search
Back to results

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

Primary Purpose

Hereditary Cerebellar Ataxia.

Status
Unknown status
Phase
Phase 2
Locations
China
Study Type
Interventional
Intervention
stem cell transplantation
Sponsored by
General Hospital of Chinese Armed Police Forces
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hereditary Cerebellar Ataxia. focused on measuring cerebellar ataxia, hereditary disease, stem cell

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Yihua An

Arms of the Study

Arm 1

Arm 2

Arm Type

No Intervention

Experimental

Arm Label

Control group

Stem cell transplantation

Arm Description

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.

10 patients in the group accept stem cell transplantation.

Outcomes

Primary Outcome Measures

blood test
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
Timed-up-and-go test ,5 minutes down-and-up test ICARS score Modified Falls Efficacy Scale Berg balance assessment Tremor Rating Scale: Part A - Tremor location/severity rating Part B -Handwriting and drawings Part C - Functional disabilities resulting from tremor Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
blood test
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
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
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
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
Timed-up-and-go test ,5 minutes down-and-up test ICARS score Modified Falls Efficacy Scale Berg balance assessment Tremor Rating Scale: Part A - Tremor location/severity rating Part B -Handwriting and drawings Part C - Functional disabilities resulting from tremor Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
Nerve functional evaluation
Timed-up-and-go test ,5 minutes down-and-up test ICARS score Modified Falls Efficacy Scale Berg balance assessment Tremor Rating Scale: Part A - Tremor location/severity rating Part B -Handwriting and drawings Part C - Functional disabilities resulting from tremor Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
Nerve functional evaluation
Timed-up-and-go test ,5 minutes down-and-up test ICARS score Modified Falls Efficacy Scale Berg balance assessment Tremor Rating Scale: Part A - Tremor location/severity rating Part B -Handwriting and drawings Part C - Functional disabilities resulting from tremor Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
Nerve functional evaluation
Timed-up-and-go test ,5 minutes down-and-up test ICARS score Modified Falls Efficacy Scale Berg balance assessment Tremor Rating Scale: Part A - Tremor location/severity rating Part B -Handwriting and drawings Part C - Functional disabilities resulting from tremor 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
proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
Electrophysiology examination
somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)
Urinal test
proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
Urinal test
proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
Urinal test
proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
Urinal test
proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
Electrophysiology examination
somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)
Electrophysiology examination
somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)
Electrophysiology examination
somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)
Electrophysiology examination
somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)

Full Information

First Posted
October 30, 2011
Last Updated
June 18, 2012
Sponsor
General Hospital of Chinese Armed Police Forces
search

1. Study Identification

Unique Protocol Identification Number
NCT01489267
Brief Title
A New Method to Treat Hereditary Cerebellar Ataxia - Umbilical Cord Mesenchymal Stem Cells Transplantation
Acronym
SCA
Official Title
The Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cells Transplantation in Hereditary Cerebellar Ataxia Patients
Study Type
Interventional

2. Study Status

Record Verification Date
June 2012
Overall Recruitment Status
Unknown status
Study Start Date
December 2011 (undefined)
Primary Completion Date
July 2013 (Anticipated)
Study Completion Date
July 2014 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
General Hospital of Chinese Armed Police Forces

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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 .

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hereditary Cerebellar Ataxia.
Keywords
cerebellar ataxia, hereditary disease, stem cell

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
20 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Control group
Arm Type
No Intervention
Arm Description
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.
Arm Title
Stem cell transplantation
Arm Type
Experimental
Arm Description
10 patients in the group accept stem cell transplantation.
Intervention Type
Other
Intervention Name(s)
stem cell transplantation
Other Intervention Name(s)
Cell therapy.
Intervention Description
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).
Primary Outcome Measure Information:
Title
blood test
Description
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
Time Frame
within one week before transplantation
Title
Nerve functional evaluation
Description
Timed-up-and-go test ,5 minutes down-and-up test ICARS score Modified Falls Efficacy Scale Berg balance assessment Tremor Rating Scale: Part A - Tremor location/severity rating Part B -Handwriting and drawings Part C - Functional disabilities resulting from tremor Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
Time Frame
within one week before transplantation
Title
blood test
Description
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
Time Frame
1 month after transplantation
Title
blood test
Description
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
Time Frame
3 months after transplantation
Title
blood test
Description
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
Time Frame
6 months after transplantation
Title
blood test
Description
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
Time Frame
12 months after transplantation
Title
Nerve functional evaluation
Description
Timed-up-and-go test ,5 minutes down-and-up test ICARS score Modified Falls Efficacy Scale Berg balance assessment Tremor Rating Scale: Part A - Tremor location/severity rating Part B -Handwriting and drawings Part C - Functional disabilities resulting from tremor Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
Time Frame
1 month after transplantation
Title
Nerve functional evaluation
Description
Timed-up-and-go test ,5 minutes down-and-up test ICARS score Modified Falls Efficacy Scale Berg balance assessment Tremor Rating Scale: Part A - Tremor location/severity rating Part B -Handwriting and drawings Part C - Functional disabilities resulting from tremor Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
Time Frame
3 months after transplantation
Title
Nerve functional evaluation
Description
Timed-up-and-go test ,5 minutes down-and-up test ICARS score Modified Falls Efficacy Scale Berg balance assessment Tremor Rating Scale: Part A - Tremor location/severity rating Part B -Handwriting and drawings Part C - Functional disabilities resulting from tremor Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
Time Frame
6 months after transplantation
Title
Nerve functional evaluation
Description
Timed-up-and-go test ,5 minutes down-and-up test ICARS score Modified Falls Efficacy Scale Berg balance assessment Tremor Rating Scale: Part A - Tremor location/severity rating Part B -Handwriting and drawings Part C - Functional disabilities resulting from tremor Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
Time Frame
12 months after transplantation
Secondary Outcome Measure Information:
Title
Urinal test
Description
proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
Time Frame
within one week before transplantation
Title
Electrophysiology examination
Description
somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)
Time Frame
within one week before transplantation
Title
Urinal test
Description
proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
Time Frame
1 month after transplantation
Title
Urinal test
Description
proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
Time Frame
3 months after transplantation
Title
Urinal test
Description
proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
Time Frame
6 months after transplantation
Title
Urinal test
Description
proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
Time Frame
12 months after transplantation
Title
Electrophysiology examination
Description
somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)
Time Frame
1 month after transplantation
Title
Electrophysiology examination
Description
somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)
Time Frame
3 months after transplantation
Title
Electrophysiology examination
Description
somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)
Time Frame
6 months after transplantation
Title
Electrophysiology examination
Description
somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)
Time Frame
12 months after transplantation

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 18-65 years of age Molecularly diagnosed SCA1 Exclusion Criteria: Cognitively impaired individuals Schizophrenics 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) Age less than 18 years, age greater than 65 years
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Yihua An, doctor
Organizational Affiliation
director of neural stem cell transplantation department in general hospital of chinese armed police forces
Official's Role
Study Director
Facility Information:
Facility Name
Yihua An
City
Beijing
Country
China

12. IPD Sharing Statement

Learn more about this trial

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

We'll reach out to this number within 24 hrs