Ignite Creation Date:
2025-12-24 @ 12:47 PM
Ignite Modification Date:
2025-12-28 @ 3:58 AM
Study NCT ID:
NCT05085561
Status:
COMPLETED
Last Update Posted:
2025-08-06
First Post:
2021-10-08
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
The Symptomatic Cerebral Cavernous Malformation Trial of REC-994
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D020786', 'term': 'Hemangioma, Cavernous, Central Nervous System'}], 'ancestors': [{'id': 'D006392', 'term': 'Hemangioma, Cavernous'}, {'id': 'D006391', 'term': 'Hemangioma'}, {'id': 'D009383', 'term': 'Neoplasms, Vascular Tissue'}, {'id': 'D009370', 'term': 'Neoplasms by Histologic Type'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D000096826', 'term': 'Cavernous Sinus Syndromes'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D020785', 'term': 'Central Nervous System Vascular Malformations'}, {'id': 'D009421', 'term': 'Nervous System Malformations'}, {'id': 'D054079', 'term': 'Vascular Malformations'}, {'id': 'D018376', 'term': 'Cardiovascular Abnormalities'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D020141', 'term': 'Hemostatic Disorders'}, {'id': 'D014652', 'term': 'Vascular Diseases'}, {'id': 'D006474', 'term': 'Hemorrhagic Disorders'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'TRIPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 62}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2022-03-17', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-07', 'completionDateStruct': {'date': '2025-06-30', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-08-01', 'studyFirstSubmitDate': '2021-10-08', 'studyFirstSubmitQcDate': '2021-10-08', 'lastUpdatePostDateStruct': {'date': '2025-08-06', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2021-10-20', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-06-30', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Change in disease-associated symptoms (size and number of lesions on MRI)', 'timeFrame': 'Up to 24 months'}], 'primaryOutcomes': [{'measure': 'Incidence and severity of adverse events (AEs)', 'timeFrame': 'Up to 24 months'}], 'secondaryOutcomes': [{'measure': 'Change in patient reported outcomes (Cerebral Cavernous Malformation Health Index)', 'timeFrame': 'Up to 24 months'}, {'measure': 'Change in patient reported outcomes (Modified Rankin Scale)', 'timeFrame': 'Up to 24 months'}, {'measure': 'Change in patient reported outcomes (SymptoMScreen Score)', 'timeFrame': 'Up to 24 months'}, {'measure': 'Change in disease-associated symptoms (number of MRI-confirmed cerebral hemorrhagic events)', 'timeFrame': 'Up to 24 months'}, {'measure': 'Plasma concentrations of REC-994', 'timeFrame': 'Up to 12 months'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Cerebral Cavernous Malformation', 'CCM'], 'conditions': ['Cerebral Cavernous Malformation']}, 'descriptionModule': {'briefSummary': '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).', 'detailedDescription': '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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion criteria:\n\n1. 18 years of age or older with anatomic CCM lesions demonstrated by brain MRI\n2. Have symptomatic CCM\n3. Have provided written informed consent to participate in the study\n4. 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\n\nExclusion Criteria:\n\n1. Symptoms deemed by the study Investigator to be caused exclusively by irreversible neuronal damage from prior stroke or neurosurgical instrumentation\n2. History of cranial irradiation or surgical/radiosurgical treatment of the primary symptomatic CCM lesion\n3. Pregnant or breast feeding\n4. Be unable or unwilling to participate in MRI assessments (e.g., claustrophobia, metal implant or implanted cardiac pacemaker)\n5. Liver dysfunction or active liver disease as defined by baseline serum transaminases \\>2x upper limit of normal (ULN)\n6. 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\n7. Have had a previous diagnosis of skeletal muscle disorders (myopathy) of any cause or have a baseline creatine kinase level \\> 5x ULN\n8. History of alcohol or substance abuse within 1 year prior to screening\n9. Clinically significant laboratory abnormality\n10. 