<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/NCT01721681</url>
  </required_header>
  <id_info>
    <org_study_id>Ten 02</org_study_id>
    <secondary_id>2012-003093-98</secondary_id>
    <nct_id>NCT01721681</nct_id>
  </id_info>
  <brief_title>A Study to Investigate Bio Product Laboratory Ltd (BPL's) Factor X in the Prophylaxis of Bleeding in Children &lt;12 Years</brief_title>
  <official_title>A Phase III Open, Multicentre Study to Investigate the Safety, Pharmacokinetics and Efficacy of BPL's High Purity Factor X in the Prophylaxis of Bleeding in Factor X Deficient Children Under the Age of 12 Years</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Bio Products Laboratory</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Bio Products Laboratory</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The primary objective of the study is to assess the efficacy of FACTOR X in the prevention of&#xD;
      bleeding when given as routine prophylaxis over 12 months.&#xD;
&#xD;
      The secondary objectives of the study are:&#xD;
&#xD;
        1. To assess the pharmacokinetics of FACTOR X after a single dose of 50 IU/kg.&#xD;
&#xD;
        2. To assess the safety of FACTOR X when given as routine prophylaxis over 6 months (26&#xD;
           weeks).&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date>April 2015</start_date>
  <completion_date type="Actual">October 2016</completion_date>
  <primary_completion_date type="Actual">October 2016</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>Prevention</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>The Number of Participants With Excellent Reduction in Bleeding When Given FACTOR X as Routine Prophylaxis Over 6 Months</measure>
    <time_frame>6 months</time_frame>
    <description>The Investigator's assessment of the efficacy of FACTOR X in reduction/prevention of bleeding when given as routine prophylaxis over 6 months.&#xD;
The efficacy was assessed according to tabulated criteria; Excellent, good, poor, unassessable.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Safety of FACTOR X: Number of Participants Experiencing Adverse Events</measure>
    <time_frame>6 months</time_frame>
    <description>One of the secondary objectives was to assess the safety of FACTOR X when given as routine prophylaxis over 6 months (26 weeks). The general strategy of the safety evaluation was to examine the summaries for any trends. No formal hypothesis was carried out. The number of participants who experienced Adverse Events is provided.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Pharmacokinetics: FX:C Incremental Recovery</measure>
    <time_frame>Baseline Visit and End of Study Visit, 30 minutes post-dose</time_frame>
    <description>One of the secondary objectives was to assess the pharmacokinetics (FX:C incremental recovery 30 minute post-dose at the Visit 1 (Baseline) and the End of Study Visit after a single dose of 50 IU/kg). The overall mean IR calculated for both visits is presented in the outcome measure table.</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">9</enrollment>
  <condition>Factor X Deficiency</condition>
  <arm_group>
    <arm_group_label>FACTOR X</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>At the Baseline Visit, eligible children will receive a bolus dose of 50 IU/kg FACTOR X. After the Baseline Visit, children will be treated with FACTOR X prophylactically for a period of 6 months (26 weeks).&#xD;
A dosing regimen of 40-50 IU/kg twice a week is recommended, but is not mandatory. Each dose of FACTOR X must not exceed 60 IU/kg.</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>FACTOR X</intervention_name>
    <arm_group_label>FACTOR X</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria&#xD;
&#xD;
          1. Children with hereditary severe or moderate FX deficiency (FX:C &lt;5 IU/dL), based on&#xD;
             their lowest reliable FX:C recorded.&#xD;
&#xD;
          2. Children under 12 years old, whose parent/guardian has given informed consent.&#xD;
&#xD;
          3. Children with a history of severe bleeding e.g.: intracranial haemorrhage, before&#xD;
             starting prophylactic therapy, OR a mutation in the F10 gene causing a documented&#xD;
             severe bleeding phenotype.&#xD;
&#xD;
        Exclusion Criteria&#xD;
&#xD;
          1. Children must not suffer from clinically significant liver disease, renal disease, or&#xD;
             other coagulopathy or thrombophilia&#xD;
&#xD;
          2. Children must have no history or suspicion of inhibitors to factor X.&#xD;
&#xD;
          3. Children who have known or suspected hypersensitivity to the investigational medicinal&#xD;
             product or its excipients.&#xD;
&#xD;
          4. Children with a history of unreliability or non-cooperation.&#xD;
&#xD;
          5. Children who are participating or have taken part in another trial within the last 30&#xD;
             days.&#xD;
&#xD;
          6. Children planning more than 4 weeks' continuous absence from the locality of the&#xD;
             investigational site, between the Screening Visit and the End of Study Visit at&#xD;
             approximately 6 months (26 weeks) post-Baseline.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>N/A</minimum_age>
    <maximum_age>11 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Ri Liesner, Dr</last_name>
    <role>Principal Investigator</role>
    <affiliation>Great Ormond Street Hospital</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Addenbrookes Hospital</name>
      <address>
        <city>Cambridge</city>
        <zip>CB2 0QQ</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Great Ormond Street Hospital</name>
      <address>
        <city>London</city>
        <zip>WC1N 3JH</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Sheffield Children's Hospital</name>
      <address>
        <city>Sheffield</city>
        <zip>S10 2TH</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United Kingdom</country>
