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

Study Description

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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

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)
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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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations 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 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