The Symptomatic Cerebral Cavernous Malformation Trial of REC-994
- Conditions
- Cerebral Cavernous Malformation
- Interventions
- Drug: Placebo
- Registration Number
- NCT05085561
- Lead Sponsor
- Recursion Pharmaceuticals Inc.
- 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).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 62
Not provided
- 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)
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- 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
Trial Locations
- 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