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

Hereditary Cerebellar Ataxia. Clinical Trial

— SCA  

Official title:

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT01489267
• Other study ID #: 20111031SCA
• Secondary ID: 20111031limin
• Status: Enrolling by invitation
• Phase: Phase 2
• First received: October 30, 2011
• Last updated: June 18, 2012
• Start date: December 2011
• Est. completion date: July 2014

Study information

• Verified date: June 2012
• Source: General Hospital of Chinese Armed Police Forces
• Contact: n/a
• Is FDA regulated: No
• Health authority: Chinese:Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

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 .

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 20
• Est. completion date: July 2014
• Est. primary completion date: July 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 65 Years

Eligibility

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

Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Ataxia
• Cerebellar Ataxia
• Hereditary Cerebellar Ataxia.
• Spinocerebellar Degenerations

Intervention

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).

Locations

Country	Name	City	State
China	Yihua An	Beijing

Sponsors (1)

Lead Sponsor	Collaborator
General Hospital of Chinese Armed Police Forces

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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)?a1- microglobulin?ß2- microglobulin;lymphotoxin(LCT). Tumor markers(AFP,CEA,CA125,CA15-3,CA19-9,CA72-4CY21-1,NSE) ,Lymphocytes classification	within one week before transplantation	Yes
Primary	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)	within one week before transplantation	Yes
Primary	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)?a1- microglobulin?ß2- microglobulin;lymphotoxin(LCT). Tumor markers(AFP,CEA,CA125,CA15-3,CA19-9,CA72-4CY21-1,NSE) ,Lymphocytes classification	1 month after transplantation	Yes
Primary	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)?a1- microglobulin?ß2- microglobulin;lymphotoxin(LCT). Tumor markers(AFP,CEA,CA125,CA15-3,CA19-9,CA72-4CY21-1,NSE) ,Lymphocytes classification	3 months after transplantation	Yes
Primary	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)?a1- microglobulin?ß2- microglobulin;lymphotoxin(LCT). Tumor markers(AFP,CEA,CA125,CA15-3,CA19-9,CA72-4CY21-1,NSE) ,Lymphocytes classification	6 months after transplantation	Yes
Primary	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)?a1- microglobulin?ß2- microglobulin;lymphotoxin(LCT). Tumor markers(AFP,CEA,CA125,CA15-3,CA19-9,CA72-4CY21-1,NSE) ,Lymphocytes classification	12 months after transplantation	Yes
Primary	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)	1 month after transplantation	Yes
Primary	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)	3 months after transplantation	Yes
Primary	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)	6 months after transplantation	Yes
Primary	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)	12 months after transplantation	Yes
Secondary	Urinal test	proteinum?akaryocyte?a1- microglobulin?ß2- microglobulin	within one week before transplantation	Yes
Secondary	Electrophysiology examination	somatosensory evoked potential(SEP)?brain stem auditory evoked potential(BAEP)	within one week before transplantation	Yes
Secondary	Urinal test	proteinum?akaryocyte?a1- microglobulin?ß2- microglobulin	1 month after transplantation	Yes
Secondary	Urinal test	proteinum?akaryocyte?a1- microglobulin?ß2- microglobulin	3 months after transplantation	Yes
Secondary	Urinal test	proteinum?akaryocyte?a1- microglobulin?ß2- microglobulin	6 months after transplantation	Yes
Secondary	Urinal test	proteinum?akaryocyte?a1- microglobulin?ß2- microglobulin	12 months after transplantation	Yes
Secondary	Electrophysiology examination	somatosensory evoked potential(SEP)?brain stem auditory evoked potential(BAEP)	1 month after transplantation	Yes
Secondary	Electrophysiology examination	somatosensory evoked potential(SEP)?brain stem auditory evoked potential(BAEP)	3 months after transplantation	Yes
Secondary	Electrophysiology examination	somatosensory evoked potential(SEP)?brain stem auditory evoked potential(BAEP)	6 months after transplantation	Yes
Secondary	Electrophysiology examination	somatosensory evoked potential(SEP)?brain stem auditory evoked potential(BAEP)	12 months after transplantation	Yes