<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/NCT03286153</url>
  </required_header>
  <id_info>
    <org_study_id>BDC Ideal Body Weight Dosing</org_study_id>
    <nct_id>NCT03286153</nct_id>
  </id_info>
  <brief_title>Comparison of Ideal vs. Actual Weight Base Factor Dosing</brief_title>
  <official_title>Comparison of Ideal vs. Actual Weight Base Factor Dosing</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Bloodworks</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Seattle Children's Hospital</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Oregon Health and Science University</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Providence Health &amp; Services</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Bloodworks</source>
  <oversight_info>
    <has_dmc>No</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>
      This is a randomized, prospective, multicenter study to examine whether or not the current&#xD;
      recommended factor dosing strategy - i.e., dosing by actual body weight - in overweight and&#xD;
      obese patients with Hemophilia A may deliver excessive clotting factor to achieve the desired&#xD;
      result of bleeding prevention and cessation. This study also examines ways to prevent&#xD;
      delivering excessive factor by using a patient's ideal body weight as a new dosing strategy&#xD;
      compared to the current dosing strategy. The hypothesis being tested is that factor dosing&#xD;
      based on ideal body weight will result in protective factor levels.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This is a randomized, prospective, multicenter, open-label, crossover study to examine&#xD;
      whether or not the current recommended factor dosing strategy, i.e., dosing by actual body&#xD;
      weight in overweight and obese patients, may deliver more clotting factor than necessary to&#xD;
      cause bleeding to stop in participants with Hemophilia A who use Factor VIII (FVIII). This&#xD;
      study also examines ways to prevent delivering too much factor by using a participant's ideal&#xD;
      body weight as a new dosing strategy compared to the current dosing strategy. The hypothesis&#xD;
      being tested is that factor dosing based on ideal body weight will result in hemostatic&#xD;
      factor levels.&#xD;
&#xD;
      The study will be conducted at the Washington Center for Bleeding Disorders (WCBD) at&#xD;
      Bloodworks Northwest, Oregon Health &amp; Science University (OHSU), Seattle Children's Hospital&#xD;
      (SCH), and Providence Sacred Heart Children's Hospital (SH). Cumulatively across the four&#xD;
      sites, up to 20 participants will be enrolled. Randomization will be performed centrally at&#xD;
      WCBD.&#xD;
&#xD;
      Participants will provide their own factor. Prior to the first study-related dose,&#xD;
      participants will stop taking any FVIII products for either 48 hours if currently using a&#xD;
      short-acting FVIII product or 72 hours for a long acting FVIII product. Factor levels will be&#xD;
      measured immediately before and at multiple points after two different factor doses. Subjects&#xD;
      will be randomized to start their dosage based either on actual body weight or ideal body&#xD;
      weight first and then crossover to receive dosage based on the other category.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Recruiting</last_known_status>
  <start_date type="Actual">January 2017</start_date>
  <completion_date type="Anticipated">December 2018</completion_date>
  <primary_completion_date type="Anticipated">December 2018</primary_completion_date>
  <phase>N/A</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Crossover Assignment</intervention_model>
    <intervention_model_description>Subjects are randomized to receive factor based either on ideal body weight or actual body weight first followed by receiving factor based on the other category next</intervention_model_description>
    <primary_purpose>Health Services Research</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Recovery</measure>
    <time_frame>Change from baseline at up to two months</time_frame>
    <description>Compare the recovery with FVIII between doses calculated on actual body weight versus ideal body weight in subjects with Hemophilia A</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Underdosing</measure>
    <time_frame>Change from baseline at up to two months</time_frame>
    <description>Determine the likelihood of underdosing when using ideal body weight</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Overdosing</measure>
    <time_frame>Change from baseline at up to two months</time_frame>
    <description>Determine the likelihood of overdosing when using actual body weight</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Effect of half-life</measure>
    <time_frame>Change from baseline at up to two months</time_frame>
    <description>Determine the effect on half-life of these dosing strategies</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Effect on hemophilia severity</measure>
    <time_frame>Change from baseline at 20-40 minutes, 5-7 hours, 20-26 hours, and 44-50 hours for both half-life and extended half-life and also at 69-75 hours, and 93-99 hours for extended half-life</time_frame>
    <description>Determine the effect of pharmacokinetic differences on hemophilia severity</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Regular half-life vs. extended half-life Regular half-life vs. extended half-life</measure>
    <time_frame>Change from baseline at up to two months</time_frame>
    <description>Determine differences in participants receiving regular half-life versus extended half-life products</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Overweight vs. obese</measure>
    <time_frame>Change from baseline at up to two months</time_frame>
    <description>Determine the differences, if any, between overweight and obese participants</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Anticipated">20</enrollment>
  <condition>Hemophilia A</condition>
  <arm_group>
    <arm_group_label>Ideal Body Weight First</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Randomized to receive factor product based on ideal body weight first</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Actual Body Weight First</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Randomized to receive factor product based on actual body weight first</description>
  </arm_group>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Ideal Body Weight First</intervention_name>
    <description>Randomized to receive 50 U/kg (+/- 20%) of the factor product participants routinely use based on ideal body weight. For participants age 12-19, ideal weight is calculated using the McLaren method. For participants age 20 and over, ideal weight is calculated using the following equation: [50kg + (2.3kg*every inch over 5 feet)].</description>
    <arm_group_label>Ideal Body Weight First</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Actual Body Weight First</intervention_name>
    <description>Randomized to receive 50 U/kg (+/- 20%) of the factor product participants routinely use based on actual body weight.</description>
    <arm_group_label>Actual Body Weight First</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Hemophilia A&#xD;
&#xD;
          -  Able and willing to comply with pharmacokinetic testing schedule&#xD;
&#xD;
          -  Either overweight or obese BMI using CDC definitions by age&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Inhibitor of &gt; 0.6 BU twice in the past, or documented abnormal recovery of less than&#xD;
             66% (of expected) in the past&#xD;
&#xD;
          -  Known other bleeding disorder&#xD;
&#xD;
          -  Known other prolongation in aPTT (lupus anticoagulant, FXII deficiency)&#xD;
&#xD;
          -  Female&#xD;
      </textblock>
    </criteria>
    <gender>Male</gender>
    <minimum_age>12 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Rebecca Kruse-Jarres, MD, MPH</last_name>
    <role>Principal Investigator</role>
    <affiliation>Washington Center for Bleeding Disorders at Bloodworks Northwest</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Heidi Thielmann, PhD</last_name>
    <phone>206-689-6234</phone>
    <email>hthielmann@bloodworksnw.org</email>
  </overall_contact>
  <overall_contact_backup>
    <last_name>Rebecca Kruse-Jarres, MD, MPH</last_name>
    <phone>206-689-6593</phone>
    <email>RebeccaKr@BloodWorksNW.org</email>
  </overall_contact_backup>
  <location>
    <facility>
      <name>Oregon Health &amp; Science University</name>
      <address>
        <city>Portland</city>
        <state>Oregon</state>
        <zip>97239-3098</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Michael Recht, MD, PhD</last_name>
      <phone>503-494-8311</phone>
      <email>rechtm@ohsu.edu</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>Washington Center for Bleeding Disorders at Bloodworks Northwest</name>
      <address>
        <city>Seattle</city>
        <state>Washington</state>
        <zip>98104</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Rebecca Kruse-Jarres, MD, MPH</last_name>
      <phone>206-689-6593</phone>
      <email>RebeccaKr@BloodWorksNW.org</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>Seattle Children's Hospital</name>
      <address>
        <city>Seattle</city>
        <state>Washington</state>
        <zip>98105</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Amanda Blair, MD</last_name>
      <phone>206-987-2106</phone>
