<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/NCT03238079</url>
  </required_header>
  <id_info>
    <org_study_id>TBI-001-IGIV</org_study_id>
    <nct_id>NCT03238079</nct_id>
  </id_info>
  <brief_title>Study of PlasmaCap IG in Adults and Children With PIDD</brief_title>
  <official_title>A Prospective, Open-Label, Multicenter Study of the Efficacy, Safety, Tolerability, and Pharmacokinetics of Therapure PlasmaCap IG in Adults and Children With Primary Immune Deficiency Diseases</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Therapure Biopharma Inc</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Therapure Biopharma Inc</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this study is to investigate the efficacy, safety, tolerability, and&#xD;
      pharmacokinetic profile of the investigational medicinal product (IMP) and to determine, on&#xD;
      the basis of historical control data, how it compares with other 10% intravenous&#xD;
      immunoglobulin (IGIV) products currently licensed in North America for the treatment of&#xD;
      subjects with primary immune deficiency diseases (PIDD).&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Active, not recruiting</last_known_status>
  <start_date type="Actual">September 5, 2017</start_date>
  <completion_date type="Anticipated">December 30, 2020</completion_date>
  <primary_completion_date type="Anticipated">August 25, 2020</primary_completion_date>
  <phase>Phase 3</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>Mean acute Serious Bacterial Infection (SBI) rate</measure>
    <time_frame>1 year</time_frame>
    <description>The primary efficacy objective of the study is to demonstrate the efficacy of the IMP by determining that the mean annual acute SBI rate (as defined in Appendix 20.1) is statistically significantly lower than one infection per subject per year.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Immunoglobulin G (IgG) trough concentration</measure>
    <time_frame>up to 12 months per subject</time_frame>
    <description>The average serum total IgG trough concentrations prior to each infusion</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Days unable to perform daily activities</measure>
    <time_frame>up to 12 months per subject</time_frame>
    <description>The number of days unable to perform daily activities</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Therapeutic IgG levels</measure>
    <time_frame>up to 12 months per subject</time_frame>
    <description>The ability of the IMP to maintain stable, therapeutic IgG levels</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">74</enrollment>
  <condition>Primary Immune Deficiency Diseases (PIDD)</condition>
  <arm_group>
    <arm_group_label>Open-Label 10% IGIV</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>IMP will be administered every 21 or 28 days in accordance with the subject's weekly regimen at screening for a period of 12 months. Subjects on a 21-day regimen will receive approximately 17 infusions, and subjects on a 28-day regimen will receive approximately 13 infusions.&#xD;
The starting dose will be the previous IGIV dose or a dose calculated from the previous SCIG dose up to a maximum of 900 mg/kg/mo.</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>10% IGIV</intervention_name>
    <description>300-900 mg/kg</description>
    <arm_group_label>Open-Label 10% IGIV</arm_group_label>
    <other_name>Human Immunoglobulin</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Subject has a confirmed clinical diagnosis of a PIDD, which requires treatment with&#xD;
             IGIV:&#xD;
&#xD;
          -  Subject/guardian has provided written informed consent (and assent, as applicable).&#xD;
&#xD;
          -  Subject is between the ages of 2 and 70 years.&#xD;
&#xD;
          -  Subject has received regular IGIV therapy at 21- or 28-day (±4 days) intervals for at&#xD;
             least three consecutive months at a dose between 300-900 mg/kg/month prior to&#xD;
             Screening or;&#xD;
&#xD;
          -  Subject has received commercial SCIG at a dose of 300-900 mg/kg/month on any dosing&#xD;
             schedule for at least 12 consecutive weeks prior to Screening. Subjects on SCIG must&#xD;
             have received and tolerated IGIV treatment prior to SCIG treatment.&#xD;
&#xD;
          -  Subject has a documented trough of ≥500 mg/dL in the 6 months prior to screening.&#xD;
&#xD;
          -  Females of childbearing potential must be willing to use an effective form of birth&#xD;
             control (eg, oral contraceptives) for the duration of the study, per IRB/REB&#xD;
             guidelines.&#xD;
&#xD;
          -  Subject agrees to comply with the requirements of the protocol.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Subject has secondary immunodeficiency.&#xD;
&#xD;
          -  Subject has history of thrombotic events, such as deep vein thrombosis, myocardial&#xD;
             infarction, cerebrovascular accident, pulmonary embolism, etc within the year prior to&#xD;
             screening.&#xD;
&#xD;
          -  Subject has had an immune globulin associated arterial or venous&#xD;
             thrombotic/thromboembolic event (TEE) within 7 days of infusion or a TEE that is not&#xD;
             associated with an immune globulin within one year of screening.&#xD;
&#xD;
          -  Subject has received blood products (except for IGIV, SCIG, or albumin) within 6&#xD;
             months of screening.&#xD;
&#xD;
          -  Subject has anemia (≤8.5 g/dL).&#xD;
&#xD;
          -  Subject has levels of alanine aminotransferase (ALT) or aspartate aminotransferase&#xD;
             (AST) &gt;3.0 times the upper limit of normal (ULN).&#xD;
&#xD;
          -  Subject has severe neutropenia (≤1000 neutrophils per mm3).&#xD;
&#xD;
          -  Subject is receiving other immunosuppressive or immunomodulatory drugs or&#xD;
             chemotherapy.&#xD;
&#xD;
          -  Subject is taking or has taken within the four weeks prior to screening prednisone at&#xD;
             ≥0.15 mg/kg/day for more than 10 days.&#xD;
&#xD;
          -  Subject has ever had a severe anaphylactic reaction to a blood or IgG product.&#xD;
&#xD;
          -  Subject has lymphoid malignancy, leukemia, or any other history of malignancy within&#xD;
             the past five years, except squamous cell or basal cell carcinoma of the skin (not&#xD;
             melanoma).&#xD;
&#xD;
          -  Subject has hypoalbuminemia, protein-losing enteropathy, or proteinuria greater than&#xD;
             300 mg/24 hours except for subjects with documented orthostatic proteinuria.&#xD;
&#xD;
          -  Subject has immunoglobulin A (IgA) deficiency with known antibodies to IgA.&#xD;
&#xD;
          -  Female who is pregnant, breastfeeding, or planning a pregnancy during the course of&#xD;
             the study (women who become pregnant during the study will be withdrawn from the&#xD;
             study).&#xD;
&#xD;
          -  Any condition that is likely to interfere with evaluation of IMP or satisfactory&#xD;
             conduct of the trial in the PI's opinion.&#xD;
&#xD;
          -  Subjects who may not be compliant or have a history of non-compliance in the opinion&#xD;
             of the PI.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>2 Years</minimum_age>
    <maximum_age>70 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Mark Krause</last_name>
    <role>Study Director</role>
    <affiliation>Therapure Biopharma</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>University of California</name>
      <address>
        <city>Los Angeles</city>
        <state>California</state>
        <zip>92697</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IMMUNOe Health &amp; Research Centers</name>
      <address>
        <city>Centennial</city>
        <state>Colorado</state>
        <zip>80112</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of South Florida</name>
      <address>
        <city>Tampa</city>
        <state>Florida</state>
        <zip>33620</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Allergy Associates of the Palm Beaches, P.A.</name>
      <address>
        <city>West Palm Beach</city>
        <state>Florida</state>
        <zip>33408</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Institute for Asthma and Allergy, PC</name>
      <address>
        <city>Chevy Chase</city>
        <state>Maryland</state>
        <zip>20815</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Optimed Research Ltd.</name>
      <address>
        <city>Little Silver</city>
        <state>New Jersey</state>
        <zip>07739</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Optimed Research LTD</name>
      <address>
        <city>Columbus</city>
        <state>Ohio</state>
        <zip>43235</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Allergy Partners of North Texas</name>
      <address>
        <city>Dallas</city>
        <state>Texas</state>
        <zip>75230</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>AARA Research Center</name>
      <address>
        <city>Dallas</city>
        <state>Texas</state>
        <zip>75231</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>AAICPA</name>
      <address>
        <city>Irving</city>
        <state>Texas</state>
        <zip>75063</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The Medical College of Wisconsin, Inc.</name>
      <address>
        <city>Milwaukee</city>
        <state>Wisconsin</state>
        <zip>53226</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>CHU Ste-Justine</name>
      <address>
        <city>Montréal</city>
        <state>Quebec</state>
        <zip>H3T 1C5</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Canada</country>
    <country>United States</country>
  </location_countries>
  <verification_date>May 2020</verification_date>
  <study_first_submitted>July 21, 2017</study_first_submitted>
  <study_first_submitted_qc>July 31, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">August 3, 2017</study_first_posted>
  <last_update_submitted>May 6, 2020</last_update_submitted>
  <last_update_submitted_qc>May 6, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">May 7, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Primary Immunodeficiency Diseases</mesh_term>
    <mesh_term>Deficiency Diseases</mesh_term>
    <mesh_term>Immunologic Deficiency Syndromes</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Immunoglobulins</mesh_term>
    <mesh_term>Immunoglobulins, Intravenous</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/NCT03238079
org study id: TBI-001-IGIV
nct id: NCT03238079
lead sponsor: 
has dmc: No
is fda regulated drug: Yes
is fda regulated device: No
The purpose of this study is to investigate the efficacy, safety, tolerability, and
      pharmacokinetic profile of the investigational medicinal product (IMP) and to determine, on
      the basis of historical control data, how it compares with other 10% intravenous
      immunoglobulin (IGIV) products currently licensed in North America for the treatment of
      subjects with primary immune deficiency diseases (PIDD).
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Mean acute Serious Bacterial Infection (SBI) rate
time frame: 1 year
description: The primary efficacy objective of the study is to demonstrate the efficacy of the IMP by determining that the mean annual acute SBI rate (as defined in Appendix 20.1) is statistically significantly lower than one infection per subject per year.
measure: Immunoglobulin G (IgG) trough concentration
time frame: up to 12 months per subject
description: The average serum total IgG trough concentrations prior to each infusion
measure: Days unable to perform daily activities
time frame: up to 12 months per subject
description: The number of days unable to perform daily activities
measure: Therapeutic IgG levels
time frame: up to 12 months per subject
description: The ability of the IMP to maintain stable, therapeutic IgG levels
arm group label: Open-Label 10% IGIV
arm group type: Experimental
description: IMP will be administered every 21 or 28 days in accordance with the subject's weekly regimen at screening for a period of 12 months. Subjects on a 21-day regimen will receive approximately 17 infusions, and subjects on a 28-day regimen will receive approximately 13 infusions.
The starting dose will be the previous IGIV dose or a dose calculated from the previous SCIG dose up to a maximum of 900 mg/kg/mo.
intervention type: Biological
intervention name: 10% IGIV
description: 300-900 mg/kg
arm group label: Open-Label 10% IGIV
other name: Human Immunoglobulin
criteria: 
gender: All
minimum age: 2 Years
maximum age: 70 Years
healthy volunteers: No
last name: Mark Krause
role: Study Director
affiliation: Therapure Biopharma
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
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facility: 
country: Canada
country: United States
responsible party type: Sponsor
mesh term: Primary Immunodeficiency Diseases
mesh term: Deficiency Diseases
mesh term: Immunologic Deficiency Syndromes
mesh term: Immunoglobulins
mesh term: Immunoglobulins, Intravenous
sharing ipd: No