Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3, Spinocerebellar Ataxia Type 6
Conditions
Keywords
Spinocerebellar Ataxia,Mesenchymal Stem Cells
Brief summary
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
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.
Interventions
PROCEDUREIntravenous infusion
Intravenous infusion of mesenchymal stem cells: 2 \* 10\^7 cells (30ml)
PROCEDUREIntrathecal injection
Intrathecal injection of mesenchymal stem cells: 2 \* 10\^7 cells (1ml)
BIOLOGICALumbilical cord mesenchymal stem cell
Treat patients with umbilical cord mesenchymal stem cell (SCLnow 19#), the dosage based on different procedure
Sponsors
Sclnow Biotechnology Co., Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
16 Years to 60 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Scale for the assessment anf rating of ataxia (SARA) | 12 months | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Image examinations | 12 months | MRI plain scan of brain. |
| Inventory of Non-Ataxia Symptoms (INAS) score | 12 months | Using Inventory of Non-Ataxia Symptoms (INAS) score to determine the presence and severity of non-ataxia signs. |
| Cerebrospinal fluid (csf) routine | 12 months | 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. |
Contacts
Primary ContactLei Guo
Outcome results
None listed