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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
Sponsor:
Information provided by (Responsible Party):
General Hospital of Chinese Armed Police Forces

Study Description
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Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Hereditary Cerebellar Ataxia. Other: stem cell transplantation Phase 2

Detailed Description:
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 Design
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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

Resource links provided by the National Library of Medicine


Arms and Interventions
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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.
Experimental: Stem cell transplantation
10 patients in the group accept stem cell transplantation.
 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.


Outcome Measures
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Primary Outcome Measures :
  1. 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

  2. Nerve functional evaluation [ Time Frame: within one week before transplantation ]
    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)

  3. 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

  4. 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

  5. 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

  6. 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

  7. Nerve functional evaluation [ Time Frame: 1 month after transplantation ]
    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)

  8. Nerve functional evaluation [ Time Frame: 3 months after transplantation ]
    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)

  9. Nerve functional evaluation [ Time Frame: 6 months after transplantation ]
    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)

  10. Nerve functional evaluation [ Time Frame: 12 months after transplantation ]
    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)


Secondary Outcome Measures :
  1. Urinal test [ Time Frame: within one week before transplantation ]
    proteinum、akaryocyte、α1- microglobulin、β2- microglobulin

  2. Electrophysiology examination [ Time Frame: within one week before transplantation ]
    somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)

  3. Urinal test [ Time Frame: 1 month after transplantation ]
    proteinum、akaryocyte、α1- microglobulin、β2- microglobulin

  4. Urinal test [ Time Frame: 3 months after transplantation ]
    proteinum、akaryocyte、α1- microglobulin、β2- microglobulin

  5. Urinal test [ Time Frame: 6 months after transplantation ]
    proteinum、akaryocyte、α1- microglobulin、β2- microglobulin

  6. Urinal test [ Time Frame: 12 months after transplantation ]
    proteinum、akaryocyte、α1- microglobulin、β2- microglobulin

  7. Electrophysiology examination [ Time Frame: 1 month after transplantation ]
    somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)

  8. Electrophysiology examination [ Time Frame: 3 months after transplantation ]
    somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)

  9. Electrophysiology examination [ Time Frame: 6 months after transplantation ]
    somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)

  10. Electrophysiology examination [ Time Frame: 12 months after transplantation ]
    somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)


Eligibility Criteria
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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
Criteria

Inclusion Criteria:

  1. 18-65 years of age
  2. Molecularly diagnosed SCA1

Exclusion Criteria:

  1. Cognitively impaired individuals
  2. Schizophrenics
  3. 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)
  4. Age less than 18 years, age greater than 65 years
Contacts and Locations
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Information from the National Library of Medicine

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


Locations
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China
Yihua An
Beijing, China
Sponsors and Collaborators
General Hospital of Chinese Armed Police Forces
Investigators
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Study Director: Yihua An, doctor director of neural stem cell transplantation department in general hospital of chinese armed police forces
More Information
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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
Keywords provided by General Hospital of Chinese Armed Police Forces:
cerebellar ataxia
hereditary disease
stem cell
Additional relevant MeSH terms:
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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