<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/NCT02208713</url>
  </required_header>
  <id_info>
    <org_study_id>Royan-Nerve-007</org_study_id>
    <nct_id>NCT02208713</nct_id>
  </id_info>
  <brief_title>Intramuscular Transplantation of Muscle Derived Stem Cell and Adipose Derived Mesenchymal Stem Cells in Patients With Facioscapulohumeral Dystrophy (FSHD)</brief_title>
  <official_title>Intramuscular Transplantation of Autologous Muscle Derived Stem Cell(MDSC) and Adipose Derived Mesenchymal Stem Cells (AD-MSC) in Patients With Facioscapulohumeral Dystrophy (FSHD), Phase I Clinical Trial</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Royan Institute</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Royan Institute</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      Facioscapulohumeral Dystrophy is a Autosomal dominant inherited dystrophy with the prevalence&#xD;
      of 1:20,000 and it is the third most common dystrophy after the dystrophinopathies and&#xD;
      myotonic dystrophy. The symptoms including: Pain, facial weakness, scapular fixator, humeral,&#xD;
      truncal, pelvic girdle and lower-extremity weakness, High frequency hearing loss, Retinal&#xD;
      telangiectasia . The existing treatments are not effective so, cell therapy is a new hope to&#xD;
      improve patients' quality of life. Therefore, We design this clinical trial to evaluate the&#xD;
      safety and feasibility of stem cell transplantation.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      In this study, we select 15 patients with FSHD based on eligibility criteria. All the&#xD;
      patients underwent physical examination, laboratory evaluations, EMG-NCV, muscle sonography&#xD;
      and muscle MRI. Then, a sample of patient's muscle is taken from Biceps Femoralis to isolate&#xD;
      and culture of MDSC. The AD-MSC is prepared from Royan Adipose Tissue Bank. The patient is&#xD;
      admitted in general hospital, and the cell suspensions are injected into biceps, triceps and&#xD;
      trapezoids muscles by neurologists. After transplantation, the patients will be under&#xD;
      observation for 5 hours and will be discharged if no side effect happen. All the patients&#xD;
      will be followed at 1,2,4,6 and 12 months after cell injection.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Recruiting</last_known_status>
  <start_date type="Actual">May 2014</start_date>
  <completion_date type="Anticipated">December 2017</completion_date>
  <primary_completion_date type="Anticipated">November 2017</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>Myalsia</measure>
    <time_frame>1month</time_frame>
    <description>Evaluation the presence of myalsia 1month after cell injection.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Mass formation</measure>
    <time_frame>6 months</time_frame>
    <description>Evaluation the probability of mass formation 6 months after cell injection.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Hematoma</measure>
    <time_frame>1 month</time_frame>
    <description>Evaluation the presence of hematoma 1 month after injection.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Muscle bulk</measure>
    <time_frame>6 months</time_frame>
    <description>Evaluation the muscle bulk changes with sonography and MRI 6 months after transplantation.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>CPK</measure>
    <time_frame>1month</time_frame>
    <description>Evaluation the decrease of CPK 1 month after cell transplantation.</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">21</enrollment>
  <condition>Dystrophy</condition>
  <arm_group>
    <arm_group_label>Stem cell recipient</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>The patients with FSHD who underwent muscle derived stem cell and Adipose derived mesenchymal stem cell with intramuscular injection.</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>Intramuscular injection</intervention_name>
    <description>Intramuscular injection of stem cells in patients with FSHD.</description>
    <arm_group_label>Stem cell recipient</arm_group_label>
    <other_name>Intramuscular cell transplantation</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Age: 18-50&#xD;
&#xD;
          2. Both gender&#xD;
&#xD;
          3. Weakness of face muscle&#xD;
&#xD;
          4. FSHD phenotype positive&#xD;
&#xD;
          5. Genetic Test confirmation for FSHD&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Co - morbidities: Heart &amp; respiratory diseases,malignancy, rheumatologic disorders&#xD;
&#xD;
          2. Progressive form of disease&#xD;
&#xD;
          3. Not able to sign the consent&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>50 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Hamid Gourabi, PhD</last_name>
    <role>Study Chair</role>
    <affiliation>Head of Royan Institute</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Nasser Aghdami, MD,PhD</last_name>
    <role>Study Director</role>
    <affiliation>Head of Department of Regenerative Medicine &amp; Cell therapy center of Royan Institute</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Mahdi Vahid Dastjerdi, MD</last_name>
    <role>Study Director</role>
    <affiliation>Scientific Board of BouAli Hospital, Azad University</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Leila Arab, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Department of Regenerative Medicine &amp; cell therapy of Royan Institute</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Nasser Aghdami, MD,PhD</last_name>
    <phone>(+98)23562000</phone>
