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/NCT05986734</url>
    </required_header>
    <id_info>
      <org_study_id>NCPHOI-2022-09</org_study_id>
      <nct_id>NCT05986734</nct_id>
    </id_info>
    <brief_title>Evaluation of Subcutaneous Immunoglobulin Product Cutaquig in Terms of Safety and Efficacy in the Treatment of Patients With Primary Immunodeficiencies</brief_title>
    <official_title>Evaluation of Subcutaneous Immunoglobulin Product Cutaquig in Terms of Safety and Efficacy in the Treatment of Patients With Primary Immunodeficiencies</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Federal Research Institute of Pediatric Hematology, Oncology and Immunology</agency>
        <agency_class>Other</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>Federal Research Institute of Pediatric Hematology, Oncology and Immunology</source>
    <oversight_info>
      <is_fda_regulated_drug>No</is_fda_regulated_drug>
      <is_fda_regulated_device>No</is_fda_regulated_device>
    </oversight_info>
    <brief_summary>
      <textblock>
        In the prospective part of the study, patients diagnosed with PID will receive replacement&#xD;
        therapy with Cutaquig for at least 6 months and will be randomised with the help of&#xD;
        physicians of the Russian PID Registry. At the beginning of the study, data on the prior&#xD;
        intravenous immunoglobulin (IVIG) therapy efficacy will be extracted from patients' medical&#xD;
        records (retrospective part of study). Also, at the beginning of the study, patients or their&#xD;
        parents will be asked to complete Quality of Life Questionnaire for children, version 4.0;&#xD;
        short form in Russian - Pediatric Quality of Life Inventory Russian, Version 4.0 (Russia),&#xD;
        PedSQL (Appendix 14.1). During the 6 months of treatment with Cutaquig, investigators will&#xD;
        enter information on infectious episodes, IgG levels (at least 3 times during the study&#xD;
        period), and definition of an adverse drug reaction / serious adverse drug reaction, in the&#xD;
        database. After 6 months, patients/their parents will be asked to complete the PedSQL,&#xD;
        Quality of Life Questionnaire again. If the patient continues to receive the drug, and&#xD;
        patient and his/her legally acceptable representatives (for patients under 14 years of age)&#xD;
        agree to continue participating in the study, similar information will be collected for&#xD;
        another 6 months. The first 6 months are planned for the main study period, 6-12 months for&#xD;
        an extended study period. Data obtained in the prospective phase of the study will be&#xD;
        compared with similar data obtained in the retrospective phase in the same patients&#xD;
      </textblock>
    </brief_summary>
    <detailed_description>
      <textblock>
        In the prospective part of the study, patients diagnosed with PID will receive replacement&#xD;
        therapy with Cutaquig for at least 6 months and will be randomised with the help of&#xD;
        physicians of the Russian PID Registry. At the beginning of the study, data on the prior&#xD;
        intravenous immunoglobulin (IVIG) therapy efficacy will be extracted from patients' medical&#xD;
        records (retrospective part of study). Also, at the beginning of the study, patients or their&#xD;
        parents will be asked to complete Quality of Life Questionnaire for children, version 4.0;&#xD;
        short form in Russian - Pediatric Quality of Life Inventory Russian, Version 4.0 (Russia),&#xD;
        PedSQL (Appendix 14.1). During the 6 months of treatment with Cutaquig, investigators will&#xD;
        enter information on infectious episodes, IgG levels (at least 3 times during the study&#xD;
        period), and definition of an adverse drug reaction / serious adverse drug reaction, in the&#xD;
        database. After 6 months, patients/their parents will be asked to complete the PedSQL,&#xD;
        Quality of Life Questionnaire again. If the patient continues to receive the drug, and&#xD;
        patient and his/her legally acceptable representatives (for patients under 14 years of age)&#xD;
        agree to continue participating in the study, similar information will be collected for&#xD;
        another 6 months. The first 6 months are planned for the main study period, 6-12 months for&#xD;
        an extended study period. Data obtained in the prospective phase of the study will be&#xD;
        compared with similar data obtained in the retrospective phase in the same patients.&#xD;
  &#xD;
        Considering the lack of the study drug marketing authorisation in the Russian Federation,&#xD;
        study drug doses and treatment regimens were chosen as per current clinical practice and&#xD;
        instructions for human use approved in other countries. Conventionally, on switching from&#xD;
        IVIG therapy, the subcutaneous immunoglobulin products are administered at the same monthly&#xD;
        dose (0.4-0.8 g/kg), evenly divided into 3-5 administrations per month, subject to monitoring&#xD;
