Ignite Creation Date:
2025-12-25 @ 12:54 AM
Ignite Modification Date:
2025-12-31 @ 1:11 PM
Study NCT ID:
NCT01489267
Status:
UNKNOWN
Last Update Posted:
2012-06-20
First Post:
2011-10-30
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A New Method to Treat Hereditary Cerebellar Ataxia - Umbilical Cord Mesenchymal Stem Cells Transplantation
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D013132', 'term': 'Spinocerebellar Degenerations'}, {'id': 'D002524', 'term': 'Cerebellar Ataxia'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}], 'ancestors': [{'id': 'D002526', 'term': 'Cerebellar Diseases'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D013118', 'term': 'Spinal Cord Diseases'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D001259', 'term': 'Ataxia'}, {'id': 'D020820', 'term': 'Dyskinesias'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D033581', 'term': 'Stem Cell Transplantation'}, {'id': 'D064987', 'term': 'Cell- and Tissue-Based Therapy'}], 'ancestors': [{'id': 'D017690', 'term': 'Cell Transplantation'}, {'id': 'D001691', 'term': 'Biological Therapy'}, {'id': 'D013812', 'term': 'Therapeutics'}, {'id': 'D014180', 'term': 'Transplantation'}, {'id': 'D013514', 'term': 'Surgical Procedures, Operative'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 20}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'ENROLLING_BY_INVITATION', 'startDateStruct': {'date': '2011-12'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2012-06', 'completionDateStruct': {'date': '2014-07', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2012-06-18', 'studyFirstSubmitDate': '2011-10-30', 'studyFirstSubmitQcDate': '2011-12-08', 'lastUpdatePostDateStruct': {'date': '2012-06-20', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2011-12-09', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2013-07', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'blood test', 'timeFrame': 'within one week before transplantation', 'description': 'white blood cell、neutrophilic granulocyte、leukomonocyte；glutamic pyruvic transaminase(GPT)、glutamic oxalacetic transaminase、lactate dehydrogenase(LDH)、;hydroxybutyrate dehydrogenase(HBDH)、phosphocreatine kinase(CK)；acidum uricum(UA)、creatinine(Cr)、α1- microglobulin、β2- microglobulin；lymphotoxin(LCT). Tumor markers(AFP,CEA,CA125,CA15-3,CA19-9,CA72-4CY21-1,NSE) ,Lymphocytes classification'}, {'measure': 'Nerve functional evaluation', 'timeFrame': 'within one week before transplantation', 'description': '1. Timed-up-and-go test ,5 minutes down-and-up test\n2. ICARS score\n3. Modified Falls Efficacy Scale\n4. Berg balance assessment\n5. Tremor Rating Scale:\n\n * Part A - Tremor location/severity rating\n * Part B -Handwriting and drawings\n * Part C - Functional disabilities resulting from tremor\n6. Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)'}, {'measure': 'blood test', 'timeFrame': '1 month after transplantation', 'description': 'white blood cell、neutrophilic granulocyte、leukomonocyte；glutamic pyruvic transaminase(GPT)、glutamic oxalacetic transaminase、lactate dehydrogenase(LDH)、;hydroxybutyrate dehydrogenase(HBDH)、phosphocreatine kinase(CK)；acidum uricum(UA)、creatinine(Cr)、α1- microglobulin、β2- microglobulin；lymphotoxin(LCT). Tumor markers(AFP,CEA,CA125,CA15-3,CA19-9,CA72-4CY21-1,NSE) ,Lymphocytes classification'}, {'measure': 'blood test', 'timeFrame': '3 months after transplantation', 'description': 'white blood cell、neutrophilic granulocyte、leukomonocyte；glutamic pyruvic transaminase(GPT)、glutamic oxalacetic transaminase、lactate dehydrogenase(LDH)、;hydroxybutyrate dehydrogenase(HBDH)、phosphocreatine kinase(CK)；acidum uricum(UA)、creatinine(Cr)、α1- microglobulin、β2- microglobulin；lymphotoxin(LCT). Tumor markers(AFP,CEA,CA125,CA15-3,CA19-9,CA72-4CY21-1,NSE) ,Lymphocytes classification'}, {'measure': 'blood test', 'timeFrame': '6 months after transplantation', 'description': 'white blood cell、neutrophilic granulocyte、leukomonocyte；glutamic