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Not yet recruiting NCT03378414

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

Phase II Interventional Spinocerebellar Ataxia Type 1 Spinocerebellar Ataxia Type 2 Spinocerebellar Ataxia Type 3 Spinocerebellar Ataxia Type 6

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Intravenous infusion, Intrathecal injection, umbilical cord mesenchymal stem cell.
Who it may be relevant to
Registry conditions: Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3, Spinocerebellar Ataxia Type 6. Basic parameters: 16 years — 60 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Center list to be confirmed — check the primary protocol.
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

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Official title

A Clinical Research on the Safety/Efficacy of Umbilical Cord Mesenchymal Stem Cells Therapy for Patients With Spinocerebellar Ataxia

Overview

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

  • Procedure Intravenous infusion
    Intravenous infusion of mesenchymal stem cells: 2 \* 10\^7 cells (30ml)
  • Procedure Intrathecal injection
    Intrathecal injection of mesenchymal stem cells: 2 \* 10\^7 cells (1ml)
  • Biological umbilical cord mesenchymal stem cell
    Treat patients with umbilical cord mesenchymal stem cell (SCLnow 19#), the dosage based on different procedure

Primary outcome measures

  • Scale for the assessment anf rating of ataxia (SARA) [Time frame: 12 months]
Secondary outcome measures (3)
  • Image examinations [Time frame: 12 months]
  • Inventory of Non-Ataxia Symptoms (INAS) score [Time frame: 12 months]
  • Cerebrospinal fluid (csf) routine [Time frame: 12 months]

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Open label
Primary purpose
Treatment

Study locations

Center list to be confirmed — check the primary protocol.

Identifiers

NCT: NCT03378414 · SCLnow-XY-03

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