<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/NCT04476550</url>
  </required_header>
  <id_info>
    <org_study_id>SRPH-LP-02/PoD</org_study_id>
    <nct_id>NCT04476550</nct_id>
  </id_info>
  <brief_title>Clinical Specimen Collection From Pompe Disease Patients</brief_title>
  <official_title>Developing a Potential Cure for Pompe Disease: Clinical Specimen Collection From Individuals With Pompe Disease</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Serhat Gumrukcu, MD PhD</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Frida Therapeutics LLC</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Seraph Research Institute</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>
      Clinical specimens are required from individuals with Pompe Disease to support process and&#xD;
      analytical development for a genetically modified autologous bone marrow cell product&#xD;
      currently in preclinical research, FTX-PD01. The intent is for this product to be&#xD;
      investigated in a subsequent clinical trial under a future FDA IND to treat Pompe Disease.&#xD;
      Enrolled participants provide a venous blood specimen (approximately 20mL) to be used in&#xD;
      preclinical studies and research and development of FTX-PD01. Subjects may eventually be&#xD;
      asked to undergo mobilized leukapheresis for bone marrow stem cell collection and their&#xD;
      specimens will be used to further develop the FTX-PD01 cell product, including a cGMP&#xD;
      compliant process to be applied under the future FDA IND.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This protocol is to collect blood and HSPC specimens from individuals with Pompe Disease. The&#xD;
      first blood draw will be done at the first study visit and if eligible, the second collection&#xD;
      will be done via mobilized leukapheresis at the second visit. The mobilized leukapheresis&#xD;
      procedures will follow the facility's standard operating procedures and protocol requirements&#xD;
      for mobilized leukapheresis.&#xD;
&#xD;
      Donors will be males or females between and including the ages of 3 years and 30 years.&#xD;
      Volunteers will provide written informed consent and meet all inclusion and exclusion&#xD;
      criteria. Each participant can be in the study for up to 120 days (3 months).&#xD;
&#xD;
      The study will be conducted in accordance with human research for the purposes of obtaining&#xD;
      clinical specimens for research. There is no endpoint for this study, however, data collected&#xD;
      from this study will include, but not be limited to, gender, demographics, medical history,&#xD;
      clinical laboratory values, and volume of the blood collected. The data will be summarized in&#xD;
      future studies reporting results from a future clinical trial under FDA IND.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Recruiting</overall_status>
  <start_date type="Anticipated">September 1, 2022</start_date>
  <completion_date type="Anticipated">May 31, 2023</completion_date>
  <primary_completion_date type="Anticipated">March 1, 2023</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Case-Only</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Collection of blood</measure>
    <time_frame>Up to 20 days</time_frame>
    <description>Collection of peripheral blood (up to 20ml)</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Collection of HSPCs</measure>
    <time_frame>Up to 100 days</time_frame>
    <description>Collection of HSPCs through G-CSF mobilized leukapheresis</description>
  </secondary_outcome>
  <enrollment type="Anticipated">12</enrollment>
  <condition>Pompe Disease</condition>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Filgrastim</intervention_name>
    <description>5 days of daily sub-cutaneous administration of 10mcg/kg filgrastim</description>
    <other_name>Granulocyte-Colony Stimulating Factor (G-CSF)</other_name>
  </intervention>
  <biospec_retention>Samples With DNA</biospec_retention>
  <biospec_descr>
    <textblock>
      Mononuclear cells with GAA gene mutation&#xD;
    </textblock>
  </biospec_descr>
  <eligibility>
    <study_pop>
      <textblock>
        Otherwise healthy individuals diagnosed with Pompe Disease.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Male of female aged 3-30&#xD;
&#xD;
          -  Documented diagnosis of Pompe Disease&#xD;
&#xD;
          -  Participants who has not participated in a cell or gene therapy trial for Pompe&#xD;
             Disease&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Active acute infection at screening&#xD;
&#xD;
          -  Uncontrolled diabetes&#xD;
&#xD;
          -  Uncontrolled hypertension&#xD;
&#xD;
          -  Active DIC, bleeding or coagulopathy which cannot be corrected with minimal&#xD;
             intervention&#xD;
&#xD;
          -  Symptomatic, uncontrolled or severe intercurrent illness that would compromise the&#xD;
             ability to tolerate blood collection or mobilized leukapheresis procedure&#xD;
&#xD;
          -  Systemic chemotherapy less than or equal to 2 weeks (6 weeks for clofarabine or&#xD;
             nitrosoureas) or radiation therapy less than or equal to 3 weeks prior to&#xD;
             leukapheresis&#xD;
&#xD;
          -  Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a&#xD;
             female after conception and until the termination of gestation, confirmed by a&#xD;
             positive hCG laboratory test at screening&#xD;
