<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/NCT04451434</url>
  </required_header>
  <id_info>
    <org_study_id>ALXN2040-HV-101</org_study_id>
    <nct_id>NCT04451434</nct_id>
  </id_info>
  <brief_title>Study of Danicopan in Participants of Japanese Descent</brief_title>
  <official_title>A Phase 1 Study to Determine the Safety, Tolerability, and Pharmacokinetics of Danicopan After Administration as an Oral Tablet in the Fed and Fasted States in Participants of Japanese Descent</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Alexion</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Alexion</source>
  <oversight_info>
    <has_dmc>Yes</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>
      This study will assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics&#xD;
      (PD) of danicopan after a single-dose oral administration under fed and fasting conditions to&#xD;
      participants of Japanese descent.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This will be an open-label, randomized, single-dose, 3-period, 3-treatment crossover study in&#xD;
      participants of Japanese descent.&#xD;
&#xD;
      On Day 1 of each period, participants will receive a single oral dose of 200 milligrams (mg)&#xD;
      danicopan under fasting conditions, 200 mg danicopan under fed conditions, or 400 mg&#xD;
      danicopan under fed conditions. Participants will receive each treatment once, according to&#xD;
      the randomization schedule. There will be a washout period of at least 7 days between each&#xD;
      dose of study intervention.&#xD;
&#xD;
      Safety will be assessed throughout the study.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">August 17, 2020</start_date>
  <completion_date type="Actual">September 28, 2020</completion_date>
  <primary_completion_date type="Actual">September 28, 2020</primary_completion_date>
  <phase>Phase 1</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>Three-way crossover</intervention_model_description>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Number Of Participants With Treatment-Emergent Adverse Events</measure>
    <time_frame>Day 1 (after first dose) through safety follow-up (10 +/- 2 days after last dose)</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Area Under The Concentration Versus Time Curve (AUC) Of Danicopan In Both Fed And Fasted States</measure>
    <time_frame>up to 72 hours postdose</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Maximum Observed Concentration (Cmax) Of Danicopan In Both Fed And Fasted States</measure>
    <time_frame>up to 72 hours postdose</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Time To Maximum Observed Concentration (Tmax) Of Danicopan In Both Fed And Fasted States</measure>
    <time_frame>up to 72 hours postdose</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Dose Proportionality Of Danicopan In Fed State Assessed by AUC</measure>
    <time_frame>up to 72 hours postdose</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Dose Proportionality Of Danicopan In Fed State Assessed by Cmax</measure>
    <time_frame>up to 72 hours postdose</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Activity Of Danicopan As Measured By Alternative Pathway Wieslab Assay</measure>
    <time_frame>up to 72 hours postdose</time_frame>
    <description>Blood samples will be collected for measurement of danicopan activity.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Complement Factor B Fraction b Levels</measure>
    <time_frame>up to 72 hours postdose</time_frame>
    <description>Blood samples will be collected for measurement of complement factor B fraction b levels.</description>
  </secondary_outcome>
  <number_of_arms>3</number_of_arms>
  <enrollment type="Actual">9</enrollment>
  <condition>Healthy</condition>
  <arm_group>
    <arm_group_label>Danicopan 200 mg Fasted</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Fasting participants will receive a single dose of 200 mg danicopan.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Danicopan 200 mg Fed</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Fed participants will receive a single dose of 200 mg danicopan.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Danicopan 400 mg Fed</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Fed participants will receive a single dose of 400 mg danicopan.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Danicopan</intervention_name>
    <description>Oral tablet</description>
    <arm_group_label>Danicopan 200 mg Fasted</arm_group_label>
    <arm_group_label>Danicopan 200 mg Fed</arm_group_label>
    <arm_group_label>Danicopan 400 mg Fed</arm_group_label>
    <other_name>ALXN2040</other_name>
    <other_name>ACH-0144471</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Participants who are of Japanese descent defined as:&#xD;
&#xD;
               -  First generation (born to 2 Japanese parents and 4 Japanese grandparents)&#xD;
&#xD;
               -  Born in Japan, and not have lived outside Japan for greater than 10 years&#xD;
&#xD;
               -  Lifestyle, including diet, must not have significantly changed since leaving&#xD;
                  Japan&#xD;
&#xD;
          2. No clinically significant history or presence of electrocardiogram findings as judged&#xD;
             by the Investigator at screening and prior to the first dose of study intervention in&#xD;
             Period 1.&#xD;
&#xD;
          3. Body mass index in the range of 18.0 to 32.0 kilograms (kg)/meters squared, inclusive,&#xD;
             with a minimum body weight of 50.0 kg at screening.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Evidence of any clinically significant deviation from normal in clinical laboratory&#xD;
             evaluations, as determined by the Investigator or designee.&#xD;
&#xD;
          2. History of any medical or psychiatric condition or disease that, in the opinion of the&#xD;
             Investigator or designee, might limit the participant's ability to complete or&#xD;
             participate in this clinical study, confound the results of the study, or pose an&#xD;
             additional risk to the participant by their participation in the study.&#xD;
&#xD;
          3. History or presence of clinically significant hypersensitivity or idiosyncratic&#xD;
             reaction to the study interventions or related compounds or commonly used&#xD;
