The Symptomatic Cerebral Cavernous Malformation Trial of REC-994

Phase 2

Active, not recruiting

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).

Recruitment & Eligibility

Inclusion Criteria

Not provided

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)

Arm && Interventions

Group	Intervention	Description
REC-994 200 mg	REC-994	REC-994 200 mg po once daily (QD) (1 200 mg REC-994 tablet, 1 matching placebo tablet)
REC-994 200 mg	Placebo	REC-994 200 mg po once daily (QD) (1 200 mg REC-994 tablet, 1 matching placebo tablet)
REC-994 400 mg	REC-994	REC-994 400 mg po QD (2 200 mg REC-994 tablets)
Placebo	Placebo	Matching Placebo po QD (2 matching placebo tablets)

Primary Outcome Measures

Name	Time	Method
Incidence and severity of adverse events (AEs)	24 months	Safety and tolerability
Incidence of clinically significant changes in physical examinations	12 months (Part 2)	Safety and tolerability

Secondary Outcome Measures

Name	Time	Method
Change in patient reported outcomes (SymptoMScreen Score)	24 months	Efficacy
Change in disease-associated symptoms (number of MRI-confirmed cerebral hemorrhagic events)	24 months	Efficacy
Incidence of clinically significant changes in physical examinations	12 months (Part 1)	Safety and tolerability
Change in patient reported outcomes (Cerebral Cavernous Malformation Health Index)	24 months	Efficacy
Change in patient reported outcomes (Modified Rankin Scale)	24 months	Efficacy
Change in disease-associated symptoms (size and number of lesions on MRI)	24 months	Efficacy

Locations (15): University of Virginia
🇺🇸
Charlottesville, Virginia, United States
University of Florida
🇺🇸
Gainesville, Florida, United States
Xenoscience Inc
🇺🇸
Phoenix, Arizona, United States
Stanford University
🇺🇸
Palo Alto, California, United States
UCLA
🇺🇸
Los Angeles, California, United States
Cleveland Clinic Florida
🇺🇸
Port Saint Lucie, Florida, United States
Valley Hospital
🇺🇸
Ridgewood, New Jersey, United States
Columbia University Medical Center
🇺🇸
New York, New York, United States
University of Rochester Medical Center
🇺🇸
Rochester, New York, United States
University of Pennsylvania
🇺🇸
Philadelphia, Pennsylvania, United States
University of Pittsburgh
🇺🇸
Pittsburgh, Pennsylvania, United States
Emory
🇺🇸
Atlanta, Georgia, United States
Lyerly Neurosurgery
🇺🇸
Jacksonville, Florida, United States
The University of Texas Southwestern Medical Center
🇺🇸
Dallas, Texas, United States
Thomas Jefferson University
🇺🇸
Philadelphia, Pennsylvania, United States