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

Phase 2

Not yet recruiting

• Conditions: Spinocerebellar Ataxia Type 3; Spinocerebellar Ataxia Type 2; Spinocerebellar Ataxia Type 1; Spinocerebellar Ataxia Type 6
• Interventions: Procedure: Intravenous infusion; Procedure: Intrathecal injection; Biological: umbilical cord mesenchymal stem cell

• Registration Number: NCT03378414
• Lead Sponsor: Sclnow Biotechnology Co., Ltd.

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.

Study Timeline

• Study First Submitted: 2017-12-12
• Study First Submitted QC: 2017-12-18
• Study First Posted: 2017-12-19
• Status Verified: 2024-04
• Last Update Submitted: 2025-04-02
• Last Update Posted: 2025-04-04
• Study Start: 2025-06-30
• Primary Completion: 2025-12-31
• Study Completion: 2026-12-31

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 45

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Intravenous infusion group	Intravenous infusion	Umbilical cord mesenchymal stem cells (SCLnow 19#)
Intravenous infusion group	umbilical cord mesenchymal stem cell	Umbilical cord mesenchymal stem cells (SCLnow 19#)
Intrathecal injection group	umbilical cord mesenchymal stem cell	Umbilical cord mesenchymal stem cells (SCLnow 19#)
Intrathecal injection group	Intrathecal injection	Umbilical cord mesenchymal stem cells (SCLnow 19#)

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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.