Clinical Trial: A New Method to Treat Hereditary Cerebellar Ataxia - Umbilical Cord Mesenchymal Stem Cells Transplantation

Study Status: Completed
Recruit Status: Unknown status
Study Type: Interventional

Official Title: The Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cells Transplantation in Hereditary Cerebellar Ataxia Patients

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.

Detailed Summary: 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 .
Sponsor: General Hospital of Chinese Armed Police Forces

Current Primary Outcome:

  • 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&#

    Original Primary Outcome: Same as current

    Current Secondary Outcome:

    • 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)


    Original Secondary Outcome: Same as current

    Information By: General Hospital of Chinese Armed Police Forces

    Dates:
    Date Received: October 30, 2011
    Date Started: December 2011
    Date Completion: July 2014
    Last Updated: June 18, 2012
    Last Verified: June 2012