  </location_countries>
  <verification_date>March 2018</verification_date>
  <study_first_submitted>October 25, 2012</study_first_submitted>
  <study_first_submitted_qc>November 2, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">November 6, 2012</study_first_posted>
  <results_first_submitted>October 11, 2017</results_first_submitted>
  <results_first_submitted_qc>February 7, 2018</results_first_submitted_qc>
  <results_first_posted type="Actual">March 7, 2018</results_first_posted>
  <last_update_submitted>March 7, 2018</last_update_submitted>
  <last_update_submitted_qc>March 7, 2018</last_update_submitted_qc>
  <last_update_posted type="Actual">April 2, 2018</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>Factor X Deficiency</mesh_term>
  </condition_browse>

  <clinical_results>

    <participant_flow>
      <recruitment_details>Two subjects completed less than 26 weeks in the study. The subjects were re-enrolled and data from their first treatment cycle was excluded from the per-protocol analysis. 9 unique subjects were enrolled event though there were 11 treatment cycles.</recruitment_details>
      <group_list>
        <group group_id="P1">
          <title>Overall Study</title>
          <description>At the Baseline Visit, eligible children received a bolus dose of 50 IU/kg FACTOR X. After the Baseline Visit, children were treated with FACTOR X prophylactically for a period of 6 months (26 weeks).&#xD;
A dosing regimen of 40-50 IU/kg twice a week was recommended, but was not mandatory. Each dose of FACTOR X was not to not exceed 60 IU/kg.</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="9"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="9"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
              </participants_list>
            </milestone>
          </milestone_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>Human Coagulation FACTOR X</title>
          <description>At the Baseline Visit, eligible children received a bolus dose of 50 IU/kg FACTOR X. After the Baseline Visit, children were treated with FACTOR X prophylactically for a period of 6 months (26 weeks).&#xD;
A dosing regimen of 40-50 IU/kg twice a week was recommended, but was not mandatory. Each dose of FACTOR X was not to not exceed 60 IU/kg.</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="9"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>&lt;=18 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="9"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Between 18 and 65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>&gt;=65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Sex: Female, Male</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Female</title>
                  <measurement_list>
                    <measurement group_id="B1" value="5"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="4"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Region of Enrollment</title>
          <units>participants</units>
          <param>Number</param>
          <class_list>
            <class>
              <title>United Kingdom</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="9"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>The Number of Participants With Excellent Reduction in Bleeding When Given FACTOR X as Routine Prophylaxis Over 6 Months</title>
        <description>The Investigator's assessment of the efficacy of FACTOR X in reduction/prevention of bleeding when given as routine prophylaxis over 6 months.&#xD;
The efficacy was assessed according to tabulated criteria; Excellent, good, poor, unassessable.</description>
        <time_frame>6 months</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Overall Study</title>
            <description>At the Baseline Visit, eligible children received a bolus dose of 50 IU/kg FACTOR X. After the Baseline Visit, children were treated with FACTOR X prophylactically for a period of 6 months (26 weeks).&#xD;
A dosing regimen of 40-50 IU/kg twice a week was recommended, but was not mandatory. Each dose of FACTOR X was not to not exceed 60 IU/kg.</description>
          </group>
        </group_list>
        <measure>
          <title>The Number of Participants With Excellent Reduction in Bleeding When Given FACTOR X as Routine Prophylaxis Over 6 Months</title>
          <description>The Investigator's assessment of the efficacy of FACTOR X in reduction/prevention of bleeding when given as routine prophylaxis over 6 months.&#xD;
The efficacy was assessed according to tabulated criteria; Excellent, good, poor, unassessable.</description>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="9"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="9"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Safety of FACTOR X: Number of Participants Experiencing Adverse Events</title>
        <description>One of the secondary objectives was to assess the safety of FACTOR X when given as routine prophylaxis over 6 months (26 weeks). The general strategy of the safety evaluation was to examine the summaries for any trends. No formal hypothesis was carried out. The number of participants who experienced Adverse Events is provided.</description>
        <time_frame>6 months</time_frame>
        <population>The safety evaluation examined the summaries for any trends. No formal hypothesis was carried out.</population>
        <group_list>
          <group group_id="O1">
            <title>Overall Study</title>
            <description>At the Baseline Visit, eligible children received a bolus dose of 50 IU/kg FACTOR X. After the Baseline Visit, children were treated with FACTOR X prophylactically for a period of 6 months (26 weeks).&#xD;
A dosing regimen of 40-50 IU/kg twice a week was recommended, but was not mandatory. Each dose of FACTOR X was not to not exceed 60 IU/kg.</description>
          </group>
        </group_list>
        <measure>
