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

The Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cells Transplantation in Hereditary Cerebellar Ataxia Patients

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.

Study Overview

• Status: Unknown
• Conditions: Hereditary Cerebellar Ataxia.
• Intervention / Treatment: Other: stem cell transplantation

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 Type: Interventional
• Enrollment (Anticipated): 20
• Phase: Phase 2

Contacts and Locations

Study Locations

• China
  • Beijing, China
    • Yihua An

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / 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 Names: Cell therapy.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	within one week before transplantation
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 ratingPart B -Handwriting and drawingsPart 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)	within one week before transplantation
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	1 month after transplantation
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	3 months after transplantation
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	6 months after transplantation
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	12 months after transplantation
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 ratingPart B -Handwriting and drawingsPart 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)	1 month after transplantation
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 ratingPart B -Handwriting and drawingsPart 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)	3 months after transplantation
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 ratingPart B -Handwriting and drawingsPart 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)	6 months after transplantation
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 ratingPart B -Handwriting and drawingsPart 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)	12 months after transplantation

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Urinal test	proteinum、akaryocyte、α1- microglobulin、β2- microglobulin	within one week before transplantation
Electrophysiology examination	somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)	within one week before transplantation
Urinal test	proteinum、akaryocyte、α1- microglobulin、β2- microglobulin	1 month after transplantation
Urinal test	proteinum、akaryocyte、α1- microglobulin、β2- microglobulin	3 months after transplantation
Urinal test	proteinum、akaryocyte、α1- microglobulin、β2- microglobulin	6 months after transplantation
Urinal test	proteinum、akaryocyte、α1- microglobulin、β2- microglobulin	12 months after transplantation
Electrophysiology examination	somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)	1 month after transplantation
Electrophysiology examination	somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)	3 months after transplantation
Electrophysiology examination	somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)	6 months after transplantation
Electrophysiology examination	somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)	12 months after transplantation

Collaborators and Investigators

• Sponsor: 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

Study record dates

Study Major Dates

• Study Start: December 1, 2011
• Primary Completion (Anticipated): July 1, 2013
• Study Completion (Anticipated): July 1, 2014

Study Registration Dates

• First Submitted: October 30, 2011
• First Submitted That Met QC Criteria: December 8, 2011
• First Posted (Estimate): December 9, 2011

Study Record Updates

• Last Update Posted (Estimate): June 20, 2012
• Last Update Submitted That Met QC Criteria: June 18, 2012
• Last Verified: June 1, 2012

More Information

Terms related to this study

• Keywords: stem cell; cerebellar ataxia; hereditary disease
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Genetic Diseases, Inborn; Neurodegenerative Diseases; Dyskinesias; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Cerebellar Diseases; Ataxia; Cerebellar Ataxia; Spinocerebellar Degenerations
• Other Study ID Numbers: 20111031SCA; 20111031limin (Other Identifier: neuralstemcell)