      <email>Amanda.Blair@seattlechildrens.org</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>Providence Sacred Heart Children's Hospital</name>
      <address>
        <city>Spokane</city>
        <state>Washington</state>
        <zip>99220-2555</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Judy Felgenhauer, MD</last_name>
      <phone>509-474-2777</phone>
      <email>Judy.Felgenhauer@providence.org</email>
    </contact>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>September 2017</verification_date>
  <study_first_submitted>August 9, 2017</study_first_submitted>
  <study_first_submitted_qc>September 13, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">September 18, 2017</study_first_posted>
  <last_update_submitted>September 13, 2017</last_update_submitted>
  <last_update_submitted_qc>September 13, 2017</last_update_submitted_qc>
  <last_update_posted type="Actual">September 18, 2017</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Hemophilia A</keyword>
  <keyword>FVIII</keyword>
  <keyword>Ideal body weight</keyword>
  <keyword>Actual body weight</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Hemophilia A</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <provided_document_section>
    <provided_document>
      <document_type>Informed Consent Form: Main</document_type>
      <document_has_protocol>No</document_has_protocol>
      <document_has_icf>Yes</document_has_icf>
      <document_has_sap>No</document_has_sap>
      <document_date>August 7, 2017</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/53/NCT03286153/ICF_000.pdf</document_url>
    </provided_document>
    <provided_document>
      <document_type>Informed Consent Form: Consent/Assent SC</document_type>
      <document_has_protocol>No</document_has_protocol>
      <document_has_icf>Yes</document_has_icf>
      <document_has_sap>No</document_has_sap>
      <document_date>August 7, 2017</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/53/NCT03286153/ICF_001.pdf</document_url>
    </provided_document>
    <provided_document>
      <document_type>Informed Consent Form: Assent Age 15 to 17</document_type>
      <document_has_protocol>No</document_has_protocol>
      <document_has_icf>Yes</document_has_icf>
      <document_has_sap>No</document_has_sap>
      <document_date>August 7, 2017</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/53/NCT03286153/ICF_002.pdf</document_url>
    </provided_document>
    <provided_document>
      <document_type>Informed Consent Form: Assent Age 7 to 14</document_type>
      <document_has_protocol>No</document_has_protocol>
      <document_has_icf>Yes</document_has_icf>
      <document_has_sap>No</document_has_sap>
      <document_date>August 7, 2017</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/53/NCT03286153/ICF_003.pdf</document_url>
    </provided_document>
    <provided_document>
      <document_type>Study Protocol</document_type>
      <document_has_protocol>Yes</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>No</document_has_sap>
      <document_date>October 12, 2016</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/53/NCT03286153/Prot_004.pdf</document_url>
    </provided_document>
  </provided_document_section>
  <!-- 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/NCT03286153
org study id: BDC Ideal Body Weight Dosing
nct id: NCT03286153
lead sponsor: 
collaborator: 
collaborator: 
collaborator: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
This is a randomized, prospective, multicenter study to examine whether or not the current
      recommended factor dosing strategy - i.e., dosing by actual body weight - in overweight and
      obese patients with Hemophilia A may deliver excessive clotting factor to achieve the desired
      result of bleeding prevention and cessation. This study also examines ways to prevent
      delivering excessive factor by using a patient's ideal body weight as a new dosing strategy
      compared to the current dosing strategy. The hypothesis being tested is that factor dosing
      based on ideal body weight will result in protective factor levels.
This is a randomized, prospective, multicenter, open-label, crossover study to examine
      whether or not the current recommended factor dosing strategy, i.e., dosing by actual body
      weight in overweight and obese patients, may deliver more clotting factor than necessary to
      cause bleeding to stop in participants with Hemophilia A who use Factor VIII (FVIII). This
      study also examines ways to prevent delivering too much factor by using a participant's ideal
      body weight as a new dosing strategy compared to the current dosing strategy. The hypothesis
      being tested is that factor dosing based on ideal body weight will result in hemostatic
      factor levels.

      The study will be conducted at the Washington Center for Bleeding Disorders (WCBD) at
      Bloodworks Northwest, Oregon Health & Science University (OHSU), Seattle Children's Hospital
      (SCH), and Providence Sacred Heart Children's Hospital (SH). Cumulatively across the four
      sites, up to 20 participants will be enrolled. Randomization will be performed centrally at
      WCBD.

      Participants will provide their own factor. Prior to the first study-related dose,
      participants will stop taking any FVIII products for either 48 hours if currently using a
      short-acting FVIII product or 72 hours for a long acting FVIII product. Factor levels will be
      measured immediately before and at multiple points after two different factor doses. Subjects
      will be randomized to start their dosage based either on actual body weight or ideal body
      weight first and then crossover to receive dosage based on the other category.
allocation: Randomized
intervention model: Crossover Assignment
intervention model description: Subjects are randomized to receive factor based either on ideal body weight or actual body weight first followed by receiving factor based on the other category next
primary purpose: Health Services Research
masking: None (Open Label)
measure: Recovery
time frame: Change from baseline at up to two months
description: Compare the recovery with FVIII between doses calculated on actual body weight versus ideal body weight in subjects with Hemophilia A
measure: Underdosing
time frame: Change from baseline at up to two months
description: Determine the likelihood of underdosing when using ideal body weight
measure: Overdosing
time frame: Change from baseline at up to two months
description: Determine the likelihood of overdosing when using actual body weight
measure: Effect of half-life
time frame: Change from baseline at up to two months
description: Determine the effect on half-life of these dosing strategies
measure: Effect on hemophilia severity
time frame: Change from baseline at 20-40 minutes, 5-7 hours, 20-26 hours, and 44-50 hours for both half-life and extended half-life and also at 69-75 hours, and 93-99 hours for extended half-life
description: Determine the effect of pharmacokinetic differences on hemophilia severity
measure: Regular half-life vs. extended half-life Regular half-life vs. extended half-life
time frame: Change from baseline at up to two months
description: Determine differences in participants receiving regular half-life versus extended half-life products
measure: Overweight vs. obese
time frame: Change from baseline at up to two months
description: Determine the differences, if any, between overweight and obese participants
arm group label: Ideal Body Weight First
arm group type: Experimental
description: Randomized to receive factor product based on ideal body weight first
arm group label: Actual Body Weight First
arm group type: Experimental
description: Randomized to receive factor product based on actual body weight first
intervention type: Other
intervention name: Ideal Body Weight First
description: Randomized to receive 50 U/kg (+/- 20%) of the factor product participants routinely use based on ideal body weight. For participants age 12-19, ideal weight is calculated using the McLaren method. For participants age 20 and over, ideal weight is calculated using the following equation: [50kg + (2.3kg*every inch over 5 feet)].
arm group label: Ideal Body Weight First
intervention type: Other
intervention name: Actual Body Weight First
description: Randomized to receive 50 U/kg (+/- 20%) of the factor product participants routinely use based on actual body weight.
arm group label: Actual Body Weight First
criteria: 
gender: Male
minimum age: 12 Years
maximum age: N/A
healthy volunteers: No
last name: Rebecca Kruse-Jarres, MD, MPH
role: Principal Investigator
affiliation: Washington Center for Bleeding Disorders at Bloodworks Northwest
last name: Heidi Thielmann, PhD
phone: 206-689-6234
email: hthielmann@bloodworksnw.org
last name: Rebecca Kruse-Jarres, MD, MPH
phone: 206-689-6593
email: RebeccaKr@BloodWorksNW.org
facility: 
status: Recruiting
contact: 
facility: 
status: Recruiting
contact: 
facility: 
status: Recruiting
contact: 
facility: 
status: Recruiting
contact: 
country: United States
responsible party type: Sponsor
mesh term: Hemophilia A
sharing ipd: No
provided document: 
provided document: 
provided document: 
provided document: 
provided document: 