NCT03378414

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.

Trial Health

65
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
45

participants targeted

Target at P25-P50 for phase_2

Timeline
12mo left

Started Dec 2026

Shorter than P25 for phase_2

Status
not yet recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

December 12, 2017

Completed
7 days until next milestone

First Posted

Study publicly available on registry

December 19, 2017

Completed
9 years until next milestone

Study Start

First participant enrolled

December 31, 2026

Expected
6 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 30, 2027

6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 31, 2027

Last Updated

April 23, 2026

Status Verified

December 1, 2025

Enrollment Period

6 months

First QC Date

December 12, 2017

Last Update Submit

April 22, 2026

Conditions

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

Keywords

Spinocerebellar Ataxia,Mesenchymal Stem Cells

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (3)

  • Image examinations

    12 months

  • Inventory of Non-Ataxia Symptoms (INAS) score

    12 months

  • Cerebrospinal fluid (csf) routine

    12 months

Study Arms (3)

Intravenous infusion group

EXPERIMENTAL

Umbilical cord mesenchymal stem cells (SCLnow 19#)

Procedure: Intravenous infusionBiological: umbilical cord mesenchymal stem cell

Intrathecal injection group

EXPERIMENTAL

Umbilical cord mesenchymal stem cells (SCLnow 19#)

Procedure: Intrathecal injectionBiological: umbilical cord mesenchymal stem cell

Control groups

NO INTERVENTION

No intervention

Interventions

Intravenous infusionPROCEDURE

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

Intravenous infusion group
Intrathecal injectionPROCEDURE

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

Intrathecal injection group
umbilical cord mesenchymal stem cellBIOLOGICAL

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

Intrathecal injection groupIntravenous infusion group

Eligibility Criteria

Age16 Years - 60 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Spinocerebellar AtaxiasMachado-Joseph Disease

Interventions

Infusions, IntravenousInjections, Spinal

Condition Hierarchy (Ancestors)

Cerebellar AtaxiaCerebellar DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesSpinocerebellar DegenerationsSpinal Cord DiseasesHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesAtaxiaDyskinesiasNeurologic ManifestationsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Administration, IntravenousDrug Administration RoutesDrug TherapyTherapeuticsInfusions, ParenteralInjections

Study Officials

  • Hong Jiang

    Xiangya Hospital of Central South University

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Lei Guo

CONTACT

861064368977georgeguo@sclnow.com

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 12, 2017

First Posted

December 19, 2017

Study Start (Estimated)

December 31, 2026

Primary Completion (Estimated)

June 30, 2027

Study Completion (Estimated)

December 31, 2027

Last Updated

April 23, 2026

Record last verified: 2025-12