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 participants with symptomatic cerebral cavernous malformation (CCM).
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
62
Xenoscience Inc
Phoenix, Arizona, United States
UCLA
Los Angeles, California, United States
Stanford University
Incidence and severity of adverse events (AEs)
Time frame: Up to 24 months
Change in patient reported outcomes (Cerebral Cavernous Malformation Health Index)
Time frame: Up to 24 months
Change in patient reported outcomes (Modified Rankin Scale)
Time frame: Up to 24 months
Change in patient reported outcomes (SymptoMScreen Score)
Time frame: Up to 24 months
Change in disease-associated symptoms (number of MRI-confirmed cerebral hemorrhagic events)
Time frame: Up to 24 months
Plasma concentrations of REC-994
Time frame: Up to 12 months
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