A New Method to Treat Hereditary Cerebellar Ataxia - Umbilical Cord Mesenchymal Stem Cells Transplantation (SCA)
This study is enrolling participants by invitation only.
Sponsor:
General Hospital of Chinese Armed Police Forces
Information provided by (Responsible Party):
General Hospital of Chinese Armed Police Forces
ClinicalTrials.gov Identifier:
NCT01489267
First received: October 30, 2011
Last updated: June 18, 2012
Last verified: June 2012
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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 Endpoint Classification: Efficacy Study Intervention Model: Single Group Assignment Masking: 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:
Genetics Home Reference related topics:
autosomal recessive cerebellar ataxia type 1
infantile-onset spinocerebellar ataxia
Marinesco-Sjögren syndrome
spinocerebellar ataxia type 1
spinocerebellar ataxia type 2
spinocerebellar ataxia type 3
spinocerebellar ataxia type 6
VLDLR-associated cerebellar hypoplasia
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 ] [ Designated as safety issue: Yes ]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 ] [ Designated as safety issue: Yes ]
- 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 [ Time Frame: 1 month after transplantation ] [ Designated as safety issue: Yes ]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 ] [ Designated as safety issue: Yes ]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 ] [ Designated as safety issue: Yes ]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 ] [ Designated as safety issue: Yes ]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 ] [ Designated as safety issue: Yes ]
- 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 [ Time Frame: 3 months after transplantation ] [ Designated as safety issue: Yes ]
- 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 [ Time Frame: 6 months after transplantation ] [ Designated as safety issue: Yes ]
- 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 [ Time Frame: 12 months after transplantation ] [ Designated as safety issue: Yes ]
- 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 [ Time Frame: within one week before transplantation ] [ Designated as safety issue: Yes ]proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
- Electrophysiology examination [ Time Frame: within one week before transplantation ] [ Designated as safety issue: Yes ]somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)
- Urinal test [ Time Frame: 1 month after transplantation ] [ Designated as safety issue: Yes ]proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
- Urinal test [ Time Frame: 3 months after transplantation ] [ Designated as safety issue: Yes ]proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
- Urinal test [ Time Frame: 6 months after transplantation ] [ Designated as safety issue: Yes ]proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
- Urinal test [ Time Frame: 12 months after transplantation ] [ Designated as safety issue: Yes ]proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
- Electrophysiology examination [ Time Frame: 1 month after transplantation ] [ Designated as safety issue: Yes ]somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)
- Electrophysiology examination [ Time Frame: 3 months after transplantation ] [ Designated as safety issue: Yes ]somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)
- Electrophysiology examination [ Time Frame: 6 months after transplantation ] [ Designated as safety issue: Yes ]somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)
- Electrophysiology examination [ Time Frame: 12 months after transplantation ] [ Designated as safety issue: Yes ]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| Ages Eligible for Study: | 18 Years to 65 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: 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
No publications provided
| Responsible Party: | General Hospital of Chinese Armed Police Forces |
| ClinicalTrials.gov Identifier: | NCT01489267 History of Changes |
| Other Study ID Numbers: | 20111031SCA, 20111031limin |
| Study First Received: | October 30, 2011 |
| Last Updated: | June 18, 2012 |
| Health Authority: | Chinese:Food and Drug Administration |
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 |
ClinicalTrials.gov processed this record on May 16, 2013