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

Study Overview
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Brief Summary
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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
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This Study is designed to evaluate the the safety and efficacy of Human Umbilical Cord Mesenchymal Stem Cells transplantation in patients with Hereditary Ataxias.

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

Conditions
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Hereditary Ataxia

Intervention / Treatment
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* Biological: human umbilical cord mesenchymal stem cells


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 16 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

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


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Human umbilical cord mesenchymal stem cells transplantation<br>Participants will be given umbilical cord mesenchymal stem cells transplantation with a 1 year follow-up. | Biological: human umbilical cord mesenchymal stem cells<br>* Participants will be given hUC-MSCs transplantation.<br>* Other names: Intervention Group;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | 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 | Measure Description | 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Human Umbilical Cord Mesenchymal Stem Cells