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Umbilical Cord Mesenchymal Stem Cells Therapy for Patients With Spinocerebellar Ataxia

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ClinicalTrials.gov Identifier: NCT03378414
Recruitment Status : Not yet recruiting
First Posted : December 19, 2017
Last Update Posted : October 27, 2020
Sponsor:
Information provided by (Responsible Party):
Sclnow Biotechnology Co., Ltd.

Tracking Information
First Submitted Date  ICMJE December 12, 2017
First Posted Date  ICMJE December 19, 2017
Last Update Posted Date October 27, 2020
Estimated Study Start Date  ICMJE October 2021
Estimated Primary Completion Date June 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 18, 2017)
Scale for the assessment anf rating of ataxia (SARA) [ Time Frame: 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.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 18, 2017)
  • Image examinations [ Time Frame: 12 months ]
    MRI plain scan of brain.
  • Inventory of Non-Ataxia Symptoms (INAS) score [ Time Frame: 12 months ]
    Using Inventory of Non-Ataxia Symptoms (INAS) score to determine the presence and severity of non-ataxia signs.
  • Cerebrospinal fluid (csf) routine [ Time Frame: 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.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Umbilical Cord Mesenchymal Stem Cells Therapy for Patients With Spinocerebellar Ataxia
Official Title  ICMJE A Clinical Research on the Safety/Efficacy of Umbilical Cord Mesenchymal Stem Cells Therapy for Patients With Spinocerebellar Ataxia
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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Spinocerebellar Ataxia Type 1
  • Spinocerebellar Ataxia Type 2
  • Spinocerebellar Ataxia Type 3
  • Spinocerebellar Ataxia Type 6
Intervention  ICMJE
  • 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
Study Arms  ICMJE
  • Experimental: Intravenous infusion group
    Umbilical cord mesenchymal stem cells (SCLnow 19#)
    Interventions:
    • Procedure: Intravenous infusion
    • Biological: umbilical cord mesenchymal stem cell
  • Experimental: Intrathecal injection group
    Umbilical cord mesenchymal stem cells (SCLnow 19#)
    Interventions:
    • Procedure: Intrathecal injection
    • Biological: umbilical cord mesenchymal stem cell
  • No Intervention: Control groups
    No intervention
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Not yet recruiting
Estimated Enrollment  ICMJE
 (submitted: December 18, 2017)
45
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2022
Estimated Primary Completion Date June 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 16 Years to 60 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Lei Guo 861064368977 georgeguo@sclnow.com
Listed Location Countries  ICMJE Not Provided
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03378414
Other Study ID Numbers  ICMJE SCLnow-XY-03
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Sclnow Biotechnology Co., Ltd.
Study Sponsor  ICMJE Sclnow Biotechnology Co., Ltd.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Hong Jiang Xiangya Hospital of Central South University
PRS Account Sclnow Biotechnology Co., Ltd.
Verification Date October 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP