Ignite Creation Date:
2025-12-24 @ 4:20 PM
Ignite Modification Date:
2026-01-06 @ 4:46 PM
Study NCT ID:
NCT00311766
Status:
TERMINATED
Last Update Posted:
2014-05-12
First Post:
2006-04-04
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Phase 2 Study on Effect of Thymosin Beta 4 on Wound Healing in Patients With Epidermolysis Bullosa
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['United States']}, 'conditionBrowseModule': {'meshes': [{'id': 'D004820', 'term': 'Epidermolysis Bullosa'}], 'ancestors': [{'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': 'D012871', 'term': 'Skin Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D012872', 'term': 'Skin Diseases, Vesiculobullous'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C033402', 'term': 'thymosin beta(4)'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'dcrockford@regenerx.com', 'phone': '617-285-5588', 'title': 'Study Director, David R. Crockford', 'organization': 'RegeneRx Biopharmaceuticals, Inc.'}, 'certainAgreement': {'restrictionType': 'LTE60', 'piSponsorEmployee': False, 'restrictiveAgreement': True}, 'limitationsAndCaveats': {'description': 'Early termination of the study leading to randomizing 30 of the 36 participants planned. This was due to the small pool of participants and therefore resulting in low availability of eligible participants.'}}, 'adverseEventsModule': {'timeFrame': 'Adverse Events (AEs) were collected within 70 days', 'eventGroups': [{'id': 'EG000', 'title': 'Placebo', 'description': 'Topical administration of placebo gel, 0% Thymosin Beta 4, qd up to 56 days\n\nPlacebo : Topical administration, 0.00% qd up to 56 days', 'otherNumAtRisk': 8, 'otherNumAffected': 0, 'seriousNumAtRisk': 8, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'Thymosin Beta 4', 'description': 'Topical administration of 0.01%, 0.03%, and 0.1% Thymosin Beta 4 gel, qd up to 56 days\n\nThymosin Beta 4 : Topical administration, 0.01%, 0.03%, and 0.1% gel, qd up to 56 days', 'otherNumAtRisk': 22, 'otherNumAffected': 1, 'seriousNumAtRisk': 22, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'cellulitis', 'notes': 'Cellulitis was resolved in 27 days', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 22, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (10.0)'}], 'frequencyThreshold': '1'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'SECONDARY', 'title': 'Number of Participants Whose Wounds Have Healed', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '22', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Topical administration of placebo gel, 0% Thymosin Beta 4, qd up to 56 days\n\nPlacebo : Topical administration, 0.00% qd up to 56 days'}, {'id': 'OG001', 'title': 'Thymosin Beta 4', 'description': 'Topical administration of 0.01%, 0.03%, and 0.1% Thymosin Beta 4 gel, qd up to 56 days\n\nThymosin Beta 4 : Topical administration, 0.01%, 0.03%, and 0.1% gel, qd up to 56 days'}], 'classes': [{'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '>0.05', 'groupIds': ['OG000', 'OG001'], 'paramType': 'ANCOVA', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.80', 'groupDescription': 'The primary population was the Full Analysis(FA)population. The FA population included all patients who were randomized and received at least one dose of study medication and who had at least one baseline efficacy parameter recorded', 'statisticalMethod': 'Fisher Exact', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}], 'paramType': 'NUMBER', 'timeFrame': '56 days', 'description': 'Wound healing means that the wound has closed without any drainage', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The population for efficacy analysis will be the Full Analysis (FA) population. The FA analysis included all patients who were randomized and received at least one dose of study medication and who had at least one baseline efficacy parameter recorded.'}, {'type': 'PRIMARY', 'title': 'Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '22', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Topical administration of placebo gel, 0% Thymosin Beta 4, qd up to 56 days\n\nPlacebo : Topical administration, 0.00% qd up to 56 days'}, {'id': 'OG001', 'title': 'Thymosin Beta 4', 'description': 'Topical administration of 0.01%, 0.03%, and 0.1% Thymosin Beta 4 gel, qd up to 56 days\n\nThymosin Beta 4 : Topical administration, 0.01%, 0.03%, and 0.1% gel, qd up to 56 days'}], 'classes': [{'title': 'Adverse Events', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'Serious Adverse Events', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '70 days', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The population for safety analysis included all patients who were randomized and received at least one dose of study medication and had at one safety parameter recorded.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Placebo', 'description': 'Topical administration of placebo gel, 0% Thymosin Beta 4, qd up to 56 days\n\nPlacebo : Topical administration, 0.00% qd up to 56 days'}, {'id': 'FG001', 'title': 'Thymosin Beta 4', 'description': 'Topical administration of 0.01%, 0.03%, and 0.1% Thymosin Beta 4 gel, qd up to 56 days\n\nThymosin Beta 4 : Topical administration, 0.01%, 0.03%, and 0.1% gel, qd up to 56 days'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '8'}, {'groupId': 'FG001', 'numSubjects': '22'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '8'}, {'groupId': 'FG001', 'numSubjects': '17'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '5'}]}]}], 'recruitmentDetails': 'First Patient In:22 February 2006; Last Patient In: 6 July 2010 Locations: Medical Centers', 'preAssignmentDetails': 'No wash out or run-in periods in this study'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'BG000'}, {'value': '22', 'groupId': 'BG001'}, {'value': '30', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Placebo', 'description': 'Topical administration of placebo gel, 0% Thymosin Beta 4, qd up to 56 days\n\nPlacebo : Topical administration, 0.00% qd up to 56 days'}, {'id': 'BG001', 'title': 'Thymosin Beta 4', 'description': 'Topical administration of 0.01%, 0.03%, and 0.1% Thymosin