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)'}, 'identificationModule': {'nctId': 'NCT05085561', 'acronym': 'SYCAMORE', 'briefTitle': 'The Symptomatic Cerebral Cavernous Malformation Trial of REC-994', 'organization': {'class': 'INDUSTRY', 'fullName': 'Recursion Pharmaceuticals Inc.'}, 'officialTitle': 'A Two-Part Study of REC-994 in the Treatment of Adults With Symptomatic Cerebral Cavernous Malformation (CCM); Part 1: A Phase 2 Randomized, Double-Blind, Placebo-Controlled Clinical Trial to Evaluate the Safety, Efficacy and Pharmacokinetics of Two Doses of REC-994; Part 2: A Long-Term Blinded Extension Clinical Trial to Evaluate Long-Term Safety Tolerability and Efficacy of REC-994', 'orgStudyIdInfo': {'id': 'REC-994-201'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'ACTIVE_COMPARATOR', 'label': 'REC-994 200 mg', 'description': 'REC-994 200 mg po once daily (QD) (1 200 mg REC-994 tablet, 1 matching placebo tablet)', 'interventionNames': ['Drug: REC-994', 'Drug: Placebo']}, {'type': 'ACTIVE_COMPARATOR', 'label': 'REC-994 400 mg', 'description': 'REC-994 400 mg po QD (2 200 mg REC-994 tablets)', 'interventionNames': ['Drug: REC-994']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Matching Placebo po QD (2 matching placebo tablets)', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'REC-994', 'type': 'DRUG', 'description': 'REC-994 200 mg tablets', 'armGroupLabels': ['REC-994 200 mg', 'REC-994 400 mg']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Placebo Tablets', 'armGroupLabels': ['Placebo', 'REC-994 200 mg']}]}, 'contactsLocationsModule': {'locations': [{'zip': '85004', 'city': 'Phoenix', 'state': 'Arizona', 'country': 'United States', 'facility': 'Xenoscience Inc', 'geoPoint': {'lat': 33.44838, 'lon': -112.07404}}, {'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'UCLA', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '94304', 'city': 'Palo Alto', 'state': 'California', 'country': 'United States', 'facility': 'Stanford University', 'geoPoint': {'lat': 37.44188, 'lon': -122.14302}}, {'zip': '32608', 'city': 'Gainesville', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Florida', 'geoPoint': {'lat': 29.65163, 'lon': -82.32483}}, {'zip': '32207-8202', 'city': 'Jacksonville', 'state': 'Florida', 'country': 'United States', 'facility': 'Lyerly Neurosurgery', 'geoPoint': {'lat': 30.33218, 'lon': -81.65565}}, {'zip': '34987', 'city': 'Port Saint Lucie', 'state': 'Florida', 'country': 'United States', 'facility': 'Cleveland Clinic Florida', 'geoPoint': {'lat': 27.29393, 'lon': -80.35033}}, {'zip': '30322', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': 'Emory', 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '07450', 'city': 'Ridgewood', 'state': 'New Jersey', 'country': 'United States', 'facility': 'Valley Hospital', 'geoPoint': {'lat': 40.97926, 'lon': -74.11653}}, {'zip': '10027', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Columbia University Medical Center', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '14642', 'city': 'Rochester', 'state': 'New York', 'country': 'United States', 'facility': 'University of Rochester Medical Center', 'geoPoint': {'lat': 43.15478, 'lon': -77.61556}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Thomas Jefferson University', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'University of Pennsylvania', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '15260', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'University of Pittsburgh', 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '75390-8855', 'city': 'Dallas', 'state': 'Texas', 'country': 'United States', 'facility': 'The University of Texas Southwestern Medical Center', 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'zip': '22908', 'city': 'Charlottesville', 'state': 'Virginia', 'country': 'United States', 'facility': 'University of Virginia', 'geoPoint': {'lat': 38.02931, 'lon': -78.47668}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Recursion Pharmaceuticals Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}