Ignite Creation Date:
2025-12-24 @ 11:40 PM
Ignite Modification Date:
2026-01-10 @ 3:04 PM
Study NCT ID:
NCT02810951
Status:
TERMINATED
Last Update Posted:
2023-02-08
First Post:
2016-06-13
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D016108', 'term': 'Epidermolysis Bullosa Dystrophica'}], 'ancestors': [{'id': 'D004820', 'term': 'Epidermolysis Bullosa'}, {'id': 'D012868', 'term': 'Skin Abnormalities'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D012873', 'term': 'Skin Diseases, Genetic'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D003095', 'term': 'Collagen Diseases'}, {'id': 'D003240', 'term': 'Connective Tissue Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D012871', 'term': 'Skin Diseases'}, {'id': 'D012872', 'term': 'Skin Diseases, Vesiculobullous'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'medinfo@castlecreekbio.com', 'phone': '(484) 713-6000', 'title': 'Chief Medical Officer', 'organization': 'Castle Creek Biosciences'}, 'certainAgreement': {'restrictionType': 'GT60', 'piSponsorEmployee': False, 'restrictiveAgreement': True}, 'limitationsAndCaveats': {'description': 'Small number of subjects analyzed for proof of mechanism analyses'}}, 'adverseEventsModule': {'timeFrame': '52 weeks', 'description': 'Adverse events were reported on the whole participant level and not segmented to treated or control wounds', 'eventGroups': [{'id': 'EG000', 'title': 'FCX-007', 'description': "In Phase I, a target of three adult subjects will be enrolled into Group A and a target of three adult subjects will be enrolled into Group B.\n\nIn Phase II the study will target enrolling subjects (aged seven (7) or older) to each arm, but will allow a disproportionate distribution of subjects between Group A and Group B to equal approximately 6 total subjects.\n\nAll subjects will receive FCX-007 into intact skin as well as to one or more paired target wounds at least one time during the study with a possible second administration pending laboratory results.\n\nOne wound in each target wound pair will be used as control for efficacy and safety evaluations. FCX-007 administered wounds will be compared within paired target wounds to untreated wounds.\n\nFCX-007: FCX-007 is a genetically modified cell product obtained from the subject's own skin cells (Autologous fibroblasts). The cells are expanded and genetically modified to produce functional collagen VII. FCX-007 cell suspension is injected intradermally.", 'otherNumAtRisk': 6, 'deathsNumAtRisk': 6, 'otherNumAffected': 3, 'seriousNumAtRisk': 6, 'deathsNumAffected': 1, 'seriousNumAffected': 3}], 'otherEvents': [{'term': 'Blister', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Injection site discolouration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Injection site erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}], 'seriousEvents': [{'term': 'Gastroenteritis viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Neoplasm progression', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Squamous cell carcinoma of skin', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Adverse Events', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Subjects Who Received FCX-007 Injections', 'description': 'All subjects who received FCX-007 injections in at least one target wound.'}], 'classes': [{'categories': [{'measurements': [{'value': '6', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '52 weeks post treatment', 'description': 'Number of subjects with adverse events.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Complete Wound Closure', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}]}, {'units': 'target wounds', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '10', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Treated Wounds', 'description': "FCX-007 is a genetically modified cell product obtained from the subject's own skin cells (Autologous fibroblasts). The cells are expanded and genetically modified to produce functional collagen 7 (COL7). FCX-007 cell suspension is injected intradermally."}, {'id': 'OG001', 'title': 'Untreated Wounds', 'description': 'No FCX-007 injected'}], 'classes': [{'title': 'Complete closure at Week 4', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}]}, {'units': 'target wounds', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '10', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '40.0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Complete closure at Week 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}]}, {'units': 'target wounds', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '80.0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Complete closure at Week 25', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}, {'units': 'target wounds', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '75.0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Complete closure at Week 52', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}, {'units': 'target wounds', 'counts': [{'value': '6', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '50.0', 'groupId': 'OG000'}, {'value': '16.7', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Through Week 52', 'description': 'Percentage of target wounds achieving complete wound closure (greater than 90%) at all post-baseline visits', 'unitOfMeasure': 'percentage of target wounds', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'target wounds', 'denomUnitsSelected': 'target wounds', 'populationDescription': 'Number analyzed represents observed wounds/participants only'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'All Study Participants', 'description': 'All subjects will receive FCX-007 intradermal injections into a treated wound in a target pair and will have no FCX-007 injections into the other control wound in the target pair.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '6'}]}, {'type': 'FCX-007 Injection', 'achievements': [{'groupId': 'FG000', 'numSubjects': '6'}]}, {'type': 'No FCX-007 Injection', 'achievements': [{'groupId': 'FG000', 'numSubjects': '6'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '5'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'FCX-007', 'description': "In Phase I, a target of three adult subjects will be enrolled into Group A and a target of three adult subjects will be enrolled into Group B.\n\nIn Phase II the study will target enrolling subjects (aged seven (7) or older) to each arm, but will allow a disproportionate distribution of subjects between Group A and Group B to equal approximately 6 total subjects.\n\nAll subjects will receive FCX-007 into intact skin as well as to one or more paired target wounds at least one time during the study with a possible second administration pending laboratory results.