A Clinical Research on the Safety/Efficacy of Umbilical Cord Mesenchymal Stem Cells Therapy for Patients With Spinocerebellar Ataxia
Overview
- Phase
- Phase 2
- Intervention
- Control groups
- Conditions
- Spinocerebellar Ataxia Type 1
- Sponsor
- Sclnow Biotechnology Co., Ltd.
- Enrollment
- 45
- Primary Endpoint
- Scale for the assessment anf rating of ataxia (SARA)
- Status
- Not yet recruiting
- Last Updated
- 9 days ago
Overview
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.
Investigators
Eligibility Criteria
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
Arms & Interventions
Control groups
No intervention
Intravenous infusion group
Umbilical cord mesenchymal stem cells (SCLnow 19#)
Intervention: umbilical cord mesenchymal stem cell
Intrathecal injection group
Umbilical cord mesenchymal stem cells (SCLnow 19#)
Intervention: umbilical cord mesenchymal stem cell
Intravenous infusion group
Umbilical cord mesenchymal stem cells (SCLnow 19#)
Intervention: Intravenous infusion
Intrathecal injection group
Umbilical cord mesenchymal stem cells (SCLnow 19#)
Intervention: Intrathecal injection
Outcomes
Primary Outcomes
Scale for the assessment anf rating of ataxia (SARA)
Time Frame: 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 Outcomes
- Image examinations(12 months)
- Inventory of Non-Ataxia Symptoms (INAS) score(12 months)
- Cerebrospinal fluid (csf) routine(12 months)