          <title>Safety of FACTOR X: Number of Participants Experiencing Adverse Events</title>
          <description>One of the secondary objectives was to assess the safety of FACTOR X when given as routine prophylaxis over 6 months (26 weeks). The general strategy of the safety evaluation was to examine the summaries for any trends. No formal hypothesis was carried out. The number of participants who experienced Adverse Events is provided.</description>
          <population>The safety evaluation examined the summaries for any trends. No formal hypothesis was carried out.</population>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="9"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="8"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Pharmacokinetics: FX:C Incremental Recovery</title>
        <description>One of the secondary objectives was to assess the pharmacokinetics (FX:C incremental recovery 30 minute post-dose at the Visit 1 (Baseline) and the End of Study Visit after a single dose of 50 IU/kg). The overall mean IR calculated for both visits is presented in the outcome measure table.</description>
        <time_frame>Baseline Visit and End of Study Visit, 30 minutes post-dose</time_frame>
        <population>Plasma concentrations were obtained for FX:C for all 9 subjects at 30 minutes post dose at Visit 1 and Visit 5.</population>
        <group_list>
          <group group_id="O1">
            <title>Overall Study</title>
            <description>At the Baseline Visit, eligible children received a bolus dose of 50 IU/kg FACTOR X. After the Baseline Visit, children were treated with FACTOR X prophylactically for a period of 6 months (26 weeks).&#xD;
A dosing regimen of 40-50 IU/kg twice a week was recommended, but was not mandatory. Each dose of FACTOR X was not to not exceed 60 IU/kg.</description>
          </group>
        </group_list>
        <measure>
          <title>Pharmacokinetics: FX:C Incremental Recovery</title>
          <description>One of the secondary objectives was to assess the pharmacokinetics (FX:C incremental recovery 30 minute post-dose at the Visit 1 (Baseline) and the End of Study Visit after a single dose of 50 IU/kg). The overall mean IR calculated for both visits is presented in the outcome measure table.</description>
          <population>Plasma concentrations were obtained for FX:C for all 9 subjects at 30 minutes post dose at Visit 1 and Visit 5.</population>
          <units>IU/dL</units>
          <param>Mean</param>
          <dispersion>95% Confidence Interval</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="9"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1.74" lower_limit="1.66" upper_limit="1.82"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>From signing of informed consent form until 28 days post-last dose. For subjects who had received FACTOR X on compassionate use prior to study entry, retrospective adverse events data was collected as part of medical history.</time_frame>
      <group_list>
        <group group_id="E1">
          <title>Human Coagulation FACTOR X</title>
          <description>At the Baseline Visit, eligible children received a bolus dose of 50 IU/kg FACTOR X. After the Baseline Visit, children were treated with FACTOR X prophylactically for a period of 6 months (26 weeks).&#xD;
A dosing regimen of 40-50 IU/kg twice a week was recommended, but was not mandatory. Each dose of FACTOR X was not to not exceed 60 IU/kg.</description>
        </group>
      </group_list>
      <serious_events>
        <default_vocab>MedDRA (19.0)</default_vocab>
        <default_assessment>Non-systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="9"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Lower Respiratory Tract Infection</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="9"/>
              </event>
              <event>
                <sub_title>Influenza A</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="9"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>5</frequency_threshold>
        <default_vocab>MedDRA (19.0)</default_vocab>
        <default_assessment>Non-systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, other adverse events</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="9"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Blood and lymphatic system disorders</title>
            <event_list>
              <event>
                <sub_title>Aneamia</sub_title>
                <counts group_id="E1" events="3" subjects_affected="3" subjects_at_risk="9"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Intermittent Low Grade Fever</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="9"/>
              </event>
              <event>
                <sub_title>High Temperature/Fever</sub_title>
                <counts group_id="E1" events="5" subjects_affected="3" subjects_at_risk="9"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Cold</sub_title>
                <counts group_id="E1" events="4" subjects_affected="3" subjects_at_risk="9"/>
              </event>
              <event>
                <sub_title>Viral Infection</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="9"/>
              </event>
              <event>
                <sub_title>Chest Infection</sub_title>
                <counts group_id="E1" events="2" subjects_affected="1" subjects_at_risk="9"/>
              </event>
              <event>
                <sub_title>Positive MRSA Swab To Groin</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="9"/>
              </event>
              <event>
                <sub_title>Influenza A</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="9"/>
              </event>
              <event>
                <sub_title>Coryzal</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="9"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Investigations</title>
            <event_list>
              <event>