pyruvic transaminase(GPT)、glutamic oxalacetic transaminase、lactate dehydrogenase(LDH)、;hydroxybutyrate dehydrogenase(HBDH)、phosphocreatine kinase(CK)；acidum uricum(UA)、creatinine(Cr)、α1- microglobulin、β2- microglobulin；lymphotoxin(LCT). Tumor markers(AFP,CEA,CA125,CA15-3,CA19-9,CA72-4CY21-1,NSE) ,Lymphocytes classification'}, {'measure': 'blood test', 'timeFrame': '12 months after transplantation', 'description': 'white blood cell、neutrophilic granulocyte、leukomonocyte；glutamic pyruvic transaminase(GPT)、glutamic oxalacetic transaminase、lactate dehydrogenase(LDH)、;hydroxybutyrate dehydrogenase(HBDH)、phosphocreatine kinase(CK)；acidum uricum(UA)、creatinine(Cr)、α1- microglobulin、β2- microglobulin；lymphotoxin(LCT). Tumor markers(AFP,CEA,CA125,CA15-3,CA19-9,CA72-4CY21-1,NSE) ,Lymphocytes classification'}, {'measure': 'Nerve functional evaluation', 'timeFrame': '1 month after transplantation', 'description': '1. Timed-up-and-go test ,5 minutes down-and-up test\n2. ICARS score\n3. Modified Falls Efficacy Scale\n4. Berg balance assessment\n5. Tremor Rating Scale:\n\n * Part A - Tremor location/severity rating\n * Part B -Handwriting and drawings\n * Part C - Functional disabilities resulting from tremor\n6. Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)'}, {'measure': 'Nerve functional evaluation', 'timeFrame': '3 months after transplantation', 'description': '1. Timed-up-and-go test ,5 minutes down-and-up test\n2. ICARS score\n3. Modified Falls Efficacy Scale\n4. Berg balance assessment\n5. Tremor Rating Scale:\n\n * Part A - Tremor location/severity rating\n * Part B -Handwriting and drawings\n * Part C - Functional disabilities resulting from tremor\n6. Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)'}, {'measure': 'Nerve functional evaluation', 'timeFrame': '6 months after transplantation', 'description': '1. Timed-up-and-go test ,5 minutes down-and-up test\n2. ICARS score\n3. Modified Falls Efficacy Scale\n4. Berg balance assessment\n5. Tremor Rating Scale:\n\n * Part A - Tremor location/severity rating\n * Part B -Handwriting and drawings\n * Part C - Functional disabilities resulting from tremor\n6. Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)'}, {'measure': 'Nerve functional evaluation', 'timeFrame': '12 months after transplantation', 'description': '1. Timed-up-and-go test ,5 minutes down-and-up test\n2. ICARS score\n3. Modified Falls Efficacy Scale\n4. Berg balance assessment\n5. Tremor Rating Scale:\n\n * Part A - Tremor location/severity rating\n * Part B -Handwriting and drawings\n * Part C - Functional disabilities resulting from tremor\n6. Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)'}], 'secondaryOutcomes': [{'measure': 'Urinal test', 'timeFrame': 'within one week before transplantation', 'description': 'proteinum、akaryocyte、α1- microglobulin、β2- microglobulin'}, {'measure': 'Electrophysiology examination', 'timeFrame': 'within one week before transplantation', 'description': 'somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)'}, {'measure': 'Urinal test', 'timeFrame': '1 month after transplantation', 'description': 'proteinum、akaryocyte、α1- microglobulin、β2- microglobulin'}, {'measure': 'Urinal test', 'timeFrame': '3 months after transplantation', 'description': 'proteinum、akaryocyte、α1- microglobulin、β2- microglobulin'}, {'measure': 'Urinal test', 'timeFrame': '6 months after transplantation', 'description': 'proteinum、akaryocyte、α1- microglobulin、β2- microglobulin'}, {'measure': 'Urinal test', 'timeFrame': '12 months after transplantation', 'description': 'proteinum、akaryocyte、α1- microglobulin、β2- microglobulin'}, {'measure': 'Electrophysiology examination', 'timeFrame': '1 month after transplantation', 'description': 'somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)'}, {'measure': 'Electrophysiology examination', 'timeFrame': '3 months after transplantation', 'description': 'somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)'}, {'measure': 'Electrophysiology examination', 'timeFrame': '6 months after transplantation', 'description': 'somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)'}, {'measure': 'Electrophysiology examination', 'timeFrame': '12 months after transplantation', 'description': 'somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['cerebellar ataxia', 'hereditary disease', 'stem cell'], 'conditions': ['Hereditary Cerebellar Ataxia.']}, 'descriptionModule': {'briefSummary': 'Hereditary cerebellar ataxia is a type of autosomal dominant genetic disease, lesions mainly involving the cerebellum, but the spinal cord and cranial nerves may also be some involvement. A total of 20 molecularly diagnosed SCA1 patients divided in two groups. One group accepted for the treatment of stem cell transplantation,the other group will be the control. Purpose of this project to prove that allogeneic umbilical cord mesenchymal stem cells are applied to clinical safely, and in the treatment of hereditary cerebellar ataxia is valid.', 'detailedDescription': 'After admission the patients accepted transplantation would receive physical examination, blood and urine tests, electrocardiogram, electrophysiologic study;evaluation of SCA1 symptoms (balance, walking, dexterity, tremor, memory, mood and concentration),then stem cell therapy：after stem cell prepared, the patients accepted 4 times stem cell transplantations through lumbar puncture, the time is 3-5days between two treatments. The control only receive evaluation of SCA1 symptoms .'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. 18-65 years of age\n2. Molecularly diagnosed SCA1\n\nExclusion Criteria:\n\n1. Cognitively impaired individuals\n2. Schizophrenics\n3. Patients with severe kidney and liver disease, epilepsy, heart disease, pulmonary disease, cardiac arrhythmia, diabetes insipidus, leukemia and some other central neural disease (such as Parkinson disease, accidence of brain vascular et al)\n4. Age less than 18 years, age greater than 65 years'}, 'identificationModule': {'nctId': 'NCT01489267', 'acronym': 'SCA', 'briefTitle': 'A New Method to Treat Hereditary Cerebellar Ataxia - Umbilical Cord Mesenchymal Stem Cells Transplantation', 'organization': {'class': 'OTHER', 'fullName': 'General Hospital of Chinese Armed Police Forces'}, 'officialTitle': 'The Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cells Transplantation in Hereditary Cerebellar Ataxia Patients', 'orgStudyIdInfo': {'id': '20111031SCA'}, 'secondaryIdInfos': [{'id': '20111031limin', 'type': 'OTHER', 'domain': 'neuralstemcell'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'NO_INTERVENTION', 'label': 'Control group', 'description': 'Ten patients in the group only receive nerve functional evaluation and electrophysiology examination before and 1,3,6,12 months after recruit. They will not accept cell therapy.'}, {'type': 'EXPERIMENTAL', 'label': 'Stem cell transplantation', 'description': '10 patients in the group accept stem cell transplantation.', 'interventionNames': ['Other: stem cell transplantation']}], 'interventions': [{'name': 'stem cell transplantation', 'type': 'OTHER', 'otherNames': ['Cell therapy.'], 'description': "The patients accepted 4 times stem cell transplantations through lumbar puncture, the time is 3-5days between two treatments and the dose is about 2 ml(including 1×10'7 cells).", 'armGroupLabels': ['Stem cell transplantation']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Beijing', 'country': 'China', 'facility': 'Yihua An', 'geoPoint': {'lat': 39.9075, 'lon': 116.39723}}], 'overallOfficials': [{'name': 'Yihua An, doctor', 'role': 'STUDY_DIRECTOR', 'affiliation': 'director of neural stem cell transplantation department in general hospital of chinese armed police forces'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'General Hospital of Chinese Armed Police Forces', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}