    <phone_ext>516</phone_ext>
    <email>nasser.aghdami@royaninstitute.org</email>
  </overall_contact>
  <overall_contact_backup>
    <last_name>Leila Arab, MD</last_name>
    <phone>(+98)23562000</phone>
    <phone_ext>414</phone_ext>
    <email>Leara91@gmail.com</email>
  </overall_contact_backup>
  <location>
    <facility>
      <name>Royan Institute</name>
      <address>
        <city>Tehran</city>
        <country>Iran, Islamic Republic of</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Nasser Aghdami, MD,PhD</last_name>
      <phone>(+98)212356000</phone>
      <phone_ext>516</phone_ext>
      <email>nasser.aghdami@royaninstitute.org</email>
    </contact>
    <contact_backup>
      <last_name>Leila Arab, MD</last_name>
      <phone>(+98)23562000</phone>
      <phone_ext>414</phone_ext>
      <email>Leara91@gmail.com</email>
    </contact_backup>
    <investigator>
      <last_name>Neda Jarooghi, MSc</last_name>
      <role>Sub-Investigator</role>
    </investigator>
    <investigator>
      <last_name>Tina Bolourieh, BSc</last_name>
      <role>Sub-Investigator</role>
    </investigator>
    <investigator>
      <last_name>Fatemeh Abbasi, MSc</last_name>
      <role>Sub-Investigator</role>
    </investigator>
  </location>
  <location_countries>
    <country>Iran, Islamic Republic of</country>
  </location_countries>
  <link>
    <url>http://Royaninstitute.org</url>
    <description>Related Info</description>
  </link>
  <verification_date>April 2017</verification_date>
  <study_first_submitted>August 4, 2014</study_first_submitted>
  <study_first_submitted_qc>August 4, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">August 5, 2014</study_first_posted>
  <last_update_submitted>April 26, 2017</last_update_submitted>
  <last_update_submitted_qc>April 26, 2017</last_update_submitted_qc>
  <last_update_posted type="Actual">April 27, 2017</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>FSHD muscle derived stem cell adipose derived mesenchymal stem cell intramuscular injection</keyword>
  <!-- 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/NCT02208713
org study id: Royan-Nerve-007
nct id: NCT02208713
lead sponsor: 
has dmc: Yes
Facioscapulohumeral Dystrophy is a Autosomal dominant inherited dystrophy with the prevalence
      of 1:20,000 and it is the third most common dystrophy after the dystrophinopathies and
      myotonic dystrophy. The symptoms including: Pain, facial weakness, scapular fixator, humeral,
      truncal, pelvic girdle and lower-extremity weakness, High frequency hearing loss, Retinal
      telangiectasia . The existing treatments are not effective so, cell therapy is a new hope to
      improve patients' quality of life. Therefore, We design this clinical trial to evaluate the
      safety and feasibility of stem cell transplantation.
In this study, we select 15 patients with FSHD based on eligibility criteria. All the
      patients underwent physical examination, laboratory evaluations, EMG-NCV, muscle sonography
      and muscle MRI. Then, a sample of patient's muscle is taken from Biceps Femoralis to isolate
      and culture of MDSC. The AD-MSC is prepared from Royan Adipose Tissue Bank. The patient is
      admitted in general hospital, and the cell suspensions are injected into biceps, triceps and
      trapezoids muscles by neurologists. After transplantation, the patients will be under
      observation for 5 hours and will be discharged if no side effect happen. All the patients
      will be followed at 1,2,4,6 and 12 months after cell injection.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Myalsia
time frame: 1month
description: Evaluation the presence of myalsia 1month after cell injection.
measure: Mass formation
time frame: 6 months
description: Evaluation the probability of mass formation 6 months after cell injection.
measure: Hematoma
time frame: 1 month
description: Evaluation the presence of hematoma 1 month after injection.
measure: Muscle bulk
time frame: 6 months
description: Evaluation the muscle bulk changes with sonography and MRI 6 months after transplantation.
measure: CPK
time frame: 1month
description: Evaluation the decrease of CPK 1 month after cell transplantation.
arm group label: Stem cell recipient
arm group type: Experimental
description: The patients with FSHD who underwent muscle derived stem cell and Adipose derived mesenchymal stem cell with intramuscular injection.
intervention type: Biological
intervention name: Intramuscular injection
description: Intramuscular injection of stem cells in patients with FSHD.
arm group label: Stem cell recipient
other name: Intramuscular cell transplantation
criteria: 
gender: All
minimum age: 18 Years
maximum age: 50 Years
healthy volunteers: No
last name: Hamid Gourabi, PhD
role: Study Chair
affiliation: Head of Royan Institute
last name: Nasser Aghdami, MD,PhD
role: Study Director
affiliation: Head of Department of Regenerative Medicine & Cell therapy center of Royan Institute
last name: Mahdi Vahid Dastjerdi, MD
role: Study Director
affiliation: Scientific Board of BouAli Hospital, Azad University
last name: Leila Arab, MD
role: Principal Investigator
affiliation: Department of Regenerative Medicine & cell therapy of Royan Institute
last name: Nasser Aghdami, MD,PhD
phone: (+98)23562000
phone ext: 516
email: nasser.aghdami@royaninstitute.org
last name: Leila Arab, MD
phone: (+98)23562000
phone ext: 414
email: Leara91@gmail.com
facility: 
status: Recruiting
contact: 
contact backup: 
investigator: 
investigator: 
investigator: 
country: Iran, Islamic Republic of
url: http://Royaninstitute.org
description: Related Info
responsible party type: Sponsor