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

Primary Purpose

Hereditary Ataxia

Status

Unknown status

Phase

Phase 1

Locations

China

Study Type

Interventional

Intervention

human umbilical cord mesenchymal stem cells

About this trial

This is an interventional treatment trial for Hereditary Ataxia focused on measuring Human Umbilical Cord Mesenchymal Stem Cells

Eligibility Criteria

16 Years - 65 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Nanjing University Medical College Affiliated Drum Tower HospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Human umbilical cord mesenchymal stem cells transplantation

Arm Description

Participants will be given umbilical cord mesenchymal stem cells transplantation with a 1 year follow-up.

Outcomes

Primary Outcome Measures

scores of International Cooperative Ataxia Rating Scale （ICARS） scale and Berg Scale

the volume of Cerebellum of Brain Magnetic Resonance Imaging （MRI） Scan

Secondary Outcome Measures

Number of Participants with Adverse Events as a Measure of Safety and Tolerability

Number of Participants with Serious Adverse Events as a Measure of Safety and Tolerability

Full Information

NCT ID

NCT01360164

First Posted

May 23, 2011

Last Updated

November 26, 2012

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 University, The Second Hospital of Nanjing Medical University

1. Study Identification

Unique Protocol Identification Number

NCT01360164

Brief Title

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

Official Title

Phase I/II Study of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Hereditary Ataxia

Study Type

Interventional

2. Study Status

Record Verification Date

November 2012

Overall Recruitment Status

Unknown status

Study Start Date

January 2010 (undefined)

Primary Completion Date

December 2012 (Anticipated)

Study Completion Date

December 2013 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Shenzhen Beike Bio-Technology Co., Ltd.

Collaborators

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Hereditary Ataxia

Keywords

Human Umbilical Cord Mesenchymal Stem Cells

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

20 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Human umbilical cord mesenchymal stem cells transplantation

Arm Type

Experimental

Arm Description

Participants will be given umbilical cord mesenchymal stem cells transplantation with a 1 year follow-up.

Intervention Type

Biological

Intervention Name(s)

human umbilical cord mesenchymal stem cells

Other Intervention Name(s)

Intervention Group

Intervention Description

Participants will be given hUC-MSCs transplantation.

Primary Outcome Measure Information:

Title

scores of International Cooperative Ataxia Rating Scale （ICARS） scale and Berg Scale

Time Frame

1 year after treatment

Title

the volume of Cerebellum of Brain Magnetic Resonance Imaging （MRI） Scan

Time Frame

1 year after treatment

Secondary Outcome Measure Information:

Title

Number of Participants with Adverse Events as a Measure of Safety and Tolerability

Time Frame

1 year after treatment

Title

Number of Participants with Serious Adverse Events as a Measure of Safety and Tolerability

Time Frame

1 year after treatment

10. Eligibility

Sex

All

Minimum Age & Unit of Time

16 Years

Maximum Age & Unit of Time

65 Years

Accepts Healthy Volunteers

Eligibility 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.

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Yun Xu

xuyun20042001@yahoo.com.cn

Facility Information:

Facility Name

Nanjing University Medical College Affiliated Drum Tower Hospital

City

Nanjing

State/Province

Jiangsu

Country

China

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Yun Xu

xuyun20042001@yahoo.com.cn

12. IPD Sharing Statement

Citations:

PubMed Identifier

23151076

Citation

Jin JL, Liu Z, Lu ZJ, Guan DN, Wang C, Chen ZB, Zhang J, Zhang WY, Wu JY, Xu Y. Safety and efficacy of umbilical cord mesenchymal stem cell therapy in hereditary spinocerebellar ataxia. Curr Neurovasc Res. 2013 Feb;10(1):11-20. doi: 10.2174/156720213804805936.

Results Reference

derived

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

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