Hereditary Cerebellar Ataxia.
Conditions
Keywords
cerebellar ataxia, hereditary disease, stem cell
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 .
Interventions
OTHERstem 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).
Sponsors
General Hospital of Chinese Armed Police Forces
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| blood test | 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 | 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) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Urinal test | within one week before transplantation | proteinum、akaryocyte、α1- microglobulin、β2- microglobulin |
| Electrophysiology examination | within one week before transplantation | somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP) |
Countries
China
Outcome results
None listed