Ignite Creation Date:
2025-12-25 @ 2:08 AM
Ignite Modification Date:
2026-04-19 @ 12:01 AM
Study NCT ID:
NCT02484560
Status:
UNKNOWN
Last Update Posted:
2015-07-01
First Post:
2015-06-16
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Efficacy of Stem Cell Therapy in Ambulatory and Non-ambulatory Children With Duchenne Muscular Dystrophy - Phase 1-2
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'submissionInfos': [{'resetDate': '2019-02-13', 'releaseDate': '2018-09-29'}], 'estimatedResultsFirstSubmitDate': '2018-09-29'}}, 'conditionBrowseModule': {'meshes': [{'id': 'D020388', 'term': 'Muscular Dystrophy, Duchenne'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D009136', 'term': 'Muscular Dystrophies'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}], 'ancestors': [{'id': 'D020966', 'term': 'Muscular Disorders, Atrophic'}, {'id': 'D009135', 'term': 'Muscular Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 10}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2015-06'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2015-06', 'completionDateStruct': {'date': '2015-12', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2015-06-30', 'studyFirstSubmitDate': '2015-06-16', 'studyFirstSubmitQcDate': '2015-06-24', 'lastUpdatePostDateStruct': {'date': '2015-07-01', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2015-06-29', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2015-12', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Degree of improvement in patients with Duchenne Muscular Dystrophy after stem cell therapy treatment administered using Northstar Ambulatory Assessment, Magnetic Resonance Imaging & Spectroscopy, muscle strength assessment equipment, and a questionnaire.', 'timeFrame': '12 Months', 'description': 'Tests Used in Assement:\n\nNorthstar ambulatory assessment CHAQ (Child Health Assessment Questionnaire) MRI/MRS Muscle Strength Assessment - Myogrip, Myopinch, and Moviplate'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Duchenne Muscular Dystrophy', 'Neuromuscular Diseases', 'Muscular Dystrophy', 'Genetic Diseases, X-linked', 'Musculoskeletal Diseases', 'Nervous System Diseases'], 'conditions': ['Duchenne Muscular Dystrophy']}, 'descriptionModule': {'briefSummary': 'Duchenne Muscular Dystrophy (DMD) is a X-linked genetic disorder primarily affecting males, resulting in an absence of dystrophin which ultimately leads to progressive muscle degeneration. Patients with DMD progressively lose functional abilities of movement, breath, and eventually the ability to circulate blood. Currently, there is no cure for DMD, although several strategies are being tested for treatment, none have yet proven to be sufficient. Children with DMD are generally divided into two groups based on severity or progression of the disease, non-ambulatory and ambulatory. Ambulatory patients are capable of walking independently while non-ambulatory patients cannot walk independently.', 'detailedDescription': 'Duchenne Muscular Dystrophy (DMD) is a X-linked genetic disorder primarily affecting males, resulting in an absence of dystrophin which ultimately leads to progressive muscle degeneration. Patients with DMD progressively lose functional abilities of movement, breath, and eventually the ability to circulate blood. Currently, there is no cure for DMD, although several strategies are being tested for treatment, none have yet proven to be sufficient. Children with DMD are generally divided into two groups based on severity or progression of the disease, non-ambulatory and ambulatory. Ambulatory patients are capable of walking independently while non-ambulatory patients cannot walk independently.\n\nThe purpose of this study is to investigate the effects of Allogenic Mesenchymal Stem Cell Therapy in Ambulatory and Non-ambulatory Children with Duchenne Muscular Dystrophy and determine its suitability as a form of treatment.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD'], 'maximumAge': '14 Years', 'minimumAge': '8 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Ambulatory and Non-ambulatory patients diagnosed with DMD that is proven both clinically and genetically and are between 5-20 years old who need partial respiratory support daily. Patients with less than or equal to stage 1 NIH, cardiac, liver, and renal function. Patients must also not present any indication of cancer, allergic disease, nor bleeding diathesis.\n\nExclusion Criteria:\n\n* Patients who require full respiratory support. Patients have stage II NIH or greater, cardiac, liver, and renal function. Patients present with signs of symptoms of cancer, allergic disease, or bleeding diathesis.'}, 'identificationModule': {'nctId': 'NCT02484560', 'briefTitle': 'Efficacy of Stem Cell Therapy in Ambulatory and Non-ambulatory Children With Duchenne Muscular Dystrophy - Phase 1-2', 'organization': {'class': 'OTHER', 'fullName': 'University of Gaziantep'}, 'officialTitle': 'Efficacy of Allogenic Mesenchymal Stem Cell Therapy in Ambulatory and Non-ambulatory Children With Duchenne Muscular Dystrophy - Phase 1-2', 'orgStudyIdInfo': {'id': '56733164/203'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Ambulatory Patients', 'description': 'Ambulatory patients receiving stem cell therapy', 'interventionNames': ['Drug: Biological: Umbilical Cord Based Allogenic Mesenchymal Stem Cell']}, {'type': 'EXPERIMENTAL', 'label': 'Non-Ambulatory Patients', 'description': 'non-ambulatory patients receiving stem cell therapy', 'interventionNames': ['Drug: Biological: Umbilical Cord Based Allogenic Mesenchymal Stem Cell']}], 'interventions': [{'name': 'Biological: Umbilical Cord Based Allogenic Mesenchymal Stem Cell', 'type': 'DRUG', 'armGroupLabels': ['Ambulatory Patients', 'Non-Ambulatory Patients']}]}, 'contactsLocationsModule': {'locations': [{'zip': '27310', 'city': 'Gaziantep', 'country': 'Turkey (Türkiye)', 'facility': 'Gaziantep University Hospital', 'geoPoint': {'lat': 37.05944, 'lon': 37.3825}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University of Gaziantep', 'class': 'OTHER'}, 'collaborators': [{'name': 'Istinye University', 'class': 'OTHER'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Lead Investigator', 'investigatorFullName': 'Alper Dai, MD', 'investigatorAffiliation': 'University of Gaziantep'}}}, 'annotationSection': {'annotationModule': {'unpostedAnnotation': {'unpostedEvents': [{'date': '2018-09-29', 'type': 'RELEASE'}, {'date': '2019-02-13', 'type': 'RESET'}], 'unpostedResponsibleParty': 'Alper Dai, MD, Lead Investigator, University of Gaziantep'}}}}