Ignite Creation Date:
2025-12-25 @ 1:41 AM
Ignite Modification Date:
2025-12-31 @ 7:43 PM
Study NCT ID:
NCT07024394
Status:
NOT_YET_RECRUITING
Last Update Posted:
2025-06-17
First Post:
2025-06-03
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Follow-up Study to Evaluate the Safety and Efficacy of FCN-159 in Pediatric Participants With Neurofibromatosis Type 1
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009456', 'term': 'Neurofibromatosis 1'}, {'id': 'D018318', 'term': 'Neurofibroma, Plexiform'}], 'ancestors': [{'id': 'D017253', 'term': 'Neurofibromatoses'}, {'id': 'D009455', 'term': 'Neurofibroma'}, {'id': 'D018317', 'term': 'Nerve Sheath Neoplasms'}, {'id': 'D009380', 'term': 'Neoplasms, Nerve Tissue'}, {'id': 'D009370', 'term': 'Neoplasms by Histologic Type'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D009386', 'term': 'Neoplastic Syndromes, Hereditary'}, {'id': 'D020752', 'term': 'Neurocutaneous Syndromes'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D010523', 'term': 'Peripheral Nervous System Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D010524', 'term': 'Peripheral Nervous System Neoplasms'}, {'id': 'D009423', 'term': 'Nervous System Neoplasms'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 65}}, 'statusModule': {'overallStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2025-06-12', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-06', 'completionDateStruct': {'date': '2029-09-23', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-06-09', 'studyFirstSubmitDate': '2025-06-03', 'studyFirstSubmitQcDate': '2025-06-09', 'lastUpdatePostDateStruct': {'date': '2025-06-17', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2025-06-17', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2029-09-23', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'long term safty', 'timeFrame': 'Through study completion, about 5 years', 'description': 'Adverse events during treatment of FCN-159 will be assessed by the investigators according to CTCAE 5.0.'}], 'secondaryOutcomes': [{'measure': 'Height in centimeters', 'timeFrame': 'Through study completion, about 5 years', 'description': 'Height will be measured in centimeters, with subsequent plotting of height growth curves and calculation of growth velocity.'}, {'measure': 'Weight in kilograms', 'timeFrame': 'Through study completion, about 5 years', 'description': 'Weight will be measured in kilograms, with subsequent plotting of weight growth curves.'}, {'measure': 'Tanner stage', 'timeFrame': 'Through study completion, about 5 years', 'description': 'Tanner stage will be examined, and the mean age for each Tanner stage will be calculated.'}, {'measure': 'Progression-Free Survival (PFS)', 'timeFrame': 'Through study completion, about 5 years', 'description': 'PFS assessed per REiNS (Response Evaluation in Neurofibromatosis and Schwannomatosis) criteria by inverstigator'}, {'measure': 'Objective response rate (ORR)', 'timeFrame': 'Through study completion, about 5 years', 'description': 'ORR assessed per REiNS (Response Evaluation in Neurofibromatosis and Schwannomatosis) criteria by inverstigator.'}, {'measure': 'Changes in pain intensity', 'timeFrame': 'Through study completion, about 5 years', 'description': 'Changes in pain intensity score relative to baseline will be assessed according to 11-point Numerical Rating Scale (NRS-11).NRS-11 quantifies pain intensity on a scale from 0 to 10, 0 represents "No pain" ,10 represents "The worst pain imaginable".'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Neurofibromatosis 1', 'Plexiform Neurofibroma', 'NF1']}, 'descriptionModule': {'briefSummary': 'FCN-159 (Luvometinib Tablets), an orally available and highly potent selective inhibitor of MEK1/2,demonstrated good tolerability and exhibited notable anti-tumor activity in pediatric pts with NF1-related PN in study NCT04954001.This study is a 5-year long-term follow-up of the FCN-159-002 study, involving all enrolled patients to further assess safety, growth and development effects, and treatment efficacy.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '20 Years', 'minimumAge': '2 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\nPatients from the pediatric cohort of the FCN-159-002 study. Willing to participate in the long-term follow-up study and capable of understanding and voluntarily signing the informed consent form.\n\nExclusion Criteria:\n\nAny clinically significant condition that, in the investigator's judgment, may interfere with study participation or compliance with safety requirements.\n\nPatients unable to comply with visit-related requirements."}, 'identificationModule': {'nctId': 'NCT07024394', 'briefTitle': 'Follow-up Study to Evaluate the Safety and Efficacy of FCN-159 in Pediatric Participants With Neurofibromatosis Type 1', 'organization': {'class': 'INDUSTRY', 'fullName': 'Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.'}, 'officialTitle': 'A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Ruvometinib Tablets (FCN-159 Tablets) in Pediatric Participants With Neurofibromatosis Type 1', 'orgStudyIdInfo': {'id': 'FCN-159-002-LT'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'FCN-159', 'description': 'Study Drug: FCN-159 Dosing Regimen: FCN-159 to be administered once daily until disease progression or study completion, whichever occurs first', 'interventionNames': ['Drug: Luvometinib Tablets']}], 'interventions': [{'name': 'Luvometinib Tablets', 'type': 'DRUG', 'otherNames': ['FCN-159'], 'description': '5mg/m² (Maximum dose does not exceed 8mg, the recommended oral dose for adults), orally, once daily, until disease progression or study completion, whichever occurs first', 'armGroupLabels': ['FCN-159']}]}, 'contactsLocationsModule': {'locations': [{'zip': '200011', 'city': 'Shanghai', 'state': 'Shanghai Municipality', 'country': 'China', 'contacts': [{'name': 'Xiaojie Hu', 'role': 'CONTACT', 'email': 'hxjie2002@hotmail.com', 'phone': '+86 13701997136'}], 'facility': "Shanghai Ninth People's Hospital", 'geoPoint': {'lat': 31.22222, 'lon': 121.45806}}], 'centralContacts': [{'name': 'Xiaojie Hu', 'role': 'CONTACT', 'email': 'hxjie2002@hotmail.com', 'phone': '+86 13701997136'}, {'name': 'Xiaoxi Lin', 'role': 'CONTACT', 'email': 'linxiaoxi@126.com', 'phone': '+86 18017681268'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}