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

Phase 1

• Conditions: Hereditary Ataxia
• Interventions: Biological: human umbilical cord mesenchymal stem cells

• Registration Number: NCT01360164
• Lead Sponsor: Shenzhen Beike Bio-Technology Co., Ltd.

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.

Study Timeline

• Study Start: 2010-01
• Study First Submitted: 2011-05-23
• Study First Submitted QC: 2011-05-24
• Study First Posted: 2011-05-25
• Status Verified: 2012-11
• Last Update Submitted: 2012-11-26
• Last Update Posted: 2012-11-28
• Primary Completion: 2012-12
• Study Completion: 2013-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 20

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Human umbilical cord mesenchymal stem cells transplantation	human umbilical cord mesenchymal stem cells	Participants will be given umbilical cord mesenchymal stem cells transplantation with a 1 year follow-up.

Primary Outcome Measures

Name	Time	Method
the volume of Cerebellum of Brain Magnetic Resonance Imaging （MRI） Scan	1 year after treatment
scores of International Cooperative Ataxia Rating Scale （ICARS） scale and Berg Scale	1 year after treatment

Secondary Outcome Measures

Name	Time	Method
Number of Participants with Adverse Events as a Measure of Safety and Tolerability	1 year after treatment
Number of Participants with Serious Adverse Events as a Measure of Safety and Tolerability	1 year after treatment

Trial Locations

Locations (1)

Nanjing University Medical College Affiliated Drum Tower Hospital; 🇨🇳 Nanjing, Jiangsu, China