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

Primary Purpose

Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3

Status
Not yet recruiting
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
Intravenous infusion
Intrathecal injection
umbilical cord mesenchymal stem cell
Sponsored by
Sclnow Biotechnology Co., Ltd.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Spinocerebellar Ataxia Type 1 focused on measuring Spinocerebellar Ataxia,Mesenchymal Stem Cells

Eligibility Criteria

16 Years - 60 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

    Arms of the Study

    Arm 1

    Arm 2

    Arm 3

    Arm Type

    Experimental

    Experimental

    No Intervention

    Arm Label

    Intravenous infusion group

    Intrathecal injection group

    Control groups

    Arm Description

    Umbilical cord mesenchymal stem cells (SCLnow 19#)

    Umbilical cord mesenchymal stem cells (SCLnow 19#)

    No intervention

    Outcomes

    Primary Outcome Measures

    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.

    Secondary Outcome Measures

    Image examinations
    MRI plain scan of brain.
    Inventory of Non-Ataxia Symptoms (INAS) score
    Using Inventory of Non-Ataxia Symptoms (INAS) score to determine the presence and severity of non-ataxia signs.
    Cerebrospinal fluid (csf) routine
    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.

    Full Information

    First Posted
    December 12, 2017
    Last Updated
    April 11, 2023
    Sponsor
    Sclnow Biotechnology Co., Ltd.
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    1. Study Identification

    Unique Protocol Identification Number
    NCT03378414
    Brief Title
    Umbilical Cord Mesenchymal Stem Cells Therapy (19#iSCLife®-SA) for Patients With Spinocerebellar Ataxia
    Official Title
    A Clinical Research on the Safety/Efficacy of Umbilical Cord Mesenchymal Stem Cells Therapy for Patients With Spinocerebellar Ataxia
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    January 2023
    Overall Recruitment Status
    Not yet recruiting
    Study Start Date
    December 2023 (Anticipated)
    Primary Completion Date
    June 2024 (Anticipated)
    Study Completion Date
    December 2024 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Sclnow Biotechnology Co., Ltd.

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    No
    Studies a U.S. FDA-regulated Device Product
    No

    5. Study Description

    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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3, Spinocerebellar Ataxia Type 6
    Keywords
    Spinocerebellar Ataxia,Mesenchymal Stem Cells

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 2
    Interventional Study Model
    Parallel Assignment
    Masking
    None (Open Label)
    Allocation
    Randomized
    Enrollment
    45 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    Intravenous infusion group
    Arm Type
    Experimental
    Arm Description
    Umbilical cord mesenchymal stem cells (SCLnow 19#)
    Arm Title
    Intrathecal injection group
    Arm Type
    Experimental
    Arm Description
    Umbilical cord mesenchymal stem cells (SCLnow 19#)
    Arm Title
    Control groups
    Arm Type
    No Intervention
    Arm Description
    No intervention
    Intervention Type
    Procedure
    Intervention Name(s)
    Intravenous infusion
    Intervention Description
    Intravenous infusion of mesenchymal stem cells: 2 * 10^7 cells (30ml)
    Intervention Type
    Procedure
    Intervention Name(s)
    Intrathecal injection
    Intervention Description
    Intrathecal injection of mesenchymal stem cells: 2 * 10^7 cells (1ml)
    Intervention Type
    Biological
    Intervention Name(s)
    umbilical cord mesenchymal stem cell
    Intervention Description
    Treat patients with umbilical cord mesenchymal stem cell (SCLnow 19#), the dosage based on different procedure
    Primary Outcome Measure Information:
    Title
    Scale for the assessment anf rating of ataxia (SARA)
    Description
    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.
    Time Frame
    12 months
    Secondary Outcome Measure Information:
    Title
    Image examinations
    Description
    MRI plain scan of brain.
    Time Frame
    12 months
    Title
    Inventory of Non-Ataxia Symptoms (INAS) score
    Description
    Using Inventory of Non-Ataxia Symptoms (INAS) score to determine the presence and severity of non-ataxia signs.
    Time Frame
    12 months
    Title
    Cerebrospinal fluid (csf) routine
    Description
    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.
    Time Frame
    12 months

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    16 Years
    Maximum Age & Unit of Time
    60 Years
    Accepts Healthy Volunteers
    No
    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
    Central Contact Person:
    First Name & Middle Initial & Last Name or Official Title & Degree
    Lei Guo
    Phone
    861064368977
    Email
    georgeguo@sclnow.com
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Hong Jiang
    Organizational Affiliation
    Xiangya Hospital of Central South University
    Official's Role
    Principal Investigator

    12. IPD Sharing Statement

    Learn more about this trial

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

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