Ignite Creation Date:
2025-12-24 @ 7:45 PM
Ignite Modification Date:
2025-12-27 @ 9:28 AM
Study NCT ID:
NCT07072403
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-07-22
First Post:
2025-07-07
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Trametinib Treatment for Complicated Vascular Anomalies
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D054079', 'term': 'Vascular Malformations'}], 'ancestors': [{'id': 'D018376', 'term': 'Cardiovascular Abnormalities'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C560077', 'term': 'trametinib'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 20}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2025-07-07', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-07', 'completionDateStruct': {'date': '2027-09-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-07-17', 'studyFirstSubmitDate': '2025-07-07', 'studyFirstSubmitQcDate': '2025-07-09', 'lastUpdatePostDateStruct': {'date': '2025-07-22', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2025-07-18', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2027-07-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'The proportion of patients achieving an objective response at month 12', 'timeFrame': '12 months', 'description': 'Objective response was defined as ≥20% reduction in lesion volume compared to that at baseline.'}], 'secondaryOutcomes': [{'measure': 'The proportion of patients achieving an objective response at month 6', 'timeFrame': '6 months', 'description': 'Objective response was defined as ≥20% reduction in lesion volume compared to that at baseline.'}, {'measure': 'lesion responses', 'timeFrame': '6 and 12 months', 'description': 'The primary endpoint was classified as follow: -Complete involution: 100% resolution of the measured lesion volume; -Nearly complete involution was defined as decrease of ≥75% and \\<100%; -Partial involution was defined as decrease of ≥20% and \\<75%; -No change was defined as \\<20% increase and \\<20% decrease in the volumes of LM lesions; -Further growth was defined as ≥20% increase in the volume of index compared with the baseline volume measured. Lesion responses were overall lesion response rate and good lesion response rate. Overall lesion response comprised complete, nearly complete and partial involutions. Good lesion response comprised complete and nearly complete involutions.'}, {'measure': 'Quality of life (QOL) in patients', 'timeFrame': '12 months', 'description': 'Pediatric Quality of Life Inventory (PedsQLTM) 4.0 Genetic Core Infant Scales (\\<2 years) or Pediatric Quality of Life Inventory (PedsQLTM) 4.0 Genetic Core Scales (2-18 years) were used.'}, {'measure': 'Disease sequelae', 'timeFrame': '12 months', 'description': "Disease sequelae were assessed by site investigators at month 12. The site investigators assessed patients' extremity swelling (if any), general physical activity and exercise levels."}, {'measure': 'Frequency of adverse events', 'timeFrame': '12 months', 'description': 'Frequency of adverse events (e.g. gastrointestinal disorders, blood and lymphatic system disorders, metabolic disorders or other abnormal laboratory results, skin disorders and general disorders, etc.) collected by investigator and reported by parents. All adverse events were collected and graded according to Common Terminology Criteria for Adverse Events, version 5.0 (CTCAE v5.0). The causality of the adverse event was determined by the multidisciplinary staff and was classified as definitively not related, probably not related, possibly related, probably related, or definitively related. Any dose reductions, interruptions, or cessations enacted at the discretion of the investigators were recorded.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['complicated vascular anomalies'], 'conditions': ['Vascular Anomalies']}, 'referencesModule': {'references': [{'pmid': '24252784', 'type': 'BACKGROUND', 'citation': 'Croteau SE, Kozakewich HP, Perez-Atayde AR, Fishman SJ, Alomari AI, Chaudry G, Mulliken JB, Trenor CC 3rd. Kaposiform lymphangiomatosis: a distinct aggressive lymphatic anomaly. J Pediatr. 2014 Feb;164(2):383-8. doi: 10.1016/j.jpeds.2013.10.013. Epub 2013 Nov 16.'}, {'pmid': '40102890', 'type': 'BACKGROUND', 'citation': 'Zhou Z, Qiu T, Zhou J, Zhang Z, Gong X, Zhang X, Lan Y, Yang C, Zhang Y, Xiang S, Ji Y. Clinical features and current management experience in Gorham-Stout disease: a systematic review. Orphanet J Rare Dis. 2025 Mar 19;20(1):134. doi: 10.1186/s13023-025-03649-9.'}, {'pmid': '32894644', 'type': 'BACKGROUND', 'citation': 'Foster JB, Li D, March ME, Sheppard SE, Adams DM, Hakonarson H, Dori Y. Kaposiform lymphangiomatosis effectively treated with MEK inhibition. EMBO Mol Med. 2020 Oct 7;12(10):e12324. doi: 10.15252/emmm.202012324. Epub 2020 Sep 7.'}, {'pmid': '37264205', 'type': 'BACKGROUND', 'citation': "Li D, Sheppard SE, March ME, Battig MR, Surrey LF, Srinivasan AS, Matsuoka LS, Tian L, Wang F, Seiler C, Dayneka J, Borst AJ, Matos MC, Paulissen SM, Krishnamurthy G, Nriagu B, Sikder T, Casey M, Williams L, Rangu S, O'Connor N, Thomas A, Pinto E, Hou C, Nguyen K, Pellegrino da Silva R, Chehimi SN, Kao C, Biroc L, Britt AD, Queenan M, Reid JR, Napoli JA, Low DM, Vatsky S, Treat J, Smith CL, Cahill AM, Snyder KM, Adams DM, Dori Y, Hakonarson H. Genomic profiling informs diagnoses and treatment in vascular anomalies. Nat Med. 2023 Jun;29(6):1530-1539. doi: 10.1038/s41591-023-02364-x. Epub 2023 Jun 1."}, {'pmid': '30542204', 'type': 'BACKGROUND', 'citation': 'Barclay SF, Inman KW, Luks VL, McIntyre JB, Al-Ibraheemi A, Church AJ, Perez-Atayde AR, Mangray S, Jeng M, Kreimer SR, Walker L, Fishman SJ, Alomari AI, Chaudry G, Trenor Iii CC, Adams D, Kozakewich HPW, Kurek KC. A somatic activating NRAS variant associated with kaposiform lymphangiomatosis. Genet Med. 2019 Jul;21(7):1517-1524. doi: 10.1038/s41436-018-0390-0. Epub 2018 Dec 13.'}]}, 'descriptionModule': {'briefSummary': 'Complicated vascular anomalies have diverse etiologies and variable clinical manifestations, and no standardized treatment protocol has been established. Since most patients present with diffuse lesions that are difficult to resect surgically, identifying effective therapeutic strategies is of critical importance. This study aims to evaluate the safety and efficacy of systemic trametinib therapy in patients with complicated vascular anomalies.', 'detailedDescription': 'In recent years, with the widespread application of molecular diagnostic technologies and the growing awareness of complicated vascular anomalies among clinicians, an increasing number of molecular alterations have been identified in these disorders. This progress has brought new hope for patients who previously lacked standardized treatment options. Multiple somatic point mutations have been discovered in complicated vascular anomalies, and case reports have demonstrated significant therapeutic benefits from mutation-based targeted therapies-particularly in patients with NRAS mutations treated with MEK inhibitors. However, to date, there have been no prospective studies evaluating the safety and efficacy of trametinib in pediatric patients with complicated vascular anomalies. Such research is urgently needed to provide robust clinical evidence to guide treatment decisions for this challenging patient population.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '1 Year', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* -Presenting a complicated vascular anomalies vascular anomalies with the following characteristics:\n\n 1. Male and female;\n 2. Between 1 and 18 years of age;\n 3. Complicated vascular anomalies vascular anomalies diagnosis was confirmed by local investigators and by consensus of our multidisciplinary vascular anomaly group at the West China Hospital of Sichuan University based on:\n\nBiopsy; Compatible MRI findings; History and clinical features.\n\nExclusion Criteria:\n\n1. Patients have allergy or contraindication to MEK inhibitor\n2. Exposure to chemotherapy, embolization, corticosteroids, propranolol, sclerotherapy or any other investigational agents within 1 weeks before enrolment on study;\n3. Patients had a history of a major surgery within 2 weeks before enrollment;\n4. Any known evidence of significant local or systemic uncontrolled infection, defined as receiving intravenous antibiotics at the time of enrollment;\n5. Impairment of gastrointestinal function or chronic gastrointestinal disease that may significantly alter the absorption of trametinib.\n6. Concurrent severe and/or uncontrolled medical diseases that could compromise participation in the study (e.g. uncontrolled diabetes, uncontrolled hypertension, severe malnutrition, chronic liver or renal disease, active upper gastrointestinal tract ulceration).\n7. Patients with inadequate liver function:\n\n Total bilirubin higher than or equal to 1.5 × the upper limit of the normal (ULN) for age and alanine aminotransferase and aspartate aminotransferase higher than or equal to 2.5 × the ULN for age.\n8. Patients with inadequate renal function:\n\n 0-5 years of age maximum serum creatinine (mg/dL) of 0.8; 6-10 years of age maximum serum creatinine (mg/dL) of 1.0; 11-14 years of age maximum serum creatinine (mg/dL) of 1.2;\n9. Adequate bone marrow function:\n\n Absolute neutrophil count lower than 1 × 109/L;\n10. History of a malignancy within 5 years;\n11. HIV infection or known immunodeficiency;\n12. Patients with an inability to participate in or follow-up during the study treatment and assessment plan;\n13. Inability to give informed consent.'}, 'identificationModule': {'nctId': 'NCT07072403', 'briefTitle': 'Trametinib Treatment for Complicated Vascular Anomalies', 'organization': {'class': 'OTHER', 'fullName': 'West China Hospital'}, 'officialTitle': 'Safety and Efficacy of Trametinib for Complicated Vascular Anomalies: A Multicenter Prospective Study', 'orgStudyIdInfo': {'id': 'RCT20250701'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Oral Trametinib', 'description': 'Patients will receive oral trametinib once daily', 'interventionNames': ['Drug: Trametinib tablet']}], 'interventions': [{'name': 'Trametinib tablet', 'type': 'DRUG', 'description': 'Drug is supplied in 0.5 mg and 2 mg tablets', 'armGroupLabels': ['Oral Trametinib']}]}, 'contactsLocationsModule': {'locations': [{'zip': '610041', 'city': 'Chengdu', 'state': 'Sichuan', 'country': 'China', 'facility': 'West China Hospital of Sichuan University', 'geoPoint': {'lat': 30.66667, 'lon': 104.06667}}], 'overallOfficials': [{'name': 'Yi Ji', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'West China Hospital'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'West China Hospital', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Clinical Professor', 'investigatorFullName': 'Yi Ji', 'investigatorAffiliation': 'West China Hospital'}}}}