\n\nOne wound in each target wound pair will be used as control for efficacy and safety evaluations. FCX-007 administered wounds will be compared within paired target wounds to untreated wounds.\n\nFCX-007: FCX-007 is a genetically modified cell product obtained from the subject's own skin cells (Autologous fibroblasts). The cells are expanded and genetically modified to produce functional collagen VII. FCX-007 cell suspension is injected intradermally."}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '5', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '28.4', 'groupId': 'BG000', 'lowerLimit': '9.1', 'upperLimit': '38.9'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'FULL_RANGE'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '5', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '4', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '5', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '6', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Open target wounds', 'classes': [{'categories': [{'measurements': [{'value': '10', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'number of wounds'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2019-10-29', 'size': 2674604, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2021-07-23T20:36', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 6}}, 'statusModule': {'whyStopped': 'Subjects were all in Long-Term Follow Up and were transferred to the ongoing EB-002 study', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2016-07-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-02', 'completionDateStruct': {'date': '2022-04-18', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-02-06', 'studyFirstSubmitDate': '2016-06-13', 'resultsFirstSubmitDate': '2021-07-23', 'studyFirstSubmitQcDate': '2016-06-20', 'lastUpdatePostDateStruct': {'date': '2023-02-08', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2021-08-18', 'studyFirstPostDateStruct': {'date': '2016-06-23', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2021-09-16', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2020-09-29', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Adverse Events', 'timeFrame': '52 weeks post treatment', 'description': 'Number of subjects with adverse events.'}], 'secondaryOutcomes': [{'measure': 'Complete Wound Closure', 'timeFrame': 'Through Week 52', 'description': 'Percentage of target wounds achieving complete wound closure (greater than 90%) at all post-baseline visits'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['RDEB'], 'conditions': ['Epidermolysis Bullosa Dystrophica, Recessive']}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to evaluate the safety of FCX-007, evaluate Type VII collagen (COL7) expression and the presence of anchoring fibrils and to analyze wound healing as a result of FCX-007 administration in subjects with recessive dystrophic epidermolysis bullosa (RDEB). Funding Source- FDA OOPD', 'detailedDescription': 'RDEB is a rare skin and connective tissue disease characterized clinically by skin fragility with easy blistering, erosion and scarring of skin and mucous membranes, and caused by the deficiency of the protein type VII collagen (COL7). The objective of this study is evaluate the safety of FCX-007 intradermal injections in RDEB subjects. Additionally, the trial will evaluate COL7 expression, the presence of anchoring fibrils, as well evidence of wound healing.\n\nApproximately twelve subjects are expected to enroll in the Phase I/II trial. Phase I will enroll approximately six adult subjects. Phase II will enroll approximately six subjects both adults and pediatric (aged seven (7) years or older). All subjects will receive FCX-007 to one or more paired target RDEB wounds. Proof of mechanism will be monitored through digital photography of target wounds and assays conducted on biopsies taken from intact skin sites where FCX-007 is administered.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '7 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria:\n\n1. Age\n\n 1. Phase I: Eighteen (18) years or older.\n 2. Phase II: Seven (7) years or older.\n2. Diagnosis of recessive dystrophic epidermolysis bullosa (RDEB)\n\nKey Exclusion Criteria:\n\n1. Medical instability limiting ability to travel to the investigative center.\n2. Active infection with HIV, hepatitis B or hepatitis C or evidence of other systemic infection\n3. Current evidence of metastatic squamous cell carcinoma at the site to be injected\n4. Clinically significant abnormal laboratory result or other significant clinical abnormalities\n5. Receipt of a chemical or biological study product for the specific treatment of RDEB in the past six months'}, 'identificationModule': {'nctId': 'NCT02810951', 'briefTitle': 'A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa (RDEB)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Castle Creek Biosciences, LLC.'}, 'officialTitle': 'A Phase I/II Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis Bullosa (RDEB)', 'orgStudyIdInfo': {'id': 'FI-EB-001'}, 'secondaryIdInfos': [{'id': 'FD-R-6113-01', 'type': 'OTHER_GRANT', 'domain': 'Office of Orphan Products Development'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'FCX-007', 'description': 'In Phase I, a target of three adult subjects will be enrolled into Group A and a target of three adult subjects will be enrolled into Group B.\n\nIn Phase II the study will target enrolling subjects (aged seven (7 years or older) to each arm, but will allow a disproportionate distribution of subjects between Group A and Group B to equal approximately 6 total subjects.\n\nAll subjects will receive FCX-007 into one or more paired target wounds as well as to intact skin at least one time during the study with a possible second administration pending laboratory results.\n\nOne wound in each target wound pair will be used as control for efficacy and safety evaluations.', 'interventionNames': ['Genetic: FCX-007']}], 'interventions': [{'name': 'FCX-007', 'type': 'GENETIC', 'otherNames': ['Genetically-Modified Autologous Human Dermal Fibroblasts'], 'description': "FCX-007 is a genetically modified cell product obtained from the subject's own skin cells (Autologous fibroblasts). The cells are expanded and genetically modified to produce functional COL7. FCX-007 cell suspension is injected intradermally.", 'armGroupLabels': ['FCX-007']}]}, 'contactsLocationsModule': {'locations': [{'zip': '94305', 'city': 'Stanford', 'state': 'California', 'country': 'United States', 'facility': 'Stanford University', 'geoPoint': {'lat': 37.42411, 'lon': -122.16608}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': "Children's Hospital Colorado", 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Castle Creek Biosciences, LLC.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}