Beta 4 gel, qd up to 56 days\n\nThymosin Beta 4 : Topical administration, 0.01%, 0.03%, and 0.1% gel, qd up to 56 days'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '12', 'groupId': 'BG001'}, {'value': '16', 'groupId': 'BG002'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '10', 'groupId': 'BG001'}, {'value': '14', 'groupId': 'BG002'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '10', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '14', 'groupId': 'BG001'}, {'value': '20', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '8', 'groupId': 'BG000'}, {'value': '22', 'groupId': 'BG001'}, {'value': '30', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 30}}, 'statusModule': {'whyStopped': 'Lack of patient availability and expiration of study drug', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2006-02'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2014-04', 'completionDateStruct': {'date': '2012-11', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2014-04-25', 'studyFirstSubmitDate': '2006-04-04', 'resultsFirstSubmitDate': '2013-05-22', 'studyFirstSubmitQcDate': '2006-04-04', 'lastUpdatePostDateStruct': {'date': '2014-05-12', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2013-06-07', 'studyFirstPostDateStruct': {'date': '2006-04-06', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2013-06-10', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2010-07', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)', 'timeFrame': '70 days'}], 'secondaryOutcomes': [{'measure': 'Number of Participants Whose Wounds Have Healed', 'timeFrame': '56 days', 'description': 'Wound healing means that the wound has closed without any drainage'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Epidermolysis Bullosa', 'cutaneous wound-healing', 'chronic wound-healing', 'Thymosin Beta 4', 'laminin-5'], 'conditions': ['Epidermolysis Bullosa']}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to investigate a treatment to enhance the healing of acute and chronic nonhealing cutaneous wounds, such as the erosions experienced by patients with Epidermolysis Bullosa (EB), by the known activity of thymosin beta 4 (Tβ4). Funding Source - FDA Office of Orphan Product Development (OOPD).', 'detailedDescription': 'EB is a group of genetic diseases characterized by skin-blistering and lesion-formation after minor trauma to the skin. This family of disorders, most of which are inherited, range in severity from mild to the severely disabling and life-threatening. Tβ4 is a synthetically-produced copy of a naturally-occurring 43 amino acid peptide that has wound healing and anti-inflammatory properties and can up-regulate the expression of laminin-5.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '2 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Informed Consent form signed by the patient or patient's legal representative; also, if the patient is under the age of majority but capable of providing assent, signed assent from the patient\n* Diagnosis of junctional or dystrophic EB.\n* Patients who present with Hallopeau-Siemens subtype may be enrolled.\n* At least one active, unroofed EB erosion on the limb or on the trunk.\n* Lesion size 5 to 50 cm2, inclusive.\n* Stable lesion present for 14-60 days before enrollment.\n* More that one member in a family can be enrolled as long as that member is treated to a different cohort with an assurance that the study medication will not be shared.\n* No clinically significant abnormalities (Grade 2 or higher on the National Cancer Institute \\[NCI\\] toxicity scale) on Screening laboratory tests, except for the following specific laboratory threshold result: albumin must be 2 g/dL or higher; hemoglobin must be 8 g/dL or higher.\n\nExclusion Criteria:\n\n* Clinical evidence of local infection of the index (targeted) lesion.\n* Use of any investigational drug within the 30 days before enrollment.\n* Use of immunotherapy or cytotoxic chemotherapy within the 60 days before enrollment.\n* Use of systemic or topical steroidal therapy within the 30 days before enrollment.Inhaled steroids are allowed.\n* Use of systemic antibiotics within the 7 days before enrollment.\n* Current or former malignancy.\n* Arterial or venous disorder resulting in ulcerated wounds.\n* Diabetes mellitus.\n* Pregnancy or breastfeeding during the study. (A serum pregnancy test will be performed at Screening for female patients of childbearing potential.)"}, 'identificationModule': {'nctId': 'NCT00311766', 'briefTitle': 'A Phase 2 Study on Effect of Thymosin Beta 4 on Wound Healing in Patients With Epidermolysis Bullosa', 'organization': {'class': 'INDUSTRY', 'fullName': 'RegeneRx Biopharmaceuticals, Inc.'}, 'officialTitle': 'A Randomized, Double-Blind, Placebo-Controlled, Dose-Response Study of the Safety and Efficacy of Thymosin Beta 4 in the Treatment of Patients With Epidermolysis Bullosa', 'orgStudyIdInfo': {'id': 'SSEB'}, 'secondaryIdInfos': [{'id': '0003031', 'type': 'OTHER', 'domain': 'FDA Office of Orphan Product Development (OOPD)'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'PLACEBO_COMPARATOR', 'label': '1', 'description': 'Placebo comparator gel does not contain any active drug. Topical administration of 0.0% Thymosin Beta 4 gel, once a day (qd) up to 56 days', 'interventionNames': ['Drug: Placebo']}, {'type': 'ACTIVE_COMPARATOR', 'label': '2', 'description': 'Topical Administration of 0.01%, 0.03%, and 0.1% Thymosin Beta 4 gel once a day (qd) up to 56 days', 'interventionNames': ['Drug: Thymosin Beta 4']}], 'interventions': [{'name': 'Thymosin Beta 4', 'type': 'DRUG', 'description': 'Topical administration, 0.01%, 0.03%, and 0.1% Thymosin Beta 4 gel, qd up to 56 days', 'armGroupLabels': ['2']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Topical administration, 0.00% Thymosin Beta 4 gel qd up to 56 days', 'armGroupLabels': ['1']}]}, 'contactsLocationsModule': {'overallOfficials': [{'name': 'David R Crockford', 'role': 'STUDY_DIRECTOR', 'affiliation': 'RegeneRx Biopharmaceuticals, Inc.'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'RegeneRx Biopharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}