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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

Tracking Information
First Submitted Date  ICMJE October 30, 2011
First Posted Date  ICMJE December 9, 2011
Last Update Posted Date June 20, 2012
Study Start Date  ICMJE December 2011
Estimated Primary Completion Date July 2013   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 8, 2011)
  • 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)
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 8, 2011)
  • 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 Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A New Method to Treat Hereditary Cerebellar Ataxia - Umbilical Cord Mesenchymal Stem Cells Transplantation
Official Title  ICMJE 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 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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Hereditary Cerebellar Ataxia.
Intervention  ICMJE 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.
Study Arms  ICMJE
  • 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.
    Intervention: Other: stem cell transplantation
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Unknown status
Estimated Enrollment  ICMJE
 (submitted: December 8, 2011)
20
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE July 2014
Estimated Primary Completion Date July 2013   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 65 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE China
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01489267
Other Study ID Numbers  ICMJE 20111031SCA
20111031limin ( Other Identifier: neuralstemcell )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party General Hospital of Chinese Armed Police Forces
Study Sponsor  ICMJE General Hospital of Chinese Armed Police Forces
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Yihua An, doctor director of neural stem cell transplantation department in general hospital of chinese armed police forces
PRS Account General Hospital of Chinese Armed Police Forces
Verification Date June 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP