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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| REC-994 200 mg | Active Comparator | REC-994 200 mg po once daily (QD) (1 200 mg REC-994 tablet, 1 matching placebo tablet) |
|
| REC-994 400 mg | Active Comparator | REC-994 400 mg po QD (2 200 mg REC-994 tablets) |
|
| Placebo | Placebo Comparator | Matching Placebo po QD (2 matching placebo tablets) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| REC-994 | Drug | REC-994 200 mg tablets |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence and severity of adverse events (AEs) | Up to 24 months |
| Measure | Description | Time Frame |
|---|---|---|
| Change in patient reported outcomes (Cerebral Cavernous Malformation Health Index) | Up to 24 months | |
| Change in patient reported outcomes (Modified Rankin Scale) | Up to 24 months | |
| Measure | Description | Time Frame |
|---|---|---|
| Change in disease-associated symptoms (size and number of lesions on MRI) | Up to 24 months |
Inclusion criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Xenoscience Inc | Phoenix | Arizona | 85004 | United States | ||
| UCLA |
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| Placebo |
| Drug |
Placebo Tablets |
|
| Change in patient reported outcomes (SymptoMScreen Score) |
| Up to 24 months |
| Change in disease-associated symptoms (number of MRI-confirmed cerebral hemorrhagic events) | Up to 24 months |
| Plasma concentrations of REC-994 | Up to 12 months |
| Los Angeles |
| California |
| 90095 |
| United States |
| Stanford University | Palo Alto | California | 94304 | United States |
| University of Florida | Gainesville | Florida | 32608 | United States |
| Lyerly Neurosurgery | Jacksonville | Florida | 32207-8202 | United States |
| Cleveland Clinic Florida | Port Saint Lucie | Florida | 34987 | United States |
| Emory | Atlanta | Georgia | 30322 | United States |
| Valley Hospital | Ridgewood | New Jersey | 07450 | United States |
| Columbia University Medical Center | New York | New York | 10027 | United States |
| University of Rochester Medical Center | Rochester | New York | 14642 | United States |
| Thomas Jefferson University | Philadelphia | Pennsylvania | 19104 | United States |
| University of Pennsylvania | Philadelphia | Pennsylvania | 19104 | United States |
| University of Pittsburgh | Pittsburgh | Pennsylvania | 15260 | United States |
| The University of Texas Southwestern Medical Center | Dallas | Texas | 75390-8855 | United States |
| University of Virginia | Charlottesville | Virginia | 22908 | United States |
| ID | Term |
|---|---|
| D020786 | Hemangioma, Cavernous, Central Nervous System |
| ID | Term |
|---|---|
| D006392 | Hemangioma, Cavernous |
| D006391 | Hemangioma |
| D009383 | Neoplasms, Vascular Tissue |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D000096826 | Cavernous Sinus Syndromes |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D020785 | Central Nervous System Vascular Malformations |
| D009421 | Nervous System Malformations |
| D054079 | Vascular Malformations |
| D018376 | Cardiovascular Abnormalities |
| D002318 | Cardiovascular Diseases |
| D020141 | Hemostatic Disorders |
| D014652 | Vascular Diseases |
| D006474 | Hemorrhagic Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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