        of the pre transfusion IgG level with target IgG values of at least 7 g/L. All this&#xD;
        information will be reflected in the Case record forms (CRF). Besides, information about&#xD;
        therapeutic strategies, such as dose change or therapy change, will also be entered in the&#xD;
        CRF.&#xD;
      </textblock>
    </detailed_description>
    <overall_status>Recruiting</overall_status>
    <start_date type="Actual">September 1, 2022</start_date>
    <completion_date type="Anticipated">December 1, 2023</completion_date>
    <primary_completion_date type="Anticipated">July 31, 2023</primary_completion_date>
    <study_type>Observational</study_type>
    <has_expanded_access>No</has_expanded_access>
    <study_design_info>
      <observational_model>Cohort</observational_model>
      <time_perspective>Prospective</time_perspective>
    </study_design_info>
    <primary_outcome>
      <measure>comparative characteristics of serum IgG concentrations in IVIG and SCIG therapy</measure>
      <time_frame>after 3 months from the start</time_frame>
      <description>analysis of the results of the study after 3 months from the start of subcutaneous immunoglobulin therapy: assessment of IgG concentration over time, box plot</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Comparison of the average number of infectious complications per person per month on IVIG and SCIG therapy</measure>
      <time_frame>after 3 months from the start</time_frame>
      <description>analysis of the results of the study after 3 months from the start of subcutaneous immunoglobulin therapy: the frequency of infectious complications, box plot</description>
    </primary_outcome>
    <secondary_outcome>
      <measure>Pre-transfusion IgG level</measure>
      <time_frame>6 months from study enrollment</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Frequency of unscheduled antibiotic use during the study drug therapy</measure>
      <time_frame>6 months from study enrollment</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Duration of unscheduled antibiotic use during the study drug therapy</measure>
      <time_frame>6 months from study enrollment</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>The number of hospitalisations due to infectious diseases during the study drug therapy.</measure>
      <time_frame>6 months from study enrollment</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Length of hospital stay due to infectious diseases during the study drug therapy</measure>
      <time_frame>6 months from study enrollment</time_frame>
    </secondary_outcome>
    <enrollment type="Anticipated">100</enrollment>
    <condition>Primary Immunodeficiency Diseases (PID)</condition>
    <intervention>
      <intervention_type>Drug</intervention_type>
      <intervention_name>Cutaquig</intervention_name>
      <description>Subcutaneous immunoglobulin products are administered at the same monthly dose (0.4-0.8 g/kg), evenly divided into 3-5 administrations per month, subject to monitoring of the pre transfusion IgG level with target IgG values of at least 7 g/L.</description>
      <other_name>subcutaneous immunoglobulin</other_name>
      <other_name>Octapharma</other_name>
    </intervention>
    <eligibility>
      <study_pop>
        <textblock>
          patients diagnosed with PID. Patients of both sexes over the age of 0 years with a verified&#xD;
          diagnosis of primary immunodeficiency, who require regular immunoglobulin replacement&#xD;
          therapy&#xD;
        </textblock>
      </study_pop>
      <sampling_method>Probability Sample</sampling_method>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
            -  Patients of both sexes over the age of 0 years with a verified diagnosis of primary&#xD;
               immunodeficiency, who require regular immunoglobulin replacement therapy&#xD;
  &#xD;
            -  Patients' and/or their legal representatives' (for patients under 14 years of age)&#xD;
               consent to participate in the study&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            -  Hypersensitivity to the study drug or any component of the study drug&#xD;
  &#xD;
            -  Active oncological disease&#xD;
  &#xD;
            -  Condition after haematopoietic stem cell transplantation&#xD;
  &#xD;
            -  Use of other immunoglobulin products during the study drug therapy.&#xD;
  &#xD;
            -  Discontinuation or irregular use of the study drug&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>1 Day</minimum_age>
      <maximum_age>18 Years</maximum_age>
    </eligibility>
    <overall_official>
      <last_name>Anna Shcherbina, MD,Phd</last_name>
      <role>Study Director</role>
      <affiliation>Chief HSCT department at Federal Research Center for pediatric hematology, oncology and immunology</affiliation>
    </overall_official>
    <overall_contact>
      <last_name>Elena f Deripapa, PD</last_name>
      <phone>89067777058</phone>
      <email>deripapa@mail.ru</email>
