<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/NCT03826498</url>
  </required_header>
  <id_info>
    <org_study_id>12102017</org_study_id>
    <nct_id>NCT03826498</nct_id>
  </id_info>
  <brief_title>Allogenic Cord Blood Transfusion in Patients With Cerebral Palsy</brief_title>
  <official_title>Efficiency Evaluation of Allogenic Umbilical Cord Blood Mononuclear Cells (UCB-MNC) Transfusion in Patients With Autism</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>State-Financed Health Facility &quot;Samara Regional Medical Center Dinasty&quot;</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>State-Financed Health Facility &quot;Samara Regional Medical Center Dinasty&quot;</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      Cerebral palsy is a disorder of movement and posture resulted from a non-progressive lesion&#xD;
      or injury of the immature brain. It is a leading cause of childhood onset disability.&#xD;
&#xD;
      Many experimental animal studies have revealed that umbilical cord blood is useful to repair&#xD;
      neurological injury in brain.&#xD;
&#xD;
      On the basis of many experimental studies, umbilical cord blood is suggested as a potential&#xD;
      therapy for cerebral palsy.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Cerebral palsy (CP) is a group of neurodevelopmental conditions with abnormal movement and&#xD;
      posture resulted from a non-progressive cerebral disturbance. It is the most common cause of&#xD;
      motor disability in childhood. Most therapies are palliative rather than restorative.&#xD;
      Umbilical cord blood (UCB) may be used as restorative approach for children with CP.&#xD;
&#xD;
      Many experimental animal studies have revealed that UCB is beneficial to improve and repair&#xD;
      neurological injuries, this effect achieved due to immune regulation and angiogenesis as well&#xD;
      as the neuroprotective effect.&#xD;
&#xD;
      Based on animal studies and some clinical trials, UCB is suggested as a potential therapy for&#xD;
      children with CP.&#xD;
&#xD;
      This study is prospective, non randomized (open label) with control group.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">January 12, 2018</start_date>
  <completion_date type="Anticipated">December 26, 2024</completion_date>
  <primary_completion_date type="Anticipated">January 26, 2024</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Non-Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Number of participants with non-serious and serious adverse events</measure>
    <time_frame>6 month</time_frame>
    <description>Safety assessment such as adverse events will be registered. Adverse events will be monitored during all trial.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Changes in Standardized Gross Motor Function 66 (GMFM-66) Score for all child.</measure>
    <time_frame>1, 3, 6 month</time_frame>
    <description>GMFM (Gross Motor Function Measure) as a standardized measurement tool for assessing Gross Motor Function consisting of sub-scales; lying &amp; rolling, sitting, crawling &amp; kneeling, standing, walking, running &amp; jumping (range: 0~100 , Higher value means better gross motor function). We reported changes of GMFM between each assessment time points. Categories of outcome table are baseline and values of just subtracting the latter raw scores from the former ones. This test will be acquired for all child.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Changes in Cognitive Neurodevelopmental Outcome for child before 3yrs</measure>
    <time_frame>1, 3, 6 month</time_frame>
    <description>Bayley Scale of Infant Development-II (K-BSID-II) Mental Scales (higher value means better mental function: 0 - worst, 178 - best). We reported changes of BSID-II Mental Scale raw score between each assessment time points. Categories of outcome data are values of subtracting the latter scores from the former ones.This test will be acquired for child before 3yrs.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Changes in The Infant Toddler Quality of Life Questionnaire for child above 3yrs.</measure>
    <time_frame>1, 3, 6 month</time_frame>
    <description>ITQOL - the 47-item short-form (ITQOL-SF47) developed for use in infants and toddlers from 2-months-to-5 years of age. Form scores physical, mental and social well being/ For each concept, item responses are scored, summed, and transformed on a scale from 0 (worst health) to 100 (best health). Changes in completed questionnaire will be assessed. This test will be acquired for child above 3yrs.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Changes in Ashworth scale score for all child.</measure>
    <time_frame>1, 3, 6 month</time_frame>
    <description>The Ashworth scale (AS) measures resistance during passive soft-tissue stretching and is used as a simple measure of spasticity scoring, where:&#xD;
0 No increase in tone;&#xD;
Slight increase in tone giving catch when the limb is moved in flexion and extension;&#xD;
More marked increase in tone, but limb is easily flexed;&#xD;
Considerable increases in tone, passive movement difficult;&#xD;
Limb rigid in flexion or extension. This test will be acquired for child above 3yrs.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Number of Participants with Chimerism (longevity) of infused cell</measure>
    <time_frame>6 month</time_frame>
    <description>Chimerism study to detect the longevity of infused donor cells and predict effectiveness of treatment. This study measure DNA of donor cells will in patients blood at the end of protocol. Absence of donor DNA suggest no chimerism.</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Anticipated">40</enrollment>
  <condition>Cerebral Palsy</condition>
  <arm_group>
    <arm_group_label>CP CB-MNC injection</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>CP CB-MNC injection from different donors and standard therapy.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Standard therapy</arm_group_label>
    <arm_group_type>Other</arm_group_type>
    <description>Patients with standard therapy as control group</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>CP CB-MNC injection</intervention_name>
    <description>CB-MNC injection from different donors. One dose consist 20-50 mil MNC/kg.The protocol include 3 injection at monthly intervals.</description>
    <arm_group_label>CP CB-MNC injection</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Standard therapy</intervention_name>
    <description>The standard therapy can include drugs, special psychology training etc.</description>
    <arm_group_label>CP CB-MNC injection</arm_group_label>
    <arm_group_label>Standard therapy</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
        - Confirmed diagnosis: Cerebral Palsy.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  the presence of the following diseases in history: heart failure in the stage of&#xD;
             decompensation, stroke in history less than 1 year ago, blood diseases;&#xD;
&#xD;
          -  decompensation of chronic and endocrinological diseases;&#xD;
&#xD;
          -  acute respiratory viral and bacterial infections, period less than 1 month after the&#xD;
             acute phase.&#xD;
&#xD;
          -  HIV infection, hepatitis B and C.&#xD;
&#xD;
          -  oncological diseases, chemotherapy in the anamnesis;&#xD;
&#xD;
          -  tuberculosis.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>1 Year</minimum_age>
    <maximum_age>10 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>STANISLAV VOLCHKOV, MD, PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Medical Centre Dinasty</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>STANISLAV VOLCHKOV, MD, PhD</last_name>
    <phone>+79608159408</phone>
    <email>bioen07@gmail.com</email>
  </overall_contact>
  <overall_contact_backup>
    <last_name>Olga Tyumina, M.D, PhD</last_name>
    <email>centr123@bk.ru</email>
  </overall_contact_backup>
  <location>
    <facility>
      <name>Medical Centre Dinasty</name>
      <address>
        <city>Samara</city>
        <zip>443095</zip>
        <country>Russian Federation</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Stanislav Volchkov, MD</last_name>
      <phone>+78469564455</phone>
      <email>quality@cordbank.ru</email>
    </contact>
    <contact_backup>
      <last_name>Pavel Ovchinnikov, MD</last_name>
      <phone>+78469564455</phone>
    </contact_backup>
    <investigator>
      <last_name>Stanislav Volchkov</last_name>
      <role>Principal Investigator</role>
    </investigator>
    <investigator>
      <last_name>Pavel Ovchinnikov</last_name>
      <role>Sub-Investigator</role>
    </investigator>
    <investigator>
      <last_name>Alexander Bugakov</last_name>
      <role>Sub-Investigator</role>
    </investigator>
  </location>
  <location_countries>
    <country>Russian Federation</country>
  </location_countries>
  <verification_date>January 2023</verification_date>
  <study_first_submitted>January 22, 2019</study_first_submitted>
  <study_first_submitted_qc>January 30, 2019</study_first_submitted_qc>
  <study_first_posted type="Actual">February 1, 2019</study_first_posted>
  <last_update_submitted>January 17, 2023</last_update_submitted>
  <last_update_submitted_qc>January 17, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">January 18, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>State-Financed Health Facility &quot;Samara Regional Medical Center Dinasty&quot;</investigator_affiliation>
    <investigator_full_name>Volchkov Stanislav</investigator_full_name>
    <investigator_title>Deputy director, Quality assurance director</investigator_title>
  </responsible_party>
  <keyword>Cerebral palsy</keyword>
  <keyword>CP</keyword>
  <keyword>Cord Blood</keyword>
  <keyword>Stem cells</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Cerebral Palsy</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/NCT03826498
org study id: 12102017
nct id: NCT03826498
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
Cerebral palsy is a disorder of movement and posture resulted from a non-progressive lesion
      or injury of the immature brain. It is a leading cause of childhood onset disability.

      Many experimental animal studies have revealed that umbilical cord blood is useful to repair
      neurological injury in brain.

      On the basis of many experimental studies, umbilical cord blood is suggested as a potential
      therapy for cerebral palsy.
Cerebral palsy (CP) is a group of neurodevelopmental conditions with abnormal movement and
      posture resulted from a non-progressive cerebral disturbance. It is the most common cause of
      motor disability in childhood. Most therapies are palliative rather than restorative.
      Umbilical cord blood (UCB) may be used as restorative approach for children with CP.

      Many experimental animal studies have revealed that UCB is beneficial to improve and repair
      neurological injuries, this effect achieved due to immune regulation and angiogenesis as well
      as the neuroprotective effect.

      Based on animal studies and some clinical trials, UCB is suggested as a potential therapy for
      children with CP.

      This study is prospective, non randomized (open label) with control group.
allocation: Non-Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Number of participants with non-serious and serious adverse events
time frame: 6 month
description: Safety assessment such as adverse events will be registered. Adverse events will be monitored during all trial.
measure: Changes in Standardized Gross Motor Function 66 (GMFM-66) Score for all child.
time frame: 1, 3, 6 month
description: GMFM (Gross Motor Function Measure) as a standardized measurement tool for assessing Gross Motor Function consisting of sub-scales; lying & rolling, sitting, crawling & kneeling, standing, walking, running & jumping (range: 0~100 , Higher value means better gross motor function). We reported changes of GMFM between each assessment time points. Categories of outcome table are baseline and values of just subtracting the latter raw scores from the former ones. This test will be acquired for all child.
measure: Changes in Cognitive Neurodevelopmental Outcome for child before 3yrs
time frame: 1, 3, 6 month
description: Bayley Scale of Infant Development-II (K-BSID-II) Mental Scales (higher value means better mental function: 0 - worst, 178 - best). We reported changes of BSID-II Mental Scale raw score between each assessment time points. Categories of outcome data are values of subtracting the latter scores from the former ones.This test will be acquired for child before 3yrs.
measure: Changes in The Infant Toddler Quality of Life Questionnaire for child above 3yrs.
time frame: 1, 3, 6 month
description: ITQOL - the 47-item short-form (ITQOL-SF47) developed for use in infants and toddlers from 2-months-to-5 years of age. Form scores physical, mental and social well being/ For each concept, item responses are scored, summed, and transformed on a scale from 0 (worst health) to 100 (best health). Changes in completed questionnaire will be assessed. This test will be acquired for child above 3yrs.
measure: Changes in Ashworth scale score for all child.
time frame: 1, 3, 6 month
description: The Ashworth scale (AS) measures resistance during passive soft-tissue stretching and is used as a simple measure of spasticity scoring, where:
0 No increase in tone;
Slight increase in tone giving catch when the limb is moved in flexion and extension;
More marked increase in tone, but limb is easily flexed;
Considerable increases in tone, passive movement difficult;
Limb rigid in flexion or extension. This test will be acquired for child above 3yrs.
measure: Number of Participants with Chimerism (longevity) of infused cell
time frame: 6 month
description: Chimerism study to detect the longevity of infused donor cells and predict effectiveness of treatment. This study measure DNA of donor cells will in patients blood at the end of protocol. Absence of donor DNA suggest no chimerism.
arm group label: CP CB-MNC injection
arm group type: Experimental
description: CP CB-MNC injection from different donors and standard therapy.
arm group label: Standard therapy
arm group type: Other
description: Patients with standard therapy as control group
intervention type: Biological
intervention name: CP CB-MNC injection
description: CB-MNC injection from different donors. One dose consist 20-50 mil MNC/kg.The protocol include 3 injection at monthly intervals.
arm group label: CP CB-MNC injection
intervention type: Other
intervention name: Standard therapy
description: The standard therapy can include drugs, special psychology training etc.
arm group label: CP CB-MNC injection
arm group label: Standard therapy
criteria: 
gender: All
minimum age: 1 Year
maximum age: 10 Years
healthy volunteers: No
last name: STANISLAV VOLCHKOV, MD, PhD
role: Principal Investigator
affiliation: Medical Centre Dinasty
last name: STANISLAV VOLCHKOV, MD, PhD
phone: +79608159408
email: bioen07@gmail.com
last name: Olga Tyumina, M.D, PhD
email: centr123@bk.ru
facility: 
status: Recruiting
contact: 
contact backup: 
investigator: 
investigator: 
investigator: 
country: Russian Federation
responsible party type: Principal Investigator
investigator affiliation: State-Financed Health Facility "Samara Regional Medical Center Dinasty"
investigator full name: Volchkov Stanislav
investigator title: Deputy director, Quality assurance director
mesh term: Cerebral Palsy