-of-concept CASH trial aims to evaluate the effectiveness of a currently approved and widely used drug, atorvastatin, in stabilizing cerebral cavernous malformations that have caused a symptomatic bleed. Atorvastatin is a statin cholesterol-lowering drug also used to prevent stroke and heart attack.

Why is this important?

Statins are a class of drugs commonly used to treat high blood pressure. Statins have wide-ranging effects in the body and are known to alter many different chemical signaling systems.

Statins inhibit the action of a protein called Rho Kinase (ROCK). In recent years, CCM researchers have shown that inhibiting the action of Rho Kinase (ROCK) may have a potential therapeutic effect on human CCM patients. In mice, statin treatment is able to reduce lesion size, number, and bleeding. These findings have led to the first clinical drug trial for cavernous malformation with symptomatic hemorrhage

Please visit the trial’s webpage at (identifier NCT02603328) for comprehensive information or view the Atorvastatin Trial Brochure.

Why not have my doctor prescribe me that drug?

It is, in fact, unclear if statins in general, and atorvastatin at the prescribed dose, are safe in the setting of cavernous malformations. It is possible that statins might not prevent bleeding in cavernous malformations in humans as they did in mice. It is even possible that the drug might worsen bleeding or cause other complications in this disease. A clinical trial is the only way to find out for sure whether the drug might be helpful and to prevent a drug from being widely used without benefit, or even with an added risk. If the drug is beneficial, it is important to also learn the optimal dosage that provides effective treatment without adding unnecessary side-effects. The trial is designed to answer the question, whether atorvastatin decreases (or worsens) bleeding in a cavernous malformation after a recent symptomatic bleed.

Am I a candidate?

You are a candidate for pre-screening if:

  1. You are between the ages of 18-80, with either familial or sporadic cavernous malformation.
  2. You have no severe disability, dependent on others with most activities.
  3. You have had a cavernous malformation hemorrhage with new symptoms in the last year and associated new bleeding on MRI.
  4. You have not been on statin therapy in the last 12 months for any reason.
  5. You have been evaluated for surgery but have opted not to have surgery to resect the lesion which bled.
  6. You have a primary healthcare physician and have medical insurance.

Please see the study’s webpage for complete information about inclusion and exclusion.

What’s involved?

Participants in the trial will be randomly assigned to atorvastatin, at a dose commonly used for stroke prevention, or to placebo. You, your doctors, and the researchers will not know whether or not you are receiving medication. This is standard practice in clinical trials.

This trial is taking place at the University of Chicago Medicine only, but there is a travel stipend to help participants with the cost of being part of the trial. You will make 3 visits to the University of Chicago over 24 months. At all visits, you’ll have an MRI that includes a research component that examines cavernous malformation leakage and bleeding. During your trial-related visits, you also will undergo a detailed assessment by the University of Chicago Medicine expert staff. Details of the trial requirements are available on the trial’s website at and in the Atorvastatin Trial Brochure.

What is the cost of participation?

The cost of the medication, initial and follow-up visits, and MRI studies are free. Routine clinical care is not covered by the trial. You may continue your general health and cavernous malformation care by your local referring doctor, as covered by your insurance.

If you live in the Chicago area, you will receive $50 per visit to help defray travel expenses. If you live more than 200 miles from Chicago, the trial offers $500 per visit stipend to help defray travel expenses.  You will receive the funds about 3-4 weeks after your visit. The trial team will provide you with a list of local discounted hotel options. Additionally, as part of our support of Centers of Excellence and research sites, Alliance to Cure Cavernous Malformation has created a sample Airbnb list that are less expensive than hotels.

If you live within 1000 miles of Chicago, you are also eligible to use Angel Flight or other volunteer pilot organizations for free air transport. Volunteer pilot organizations (VPO) are nonprofit, regional volunteer-driven groups that arrange non-emergency air travel for children and adults with serious medical conditions and other compelling needs. Most of these groups are regional, but they are able to coordinate with each other to fly families across regional boundaries. We have provided Sample AngelFlight Application. This application is only an example of what would be required of you. Please contact the volunteer pilot organization near you for their specific flight request application.

Alliance to Cure Cavernous Malformation will also help you to create a crowdfunding webpage if your travel will cost more than the stipend, e.g. if you plan to have a family member travel with you. We can provide you with the language you can use to tell your friends and family about the trial and what it means to you and the Alliance to Cure Cavernous Malformation community.

Contact THE Trial

University of Chicago Medicine
Neurovascular Surgery Research
Justine Lee, RN, BSN Trial Nurse
5841 S. Maryland, MC 3026/J341
Chicago, IL 60637

Phone: 773-326-9839

Or visit


Last update 6.16.2021. Reviewed 4.17.22