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Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Hereditary Ataxia

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ClinicalTrials.gov Identifier: NCT01360164
Recruitment Status : Unknown
Verified November 2012 by Shenzhen Beike Bio-Technology Co., Ltd..
Recruitment status was:  Recruiting
First Posted : May 25, 2011
Last Update Posted : November 28, 2012
Sponsor:
Collaborators:
The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School
The Affiliated Nanjing Brain Hospital of Nanjing University Medical School
Nanjing University Medical College Affiliated Wuxi Second Hospital
Xuzhou Medical University
The Second Hospital of Nanjing Medical University
Information provided by (Responsible Party):
Shenzhen Beike Bio-Technology Co., Ltd.

Study Description
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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Brief Summary:
The Hereditary Ataxias are a group of genetic disorders characterized by slowly progressive incoordination of gait and often associated with poor coordination of hands, speech, and eye movements. Current treatments for Hereditary Ataxias are mainly pharmacological, rehabilitative, or psychological treatments，while no effective treatment available. Stem Cell therapy is a novel and promising therapeutic strategy for Hereditary Ataxias treatment. In this study, the safety and efficacy of Human Umbilical Cord Mesenchymal Stem Cells transplantation will be evaluated in patients with Hereditary Ataxias.

Condition or disease Intervention/treatment Phase
Hereditary Ataxia Biological: human umbilical cord mesenchymal stem cells Phase 1 Phase 2

Detailed Description:
This Study is designed to evaluate the the safety and efficacy of Human Umbilical Cord Mesenchymal Stem Cells transplantation in patients with Hereditary Ataxias.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase I/II Study of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Hereditary Ataxia
Study Start Date : January 2010
Estimated Primary Completion Date : December 2012
Estimated Study Completion Date : December 2013

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arms and Interventions
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Arm Intervention/treatment
Experimental: Human umbilical cord mesenchymal stem cells transplantation
Participants will be given umbilical cord mesenchymal stem cells transplantation with a 1 year follow-up.
 Biological: human umbilical cord mesenchymal stem cells
Participants will be given hUC-MSCs transplantation.
Other Name: Intervention Group


Outcome Measures
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Primary Outcome Measures :
  1. scores of International Cooperative Ataxia Rating Scale （ICARS） scale and Berg Scale [ Time Frame: 1 year after treatment ]
  2. the volume of Cerebellum of Brain Magnetic Resonance Imaging （MRI） Scan [ Time Frame: 1 year after treatment ]

Secondary Outcome Measures :
  1. Number of Participants with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 1 year after treatment ]
  2. Number of Participants with Serious Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 1 year after treatment ]

Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   16 Years to 65 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Aged 16-65 years.
  • Harding Diagnosis of SCAs, gene type confirmed.
  • Candidates who did not receive any stem cell therapy in past 6months.
  • sign the consent form and follow the clinic trail procedure.

Exclusion Criteria:

  • Cardiac insufficiency; Renal insufficiency; hepatic insufficiency; Total bilirubin higher than 1.5 times of upper limit of normal value; AST /ALT higher than 2.5 times of upper limit of normal value；
  • Routine Blood Test: WBC count <3.0×109/ L; PLT count <5×109/L ; or Hemoglobin <100g/L；
  • Combined Pneumonia or other Severe systemic bacteria infection；
  • Severe drug allergic history or anaphylaxis to 2 or more food or medicine；
  • Other brain organic disease (eg. Brain cancer)；
  • HIV+, Tumor Markers + ；
  • Severe psychotic patients, cognitive dysfunction， or can not understand or sign the Consent Form；
  • Other severe systemic or organic disease；
  • Uncontrolled hypertension，blood pressure≥180mmHg/110 mmHg after treatment；
  • Pregnancy；
  • Enrollment in other trials in the last 3 months；
  • Other criteria the investigator consider improper for inclusion.
Contacts and Locations
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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01360164


Contacts
Contact: Yun Xu xuyun20042001@yahoo.com.cn

Locations
China, Jiangsu
Nanjing University Medical College Affiliated Drum Tower Hospital Recruiting
Nanjing, Jiangsu, China
Contact: Yun Xu       xuyun20042001@yahoo.com.cn   
Sponsors and Collaborators
Shenzhen Beike Bio-Technology Co., Ltd.
The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School
The Affiliated Nanjing Brain Hospital of Nanjing University Medical School
Nanjing University Medical College Affiliated Wuxi Second Hospital
Xuzhou Medical University
The Second Hospital of Nanjing Medical University
More Information
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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Shenzhen Beike Bio-Technology Co., Ltd.
ClinicalTrials.gov Identifier: NCT01360164     History of Changes
Other Study ID Numbers: BKCR-HA-1.0(2010)
First Posted: May 25, 2011    Key Record Dates
Last Update Posted: November 28, 2012
Last Verified: November 2012

Keywords provided by Shenzhen Beike Bio-Technology Co., Ltd.:
Human Umbilical Cord Mesenchymal Stem Cells

Additional relevant MeSH terms:
Ataxia
Cerebellar Ataxia
Spinocerebellar Degenerations
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
 Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn