<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT02987413</url>
  </required_header>
  <id_info>
    <org_study_id>HospitalMCG</org_study_id>
    <secondary_id>IEPSaoLucas</secondary_id>
    <nct_id>NCT02987413</nct_id>
  </id_info>
  <brief_title>Escalated Application of Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis Patients</brief_title>
  <official_title>Safety and Efficacy of Mesenchymal Stem Cells Escalated Application in Amyotrophic Lateral Sclerosis Patients: Study Design of a Phase I Trial</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Hospital e Maternidade Dr. Christóvão da Gama</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>IEP São Lucas - Instituto de Ensino e Pesquisa</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Clinica Jordy Sinapse</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>TECHLIFE - Centro de Tecnologia Celular</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Hospital e Maternidade Dr. Christóvão da Gama</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      Background: Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that&#xD;
      selectively affects motor neurons in the brain and spinal cord, leading to bulbar,&#xD;
      respiratory, and limb weakness. There is no effective treatment, and the disease usually&#xD;
      progresses to death within 2 to 4 years. The therapeutic plasticity of mesenchymal stem cells&#xD;
      (MSCs) may be an attractive therapy to this complex disease, turning MSCs strong candidates&#xD;
      for cellular therapy in ALS.&#xD;
&#xD;
      Design-A phase 1 open-safety clinical trial. 4 patients will be selected according to a&#xD;
      restricted inclusion and exclusion criteria and after 2 escalated infusions of MSCs, there&#xD;
      will be a follow up period of one year Methods - Primary endpoint: safety of mesenchymal&#xD;
      autologous stem cells infusions escalated in two intrathecal administrations in patients with&#xD;
      ALS defined as severe adverse events (SAe). Secondary endpoints: clinical response,&#xD;
      laboratorial and magnetic resonance imaging of patients submitted to cellular escalating&#xD;
      doses applied in the study. Quality of life, according to El Escorial criteria, ALSFR scale&#xD;
      and functional scales.&#xD;
&#xD;
      Conclusion: This study is a primary step before a large randomized double-blind clinical&#xD;
      trial for ALS. It is expected to confirm the safety of escalated MSCs therapy in ALS&#xD;
      patients, initial data of efficacy in addition to improved quality of life.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">April 28, 2015</start_date>
  <completion_date type="Actual">April 5, 2017</completion_date>
  <primary_completion_date type="Actual">March 2016</primary_completion_date>
  <phase>Phase 1</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Serious Adverse Events related to the treatment</measure>
    <time_frame>12 months</time_frame>
    <description>Serious Adverse Events (Death, Life-threatening, Disability or Permanent Damage) will be monitored and documented during follow up</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Revised ALS Functional Rating Scale (ALSFRS-R)</measure>
    <time_frame>12 months</time_frame>
    <description>Quality of life from patients according to functional scales will be evaluated before and after interventions with 1, 3, 6 and 12 months.</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">3</enrollment>
  <condition>Motor Neuron Disease</condition>
  <arm_group>
    <arm_group_label>Autologous Mesenchymal stem cells</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Two escalated intrathecal infusions of autologous mesenchymal stem cell</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>Autologous Mesenchymal stem cells (MSCs)</intervention_name>
    <description>2 intrathecal autologous MSCs infusions (1x10^8 cells) will be performed, escalated from 30 days apart</description>
    <arm_group_label>Autologous Mesenchymal stem cells</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Women and males over 18-year-old.&#xD;
&#xD;
          -  Diagnosis of ALS in agreement with the criteria of &quot;EL SCORE&quot;&#xD;
&#xD;
          -  Less than 24 months of evolution of the disease (from the beginning of the symptoms).&#xD;
&#xD;
          -  Good understanding of the protocol and aptitude to grant the informed consent&#xD;
&#xD;
          -  Infertile women (post-menopause or hysterectomized)&#xD;
&#xD;
          -  Brazilian citizen and permanent resident.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Any significant medical condition (congestive heart failure, angina, respiratory&#xD;
             failure, and others)&#xD;
&#xD;
          -  Any auto-immune disease&#xD;
&#xD;
          -  Any malignant diseases&#xD;
&#xD;
          -  Systemic infection&#xD;
&#xD;
          -  Mental illness&#xD;
&#xD;
          -  Depressive state&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Leandro B Agati, PhD</last_name>
    <role>Study Director</role>
    <affiliation>Hospital e Maternidade Dr Christovao da Gama</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Eliseo J Sekiya, MD, PhD</last_name>
    <role>Study Chair</role>
    <affiliation>Instituto de Ensino e Pesquisas - IEP-São Lucas</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Adelson A Silva, MD</last_name>
    <role>Study Chair</role>
    <affiliation>Instituto de Ensino e Pesquisas - IEP-São Lucas</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Andresa Forte, MSc</last_name>
    <role>Study Chair</role>
    <affiliation>TECHLIFE - Centro de Tecnologia Celular</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Sergio S Jordy, MD</last_name>
    <role>Study Chair</role>
    <affiliation>Clínica Jordy Sinapse</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Hospital e Maternidade Dr Christovao da Gama</name>
      <address>
        <city>Santo Andre</city>
        <state>Sao Paulo</state>
        <zip>09030010</zip>
        <country>Brazil</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Instituto de Ensino e Pesquisas - IEP-São Lucas</name>
      <address>
        <city>Sao Paulo</city>
        <state>SP</state>
        <zip>01236-030</zip>
        <country>Brazil</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Brazil</country>
  </location_countries>
  <link>
    <url>http://www.hmcg.com.br</url>
    <description>Institutional website</description>
  </link>
  <verification_date>August 2017</verification_date>
  <study_first_submitted>July 1, 2016</study_first_submitted>
  <study_first_submitted_qc>December 8, 2016</study_first_submitted_qc>
  <study_first_posted type="Estimate">December 9, 2016</study_first_posted>
  <last_update_submitted>August 4, 2017</last_update_submitted>
  <last_update_submitted_qc>August 4, 2017</last_update_submitted_qc>
  <last_update_posted type="Actual">August 7, 2017</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Amyotrophic Lateral Sclerosis</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Motor Neuron Disease</mesh_term>
    <mesh_term>Amyotrophic Lateral Sclerosis</mesh_term>
    <mesh_term>Sclerosis</mesh_term>
  </condition_browse>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT02987413
org study id: HospitalMCG
secondary id: IEPSaoLucas
nct id: NCT02987413
lead sponsor: 
collaborator: 
collaborator: 
collaborator: 
has dmc: Yes
Background: Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that
      selectively affects motor neurons in the brain and spinal cord, leading to bulbar,
      respiratory, and limb weakness. There is no effective treatment, and the disease usually
      progresses to death within 2 to 4 years. The therapeutic plasticity of mesenchymal stem cells
      (MSCs) may be an attractive therapy to this complex disease, turning MSCs strong candidates
      for cellular therapy in ALS.

      Design-A phase 1 open-safety clinical trial. 4 patients will be selected according to a
      restricted inclusion and exclusion criteria and after 2 escalated infusions of MSCs, there
      will be a follow up period of one year Methods - Primary endpoint: safety of mesenchymal
      autologous stem cells infusions escalated in two intrathecal administrations in patients with
      ALS defined as severe adverse events (SAe). Secondary endpoints: clinical response,
      laboratorial and magnetic resonance imaging of patients submitted to cellular escalating
      doses applied in the study. Quality of life, according to El Escorial criteria, ALSFR scale
      and functional scales.

      Conclusion: This study is a primary step before a large randomized double-blind clinical
      trial for ALS. It is expected to confirm the safety of escalated MSCs therapy in ALS
      patients, initial data of efficacy in addition to improved quality of life.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Serious Adverse Events related to the treatment
time frame: 12 months
description: Serious Adverse Events (Death, Life-threatening, Disability or Permanent Damage) will be monitored and documented during follow up
measure: Revised ALS Functional Rating Scale (ALSFRS-R)
time frame: 12 months
description: Quality of life from patients according to functional scales will be evaluated before and after interventions with 1, 3, 6 and 12 months.
arm group label: Autologous Mesenchymal stem cells
arm group type: Experimental
description: Two escalated intrathecal infusions of autologous mesenchymal stem cell
intervention type: Biological
intervention name: Autologous Mesenchymal stem cells (MSCs)
description: 2 intrathecal autologous MSCs infusions (1x10^8 cells) will be performed, escalated from 30 days apart
arm group label: Autologous Mesenchymal stem cells
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Leandro B Agati, PhD
role: Study Director
affiliation: Hospital e Maternidade Dr Christovao da Gama
last name: Eliseo J Sekiya, MD, PhD
role: Study Chair
affiliation: Instituto de Ensino e Pesquisas - IEP-São Lucas
last name: Adelson A Silva, MD
role: Study Chair
affiliation: Instituto de Ensino e Pesquisas - IEP-São Lucas
last name: Andresa Forte, MSc
role: Study Chair
affiliation: TECHLIFE - Centro de Tecnologia Celular
last name: Sergio S Jordy, MD
role: Study Chair
affiliation: Clínica Jordy Sinapse
facility: 
facility: 
country: Brazil
url: http://www.hmcg.com.br
description: Institutional website
responsible party type: Sponsor
mesh term: Motor Neuron Disease
mesh term: Amyotrophic Lateral Sclerosis
mesh term: Sclerosis