NCT01489267

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.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
20

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Dec 2011

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

October 30, 2011

Completed
1 month until next milestone

Study Start

First participant enrolled

December 1, 2011

Completed
8 days until next milestone

First Posted

Study publicly available on registry

December 9, 2011

Completed
1.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2013

Completed
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2014

Completed
Last Updated

June 20, 2012

Status Verified

June 1, 2012

Enrollment Period

1.6 years

First QC Date

October 30, 2011

Last Update Submit

June 18, 2012

Conditions

Hereditary Cerebellar Ataxia.

Keywords

cerebellar ataxiahereditary diseasestem cell

Outcome Measures

Primary Outcomes (10)

  • 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

    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)

    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

    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)

    1 month after transplantation

  • Nerve functional evaluation

    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)

    3 months after transplantation

  • Nerve functional evaluation

    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)

    6 months after transplantation

  • Nerve functional evaluation

    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)

    12 months after transplantation

Secondary Outcomes (10)

  • Urinal test

    within one week before transplantation

  • Electrophysiology examination

    within one week before transplantation

  • Urinal test

    1 month after transplantation

  • Urinal test

    3 months after transplantation

  • Urinal test

    6 months after transplantation

  • +5 more secondary outcomes

Study Arms (2)

Control group

NO INTERVENTION

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.

Stem cell transplantation

EXPERIMENTAL

10 patients in the group accept stem cell transplantation.

Other: stem cell transplantation

Interventions

stem cell transplantationOTHER

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).

Also known as: Cell therapy.
Stem cell transplantation

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • years of age
  • Molecularly diagnosed SCA1

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Yihua An

Beijing, China

Location

MeSH Terms

Conditions

Spinocerebellar DegenerationsCerebellar AtaxiaGenetic Diseases, Inborn

Interventions

Stem Cell TransplantationCell- and Tissue-Based Therapy

Condition Hierarchy (Ancestors)

Cerebellar DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesSpinal Cord DiseasesHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesAtaxiaDyskinesiasNeurologic ManifestationsSigns and SymptomsPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Cell TransplantationBiological TherapyTherapeuticsTransplantationSurgical Procedures, Operative

Study Officials

  • Yihua An, doctor

    director of neural stem cell transplantation department in general hospital of chinese armed police forces

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 30, 2011

First Posted

December 9, 2011

Study Start

December 1, 2011

Primary Completion

July 1, 2013

Study Completion

July 1, 2014

Last Updated

June 20, 2012

Record last verified: 2012-06

Locations

CN(1)