    </overall_contact>
    <location>
      <facility>
        <name>Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology</name>
        <address>
          <city>Moscow</city>
          <zip>117997</zip>
          <country>Russian Federation</country>
        </address>
      </facility>
      <status>Recruiting</status>
      <contact>
        <last_name>Anna Shcherbina, MD, PhD</last_name>
        <phone>+7(495)2876570</phone>
        <phone_ext>6299</phone_ext>
        <email>shcher26@hotmail.com</email>
      </contact>
    </location>
    <location_countries>
      <country>Russian Federation</country>
    </location_countries>
    <verification_date>March 2023</verification_date>
    <study_first_submitted>March 6, 2023</study_first_submitted>
    <study_first_submitted_qc>August 8, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">August 14, 2023</study_first_posted>
    <last_update_submitted>August 8, 2023</last_update_submitted>
    <last_update_submitted_qc>August 8, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">August 14, 2023</last_update_posted>
    <responsible_party>
      <responsible_party_type>Sponsor</responsible_party_type>
    </responsible_party>
    <keyword>Primary immunodeficiency diseases</keyword>
    <keyword>Cutaquig</keyword>
    <keyword>intravenous immunoglobulin</keyword>
    <keyword>subcutaneous immunoglobulin</keyword>
    <condition_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Primary Immunodeficiency 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>
    </intervention_browse>
    <patient_data>
      <sharing_ipd>No</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/NCT05986734
  org study id: NCPHOI-2022-09
  nct id: NCT05986734
  lead sponsor: 
  is fda regulated drug: No
  is fda regulated device: No
  In the prospective part of the study, patients diagnosed with PID will receive replacement
        therapy with Cutaquig for at least 6 months and will be randomised with the help of
        physicians of the Russian PID Registry. At the beginning of the study, data on the prior
        intravenous immunoglobulin (IVIG) therapy efficacy will be extracted from patients' medical
        records (retrospective part of study). Also, at the beginning of the study, patients or their
        parents will be asked to complete Quality of Life Questionnaire for children, version 4.0;
        short form in Russian - Pediatric Quality of Life Inventory Russian, Version 4.0 (Russia),
        PedSQL (Appendix 14.1). During the 6 months of treatment with Cutaquig, investigators will
        enter information on infectious episodes, IgG levels (at least 3 times during the study
        period), and definition of an adverse drug reaction / serious adverse drug reaction, in the
        database. After 6 months, patients/their parents will be asked to complete the PedSQL,
        Quality of Life Questionnaire again. If the patient continues to receive the drug, and
        patient and his/her legally acceptable representatives (for patients under 14 years of age)
        agree to continue participating in the study, similar information will be collected for
        another 6 months. The first 6 months are planned for the main study period, 6-12 months for
        an extended study period. Data obtained in the prospective phase of the study will be
        compared with similar data obtained in the retrospective phase in the same patients
  In the prospective part of the study, patients diagnosed with PID will receive replacement
        therapy with Cutaquig for at least 6 months and will be randomised with the help of
        physicians of the Russian PID Registry. At the beginning of the study, data on the prior
        intravenous immunoglobulin (IVIG) therapy efficacy will be extracted from patients' medical
        records (retrospective part of study). Also, at the beginning of the study, patients or their
        parents will be asked to complete Quality of Life Questionnaire for children, version 4.0;
        short form in Russian - Pediatric Quality of Life Inventory Russian, Version 4.0 (Russia),
        PedSQL (Appendix 14.1). During the 6 months of treatment with Cutaquig, investigators will
        enter information on infectious episodes, IgG levels (at least 3 times during the study
        period), and definition of an adverse drug reaction / serious adverse drug reaction, in the
        database. After 6 months, patients/their parents will be asked to complete the PedSQL,
        Quality of Life Questionnaire again. If the patient continues to receive the drug, and
        patient and his/her legally acceptable representatives (for patients under 14 years of age)
        agree to continue participating in the study, similar information will be collected for
        another 6 months. The first 6 months are planned for the main study period, 6-12 months for
        an extended study period. Data obtained in the prospective phase of the study will be
        compared with similar data obtained in the retrospective phase in the same patients.
