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A New Method to Treat Hereditary Cerebellar Ataxia - Umbilical Cord Mesenchymal Stem Cells Transplantation (SCA)

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.
Verified June 2012 by General Hospital of Chinese Armed Police Forces.
Recruitment status was:  Enrolling by invitation
Sponsor:
ClinicalTrials.gov Identifier:
NCT01489267
First Posted: December 9, 2011
Last Update Posted: June 20, 2012
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
General Hospital of Chinese Armed Police Forces
  Purpose
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 Intervention Phase
Hereditary Cerebellar Ataxia. Other: stem cell transplantation Phase 2

Study Type: Interventional
Study Design: 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

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by General Hospital of Chinese Armed Police Forces:

Primary Outcome Measures:
  • 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 ]
    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)

  • 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 ]
    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)

  • 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)

  • 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)

  • 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:
  • 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)
Estimated Enrollment: 20
Study Start Date: December 2011
Estimated Study Completion Date: July 2014
Estimated Primary Completion Date: July 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.

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 .
  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (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
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
China
Yihua An
Beijing, China
Sponsors and Collaborators
General Hospital of Chinese Armed Police Forces
Investigators
Study Director: Yihua An, doctor director of neural stem cell transplantation department in general hospital of chinese armed police forces
  More Information

Responsible Party: General Hospital of Chinese Armed Police Forces
ClinicalTrials.gov Identifier: NCT01489267     History of Changes
Other Study ID Numbers: 20111031SCA
20111031limin ( Other Identifier: neuralstemcell )
First Submitted: October 30, 2011
First Posted: December 9, 2011
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:
Ataxia
Cerebellar Ataxia
Spinocerebellar Degenerations
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
 Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn