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

This study is currently recruiting participants.

Verified October 2011 by Shenzhen Beike Bio-Technology Co., Ltd.

First Received on May 23, 2011. Last Updated on October 13, 2011 History of Changes

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 Affiliated Hospital of 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

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: Friedreich ataxia infantile-onset spinocerebellar ataxia spinocerebellar ataxia type 1 spinocerebellar ataxia type 2 spinocerebellar ataxia type 3 spinocerebellar ataxia type 6 Help Me Understand Genetics

MedlinePlus related topics: MRI Scans

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 2012
Estimated Primary Completion Date:	December 2011 (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

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

Affiliated Hospital of Xuzhou Medical College

The Second Hospital of Nanjing Medical University

More Information

No publications provided

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:	October 13, 2011
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:

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

ClinicalTrials.gov processed this record on May 23, 2012