Clinical trial · Interventional
Atorvastatin Treatment of Cavernous Angiomas With Symptomatic Hemorrhage Exploratory Proof of Concept (AT CASH EPOC) Trial
Phase I-II Randomized, Placebo-Controlled, Single-Blinded, Single-Site Clinical Trial of Atorvastatin in the Treatment of Cavernous Angiomas With Symptomatic Hemorrhage Exploratory Proof of Concept (AT CASH EPOC)
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Cerebral Cavernous Malformation | — | UNRESOLVED | — |
Interventions
Interventions (2)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Atorvastatin | Drug | — | UNRESOLVED |
| Placebo | Other | — | UNRESOLVED |
Design
Arms and outcomes
Arms (2)
- type
- ACTIVE_COMPARATOR
- label
- Treatment
- description
- Atorvastatin 80mg OD (optimal dose). Treatment dose will be de-escalated to 40mg based on reported adverse events.
- interventionNames
- Drug: Atorvastatin
- type
- PLACEBO_COMPARATOR
- label
- Placebo
- description
- Identically looking capsules containing no active ingredient
- interventionNames
- Other: Placebo
Primary outcomes (1)
- measure
- Percent Change in Mean Lesional QSM (QSM Change Score)
- timeFrame
- 2 years of follow-up
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
- Maximum age
- 80 Years
Show eligibility criteria text
Inclusion Criteria: 1. Diagnosis of CCM of any genotype supported by relevant imaging studies. 2. Symptomatic CCM bleeding event within 1 year prior to enrollment. 3. Must be willing/able to travel to the study site for all study visits (baseline, 12 months, and 24 months) over the course of the study period. Exclusion Criteria: 1. Pre-menopausal women who are breastfeeding, pregnant or likely to get pregnant during the study period. 2. Previous cranial irradiation or surgical/radiosurgical treatment of CCM lesion. 3. Failure to pass MRI safety screening (claustrophobia, metal implant . . . etc) 4. Known allergy or intolerance to gadolinium. 5. Severely impaired renal function (eGFR \< 60ml/min), active renal disease or status post-kidney transplants. 6. Statin therapy, for any indication, for more than 7 continuous days or greater than 14 total days within 12 months preceding enrollment. 7. Indication to use statin medication for current approved indication, unrelated to CCM 8. Known allergy or intolerance to statins 9. Liver dysfunction or active liver disease (including chronic viral hepatitis) defined as baseline serum transaminases levels twice the upper range of normal. 10. Previous diagnosis of skeletal muscle disorders of any cause (myopathy), or baseline creatine kinase level five times the upper range of normal. 11. Currently treated with or likely to need treatment with one or more of prohibited medications listed in the protocol. 12. Active drug or alcohol use or dependence that, in the opinion of the site investigator, would interfere with adherence to study requirements. 13. Serious illness (requiring systemic treatment and/or hospitalization) until subject either completes therapy or is clinically stable on therapy, in the opinion of the site investigator, for at least 30 days prior to study entry. 14. Any other condition that the investigator believes would pose a significant hazard to the subject if the investigational therapy were initiated, including conditions resulting in or precipitating myopathy (e.g. HIV, uncontrolled hypothyroidism). 15. In the investigator's opinion, the patient is unstable, and would benefit from a specific intervention rather than treatment with atorvastatin. 16. Inability or unwillingness of subject or legal guardian/representative to give written informed consent. 17. No documentation of valid healthcare insurance. 18. No medical record confirmation of primary care physician.
References
Publications (3)
- DERIVEDAli B, Shenkar R, Lee J, Alcazar-Felix RJ, Thompson RE, Stadnik A, Sader G, Polster SP, Flemming KD, Liao JK, Sorrentino M, Girard R, Hanley DF, Awad IA. Recurrent Symptomatic Hemorrhage in Cerebral Cavernous Malformations After Discontinuation of Atorvastatin or Placebo. J Am Heart Assoc. 2026 Feb 3;15(3):e46943. doi: 10.1161/JAHA.125.046943. Epub 2026 Jan 29. PMID 41608890
- DERIVEDAlcazar-Felix RJ, Thompson RE, Stadnik A, Kinkade S, Sader G, Jhaveri A, Lee J, Iqbal J, Polster SP, Shenkar R, Flemming KD, Girard R, Carroll TJ, Hanley DF, Awad IA. Relevance of Lesion Volume as an Outcome in Cerebral Cavernous Malformation Drug Trial: Exploratory Analyses in Atorvastatin Treatment of Cavernous Angiomas with Symptomatic Hemorrhage Exploratory Proof of Concept. Neurosurgery. 2026 Jun 1;98(6):1395-1401. doi: 10.1227/neu.0000000000003814. Epub 2025 Oct 21. PMID 41117517
- DERIVEDAwad IA, Alcazar-Felix RJ, Stadnik A, Kinkade S, Jhaveri A, Lee J, Hage S, Iqbal J, Polster SP, Shenkar R, Treine K, McBee N, Ostapkovich N, Lane K, Liao JK, Sorrentino M, Lee C, Flemming KD, Girard R, Carroll TJ, Thompson RE, Hanley DF. Safety and efficacy of atorvastatin for rebleeding in cerebral cavernous malformations (AT CASH EPOC): a phase 1/2a, randomised placebo-controlled trial. Lancet Neurol. 2025 Apr;24(4):295-304. doi: 10.1016/S1474-4422(25)00036-5. PMID 40120614