<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/NCT03005327</url>
  </required_header>
  <id_info>
    <org_study_id>X4P-001-MKKA</org_study_id>
    <secondary_id>2016-005028-26</secondary_id>
    <nct_id>NCT03005327</nct_id>
  </id_info>
  <brief_title>A Dose Determination and Safety Study of X4P-001 (Mavorixafor) in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome</brief_title>
  <official_title>A Phase 2, Open-Label, Multi-Center Trial of Mavorixafor in Patients With WHIM Syndrome</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>X4 Pharmaceuticals</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>X4 Pharmaceuticals</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 is a Phase 2 study with an initial 24-week Treatment Period and an Extension Phase. The&#xD;
      primary objectives of this Phase 2 study are to determine the safety, tolerability, and dose&#xD;
      selection of mavorixafor in participants with WHIM syndrome. Participants are allowed to&#xD;
      continue treatment in an Extension Phase, if regionally applicable, until it becomes&#xD;
      commercially available or until the study is terminated by the Sponsor.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">December 2016</start_date>
  <completion_date type="Actual">June 16, 2022</completion_date>
  <primary_completion_date type="Actual">June 16, 2022</primary_completion_date>
  <phase>Phase 2</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 AUCANC and/or AUCALC</measure>
    <time_frame>Time 0 (-15 minutes [min] pre-dose), 30, 60, and 90 min (each ± 5 min) and 2, 3, 4, 8, 12, 16, and 24 hours (each ±15 min) at Weeks 5, 13, and 21</time_frame>
  </primary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">8</enrollment>
  <condition>WHIM Syndrome</condition>
  <arm_group>
    <arm_group_label>X4P-001</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Initial Treatment Phase: Participants will initiate treatment with mavorixafor at 50 milligrams (mg) once daily (QD) orally or a higher dose, with potential escalation based on area under the curve for absolute neutrophil count and absolute leukocyte count (AUCANC/ALC) values to a maximum total daily dose of 400 mg. Participants are expected to receive treatment for 24 weeks in the initial Treatment Period or until development of a treatment-limiting toxicity (TLT).&#xD;
Extension Phase: All participants will receive mavorixafor; the dose will not exceed 400 mg. In the Extension Phase, treatment may continue until mavorixafor becomes available via an alternative mechanism (for example, drug is commercially available, an expanded access program, etc.) or until the study is terminated by the sponsor.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>X4P-001</intervention_name>
    <description>Mavorixafor will be provided as either 25 mg or 100 mg capsules.</description>
    <arm_group_label>X4P-001</arm_group_label>
    <other_name>AMD11070, Mavorixafor</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
        Participants with a clinical diagnosis of WHIM syndrome must meet all of the following&#xD;
        criteria to be eligible for study participation:&#xD;
&#xD;
          1. Be at least 18 years of age.&#xD;
&#xD;
          2. Has signed the current approved informed consent form.&#xD;
&#xD;
          3. Has a genotype-confirmed mutation of chemokine receptor type 4 (CXCR4) consistent with&#xD;
             WHIM syndrome.&#xD;
&#xD;
          4. Agree to use effective contraception.&#xD;
&#xD;
          5. Be willing and able to comply with this protocol.&#xD;
&#xD;
          6. Has confirmed ANC less than or equal to (≤) 400/µL or ALC ≤650/µL or both.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
        Participants with any of the following will be excluded from participation in the study:&#xD;
&#xD;
          1. Has known systemic hypersensitivity to the mavorixafor drug substance or its inactive&#xD;
             ingredients.&#xD;
&#xD;
          2. Is pregnant or nursing.&#xD;
&#xD;
          3. Has a known history of a positive serology or viral load for human immunodeficiency&#xD;
             virus (HIV) or a known history of acquired immunodeficiency syndrome (AIDS).&#xD;
&#xD;
          4. Has, at Screening, laboratory tests meeting one or more of the following criteria:&#xD;
&#xD;
               -  A positive antibody test for hepatitis C virus (HCV), unless documented to have&#xD;
                  no detectable viral load on 2 independent samples.&#xD;
&#xD;
               -  A positive test for hepatitis B surface antigen (HBsAg).&#xD;
&#xD;
          5. Has any medical or personal condition that, in the opinion of the Investigator, may&#xD;
             potentially compromise the safety or compliance of the participant, or may preclude&#xD;
             the participant's successful completion of the clinical study.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>University of Washingington Medical Center</name>
      <address>
        <city>Seattle</city>
        <state>Washington</state>
        <zip>98195</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>St. Vincent's Hospital</name>
      <address>
        <city>Fitzroy</city>
        <state>Victoria</state>
        <zip>3065</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Australia</country>
    <country>United States</country>
  </location_countries>
  <verification_date>September 2023</verification_date>
  <study_first_submitted>December 20, 2016</study_first_submitted>
  <study_first_submitted_qc>December 23, 2016</study_first_submitted_qc>
  <study_first_posted type="Estimate">December 29, 2016</study_first_posted>
  <last_update_submitted>September 5, 2023</last_update_submitted>
  <last_update_submitted_qc>September 5, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">September 7, 2023</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>Syndrome</mesh_term>
  </condition_browse>
  <!-- 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/NCT03005327
org study id: X4P-001-MKKA
secondary id: 2016-005028-26
nct id: NCT03005327
lead sponsor: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
This is a Phase 2 study with an initial 24-week Treatment Period and an Extension Phase. The
      primary objectives of this Phase 2 study are to determine the safety, tolerability, and dose
      selection of mavorixafor in participants with WHIM syndrome. Participants are allowed to
      continue treatment in an Extension Phase, if regionally applicable, until it becomes
      commercially available or until the study is terminated by the Sponsor.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Mean AUCANC and/or AUCALC
time frame: Time 0 (-15 minutes [min] pre-dose), 30, 60, and 90 min (each ± 5 min) and 2, 3, 4, 8, 12, 16, and 24 hours (each ±15 min) at Weeks 5, 13, and 21
arm group label: X4P-001
arm group type: Experimental
description: Initial Treatment Phase: Participants will initiate treatment with mavorixafor at 50 milligrams (mg) once daily (QD) orally or a higher dose, with potential escalation based on area under the curve for absolute neutrophil count and absolute leukocyte count (AUCANC/ALC) values to a maximum total daily dose of 400 mg. Participants are expected to receive treatment for 24 weeks in the initial Treatment Period or until development of a treatment-limiting toxicity (TLT).
Extension Phase: All participants will receive mavorixafor; the dose will not exceed 400 mg. In the Extension Phase, treatment may continue until mavorixafor becomes available via an alternative mechanism (for example, drug is commercially available, an expanded access program, etc.) or until the study is terminated by the sponsor.
intervention type: Drug
intervention name: X4P-001
description: Mavorixafor will be provided as either 25 mg or 100 mg capsules.
arm group label: X4P-001
other name: AMD11070, Mavorixafor
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
facility: 
facility: 
country: Australia
country: United States
responsible party type: Sponsor
mesh term: Syndrome