<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/NCT04409145</url>
  </required_header>
  <id_info>
    <org_study_id>VT30-101</org_study_id>
    <nct_id>NCT04409145</nct_id>
  </id_info>
  <brief_title>First in Human Trial of Topical VT30 in Pts With Venous/Lymphatic Malformations Assoc With PIK3CA or TEK Gene Mutations</brief_title>
  <official_title>Open-Label, Intra Subject, Dose Escalation (Part 1) Followed by Randomized, Double Blind, Placebo Controlled (Part 2) Trial of Topical VT30 in Pts With Venous, Lymphatic or Mixed Malformations Associated With PIK3CA or TEK Genetic Mutations</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Venthera, Inc., a BridgeBio company</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Venthera, Inc., a BridgeBio company</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>
      VT30-101 is a 2-part first-in-human trial of topically administered VT30 to subjects with&#xD;
      cutaneous venous malformations, lymphatic malformations, or mixed venolymphatic malformations&#xD;
      associated with PIK3CA or TEK mutations.&#xD;
&#xD;
      Part 1 is a 4-week treatment, open-label, 4-sequence, escalating repeat-application cohort&#xD;
      study, with intra-subject and inter-cohort dose escalation.&#xD;
&#xD;
      Part 2 is a 12-week treatment, randomized, placebo-controlled, double-blind, safety and&#xD;
      exploratory efficacy study. Part 2 will be initiated only after the successful completion of&#xD;
      Part 1 with results that demonstrate the general safety and tolerability of topically applied&#xD;
      VT30. Up to 12 subjects who complete Part 1 may be enrolled into Part 2 of the study.&#xD;
&#xD;
      The primary objective is to evaluate the safety and tolerability of VT30. The study will also&#xD;
      determine the dose and regimen of VT30 to be carried into Part 2 of the protocol. Other aims&#xD;
      include documenting plasma drug levels of VT30 and VT10 and, on an exploratory basis,&#xD;
      examining pharmacologic target engagement and change in potential efficacy readouts.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      VT30-101 is designed as a Phase 1/2, first-in-human study of topically administered VT30 to&#xD;
      subjects with cutaneous venous malformations (VMs), lymphatic malformations (LMs), or mixed&#xD;
      venolymphatic malformations (VLMs) associated with phosphatidylinositol 3-kinase catalytic&#xD;
      alpha polypeptide (PIK3CA) or tyrosine receptor kinase (TEK) mutations. Capillary involvement&#xD;
      and/or extension of the lesion into subcutaneous tissues is permitted.&#xD;
&#xD;
      The study will occur in 2 parts, and in both study parts, subjects will participate in a&#xD;
      Screening Period (up to 6 weeks) before beginning the indicated Treatment Period.&#xD;
&#xD;
      Part 1 will be an open-label, 4-sequence, escalating repeat-application study comprised of up&#xD;
      to 4 cohorts (3 subjects per cohort, with 3 up to 6 in Cohort 4, or the final Part 1 cohort).&#xD;
      In each cohort, subjects will be given topical VT30 for a 4-week Treatment Period. Subjects&#xD;
      will begin treatment with the designated dose on Day 1. After 2 weeks, the Investigator will&#xD;
      examine the treated surface area and determine if the formulation is tolerated such that the&#xD;
      subject may apply the next dose strength of VT30 gel for the remaining duration of the&#xD;
      Treatment Period.&#xD;
&#xD;
      Specifically, the following gel dose strengths (concentrations) are planned for Cohorts 1&#xD;
      through 3 in Part 1:&#xD;
&#xD;
        -  Cohort 1: initiate dosing with 0.12% (w/w) gel and progress to 0.6% (w/w) gel for the&#xD;
           final 2 weeks of the Part 1 Treatment Period (if the lower dose is tolerated)&#xD;
&#xD;
        -  Cohort 2: initiate dosing with 0.6% (w/w) gel and progress to 1.2% (w/w) gel for the&#xD;
           final 2 weeks of the Part 1 Treatment Period (if the lower dose is tolerated)&#xD;
&#xD;
        -  Cohort 3: initiate dosing with 1.2% (w/w) gel and progress to 2.3% (w/w) gel for the&#xD;
           final 2 weeks of the Part 1 Treatment Period (if the lower dose is tolerated)&#xD;
&#xD;
      A Safety Review Committee (SRC), with sponsor, investigator and independent medical&#xD;
      representation, will provide oversight and guidance for study conduct. After 3 subjects have&#xD;
      been dosed for at least 21 days in any of the first three Part 1 cohorts, the SRC may request&#xD;
      additional data, approve initiation of the subsequent cohort, or mandate that additional&#xD;
      subjects to be enrolled at either the higher or lower dose within the cohort.&#xD;
&#xD;
      After 9 subjects in Cohorts 1 through 3 (3 subjects per cohort) have completed the 4-week&#xD;
      Treatment Period, the SRC will determine if additional subjects should be assigned to Cohort&#xD;
      4 (or the final Part 1 cohort) to receive the MTD (maximum tolerated dose) or MFD (maximum&#xD;
      feasible dose) strength of VT30. Subjects in Cohort 4 will receive the designated dose&#xD;
      strength and regimen of VT30 for a full 4 weeks.&#xD;
&#xD;
      Following the completion of Cohort 4 in Part 1, the SRC will determine whether to authorize&#xD;
