Ignite Creation Date:
2025-12-26 @ 1:16 PM
Ignite Modification Date:
2026-03-01 @ 3:21 AM
Study NCT ID:
NCT03085706
Status:
COMPLETED
Last Update Posted:
2018-02-09
First Post:
2017-03-15
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Transplantation of Autologous Peripheral Blood Mononuclear Cells for Amyotrophic Lateral Sclerosis
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000690', 'term': 'Amyotrophic Lateral Sclerosis'}], 'ancestors': [{'id': 'D013118', 'term': 'Spinal Cord Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D016472', 'term': 'Motor Neuron Disease'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D057177', 'term': 'TDP-43 Proteinopathies'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D057165', 'term': 'Proteostasis Deficiencies'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['NA'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': '14 patients conducted at the First Affiliated Hospital of Dalian Medical University, China were eligible if they had definite or probable sporadic amyotrophic lateral sclerosis (ALS).'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 14}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2010-10', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2018-02', 'completionDateStruct': {'date': '2013-06', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2018-02-07', 'studyFirstSubmitDate': '2017-03-15', 'studyFirstSubmitQcDate': '2017-03-15', 'lastUpdatePostDateStruct': {'date': '2018-02-09', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2017-03-21', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2012-12', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'adverse events of autologous peripheral blood mononuclear cell mobilization', 'timeFrame': '1 week after operation', 'description': 'To assess the safety of autologous peripheral blood mononuclear cell transplantation'}], 'secondaryOutcomes': [{'measure': 'Functional independence measurement(FIM)', 'timeFrame': 'changes of preoperation and week 1, week 2, week 4, week 12 after operation', 'description': 'To assess the self-care ability of daily living'}, {'measure': 'Berg Balance Scale', 'timeFrame': 'changes of preoperation and week 1, week 2, week 4, week 12 after operation', 'description': 'To assess the trunk balance capability and limb movement function'}, {'measure': 'Dysarthria Assessment Scale', 'timeFrame': 'changes of preoperation and week 1, week 2, week 4, week 12 after operation', 'description': 'To assess the progression of bulbar paralysis'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Amyotrophic Lateral Sclerosis']}, 'descriptionModule': {'briefSummary': 'To assess the safety of peripheral blood mononuclear cell transplantation into the subarachnoid space for the treatment of amyotrophic lateral sclerosis.', 'detailedDescription': 'Amyotrophic lateral sclerosis (ALS) is a rapidly evolving, fatal neurodegenerative disease resulting from the degeneration of cortical, bulbar and spinal motor neurons. The disease progresses inexorably to death, usually because by failure of respiratory function, with a median duration of 3 years.\n\nRecent clinical trials using various types of stem cells, including mesenchymal stromal cells, neural stem cells, and peripheral blood mononuclear cells (PBMCs), represent promising strategies for stem cell-based treatment in ALS. It has been demonstrated that the inflammation and neuronal death were reduced in ALS patients after bone marrow transplantation. In addition, the incidence of immune response was decreased by autologous transplantation of bone marrow cells in ALS patients. PBMCs are multi-potent stem cells that are very attractive for a cell therapy approach in ALS because of their plasticity and ability to provide the host tissue with growth factors or modulate the host immune system. PBMCs were used clinically and few adverse effects were attributed to their administration. Early clinical investigations indicated that the transplantation of autologous PBMCs into the dura is feasible in ALS patients; however, one study was limited to three patients and the other recruited eight patients. There are still many questions regarding the intrathecal transplantation of PBMCs for ALS. Therefore, a retrospective study was performed to assess further the safety and efficacy of the procedure and to test the impact of a cell therapy approach in ALS patients.\n\nStatistical analysis Data, expressed as the mean ± SD, were analyzed using SPSS version 17.0 for Windows (SPSS Inc., Chicago, IL, USA). Statistical analyses were performed by paired sample t-test. A value of P \\< 0.05 was considered statistically significant.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '75 Years', 'minimumAge': '31 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* all subjects had a verifiable diagnosis of ALS for 0.5 to 2 years based on a diagnosis using the Revised Criteria of the World Federation of Neurology. The grades of diagnosis were clinically definite ALS or clinically probable ALS;\n* ALS was mild-to-moderate based on the ALS Functional Rating Scale-Revised. Electrophysiological features showed compound muscle action potential (CMAP) amplitude of motor nerve normal or mild declining;\n* serum creatine kinase was normal or mild upper, less than 500 U/L.\n\nExclusion Criteria:\n\n* use of any other investigational agent within 30 days before treatment;\n* severe cardiac, pulmonary, hepatic or/and hematic disease;\n* human immunodeficiency virus positivity or signs and symptoms consistent with human immunodeficiency virus infection;\n* pregnant or nursing women;\n* history of cancer with less than 5 years documentation of a disease-free state;\n* history of anaphylactic reaction or hypersensitivity to granulocyte colony- stimulating factor (G-CSF);\n* alcohol or drug abuse in recent 1 year;\n* cannot understand or obey the rules of treatment;\n* blood donor in recent 30 days.'}, 'identificationModule': {'nctId': 'NCT03085706', 'briefTitle': 'Transplantation of Autologous Peripheral Blood Mononuclear Cells for Amyotrophic Lateral Sclerosis', 'organization': {'class': 'OTHER', 'fullName': 'The First Affiliated Hospital of Dalian Medical University'}, 'officialTitle': 'Transplantation of Autologous Peripheral Blood Mononuclear Cells in the Subarachnoid Space for Amyotrophic Lateral Sclerosis: a Safety Analysis of 14 Patients', 'orgStudyIdInfo': {'id': 'LCKY2016-58'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'PBMC autotransplantation', 'description': 'Fourteen amyotrophic lateral sclerosis (ALS) patients are received peripheral blood mononuclear cell (PBMC) autotransplantation.', 'interventionNames': ['Biological: PBMC autotransplantation']}], 'interventions': [{'name': 'PBMC autotransplantation', 'type': 'BIOLOGICAL', 'description': 'Fourteen amyotrophic lateral sclerosis (ALS) patients are received peripheral blood mononuclear cell (PBMC) autotransplantation.', 'armGroupLabels': ['PBMC autotransplantation']}]}, 'contactsLocationsModule': {'locations': [{'zip': '116011', 'city': 'Dalian', 'state': 'Liaoning', 'country': 'China', 'facility': 'The First Affiliated Hospital of Dalian Medical University', 'geoPoint': {'lat': 38.91222, 'lon': 121.60222}}], 'overallOfficials': [{'name': 'Jing Liu, Ph.D.', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'The First Affiliated Hospital of Dalian Medical University'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'The First Affiliated Hospital of Dalian Medical University', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Chief Physician', 'investigatorFullName': 'Liu Jing', 'investigatorAffiliation': 'The First Affiliated Hospital of Dalian Medical University'}}}}