Ignite Creation Date:
2025-12-24 @ 9:22 PM
Ignite Modification Date:
2025-12-29 @ 7:01 PM
Study NCT ID:
NCT01005004
Status:
COMPLETED
Last Update Posted:
2015-01-15
First Post:
2009-10-28
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D020371', 'term': 'Pelizaeus-Merzbacher Disease'}], 'ancestors': [{'id': 'D020279', 'term': 'Hereditary Central Nervous System Demyelinating Diseases'}, {'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D056784', 'term': 'Leukoencephalopathies'}, {'id': 'D003711', 'term': 'Demyelinating Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 4}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2009-11'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2015-01', 'completionDateStruct': {'date': '2012-12', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2015-01-13', 'studyFirstSubmitDate': '2009-10-28', 'studyFirstSubmitQcDate': '2009-10-28', 'lastUpdatePostDateStruct': {'date': '2015-01-15', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2009-10-30', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2012-10', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Safety assessment through clinical neurological and MRI evaluation.', 'timeFrame': 'one year post transplant'}], 'secondaryOutcomes': [{'measure': 'MRI examination for post-transplant myelination', 'timeFrame': 'one year post transplant'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['PMD', 'stem cells', 'Pelizaeus-Merzbacher Disease', 'human stem cells', 'central nervous system stem cells'], 'conditions': ['Pelizaeus-Merzbacher Disease']}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to determine the safety and preliminary effectiveness of human central nervous system stem cells (HuCNS-SC®) transplantation in patients with Connatal Pelizaeus-Merzbacher Disease (PMD).', 'detailedDescription': 'Enrolled subjects will be transplanted with HuCNS-SC cells into the brain and will receive immunosuppression for nine months. The study observation period is for one year after transplant surgery. Thereafter, subjects will be enrolled in a long-term observational follow-up study for four years.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD'], 'maximumAge': '5 Years', 'minimumAge': '6 Months', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Confirmed clinical diagnosis of connatal PMD\n* Molecular genetic confirmation of mutation in the proteolipid protein 1 (PLP1) gene\n* MRI consistent with PMD as interpreted by a qualified neuroradiologist\n\nExclusion Criteria:\n\n* Other significant congenital brain abnormality not related to PMD\n* Previous participation in gene transfer or cell transplant trial\n* Presence of neurological signs and symptoms not consistent with PMD\n* Current or prior malignancy\n* Prior organ, tissue or bone marrow transplant'}, 'identificationModule': {'nctId': 'NCT01005004', 'briefTitle': 'Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects', 'organization': {'class': 'INDUSTRY', 'fullName': 'StemCells, Inc.'}, 'officialTitle': 'Phase I Study of the Safety and Preliminary Efficacy of Intracerebral Transplantation of HuCNS-SC® Cells for Connatal Pelizaeus-Merzbacher Disease (PMD)', 'orgStudyIdInfo': {'id': 'CL-N01-PMD'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'HuCNS-SC cells', 'description': 'Intracerebral implantation of HuCNS-SC via direct injection during surgery', 'interventionNames': ['Biological: HuCNS-SC cells']}], 'interventions': [{'name': 'HuCNS-SC cells', 'type': 'BIOLOGICAL', 'otherNames': ['Human central nervous system stem cells'], 'description': 'intracerebral transplantation', 'armGroupLabels': ['HuCNS-SC cells']}]}, 'contactsLocationsModule': {'locations': [{'zip': '94143', 'city': 'San Francisco', 'state': 'California', 'country': 'United States', 'facility': 'University of California, San Francisco', 'geoPoint': {'lat': 37.77493, 'lon': -122.41942}}], 'overallOfficials': [{'name': 'Stephen Huhn, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'StemCells, Inc.'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'StemCells, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}