Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Hereditary Ataxia
The recruitment status of this study is unknown because the information has not been verified recently.
Verified November 2012 by Shenzhen Beike Bio-Technology Co., Ltd..
Recruitment status was Recruiting
Recruitment status was Recruiting
Sponsor:
Shenzhen Beike Bio-Technology Co., Ltd.
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 College
The Second Hospital of Nanjing Medical University
Information provided by (Responsible Party):
Shenzhen Beike Bio-Technology Co., Ltd.
ClinicalTrials.gov Identifier:
NCT01360164
First received: May 23, 2011
Last updated: November 26, 2012
Last verified: November 2012
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Purpose
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 | Intervention | Phase |
|---|---|---|
|
Hereditary Ataxia |
Biological: human umbilical cord mesenchymal stem cells |
Phase 1 Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Phase I/II Study of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Hereditary Ataxia |
Resource links provided by NLM:
Genetics Home Reference related topics:
infantile-onset spinocerebellar ataxia
spinocerebellar ataxia type 1
spinocerebellar ataxia type 2
spinocerebellar ataxia type 3
spinocerebellar ataxia type 6
U.S. FDA Resources
Further study details as provided by Shenzhen Beike Bio-Technology Co., Ltd.:
Primary Outcome Measures:
- scores of International Cooperative Ataxia Rating Scale （ICARS） scale and Berg Scale [ Time Frame: 1 year after treatment ] [ Designated as safety issue: No ]
- the volume of Cerebellum of Brain Magnetic Resonance Imaging （MRI） Scan [ Time Frame: 1 year after treatment ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- Number of Participants with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 1 year after treatment ] [ Designated as safety issue: Yes ]
- Number of Participants with Serious Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 1 year after treatment ] [ Designated as safety issue: Yes ]
| Estimated Enrollment: | 20 |
| Study Start Date: | January 2010 |
| Estimated Study Completion Date: | December 2013 |
| Estimated Primary Completion Date: | December 2012 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
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
|
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.
Eligibility| Ages Eligible for Study: | 16 Years to 65 Years |
| Genders Eligible for Study: | Both |
| 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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01360164
Please refer to this study by its ClinicalTrials.gov identifier: 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 College
The Second Hospital of Nanjing Medical University
More Information
No publications provided by Shenzhen Beike Bio-Technology Co., Ltd.
Additional 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) |
| Study First Received: | May 23, 2011 |
| Last Updated: | November 26, 2012 |
| Health Authority: | China: Ministry of Health |
Keywords provided by Shenzhen Beike Bio-Technology Co., Ltd.:
|
Human Umbilical Cord Mesenchymal Stem Cells |
Additional relevant MeSH terms:
|
Spinocerebellar Degenerations Brain Diseases Central Nervous System Diseases Cerebellar Diseases Genetic Diseases, Inborn |
Heredodegenerative Disorders, Nervous System Nervous System Diseases Neurodegenerative Diseases Spinal Cord Diseases |
ClinicalTrials.gov processed this record on March 03, 2015