xml: <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/NCT06017869</url>
    </required_header>
    <id_info>
      <org_study_id>MNV-010</org_study_id>
      <nct_id>NCT06017869</nct_id>
    </id_info>
    <brief_title>A Phase I to Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-201 in Pediatric Patients With Pearson Syndrome</brief_title>
    <official_title>A Phase I, Open Label, Single Dose Clinical Study to Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-201 (Autologous CD34+ Cells Enriched With Allogenic Placenta Derived Mitochondria) in Pediatric Patients With Pearson Syndrome</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Minovia Therapeutics Ltd.</agency>
        <agency_class>Industry</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>Minovia Therapeutics Ltd.</source>
    <oversight_info>
      <has_dmc>Yes</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>
        Primary Mitochondrial diseases are a clinically and genetically heterogeneous group of&#xD;
        disorders caused by mutations in genes encoded by nuclear Deoxyribonucleic Acid (DNA) or by&#xD;
        mutations and/or deletions in the mitochondrial DNA (mtDNA). While some mitochondrial&#xD;
        disorders only affect a single organ (e.g., the eye in Leber hereditary optic neuropathy&#xD;
        [LHON]), many involve multiple organs. Mitochondrial disorders may present at any age and a&#xD;
        frequent feature is the increasing number of organs involved in the course of the disease.&#xD;
  &#xD;
        Minovia Therapeutics Ltd. (&quot;Minovia&quot;) is a biotech company developing novel therapeutics&#xD;
        based on its mitochondrial augmentation technology (MAT). MNV-201 is a cell therapy produced&#xD;
        by MAT that consists of the participant's autologous CD34+ hematopoietic stem and progenitor&#xD;
        cells (HSPCs) enriched with allogeneic placental-derived mitochondria, manufactured in&#xD;
        Minovia's GMP facility.&#xD;
      </textblock>
    </brief_summary>
    <overall_status>Recruiting</overall_status>
    <start_date type="Actual">July 31, 2023</start_date>
    <completion_date type="Anticipated">December 2027</completion_date>
    <primary_completion_date type="Anticipated">December 2026</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>Safety Primary Endpoint</measure>
      <time_frame>12 months post treatment.</time_frame>
      <description>Occurrence of treatment-related adverse events as assessed by CTCAE v5.0 following MNV-201 infusion.</description>
    </primary_outcome>
    <secondary_outcome>
      <measure>Improvement in anemia or thrombocytopenia, or leukopenia, and/or decrease in frequency of blood transfusions</measure>
      <time_frame>12 months post treatment</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Stabilization or improvement in Quality of Life (QoL) questionnaire IPMDS (International Pediatric Mitochondrial Disease Scale) scores</measure>
      <time_frame>12 months post treatment</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>• Reduction in frequency and lengths of hospitalization during the 12 months after MAT compared to the 12 months period before MAT</measure>
      <time_frame>12 months post treatment</time_frame>
    </secondary_outcome>
    <number_of_arms>1</number_of_arms>
    <enrollment type="Anticipated">5</enrollment>
    <condition>Mitochondrial Diseases</condition>
    <condition>Pearson Syndrome</condition>
    <arm_group>
      <arm_group_label>Autologous CD34+ cells enriched with allogenic placenta-derived mitochondria</arm_group_label>
      <arm_group_type>Experimental</arm_group_type>
    </arm_group>
    <intervention>
      <intervention_type>Biological</intervention_type>
      <intervention_name>MNV-201</intervention_name>
      <description>Autologous CD34+ cells are isolated from the participant's peripheral blood after mobilization by leukapheresis. Allogeneic mitochondria are isolated under aseptic conditions from healthy donor placenta, cryopreserved and qualified before use.</description>
      <arm_group_label>Autologous CD34+ cells enriched with allogenic placenta-derived mitochondria</arm_group_label>
      <other_name>CD34+ cells enriched with allogeneic placenta derived mitochondria</other_name>
    </intervention>
    <eligibility>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
            1. Male or female participants aged from 1 to 18 years old.&#xD;
  &#xD;
            2. Diagnosis of Pearson Syndrome, mtDNA deletion verified by NGS.&#xD;
  &#xD;
            3. Body weight ≥ 10 kg.&#xD;
  &#xD;
            4. Participant has anemia or thrombocytopenia, or leukopenia and/or blood transfusion&#xD;
               dependent (receives blood transfusions every 6 weeks or less).&#xD;
  &#xD;
            5. Participant is medically able to undergo the study interventions, as determined by the&#xD;
               investigator.&#xD;
  &#xD;
            6. Participant's living parent(s) and/or legal guardian(s) able to understand and provide&#xD;
               voluntary written informed consent.&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            1. History of infection with HIV-1, HIV-2, or HTLV I/II.&#xD;
  &#xD;
            2. Current active infection with HBV (including HBcore and HBsAg positive), HCV, HTLV&#xD;
               I/II, Treponema Pallidum or HIV I-II&#xD;
  &#xD;
            3. Participant has been diagnosed with Myelodysplastic Syndrome, by FISH and/or&#xD;
               karyotype.&#xD;
  &#xD;
            4. Participant is unable to undergo leukapheresis.&#xD;
  &#xD;
            5. Total number of CD34+ cells collected is lower than 20x106 cells&#xD;
  &#xD;
            6. Participant has known hypersensitivity to murine proteins or iron-dextran.&#xD;
  &#xD;
            7. Participant has chronic severe infection.&#xD;
  &#xD;
            8. Participant has disease or condition that may risk the participant or interfere with&#xD;
               the ability to interpret the study results.&#xD;
  &#xD;
            9. History of malignancy&#xD;
  &#xD;
           10. History of treatment with gene therapy, bone marrow or allogeneic cord blood&#xD;
               transplantation.&#xD;
  &#xD;
           11. Currently participating in another clinical trial, or participation in another&#xD;
               clinical trial within 1 year prior to study enrollment.&#xD;
  &#xD;
           12. In the opinion of the Investigator, the participant is unsuitable for participating in&#xD;
               the study for any reason.&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>1 Year</minimum_age>
      <maximum_age>18 Years</maximum_age>
      <healthy_volunteers>No</healthy_volunteers>
    </eligibility>
    <overall_contact>
      <last_name>Natalie Yivgi Ohana, PhD</last_name>
      <phone>+ 972 54 5833727</phone>
      <email>natalie@minoviatx.com</email>
    </overall_contact>
    <location>
      <facility>
        <name>Sheba Medical Center</name>
        <address>
          <city>Ramat Gan</city>
          <zip>5266202</zip>
          <country>Israel</country>
        </address>
      </facility>
      <status>Recruiting</status>
      <contact>
        <last_name>Elad Jacoby, MD</last_name>
        <phone>+972 526668355</phone>
        <email>elad.jacoby@sheba.health.gov.il</email>
      </contact>
      <contact_backup>
        <last_name>Moran Levin</last_name>
        <phone>+972523923147</phone>
        <email>moran.levin@sheba.health.gov.il</email>
      </contact_backup>
    </location>
    <location_countries>
      <country>Israel</country>
    </location_countries>
    <verification_date>August 2023</verification_date>
    <study_first_submitted>August 24, 2023</study_first_submitted>
    <study_first_submitted_qc>August 24, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">August 30, 2023</study_first_posted>
    <last_update_submitted>August 24, 2023</last_update_submitted>
    <last_update_submitted_qc>August 24, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">August 30, 2023</last_update_posted>
    <responsible_party>
      <responsible_party_type>Sponsor</responsible_party_type>
    </responsible_party>
    <keyword>Autologous stem cell transplantation Mitochondrial Diseases Pearson Syndrome Rare Diseases</keyword>
    <condition_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Muscular Diseases</mesh_term>
      <mesh_term>Lipid Metabolism, Inborn Errors</mesh_term>
      <mesh_term>Mitochondrial Diseases</mesh_term>
      <mesh_term>Syndrome</mesh_term>
    </condition_browse>
    <patient_data>
      <sharing_ipd>Undecided</sharing_ipd>
    </patient_data>
    <!-- Results have not yet been posted for this study                                          -->
  </clinical_study>
  
  
text: 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/NCT06017869
  org study id: MNV-010
  nct id: NCT06017869
  lead sponsor: 
  has dmc: Yes
  is fda regulated drug: No
  is fda regulated device: No
  Primary Mitochondrial diseases are a clinically and genetically heterogeneous group of
        disorders caused by mutations in genes encoded by nuclear Deoxyribonucleic Acid (DNA) or by
        mutations and/or deletions in the mitochondrial DNA (mtDNA). While some mitochondrial
        disorders only affect a single organ (e.g., the eye in Leber hereditary optic neuropathy
        [LHON]), many involve multiple organs. Mitochondrial disorders may present at any age and a
        frequent feature is the increasing number of organs involved in the course of the disease.
  
        Minovia Therapeutics Ltd. ("Minovia") is a biotech company developing novel therapeutics
        based on its mitochondrial augmentation technology (MAT). MNV-201 is a cell therapy produced
        by MAT that consists of the participant's autologous CD34+ hematopoietic stem and progenitor
        cells (HSPCs) enriched with allogeneic placental-derived mitochondria, manufactured in
        Minovia's GMP facility.
  allocation: N/A
  intervention model: Single Group Assignment
  primary purpose: Treatment
  masking: None (Open Label)
  measure: Safety Primary Endpoint
  time frame: 12 months post treatment.
  description: Occurrence of treatment-related adverse events as assessed by CTCAE v5.0 following MNV-201 infusion.
  measure: Improvement in anemia or thrombocytopenia, or leukopenia, and/or decrease in frequency of blood transfusions
  time frame: 12 months post treatment
  measure: Stabilization or improvement in Quality of Life (QoL) questionnaire IPMDS (International Pediatric Mitochondrial Disease Scale) scores
  time frame: 12 months post treatment
  measure: • Reduction in frequency and lengths of hospitalization during the 12 months after MAT compared to the 12 months period before MAT
  time frame: 12 months post treatment
  arm group label: Autologous CD34+ cells enriched with allogenic placenta-derived mitochondria
  arm group type: Experimental
  intervention type: Biological
  intervention name: MNV-201
  description: Autologous CD34+ cells are isolated from the participant's peripheral blood after mobilization by leukapheresis. Allogeneic mitochondria are isolated under aseptic conditions from healthy donor placenta, cryopreserved and qualified before use.
  arm group label: Autologous CD34+ cells enriched with allogenic placenta-derived mitochondria
  other name: CD34+ cells enriched with allogeneic placenta derived mitochondria
  criteria: 
  gender: All
  minimum age: 1 Year
  maximum age: 18 Years
  healthy volunteers: No
  last name: Natalie Yivgi Ohana, PhD
  phone: + 972 54 5833727
  email: natalie@minoviatx.com
  facility: 
  status: Recruiting
  contact: 
  contact backup: 
  country: Israel
  responsible party type: Sponsor
  mesh term: Muscular Diseases
  mesh term: Lipid Metabolism, Inborn Errors
  mesh term: Mitochondrial Diseases
  mesh term: Syndrome
  sharing ipd: Undecided