                <sub_title>Parvovirus IgM, no symptoms</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="9"/>
              </event>
              <event>
                <sub_title>Increased Temperature</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="9"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Metabolism and nutrition disorders</title>
            <event_list>
              <event>
                <sub_title>Decreased Appetite</sub_title>
                <counts group_id="E1" events="2" subjects_affected="1" subjects_at_risk="9"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Musculoskeletal and connective tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Pain In Back Of Leg</sub_title>
                <counts group_id="E1" events="2" subjects_affected="1" subjects_at_risk="9"/>
              </event>
              <event>
                <sub_title>Pain In Left Arm</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="9"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>Headache</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="9"/>
              </event>
              <event>
                <sub_title>Lethargy</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="9"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Reproductive system and breast disorders</title>
            <event_list>
              <event>
                <sub_title>Dysmenorrhoea</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="9"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Respiratory, thoracic and mediastinal disorders</title>
            <event_list>
              <event>
                <sub_title>Cough</sub_title>
                <counts group_id="E1" events="4" subjects_affected="3" subjects_at_risk="9"/>
              </event>
              <event>
                <sub_title>Wheeze</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="9"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Skin and subcutaneous tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Vitiligo</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="9"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>Principal Investigators are NOT employed by the organization sponsoring the study.</pi_employee>
      <restrictive_agreement>The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.</restrictive_agreement>
    </certain_agreements>
    <limitations_and_caveats>2 subjects completed less than 26 weeks in the study. The subjects were re-enrolled and data from their first treatment cycle was excluded from the Per-Protocol analysis. 9 unique subjects were enrolled even though there were 11 treatment cycles</limitations_and_caveats>
    <point_of_contact>
      <name_or_title>Head of Medical Affairs</name_or_title>
      <organization>Bio Products Laboratory Ltd</organization>
      <phone>+44 20 8957 2200</phone>
      <email>medinfo@bpl.co.uk</email>
    </point_of_contact>
  </clinical_results>
</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/NCT01721681
org study id: Ten 02
secondary id: 2012-003093-98
nct id: NCT01721681
lead sponsor: 
has dmc: No
The primary objective of the study is to assess the efficacy of FACTOR X in the prevention of
      bleeding when given as routine prophylaxis over 12 months.

      The secondary objectives of the study are:

        1. To assess the pharmacokinetics of FACTOR X after a single dose of 50 IU/kg.

        2. To assess the safety of FACTOR X when given as routine prophylaxis over 6 months (26
           weeks).
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Prevention
masking: None (Open Label)
measure: The Number of Participants With Excellent Reduction in Bleeding When Given FACTOR X as Routine Prophylaxis Over 6 Months
time frame: 6 months
description: The Investigator's assessment of the efficacy of FACTOR X in reduction/prevention of bleeding when given as routine prophylaxis over 6 months.
The efficacy was assessed according to tabulated criteria; Excellent, good, poor, unassessable.
measure: Safety of FACTOR X: Number of Participants Experiencing Adverse Events
time frame: 6 months
description: One of the secondary objectives was to assess the safety of FACTOR X when given as routine prophylaxis over 6 months (26 weeks). The general strategy of the safety evaluation was to examine the summaries for any trends. No formal hypothesis was carried out. The number of participants who experienced Adverse Events is provided.
measure: Pharmacokinetics: FX:C Incremental Recovery
time frame: Baseline Visit and End of Study Visit, 30 minutes post-dose
description: One of the secondary objectives was to assess the pharmacokinetics (FX:C incremental recovery 30 minute post-dose at the Visit 1 (Baseline) and the End of Study Visit after a single dose of 50 IU/kg). The overall mean IR calculated for both visits is presented in the outcome measure table.
arm group label: FACTOR X
arm group type: Experimental
description: At the Baseline Visit, eligible children will receive a bolus dose of 50 IU/kg FACTOR X. After the Baseline Visit, children will be treated with FACTOR X prophylactically for a period of 6 months (26 weeks).
A dosing regimen of 40-50 IU/kg twice a week is recommended, but is not mandatory. Each dose of FACTOR X must not exceed 60 IU/kg.
intervention type: Biological
intervention name: FACTOR X
arm group label: FACTOR X
criteria: 
gender: All
minimum age: N/A
maximum age: 11 Years
healthy volunteers: No
last name: Ri Liesner, Dr
role: Principal Investigator
affiliation: Great Ormond Street Hospital
facility: 
facility: 
facility: 
country: United Kingdom
responsible party type: Sponsor
mesh term: Factor X Deficiency
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
limitations and caveats: 2 subjects completed less than 26 weeks in the study. The subjects were re-enrolled and data from their first treatment cycle was excluded from the Per-Protocol analysis. 9 unique subjects were enrolled even though there were 11 treatment cycles
point of contact: 