NCT02603328: AT CASH EPOC

Atorvastatin Treatment of Cavernous Angiomas With Symptomatic Hemorrhage Exploratory Proof of Concept (AT CASH EPOC) Trial

Completed Phase 1, PHASE2 Results posted Last updated 22 August 2025

What this trial tests

Phase 1, PHASE2 trial testing Atorvastatin in Cerebral Cavernous Malformation in 80 participants. Completed in 31 March 2025.

Timeline

17 July 2018

Primary endpoint
31 July 2024

31 March 2025

Quick facts

Lead sponsor	University of Chicago
Phase	Phase 1, PHASE2
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	quadruple
Primary purpose	treatment
Enrollment	80
Start date	17 July 2018
Primary completion	31 July 2024
Estimated completion	31 March 2025
Sites	1 location across United States

Drugs / interventions tested

• Atorvastatin (atorvastatin) — full drug profile →
• Placebo

Conditions studied

• Cerebral Cavernous Malformation — all drugs for Cerebral Cavernous Malformation →

Sponsor

University of Chicago

Who can join

Adults 18 to 80, any sex, with Cerebral Cavernous Malformation. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This phase I/II randomized, placebo-controlled, double-blinded, single-site clinical trial is designed to investigate the effect of a prolonged course of atorvastatin versus placebo on CCM lesional iron deposition assessed by validated quantitative susceptibility mapping (QSM) MRI studies in patients who suffered a symptomatic bleed within the preceding one year.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Familial Cerebral Cavernous Malformations.
   Zafar A, Quadri SA, Farooqui M, Ikram A, et al · · 2019 · cited 105× · PMID 30909834 · DOI 10.1161/strokeaha.118.022314
2. Atorvastatin Treatment of Cavernous Angiomas with Symptomatic Hemorrhage Exploratory Proof of Concept (AT CASH EPOC) Trial.
   Polster SP, Stadnik A, Akers AL, Cao Y, et al · · 2019 · cited 64× · PMID 30476251 · DOI 10.1093/neuros/nyy539
3. Rho Kinase Inhibition Blunts Lesion Development and Hemorrhage in Murine Models of Aggressive Pdcd10/Ccm3 Disease.
   Shenkar R, Peiper A, Pardo H, Moore T, et al · · 2019 · cited 50× · PMID 30744543 · DOI 10.1161/strokeaha.118.024058
4. Propranolol for familial cerebral cavernous malformation (Treat_CCM): study protocol for a randomized controlled pilot trial.
   Lanfranconi S, Scola E, Bertani GA, Zarino B, et al · · 2020 · cited 35× · PMID 32398113 · DOI 10.1186/s13063-020-4202-x
5. A Brain-Targeted Orally Available ROCK2 Inhibitor Benefits Mild and Aggressive Cavernous Angioma Disease.
   McKerracher L, Shenkar R, Abbinanti M, Cao Y, et al · · 2020 · cited 35× · PMID 31446620 · DOI 10.1007/s12975-019-00725-8
6. Cerebral Cavernous Malformation Proteins in Barrier Maintenance and Regulation.
   Wei S, Li Y, Polster SP, Weber CR, et al · · 2020 · cited 19× · PMID 31968585 · DOI 10.3390/ijms21020675
7. Association Between Beta-Blocker or Statin Drug Use and the Risk of Hemorrhage From Cerebral Cavernous Malformations.
   Zuurbier SM, Hickman CR, Rinkel LA, Berg R, et al · · 2022 · cited 17× · PMID 35410492 · DOI 10.1161/strokeaha.121.037009
8. Baseline Characteristics of Patients With Cavernous Angiomas With Symptomatic Hemorrhage in Multisite Trial Readiness Project.
   Kim H, Flemming KD, Nelson JA, Lui A, et al · · 2021 · cited 16× · PMID 34525838 · DOI 10.1161/strokeaha.120.033487