&#xD;
          -  Any patient that in the opinion of the investigator is not medically stable to undergo&#xD;
             the leukapheresis procedure or will not comply with the visit schedules or procedures&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>3 Years</minimum_age>
    <maximum_age>30 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Serhat Gumrukcu, MD PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Seraph Research Institute</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Gregory T Howell, BA Psy</last_name>
    <phone>424-274-3211</phone>
    <email>greg@seraphmed.com</email>
  </overall_contact>
  <location>
    <facility>
      <name>Seraph Research Institute</name>
      <address>
        <city>Toluca Lake</city>
        <state>California</state>
        <zip>91602</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Anna Hurtado</last_name>
      <phone>424-274-3211</phone>
      <email>anna@seraphmd.com</email>
    </contact>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>May 2022</verification_date>
  <study_first_submitted>February 26, 2020</study_first_submitted>
  <study_first_submitted_qc>July 14, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">July 20, 2020</study_first_posted>
  <last_update_submitted>May 17, 2022</last_update_submitted>
  <last_update_submitted_qc>May 17, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">May 19, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor-Investigator</responsible_party_type>
    <investigator_affiliation>Seraph Research Institute</investigator_affiliation>
    <investigator_full_name>Serhat Gumrukcu, MD PhD</investigator_full_name>
    <investigator_title>Sponsor-Investigator</investigator_title>
  </responsible_party>
  <keyword>Pompe</keyword>
  <keyword>Pompe Disease</keyword>
  <keyword>HSPC</keyword>
  <keyword>modified bone marrow stem cell</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Glycogen Storage Disease Type II</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Lenograstim</mesh_term>
  </intervention_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <!-- 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/NCT04476550
org study id: SRPH-LP-02/PoD
nct id: NCT04476550
lead sponsor: 
collaborator: 
has dmc: Yes
is fda regulated drug: No
is fda regulated device: No
Clinical specimens are required from individuals with Pompe Disease to support process and
      analytical development for a genetically modified autologous bone marrow cell product
      currently in preclinical research, FTX-PD01. The intent is for this product to be
      investigated in a subsequent clinical trial under a future FDA IND to treat Pompe Disease.
      Enrolled participants provide a venous blood specimen (approximately 20mL) to be used in
      preclinical studies and research and development of FTX-PD01. Subjects may eventually be
      asked to undergo mobilized leukapheresis for bone marrow stem cell collection and their
      specimens will be used to further develop the FTX-PD01 cell product, including a cGMP
      compliant process to be applied under the future FDA IND.
This protocol is to collect blood and HSPC specimens from individuals with Pompe Disease. The
      first blood draw will be done at the first study visit and if eligible, the second collection
      will be done via mobilized leukapheresis at the second visit. The mobilized leukapheresis
      procedures will follow the facility's standard operating procedures and protocol requirements
      for mobilized leukapheresis.

      Donors will be males or females between and including the ages of 3 years and 30 years.
      Volunteers will provide written informed consent and meet all inclusion and exclusion
      criteria. Each participant can be in the study for up to 120 days (3 months).

      The study will be conducted in accordance with human research for the purposes of obtaining
      clinical specimens for research. There is no endpoint for this study, however, data collected
      from this study will include, but not be limited to, gender, demographics, medical history,
      clinical laboratory values, and volume of the blood collected. The data will be summarized in
      future studies reporting results from a future clinical trial under FDA IND.
observational model: Case-Only
time perspective: Prospective
measure: Collection of blood
time frame: Up to 20 days
description: Collection of peripheral blood (up to 20ml)
measure: Collection of HSPCs
time frame: Up to 100 days
description: Collection of HSPCs through G-CSF mobilized leukapheresis
intervention type: Drug
intervention name: Filgrastim
description: 5 days of daily sub-cutaneous administration of 10mcg/kg filgrastim
other name: Granulocyte-Colony Stimulating Factor (G-CSF)
Mononuclear cells with GAA gene mutation
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 3 Years
maximum age: 30 Years
healthy volunteers: No
last name: Serhat Gumrukcu, MD PhD
role: Principal Investigator
affiliation: Seraph Research Institute
last name: Gregory T Howell, BA Psy
phone: 424-274-3211
email: greg@seraphmed.com
facility: 
status: Recruiting
contact: 
country: United States
responsible party type: Sponsor-Investigator
investigator affiliation: Seraph Research Institute
investigator full name: Serhat Gumrukcu, MD PhD
investigator title: Sponsor-Investigator
mesh term: Glycogen Storage Disease Type II
mesh term: Lenograstim
sharing ipd: No