<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/NCT02985346</url>
  </required_header>
  <id_info>
    <org_study_id>WTan</org_study_id>
    <nct_id>NCT02985346</nct_id>
  </id_info>
  <brief_title>Efficacy of Bone Marrow Mesenchymal Stem Cell in Pulmonary Hemosiderosis</brief_title>
  <official_title>Sun Yat-sen Memorial Hospital</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Sun Yat-sen University</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Sun Yat-sen University</source>
  <brief_summary>
    <textblock>
      Pulmonary hemosiderosis (PH) is a pulmonary hemosiderin deposition which caused by alveolar&#xD;
      capillary hemorrhage. PH is easy to recurrent and can lead to pulmonary fibrosis and&#xD;
      insufficiency if the disease was poor controlled. Steroid is the most common drug that was&#xD;
      administered in acute phase of the disease. However, considered the side-effects, steroid is&#xD;
      not suitable for long-time maintenance. Therefore, it is necessary to explore a new therapy.&#xD;
      Bone marrow mesenchymal stem cells (BMSC) are a kind of adult stem cells with high&#xD;
      self-renewal and multi-directional differentiation potential in bone marrow. It has become a&#xD;
      hot topic in immunosuppressive and tissue repair therapy in recent years. To date, homing,&#xD;
      colonization and differentiation of BMSCs in the lung have been observed in animal models of&#xD;
      pulmonary hypertension, radiation pneumonitis and pulmonary fibrosis. It had been reported&#xD;
      that BMSC transplantation in acute lung injury in mice, inflammation of lung injury can&#xD;
      significantly improve. The aim of this study is to explore the effect of BMSC on PH and its&#xD;
      mechanism, and to explore a new way to promote the repair of IPH. It is expected to improve&#xD;
      the status of IPH therapy in children, especially improve the prognosis of refractory PH.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Not yet recruiting</last_known_status>
  <start_date>January 2017</start_date>
  <primary_completion_date type="Anticipated">December 2020</primary_completion_date>
  <phase>Early Phase 1</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>Complete remission</measure>
    <time_frame>3-6 months</time_frame>
  </primary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Anticipated">100</enrollment>
  <condition>Idiopathic Pulmonary Hemosiderosis</condition>
  <arm_group>
    <arm_group_label>BMSC group</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Patients received Bone marrow mesenchymal stem cells (BMSC) plus standard treatment</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Control group</arm_group_label>
    <arm_group_type>Active Comparator</arm_group_type>
    <description>Patients received standard treatment</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>Bone marrow mesenchymal stem cells in treatment of Idiopathic pulmonary hemosiderosis</intervention_name>
    <arm_group_label>BMSC group</arm_group_label>
    <arm_group_label>Control group</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Patients diagnosed with Pulmonary hemosiderosis at an age less than 18 years.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Patients who cannot finish the established causes or die during the causes.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>1 Month</minimum_age>
    <maximum_age>18 Years</maximum_age>
    <healthy_volunteers>Accepts Healthy Volunteers</healthy_volunteers>
  </eligibility>
  <reference>
    <citation>Byrd RB, Gracey DR. Immunosuppressive treatment of idiopathic pulmonary hemosiderosis. JAMA. 1973 Oct 22;226(4):458-9. No abstract available.</citation>
    <PMID>4800237</PMID>
  </reference>
  <reference>
    <citation>Airaghi L, Ciceri L, Giannini S, Ferrero S, Meroni PL, Tedeschi A. Idiopathic pulmonary hemosiderosis in an adult. Favourable response to azathioprine. Monaldi Arch Chest Dis. 2001 Jun;56(3):211-3.</citation>
    <PMID>11665500</PMID>
  </reference>
  <reference>
    <citation>Calabrese F, Giacometti C, Rea F, Loy M, Sartori F, Di Vittorio G, Abudureheman A, Thiene G, Valente M. Recurrence of idiopathic pulmonary hemosiderosis in a young adult patient after bilateral single-lung transplantation. Transplantation. 2002 Dec 15;74(11):1643-5. doi: 10.1097/00007890-200212150-00027.</citation>
