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

Phase 2

• Conditions: Hereditary Cerebellar Ataxia.
• Interventions: Other: stem cell transplantation

• Registration Number: NCT01489267
• Lead Sponsor: General Hospital of Chinese Armed Police Forces

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 Timeline

• Study First Submitted: 2011-10-30
• Study Start: 2011-12
• Study First Submitted QC: 2011-12-08
• Study First Posted: 2011-12-09
• Status Verified: 2012-06
• Last Update Submitted: 2012-06-18
• Last Update Posted: 2012-06-20
• Primary Completion: 2013-07
• Study Completion: 2014-07

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 20

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Stem cell transplantation	stem cell transplantation	10 patients in the group accept stem cell transplantation.

Primary Outcome Measures

Name	Time	Method
blood test	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	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

Name	Time	Method
Urinal test	12 months after transplantation	proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
Electrophysiology examination	12 months after transplantation	somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)

Trial Locations

Locations (1)

Yihua An; 🇨🇳 Beijing, China