Ignite Creation Date:
2025-12-24 @ 10:47 PM
Ignite Modification Date:
2026-01-29 @ 8:30 AM
Study NCT ID:
NCT02178969
Status:
COMPLETED
Last Update Posted:
2021-03-17
First Post:
2014-06-04
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Short Term Observational Study in DEB Patients
Sponsor:
Organization:
Raw JSON
{'hasResults': False, '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'}]}}, 'protocolSection': {'designModule': {'bioSpec': {'retention': 'SAMPLES_WITH_DNA', 'description': 'Optional genetic test for DEB diagnosis'}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 29}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2014-06-24', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-03', 'completionDateStruct': {'date': '2015-02-23', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2021-03-15', 'studyFirstSubmitDate': '2014-06-04', 'studyFirstSubmitQcDate': '2014-06-27', 'lastUpdatePostDateStruct': {'date': '2021-03-17', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2014-07-01', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2015-02-23', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Wound Surface Area (WSA) of patient- and investigator-selected wounds', 'timeFrame': 'Over 4 weeks'}, {'measure': 'Wound Surface Area (WSA) as a percentage of Body Surface Area (BSA) in patients with DEB', 'timeFrame': 'Over 4 weeks'}], 'secondaryOutcomes': [{'measure': 'The number, severity and relationship to study procedures of adverse events (AEs) and serious AEs (SAEs)', 'timeFrame': 'Over 4 weeks'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'conditions': ['Dystrophic Epidermolysis Bullosa']}, 'referencesModule': {'references': [{'pmid': '35964087', 'type': 'DERIVED', 'citation': 'Paller AS, Pope E, Rudin D, Malyala A, Ramsdell D, Johnson R, Landy H, Murrell DF; DEB Investigators. A prospective short-term study to evaluate methodologies for the assessment of disease extent, impact, and wound evolution in patients with dystrophic epidermolysis bullosa. Orphanet J Rare Dis. 2022 Aug 13;17(1):314. doi: 10.1186/s13023-022-02461-z.'}], 'seeAlsoLinks': [{'url': 'https://clinicaltrials.takeda.com/study-detail/5f6b5fd54db2bf003ab46de2', 'label': 'To obtain more information on the study, click here/on this link.'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to better understand disease extent and to identify appropriate methodologies to evaluate (dystrophic epidermolysis bullosa) DEB in a quantitative and qualitative manner.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Patients with DEB.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Written informed consent is provided. Patients 18 years of age and older, and parent(s)/legal guardian(s) of patients younger than 18 years of age, must provide written informed consent prior to participating in the study; additionally, informed assent will be obtained from patients younger than 18 years of age as specified by local requirements.\n2. Patient must have a documented diagnosis of DEB based on genetic analysis showing a mutation in COL7A1 or, in the opinion of the investigator, a confirmed diagnosis of DEB based on histologic criteria (antigen mapping or electron microscopy).\n3. Patient must have at least 5 wounds that are suitable for imaging, in the opinion of the investigator, at the time of enrollment.\n4. Patient is willing and able to undergo the protocol-specified procedures.\n\nExclusion Criteria:\n\n1. Patient has used or is currently using experimental treatment for DEB including, but not limited to, bone marrow transplantation, systemic immune suppression, or experimental procedures that involve live cells, with potential for systemic spread such as gene transfer, stem cell infusions, or other cell type injections such that, in the opinion of the investigator, inclusion poses an unacceptable risk to the patient or interpretation of these study data.\n2. Patient has squamous cell carcinoma with evidence of locally invasive disease or distant metastases.\n3. Patient is pregnant.'}, 'identificationModule': {'nctId': 'NCT02178969', 'briefTitle': 'Short Term Observational Study in DEB Patients', 'organization': {'class': 'INDUSTRY', 'fullName': 'Takeda'}, 'officialTitle': 'A Prospective Short-term Study to Evaluate Methodologies for the Assessment of Disease Extent, Impact, and Wound Evolution in Patients With Dystrophic Epidermolysis Bullosa (DEB)', 'orgStudyIdInfo': {'id': 'SHP-608-002'}}, 'contactsLocationsModule': {'locations': [{'zip': '60611', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'Ann and Robert H Lurie Childrens Hospital of Chicago', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '10032', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Columbia University Medical Center', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': 'Cincinnati Childrens Hospital Medical Center', 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '2217', 'city': 'Kogarah', 'country': 'Australia', 'facility': 'Premier Specialists Pty Ltd', 'geoPoint': {'lat': -33.9681, 'lon': 151.13564}}, {'zip': '5020', 'city': 'Salzburg', 'country': 'Austria', 'facility': 'Salzburger Landeskliniken', 'geoPoint': {'lat': 47.79941, 'lon': 13.04399}}, {'zip': '613 00', 'city': 'Brno', 'country': 'Czechia', 'facility': 'Fakultni nemocnice Brno', 'geoPoint': {'lat': 49.19522, 'lon': 16.60796}}, {'city': 'Paris', 'country': 'France', 'facility': 'Service de Génétique et INSERM UMR 1163, Laboratoire de Génétique des maladies cutanées - Institut Imagine', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '79104', 'city': 'Freiburg im Breisgau', 'country': 'Germany', 'facility': 'Universitätsklinikum Freiburg', 'geoPoint': {'lat': 47.9959, 'lon': 7.85222}}, {'zip': '00165', 'city': 'Roma', 'country': 'Italy', 'facility': 'Ospedale Pediatrico Bambino Gesù', 'geoPoint': {'lat': 44.99364, 'lon': 11.10642}}, {'zip': '00167', 'city': 'Rome', 'country': 'Italy', 'facility': "Istituto Dermopatico Dell'immacolata IRCCS", 'geoPoint': {'lat': 41.89193, 'lon': 12.51133}}], 'overallOfficials': [{'name': 'Study Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Takeda'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Shire', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}