    <PMID>12490803</PMID>
  </reference>
  <reference>
    <citation>Chen RL, Chuang SS. Silent idiopathic pulmonary hemosiderosis with iron-deficiency anemia but normal serum ferritin. J Pediatr Hematol Oncol. 2007 Jul;29(7):509-11. doi: 10.1097/MPH.0b013e3180950372. No abstract available.</citation>
    <PMID>17609633</PMID>
  </reference>
  <results_reference>
    <citation>Rojas M, Xu J, Woods CR, Mora AL, Spears W, Roman J, Brigham KL. Bone marrow-derived mesenchymal stem cells in repair of the injured lung. Am J Respir Cell Mol Biol. 2005 Aug;33(2):145-52. doi: 10.1165/rcmb.2004-0330OC. Epub 2005 May 12.</citation>
    <PMID>15891110</PMID>
  </results_reference>
  <results_reference>
    <citation>Wang G, Bunnell BA, Painter RG, Quiniones BC, Tom S, Lanson NA Jr, Spees JL, Bertucci D, Peister A, Weiss DJ, Valentine VG, Prockop DJ, Kolls JK. Adult stem cells from bone marrow stroma differentiate into airway epithelial cells: potential therapy for cystic fibrosis. Proc Natl Acad Sci U S A. 2005 Jan 4;102(1):186-91. doi: 10.1073/pnas.0406266102. Epub 2004 Dec 22.</citation>
    <PMID>15615854</PMID>
  </results_reference>
  <results_reference>
    <citation>Lee JW, Fang X, Gupta N, Serikov V, Matthay MA. Allogeneic human mesenchymal stem cells for treatment of E. coli endotoxin-induced acute lung injury in the ex vivo perfused human lung. Proc Natl Acad Sci U S A. 2009 Sep 22;106(38):16357-62. doi: 10.1073/pnas.0907996106. Epub 2009 Aug 31.</citation>
    <PMID>19721001</PMID>
  </results_reference>
  <results_reference>
    <citation>Knight DA, Rossi FM, Hackett TL. Mesenchymal stem cells for repair of the airway epithelium in asthma. Expert Rev Respir Med. 2010 Dec;4(6):747-58. doi: 10.1586/ers.10.72.</citation>
    <PMID>21128750</PMID>
  </results_reference>
  <results_reference>
    <citation>Hoffman AM, Paxson JA, Mazan MR, Davis AM, Tyagi S, Murthy S, Ingenito EP. Lung-derived mesenchymal stromal cell post-transplantation survival, persistence, paracrine expression, and repair of elastase-injured lung. Stem Cells Dev. 2011 Oct;20(10):1779-92. doi: 10.1089/scd.2011.0105. Epub 2011 Jul 6.</citation>
    <PMID>21585237</PMID>
  </results_reference>
  <results_reference>
    <citation>Liu AR, Liu L, Chen S, Yang Y, Zhao HJ, Liu L, Guo FM, Lu XM, Qiu HB. Activation of canonical wnt pathway promotes differentiation of mouse bone marrow-derived MSCs into type II alveolar epithelial cells, confers resistance to oxidative stress, and promotes their migration to injured lung tissue in vitro. J Cell Physiol. 2013 Jun;228(6):1270-83. doi: 10.1002/jcp.24282.</citation>
    <PMID>23154940</PMID>
  </results_reference>
  <verification_date>December 2016</verification_date>
  <study_first_submitted>December 3, 2016</study_first_submitted>
  <study_first_submitted_qc>December 6, 2016</study_first_submitted_qc>
  <study_first_posted type="Estimate">December 7, 2016</study_first_posted>
  <last_update_submitted>December 6, 2016</last_update_submitted>
  <last_update_submitted_qc>December 6, 2016</last_update_submitted_qc>
  <last_update_posted type="Estimate">December 7, 2016</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Sun Yat-sen University</investigator_affiliation>
    <investigator_full_name>Weiping Tan</investigator_full_name>
    <investigator_title>Sun Yat-sen Memorial Hospital</investigator_title>
  </responsible_party>
  <keyword>Idiopathic pulmonary hemosiderosis</keyword>
  <keyword>Bone marrow mesenchymal stem cells</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Lung Diseases</mesh_term>
    <mesh_term>Hemochromatosis</mesh_term>
    <mesh_term>Hemosiderosis</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/NCT02985346
org study id: WTan
nct id: NCT02985346
lead sponsor: 
Pulmonary hemosiderosis (PH) is a pulmonary hemosiderin deposition which caused by alveolar
      capillary hemorrhage. PH is easy to recurrent and can lead to pulmonary fibrosis and
      insufficiency if the disease was poor controlled. Steroid is the most common drug that was
      administered in acute phase of the disease. However, considered the side-effects, steroid is
      not suitable for long-time maintenance. Therefore, it is necessary to explore a new therapy.
