A New Method to Treat Hereditary Cerebellar Ataxia - Umbilical Cord Mesenchymal Stem Cells Transplantation (SCA)
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ClinicalTrials.gov Identifier: NCT01489267 |
Recruitment Status : Unknown
Verified June 2012 by General Hospital of Chinese Armed Police Forces.
Recruitment status was: Enrolling by invitation
First Posted : December 9, 2011
Last Update Posted : June 20, 2012
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Condition or disease | Intervention/treatment | Phase |
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Hereditary Cerebellar Ataxia. | Other: stem cell transplantation | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 20 participants |
Allocation: | Non-Randomized |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | The Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cells Transplantation in Hereditary Cerebellar Ataxia Patients |
Study Start Date : | December 2011 |
Estimated Primary Completion Date : | July 2013 |
Estimated Study Completion Date : | July 2014 |

Arm | Intervention/treatment |
---|---|
No Intervention: Control group
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.
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Experimental: Stem cell transplantation
10 patients in the group accept stem cell transplantation.
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Other: stem cell transplantation
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).
Other Name: Cell therapy. |
- blood test [ Time Frame: within one week before transplantation ]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
- Nerve functional evaluation [ Time Frame: within one week before transplantation ]
- Timed-up-and-go test ,5 minutes down-and-up test
- ICARS score
- Modified Falls Efficacy Scale
- Berg balance assessment
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Tremor Rating Scale:
- Part A - Tremor location/severity rating
- Part B -Handwriting and drawings
- Part C - Functional disabilities resulting from tremor
- Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
- blood test [ Time Frame: 1 month after transplantation ]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
- blood test [ Time Frame: 3 months after transplantation ]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
- blood test [ Time Frame: 6 months after transplantation ]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
- blood test [ Time Frame: 12 months after transplantation ]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
- Nerve functional evaluation [ Time Frame: 1 month after transplantation ]
- Timed-up-and-go test ,5 minutes down-and-up test
- ICARS score
- Modified Falls Efficacy Scale
- Berg balance assessment
-
Tremor Rating Scale:
- Part A - Tremor location/severity rating
- Part B -Handwriting and drawings
- Part C - Functional disabilities resulting from tremor
- Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
- Nerve functional evaluation [ Time Frame: 3 months after transplantation ]
- Timed-up-and-go test ,5 minutes down-and-up test
- ICARS score
- Modified Falls Efficacy Scale
- Berg balance assessment
-
Tremor Rating Scale:
- Part A - Tremor location/severity rating
- Part B -Handwriting and drawings
- Part C - Functional disabilities resulting from tremor
- Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
- Nerve functional evaluation [ Time Frame: 6 months after transplantation ]
- Timed-up-and-go test ,5 minutes down-and-up test
- ICARS score
- Modified Falls Efficacy Scale
- Berg balance assessment
-
Tremor Rating Scale:
- Part A - Tremor location/severity rating
- Part B -Handwriting and drawings
- Part C - Functional disabilities resulting from tremor
- Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
- Nerve functional evaluation [ Time Frame: 12 months after transplantation ]
- Timed-up-and-go test ,5 minutes down-and-up test
- ICARS score
- Modified Falls Efficacy Scale
- Berg balance assessment
-
Tremor Rating Scale:
- Part A - Tremor location/severity rating
- Part B -Handwriting and drawings
- Part C - Functional disabilities resulting from tremor
- Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
- Urinal test [ Time Frame: within one week before transplantation ]proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
- Electrophysiology examination [ Time Frame: within one week before transplantation ]somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)
- Urinal test [ Time Frame: 1 month after transplantation ]proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
- Urinal test [ Time Frame: 3 months after transplantation ]proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
- Urinal test [ Time Frame: 6 months after transplantation ]proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
- Urinal test [ Time Frame: 12 months after transplantation ]proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
- Electrophysiology examination [ Time Frame: 1 month after transplantation ]somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)
- Electrophysiology examination [ Time Frame: 3 months after transplantation ]somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)
- Electrophysiology examination [ Time Frame: 6 months after transplantation ]somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)
- Electrophysiology examination [ Time Frame: 12 months after transplantation ]somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)

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Ages Eligible for Study: | 18 Years to 65 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- 18-65 years of age
- Molecularly diagnosed SCA1
Exclusion Criteria:
- Cognitively impaired individuals
- Schizophrenics
- 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)
- Age less than 18 years, age greater than 65 years

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01489267
China | |
Yihua An | |
Beijing, China |
Study Director: | Yihua An, doctor | director of neural stem cell transplantation department in general hospital of chinese armed police forces |
Responsible Party: | General Hospital of Chinese Armed Police Forces |
ClinicalTrials.gov Identifier: | NCT01489267 |
Other Study ID Numbers: |
20111031SCA 20111031limin ( Other Identifier: neuralstemcell ) |
First Posted: | December 9, 2011 Key Record Dates |
Last Update Posted: | June 20, 2012 |
Last Verified: | June 2012 |
cerebellar ataxia hereditary disease stem cell |
Ataxia Cerebellar Ataxia Spinocerebellar Degenerations Dyskinesias Neurologic Manifestations Nervous System Diseases Cerebellar Diseases |
Brain Diseases Central Nervous System Diseases Spinal Cord Diseases Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Genetic Diseases, Inborn |