The Symptomatic Cerebral Cavernous Malformation Trial of REC-994

About

Brief Summary

This is a two-part, multi-center, randomized, double-blind, placebo-controlled study to investigate the safety, efficacy and pharmacokinetics of REC-994 (200 mg and 400 mg) compared to placebo in subjects with symptomatic cerebral cavernous malformation (CCM).

Primary Purpose

Treatment

Study Type

Interventional

Phase

Phase 2

Eligibility

Gender

All

Healthy Volunteers

Minimum Age

18 Years

Maximum Age

N/A

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)

Join this Trial

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