      Bone marrow mesenchymal stem cells (BMSC) are a kind of adult stem cells with high
      self-renewal and multi-directional differentiation potential in bone marrow. It has become a
      hot topic in immunosuppressive and tissue repair therapy in recent years. To date, homing,
      colonization and differentiation of BMSCs in the lung have been observed in animal models of
      pulmonary hypertension, radiation pneumonitis and pulmonary fibrosis. It had been reported
      that BMSC transplantation in acute lung injury in mice, inflammation of lung injury can
      significantly improve. The aim of this study is to explore the effect of BMSC on PH and its
      mechanism, and to explore a new way to promote the repair of IPH. It is expected to improve
      the status of IPH therapy in children, especially improve the prognosis of refractory PH.
allocation: Non-Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Complete remission
time frame: 3-6 months
arm group label: BMSC group
arm group type: Experimental
description: Patients received Bone marrow mesenchymal stem cells (BMSC) plus standard treatment
arm group label: Control group
arm group type: Active Comparator
description: Patients received standard treatment
intervention type: Biological
intervention name: Bone marrow mesenchymal stem cells in treatment of Idiopathic pulmonary hemosiderosis
arm group label: BMSC group
arm group label: Control group
criteria: 
gender: All
minimum age: 1 Month
maximum age: 18 Years
healthy volunteers: Accepts Healthy Volunteers
citation: Byrd RB, Gracey DR. Immunosuppressive treatment of idiopathic pulmonary hemosiderosis. JAMA. 1973 Oct 22;226(4):458-9. No abstract available.
PMID: 4800237
citation: Airaghi L, Ciceri L, Giannini S, Ferrero S, Meroni PL, Tedeschi A. Idiopathic pulmonary hemosiderosis in an adult. Favourable response to azathioprine. Monaldi Arch Chest Dis. 2001 Jun;56(3):211-3.
PMID: 11665500
citation: Calabrese F, Giacometti C, Rea F, Loy M, Sartori F, Di Vittorio G, Abudureheman A, Thiene G, Valente M. Recurrence of idiopathic pulmonary hemosiderosis in a young adult patient after bilateral single-lung transplantation. Transplantation. 2002 Dec 15;74(11):1643-5. doi: 10.1097/00007890-200212150-00027.
PMID: 12490803
citation: Chen RL, Chuang SS. Silent idiopathic pulmonary hemosiderosis with iron-deficiency anemia but normal serum ferritin. J Pediatr Hematol Oncol. 2007 Jul;29(7):509-11. doi: 10.1097/MPH.0b013e3180950372. No abstract available.
PMID: 17609633
citation: Rojas M, Xu J, Woods CR, Mora AL, Spears W, Roman J, Brigham KL. Bone marrow-derived mesenchymal stem cells in repair of the injured lung. Am J Respir Cell Mol Biol. 2005 Aug;33(2):145-52. doi: 10.1165/rcmb.2004-0330OC. Epub 2005 May 12.
PMID: 15891110
citation: Wang G, Bunnell BA, Painter RG, Quiniones BC, Tom S, Lanson NA Jr, Spees JL, Bertucci D, Peister A, Weiss DJ, Valentine VG, Prockop DJ, Kolls JK. Adult stem cells from bone marrow stroma differentiate into airway epithelial cells: potential therapy for cystic fibrosis. Proc Natl Acad Sci U S A. 2005 Jan 4;102(1):186-91. doi: 10.1073/pnas.0406266102. Epub 2004 Dec 22.
PMID: 15615854
citation: Lee JW, Fang X, Gupta N, Serikov V, Matthay MA. Allogeneic human mesenchymal stem cells for treatment of E. coli endotoxin-induced acute lung injury in the ex vivo perfused human lung. Proc Natl Acad Sci U S A. 2009 Sep 22;106(38):16357-62. doi: 10.1073/pnas.0907996106. Epub 2009 Aug 31.
PMID: 19721001
citation: Knight DA, Rossi FM, Hackett TL. Mesenchymal stem cells for repair of the airway epithelium in asthma. Expert Rev Respir Med. 2010 Dec;4(6):747-58. doi: 10.1586/ers.10.72.
PMID: 21128750
citation: Hoffman AM, Paxson JA, Mazan MR, Davis AM, Tyagi S, Murthy S, Ingenito EP. Lung-derived mesenchymal stromal cell post-transplantation survival, persistence, paracrine expression, and repair of elastase-injured lung. Stem Cells Dev. 2011 Oct;20(10):1779-92. doi: 10.1089/scd.2011.0105. Epub 2011 Jul 6.
PMID: 21585237
citation: Liu AR, Liu L, Chen S, Yang Y, Zhao HJ, Liu L, Guo FM, Lu XM, Qiu HB. Activation of canonical wnt pathway promotes differentiation of mouse bone marrow-derived MSCs into type II alveolar epithelial cells, confers resistance to oxidative stress, and promotes their migration to injured lung tissue in vitro. J Cell Physiol. 2013 Jun;228(6):1270-83. doi: 10.1002/jcp.24282.
PMID: 23154940
responsible party type: Principal Investigator
investigator affiliation: Sun Yat-sen University
investigator full name: Weiping Tan
investigator title: Sun Yat-sen Memorial Hospital
mesh term: Lung Diseases
mesh term: Hemochromatosis
mesh term: Hemosiderosis