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

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

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.

Study Overview

• Status: Unknown
• Conditions: Hereditary Ataxia
• Intervention / Treatment: Biological: human umbilical cord mesenchymal stem cells

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 Type: Interventional
• Enrollment (Anticipated): 20
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• China
  • Jiangsu
    • Nanjing, Jiangsu, China
      • Recruiting
      • Nanjing University Medical College Affiliated Drum Tower Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 65 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / 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 Names: Intervention Group

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure	Time Frame
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

Collaborators and Investigators

• Sponsor: Shenzhen Beike Bio-Technology Co., Ltd.
• Collaborators: The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School; Xuzhou Medical University; The Second Hospital of Nanjing Medical University; The Affiliated Nanjing Brain Hospital of Nanjing University Medical School; Nanjing University Medical College Affiliated Wuxi Second Hospital

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start: January 1, 2010
• Primary Completion (Anticipated): December 1, 2012
• Study Completion (Anticipated): December 1, 2013

Study Registration Dates

• First Submitted: May 23, 2011
• First Submitted That Met QC Criteria: May 24, 2011
• First Posted (Estimate): May 25, 2011

Study Record Updates

• Last Update Posted (Estimate): November 28, 2012
• Last Update Submitted That Met QC Criteria: November 26, 2012
• Last Verified: November 1, 2012

More Information

Terms related to this study

• Keywords: Human Umbilical Cord Mesenchymal Stem Cells
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Genetic Diseases, Inborn; Neurodegenerative Diseases; Dyskinesias; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Cerebellar Diseases; Ataxia; Cerebellar Ataxia; Spinocerebellar Degenerations
• Other Study ID Numbers: BKCR-HA-1.0(2010)