The Symptomatic Cerebral Cavernous Malformation Trial of REC-994

Part 1: Participants will receive treatment over a period of 12 months. Part 2: Optional long-term extension (LTE) for participants completing Part 1 and who are eligible for extended treatment with REC-994.

Inclusion criteria

• 18 years of age or older with anatomic CCM lesions demonstrated by brain MRI
• Have symptomatic CCM
• Have provided written informed consent to participate in the study
• Have not participated in a clinical trial utilizing an investigational agent within 28 days or within 5 half-lives of the investigational drug (whichever is longer) prior to Screening

Exclusion criteria

• Symptoms deemed by the study Investigator to be caused exclusively by irreversible neuronal damage from prior stroke or neurosurgical instrumentation
• History of cranial irradiation or surgical/radiosurgical treatment of the primary symptomatic CCM lesion
• Pregnant or breast feeding
• Be unable or unwilling to participate in MRI assessments (e.g., claustrophobia, metal implant or implanted cardiac pacemaker)
• Liver dysfunction or active liver disease as defined by baseline serum transaminases >2x upper limit of normal (ULN)
• Have moderately or severely impaired renal function (estimated glomerular filtration rate [eGFR] <60ml/min) or active renal disease or have previously received a kidney transplant
• Have had a previous diagnosis of skeletal muscle disorders (myopathy) of any cause or have a baseline creatine kinase level > 5x ULN
• History of alcohol or substance abuse within 1 year prior to screening
• Clinically significant laboratory abnormality
• Have had an intracerebral hemorrhage within 3 months of screening or any brain surgery within 6 months of screening (not including the primary symptomatic CCM lesion)