             antibacterial agents.&#xD;
&#xD;
          4. History of meningococcal infection, or a first-degree relative with a history of&#xD;
             meningococcal infection.&#xD;
&#xD;
          5. History of febrile illness, or other evidence of infection, within 14 days prior to&#xD;
             the first dose of study intervention.&#xD;
&#xD;
          6. Any major surgery within 4 weeks of the first dose of study intervention.&#xD;
&#xD;
          7. Diagnosis or history of Gilbert's syndrome, in the opinion of the Investigator or&#xD;
             designee.&#xD;
&#xD;
          8. Unable to refrain from or anticipates the use of any drug.&#xD;
&#xD;
          9. Receipt of a vaccine within 30 days prior to the first dose of study intervention.&#xD;
&#xD;
         10. Receipt of blood products within 6 months prior to the first dose of study&#xD;
             intervention.&#xD;
&#xD;
         11. Participation in any other investigational study drug trial in which receipt of an&#xD;
             investigational study drug occurred within 5 half-lives (if known) or 30 days prior to&#xD;
             the first dose of study intervention, whichever is longer.&#xD;
&#xD;
         12. Seated blood pressure is less than 90/40 millimeters of mercury (mmHg) or greater than&#xD;
             140/90 mmHg at screening.&#xD;
&#xD;
         13. Participants who test positive for human immunodeficiency virus, hepatitis B virus,&#xD;
             and/or hepatitis C virus.&#xD;
&#xD;
         14. Current tobacco users and smokers or a positive cotinine test at screening.&#xD;
&#xD;
         15. Donation of whole blood from 3 months prior to the first dose of study intervention,&#xD;
             or of plasma from 30 days prior to the first dose of study intervention.&#xD;
&#xD;
         16. Positive drugs of abuse screen at screening or Day -1 of Period 1.&#xD;
&#xD;
         17. Positive results for alcohol screen at screening or Day -1 of Period 1.&#xD;
&#xD;
         18. Is a female with a positive pregnancy test at screening or Day -1 of Period 1 or who&#xD;
             is lactating, or who plan to become pregnant (within 5 months of screening).&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>55 Years</maximum_age>
    <healthy_volunteers>Accepts Healthy Volunteers</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>Clinical Study Site</name>
      <address>
        <city>Brisbane</city>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Australia</country>
  </location_countries>
  <verification_date>February 2021</verification_date>
  <study_first_submitted>June 19, 2020</study_first_submitted>
  <study_first_submitted_qc>June 25, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 30, 2020</study_first_posted>
  <last_update_submitted>February 11, 2021</last_update_submitted>
  <last_update_submitted_qc>February 11, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">February 15, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Factor D inhibitor</keyword>
  <keyword>Complement</keyword>
  <keyword>Danicopan</keyword>
  <keyword>Pharmacokinetics</keyword>
  <keyword>Japanese</keyword>
  <keyword>Food</keyword>
  <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/NCT04451434
org study id: ALXN2040-HV-101
nct id: NCT04451434
lead sponsor: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
This study will assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics
      (PD) of danicopan after a single-dose oral administration under fed and fasting conditions to
      participants of Japanese descent.
This will be an open-label, randomized, single-dose, 3-period, 3-treatment crossover study in
      participants of Japanese descent.

      On Day 1 of each period, participants will receive a single oral dose of 200 milligrams (mg)
      danicopan under fasting conditions, 200 mg danicopan under fed conditions, or 400 mg
      danicopan under fed conditions. Participants will receive each treatment once, according to
      the randomization schedule. There will be a washout period of at least 7 days between each
      dose of study intervention.

      Safety will be assessed throughout the study.
allocation: Randomized
intervention model: Crossover Assignment
intervention model description: Three-way crossover
primary purpose: Treatment
masking: None (Open Label)
measure: Number Of Participants With Treatment-Emergent Adverse Events
time frame: Day 1 (after first dose) through safety follow-up (10 +/- 2 days after last dose)
measure: Area Under The Concentration Versus Time Curve (AUC) Of Danicopan In Both Fed And Fasted States
time frame: up to 72 hours postdose
measure: Maximum Observed Concentration (Cmax) Of Danicopan In Both Fed And Fasted States
time frame: up to 72 hours postdose
measure: Time To Maximum Observed Concentration (Tmax) Of Danicopan In Both Fed And Fasted States
time frame: up to 72 hours postdose
measure: Dose Proportionality Of Danicopan In Fed State Assessed by AUC
time frame: up to 72 hours postdose
measure: Dose Proportionality Of Danicopan In Fed State Assessed by Cmax
time frame: up to 72 hours postdose
measure: Activity Of Danicopan As Measured By Alternative Pathway Wieslab Assay
time frame: up to 72 hours postdose
description: Blood samples will be collected for measurement of danicopan activity.
measure: Complement Factor B Fraction b Levels
time frame: up to 72 hours postdose
description: Blood samples will be collected for measurement of complement factor B fraction b levels.
arm group label: Danicopan 200 mg Fasted
arm group type: Experimental
description: Fasting participants will receive a single dose of 200 mg danicopan.
arm group label: Danicopan 200 mg Fed
arm group type: Experimental
description: Fed participants will receive a single dose of 200 mg danicopan.
arm group label: Danicopan 400 mg Fed
arm group type: Experimental
description: Fed participants will receive a single dose of 400 mg danicopan.
intervention type: Drug
intervention name: Danicopan
description: Oral tablet
arm group label: Danicopan 200 mg Fasted
arm group label: Danicopan 200 mg Fed
arm group label: Danicopan 400 mg Fed
other name: ALXN2040
other name: ACH-0144471
criteria: 
gender: All
minimum age: 18 Years
maximum age: 55 Years
healthy volunteers: Accepts Healthy Volunteers
facility: 
country: Australia
responsible party type: Sponsor
sharing ipd: No