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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.

Study Description
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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.

Condition or disease Intervention/treatment Phase
Spinocerebellar Ataxia Type 1 Spinocerebellar Ataxia Type 2 Spinocerebellar Ataxia Type 3 Spinocerebellar Ataxia Type 6 Procedure: Intravenous infusion Procedure: Intrathecal injection Biological: umbilical cord mesenchymal stem cell Phase 2

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 Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 45 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Clinical Research on the Safety/Efficacy of Umbilical Cord Mesenchymal Stem Cells Therapy for Patients With Spinocerebellar Ataxia
Estimated Study Start Date : October 2021
Estimated Primary Completion Date : June 2022
Estimated Study Completion Date : December 2022

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Arms and Interventions
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Arm Intervention/treatment
Experimental: Intravenous infusion group
Umbilical cord mesenchymal stem cells (SCLnow 19#)
 Procedure: Intravenous infusion
Intravenous infusion of mesenchymal stem cells: 2 * 10^7 cells (30ml)

Biological: umbilical cord mesenchymal stem cell
Treat patients with umbilical cord mesenchymal stem cell (SCLnow 19#), the dosage based on different procedure
Experimental: Intrathecal injection group
Umbilical cord mesenchymal stem cells (SCLnow 19#)
 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
No Intervention: Control groups
No intervention


Outcome Measures
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Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. Image examinations [ Time Frame: 12 months ]
    MRI plain scan of brain.

  2. 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.

  3. 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.


Eligibility Criteria
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Ages Eligible for Study:   16 Years to 60 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03378414


Contacts
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Contact: Lei Guo 861064368977 georgeguo@sclnow.com

Sponsors and Collaborators
Sclnow Biotechnology Co., Ltd.
Investigators
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Principal Investigator: Hong Jiang Xiangya Hospital of Central South University
More Information
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Responsible Party: Sclnow Biotechnology Co., Ltd.
ClinicalTrials.gov Identifier: NCT03378414    
Other Study ID Numbers: SCLnow-XY-03
First Posted: December 19, 2017    Key Record Dates
Last Update Posted: October 27, 2020
Last Verified: October 2020

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Sclnow Biotechnology Co., Ltd.:
Spinocerebellar Ataxia,Mesenchymal Stem Cells
Additional relevant MeSH terms:
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Ataxia
Cerebellar Ataxia
Spinocerebellar Ataxias
Spinocerebellar Degenerations
Machado-Joseph Disease
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
 Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn