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.
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 .
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
20
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).
Yihua An
Beijing, China
blood test
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
Time frame: within one week before transplantation
Nerve functional evaluation
1. Timed-up-and-go test ,5 minutes down-and-up test 2. ICARS score 3. Modified Falls Efficacy Scale 4. Berg balance assessment 5. Tremor Rating Scale: * Part A - Tremor location/severity rating * Part B -Handwriting and drawings * Part C - Functional disabilities resulting from tremor 6. Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
Time frame: within one week before transplantation
blood test
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
Time frame: 1 month after transplantation
blood test
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
Time frame: 3 months after transplantation
blood test
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
Time frame: 6 months after transplantation
blood test
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
Time frame: 12 months after transplantation
Nerve functional evaluation
1. Timed-up-and-go test ,5 minutes down-and-up test 2. ICARS score 3. Modified Falls Efficacy Scale 4. Berg balance assessment 5. Tremor Rating Scale: * Part A - Tremor location/severity rating * Part B -Handwriting and drawings * Part C - Functional disabilities resulting from tremor 6. Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
Time frame: 1 month after transplantation
Nerve functional evaluation
1. Timed-up-and-go test ,5 minutes down-and-up test 2. ICARS score 3. Modified Falls Efficacy Scale 4. Berg balance assessment 5. Tremor Rating Scale: * Part A - Tremor location/severity rating * Part B -Handwriting and drawings * Part C - Functional disabilities resulting from tremor 6. Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
Time frame: 3 months after transplantation
Nerve functional evaluation
1. Timed-up-and-go test ,5 minutes down-and-up test 2. ICARS score 3. Modified Falls Efficacy Scale 4. Berg balance assessment 5. Tremor Rating Scale: * Part A - Tremor location/severity rating * Part B -Handwriting and drawings * Part C - Functional disabilities resulting from tremor 6. Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
Time frame: 6 months after transplantation
Nerve functional evaluation
1. Timed-up-and-go test ,5 minutes down-and-up test 2. ICARS score 3. Modified Falls Efficacy Scale 4. Berg balance assessment 5. Tremor Rating Scale: * Part A - Tremor location/severity rating * Part B -Handwriting and drawings * Part C - Functional disabilities resulting from tremor 6. Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
Time frame: 12 months after transplantation
Urinal test
proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
Time frame: within one week before transplantation
Electrophysiology examination
somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)
Time frame: within one week before transplantation
Urinal test
proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
Time frame: 1 month after transplantation
Urinal test
proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
Time frame: 3 months after transplantation
Urinal test
proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
Time frame: 6 months after transplantation
Urinal test
proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
Time frame: 12 months after transplantation
Electrophysiology examination
somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)
Time frame: 1 month after transplantation
Electrophysiology examination
somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)
Time frame: 3 months after transplantation
Electrophysiology examination
somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)
Time frame: 6 months after transplantation
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Electrophysiology examination
somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)
Time frame: 12 months after transplantation