  
        Considering the lack of the study drug marketing authorisation in the Russian Federation,
        study drug doses and treatment regimens were chosen as per current clinical practice and
        instructions for human use approved in other countries. Conventionally, on switching from
        IVIG therapy, the subcutaneous immunoglobulin products are administered at the same monthly
        dose (0.4-0.8 g/kg), evenly divided into 3-5 administrations per month, subject to monitoring
        of the pre transfusion IgG level with target IgG values of at least 7 g/L. All this
        information will be reflected in the Case record forms (CRF). Besides, information about
        therapeutic strategies, such as dose change or therapy change, will also be entered in the
        CRF.
  observational model: Cohort
  time perspective: Prospective
  measure: comparative characteristics of serum IgG concentrations in IVIG and SCIG therapy
  time frame: after 3 months from the start
  description: analysis of the results of the study after 3 months from the start of subcutaneous immunoglobulin therapy: assessment of IgG concentration over time, box plot
  measure: Comparison of the average number of infectious complications per person per month on IVIG and SCIG therapy
  time frame: after 3 months from the start
  description: analysis of the results of the study after 3 months from the start of subcutaneous immunoglobulin therapy: the frequency of infectious complications, box plot
  measure: Pre-transfusion IgG level
  time frame: 6 months from study enrollment
  measure: Frequency of unscheduled antibiotic use during the study drug therapy
  time frame: 6 months from study enrollment
  measure: Duration of unscheduled antibiotic use during the study drug therapy
  time frame: 6 months from study enrollment
  measure: The number of hospitalisations due to infectious diseases during the study drug therapy.
  time frame: 6 months from study enrollment
  measure: Length of hospital stay due to infectious diseases during the study drug therapy
  time frame: 6 months from study enrollment
  intervention type: Drug
  intervention name: Cutaquig
  description: Subcutaneous immunoglobulin products are administered at the same monthly dose (0.4-0.8 g/kg), evenly divided into 3-5 administrations per month, subject to monitoring of the pre transfusion IgG level with target IgG values of at least 7 g/L.
  other name: subcutaneous immunoglobulin
  other name: Octapharma
  study pop: 
  sampling method: Probability Sample
  criteria: 
  gender: All
  minimum age: 1 Day
  maximum age: 18 Years
  last name: Anna Shcherbina, MD,Phd
  role: Study Director
  affiliation: Chief HSCT department at Federal Research Center for pediatric hematology, oncology and immunology
  last name: Elena f Deripapa, PD
  phone: 89067777058
  email: deripapa@mail.ru
  facility: 
  status: Recruiting
  contact: 
  country: Russian Federation
  responsible party type: Sponsor
  mesh term: Primary Immunodeficiency Diseases
  mesh term: Immunologic Deficiency Syndromes
  mesh term: Immunoglobulins
  sharing ipd: No