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

NCT ID: NCT03378414

Last Updated: 2025-12-04

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 45 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-12-31
• Study Completion Date: 2026-12-31

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.

Conditions

Spinocerebellar Ataxia Type 1; Spinocerebellar Ataxia Type 2; Spinocerebellar Ataxia Type 3; Spinocerebellar Ataxia Type 6

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Intravenous infusion group

Umbilical cord mesenchymal stem cells (SCLnow 19#)

Group Type: EXPERIMENTAL

Intravenous infusion

Intervention Type: PROCEDURE

Intravenous infusion of mesenchymal stem cells: 2 \* 10\^7 cells (30ml)

umbilical cord mesenchymal stem cell

Intervention Type: BIOLOGICAL

Treat patients with umbilical cord mesenchymal stem cell (SCLnow 19#), the dosage based on different procedure

Intrathecal injection group

Umbilical cord mesenchymal stem cells (SCLnow 19#)

Group Type: EXPERIMENTAL

Intrathecal injection

Intervention Type: PROCEDURE

Intrathecal injection of mesenchymal stem cells: 2 \* 10\^7 cells (1ml)

umbilical cord mesenchymal stem cell

Intervention Type: BIOLOGICAL

Treat patients with umbilical cord mesenchymal stem cell (SCLnow 19#), the dosage based on different procedure

Control groups

No intervention

Group Type: NO_INTERVENTION

No interventions assigned to this group

Interventions

Intravenous infusion

Intravenous infusion of mesenchymal stem cells: 2 \* 10\^7 cells (30ml)

Intervention Type: PROCEDURE

Intrathecal injection

Intrathecal injection of mesenchymal stem cells: 2 \* 10\^7 cells (1ml)

Intervention Type: PROCEDURE

umbilical cord mesenchymal stem cell

Treat patients with umbilical cord mesenchymal stem cell (SCLnow 19#), the dosage based on different procedure

Intervention Type: BIOLOGICAL

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
• With some other conditions that doctor propose not to participate

• Minimum Eligible Age: 16 Years
• Maximum Eligible Age: 60 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Sclnow Biotechnology Co., Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Hong Jiang

Role: PRINCIPAL_INVESTIGATOR

Xiangya Hospital of Central South University

Central Contacts

Lei Guo

Role: CONTACT

georgeguo@sclnow.com

861064368977

Other Identifiers

SCLnow-XY-03

Identifier Type: -

Identifier Source: org_study_id