Umbilical Cord Mesenchymal Stem Cells Therapy (19#iSCLife®-SA) for Patients With Spinocerebellar Ataxia

Study Overview
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Brief Summary
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The purpose of this study is verify the safety and efficacy of Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC) therapy for patients with Spinocerebellar Ataxia, and in addition, explore the possible mechanisms of UC-MSC therapy in Spinocerebellar Ataxia.

Detailed Description
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This is a random, open label, and parallel controled experiment. 45 patients are selected and sign consent forms, then divided into three groups. Doctors collect the basic information of patient (including age,height, mental condition, vital sign, history of disease, pharmaco-history, genotype test, and so on.) All patients receive laboratory and image examination as baseline. Then, they will give cell treatment based on the clinical protocol. Doctors have follow-up visit on 1, 2, 3, 6, and 12 months after treatment, and do efficacy evaluation.

Official Title
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A Clinical Research on the Safety/Efficacy of Umbilical Cord Mesenchymal Stem Cells Therapy for Patients With Spinocerebellar Ataxia

Conditions
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Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3, Spinocerebellar Ataxia Type 6

Intervention / Treatment
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* Procedure: Intravenous infusion
* Procedure: Intrathecal injection
* Biological: umbilical cord mesenchymal stem cell


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Clinical and genomic test diagnoses as spinocerebellar ataxias (SCA) (include SCA 1, SCA 2, SCA 3, SCA 6), SARA (Scale for Assessment and Rating of Ataxia) score is 2-5, can complete 8-meter walking test（8MW） Do not receive stem cells treatment in 6 months Participants sign the consent form based on the experiment process and statement Exclusion Criteria: Cardiac, renal, hepatic insufficiency; total bilirubin is 1.5 times higher than normal value, aspartate transaminase（AST）or alanine aminotransferase（ALT）is 2.5 times higher than normal value Hemogram: total white blood cells <3.0 * 10^9 cells/L, blood platelet <75 * 10^9/L, hemoglobin <100g/L pneumonia, or severe infection With severe allergic history Brain organic disorder, like brain tumor Serum with HIV, syphilis antibody positive Severe mental disease, cognitive disorder patients Other severe system or organ organic disease Pregnant, breast feeding, or planning pregnant women Participate other clinical experiments in 3 months With some other conditions that doctor propose not to participate

Ages Eligible for Study
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Minimum Age: 16 Years
Maximum Age: 60 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: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Intravenous infusion group<br>Umbilical cord mesenchymal stem cells (SCLnow 19#) | Procedure: Intravenous infusion<br>* Intravenous infusion of mesenchymal stem cells: 2 * 10^7 cells (30ml)<br>Biological: umbilical cord mesenchymal stem cell<br>* Treat patients with umbilical cord mesenchymal stem cell (SCLnow 19#), the dosage based on different procedure<br>|
| Experimental: Intrathecal injection group<br>Umbilical cord mesenchymal stem cells (SCLnow 19#) | Procedure: Intrathecal injection<br>* Intrathecal injection of mesenchymal stem cells: 2 * 10^7 cells (1ml)<br>Biological: umbilical cord mesenchymal stem cell<br>* Treat patients with umbilical cord mesenchymal stem cell (SCLnow 19#), the dosage based on different procedure<br>|
| No Intervention: Control groups<br>No intervention | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Scale for the assessment anf rating of ataxia (SARA) | Based on SARA score to calculate treatment efficacy. The equation is as follow, Efficacy = (patients who accepted treatment are effective/patients who accepted treatment)* 100% Effective: after 12 months, score of patient decrease 1 point or more; Ineffective: after 12 months, score decrease less than 1 point or increase. | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Image examinations | MRI plain scan of brain. | 12 months | 
| Inventory of Non-Ataxia Symptoms (INAS) score | Using Inventory of Non-Ataxia Symptoms (INAS) score to determine the presence and severity of non-ataxia signs. | 12 months | 
| Cerebrospinal fluid (csf) routine | Patients is observed by professionals, compare the changes of each observation point and baseline. Baseline is the data acquire from patients before stem cells treatment. | 12 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Spinocerebellar Ataxia,Mesenchymal Stem Cells