August 1, 2022 – The atorvastatin experimental proof of concept trial to treat cavernous malformations with symptomatic hemorrhage, hosted at the University of Chicago, has completed enrollment. We congratulate and are grateful to all those who are participating! Along with the atorvastatin trial, the parallel Trial Readiness project at the University of Chicago has also completed enrolling.
From Dr. Issam Awad, “We thank the patient community for their engagement in these projects, and urge all enrolled subjects to maintain compliance (atorvastatin) and to complete the required follow-ups (atorvastatin and Trial Readiness) to allow us to test the hypotheses which are critical to further progress in treating CCM patients.”
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. In recent years, CCM researchers have shown that the Rho Kinase (ROCK) inhibiting activity of statins may have potential therapeutic effects in human CCM patients. In mice, statin treatment can reduce lesion size, number, and bleeding. These findings have led to the first clinical drug trial for cavernous malformation with symptomatic hemorrhage
The object of the trial is to evaluate the effectiveness of a currently approved and widely used drug, atorvastatin, in stabilizing cerebral cavernous malformations that have caused a symptomatic bleed, for which a decision was made not to surgically resect the hemorrhagic lesion. Atorvastatin is a statin cholesterol-lowering drug also used to prevent stroke and heart attack. Please visit the trial’s webpage at clinicaltrials.gov (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 determine whether the drug might be helpful and prevent a drug from being widely used without benefit or even with an added risk. If the drug is beneficial, it is also important to learn the optimal dosage that provides effective treatment without adding unnecessary side effects. The trial is designed to answer the question of whether atorvastatin decreases (or worsens) bleeding in a cavernous malformation after a recent symptomatic bleed.
Participants in the trial have been randomly assigned to atorvastatin, at a dose commonly used for stroke prevention, or to a placebo. The participants, their doctors, and the researchers do not know whether or not you are receiving medication. This is standard practice in clinical trials.
Participants make three visits to the University of Chicago over 24 months. At all visits, they receive an MRI with a research component to examine cavernous malformation leakage and bleeding. During trial-related visits, they also will undergo a detailed assessment by the University of Chicago Medicine expert staff. Trial details are available on the trial’s website at clinicaltrials.gov and in the Atorvastatin Trial Brochure.
Last update 8.13.22