      initiation of Part 2 and will confirm the dose level and regimen to be administered in Part&#xD;
      2.&#xD;
&#xD;
      Part 2 will be a randomized, placebo-controlled, double-blind study containing 36 subjects&#xD;
      assigned in a 2:1 ratio to receive either VT30 or placebo. Up to 12 subjects who complete&#xD;
      Part 1 may enroll in Part 2, provided they meet all Part 2-specific inclusion criteria with&#xD;
      no applicable exclusions.&#xD;
&#xD;
      After the first 12 subjects in Part 2 have completed 4 weeks of treatment, the SRC will&#xD;
      conduct a review of blinded safety data to confirm no revisions or changes are needed to the&#xD;
      protocol. Subsequent reviews may also be conducted to ensure continued acceptable safety and&#xD;
      tolerability.&#xD;
&#xD;
      After subjects complete their designated Treatment Period (in Part 1 or Part 2), they will&#xD;
      participate in a 4-week post treatment Follow-up Period.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Terminated</overall_status>
  <why_stopped>
    Part 1 complete; Part 2 will not be completed.&#xD;
  </why_stopped>
  <start_date type="Actual">October 1, 2020</start_date>
  <completion_date type="Actual">April 13, 2022</completion_date>
  <primary_completion_date type="Actual">April 13, 2022</primary_completion_date>
  <phase>Phase 1/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>
    <intervention_model_description>Part 1: Open-label, 4-sequence, escalating repeat-application cohort study, with intra-subject and inter-cohort dose escalation to determine safety and tolerability, and maximum feasible dose/maximum tolerable dose Part 2: Randomized, placebo-controlled, double-blind, safety and exploratory efficacy study</intervention_model_description>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
    <masking_description>Part 1: Open label Part 2: Double blind</masking_description>
  </study_design_info>
  <primary_outcome>
    <measure>Evaluation of safety and tolerability</measure>
    <time_frame>From pre-treatment to 4 weeks of treatment</time_frame>
    <description>Composite of adverse events and changes in physical exam findings, vital signs, lab tests, and electrocardiogram evaluations</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Maximum feasible dose / maximum tolerable dose</measure>
    <time_frame>From pre-treatment to 4 weeks</time_frame>
    <description>The MTD or MFD strength will be determined based on results from Part 1, and will inform the dose strength to be used in Part 2</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Tissue and serum drug levels</measure>
    <time_frame>From pre-treatment to 4 weeks</time_frame>
    <description>Plasma levels of VT30 and VT10 will be assessed following topical administration of VT30 to determine level of systemic exposure</description>
  </secondary_outcome>
  <other_outcome>
    <measure>Maximum tissue concentration of study drug</measure>
    <time_frame>From pre-treatment to 4 weeks</time_frame>
    <description>As assessed by treated lesion tissue levels of phosphoproteins, as an indicator of local target engagement</description>
  </other_outcome>
  <other_outcome>
    <measure>Changes in Pain</measure>
    <time_frame>From pre-treatment to 4 weeks</time_frame>
    <description>Assessed by subjects' self-reporting their pain, related to the treated lesion, on a Numerical Rating Scale</description>
  </other_outcome>
  <other_outcome>
    <measure>Changes in lesion</measure>
    <time_frame>From pre-treatment to 4 weeks</time_frame>
    <description>Assessed by change in appearance of the treated lesion</description>
  </other_outcome>
  <other_outcome>
    <measure>Changes in management of lesion bleeding, oozing, or discharge</measure>
    <time_frame>From pre-treatment to 4 weeks</time_frame>
    <description>Assessed by subject-reported difficulty managing bleeding, oozing, or discharge from the treated lesion</description>
  </other_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">15</enrollment>
  <condition>Venous Malformation</condition>
  <condition>Lymphatic Malformation</condition>
  <condition>Venolymphatic Malformation</condition>
  <arm_group>
    <arm_group_label>VT30</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>VT30 is a PI3K-inhibitor prodrug, formulated as a topical gel and dispensed from a metered dose pump; administration is once or twice daily, applied to target-treatment area(s) on the skin. One pump action dispenses 250 µL of gel, intended to treat an area of 140 cm2.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>VT30</intervention_name>
    <description>VT30 gel is intended as a topical treatment of cutaneous VMs, LMs, or VLMs that driven by inappropriate PI3K activation. In the skin, VT30 is rapidly metabolized to VT10, an active drug form, and is intended to sufficiently permeate the stratum corneum and achieve target engagement. It is expected that VT30 will lead to amelioration of the signs and symptoms of cutaneous VMs, LMs and/or VLMs.</description>
    <arm_group_label>VT30</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Have signed the current approved informed consent form&#xD;
&#xD;
          2. Have a clinically or phenotypically defined VM, LM, or mixed VLM affecting the skin&#xD;
&#xD;
          3. Lesion genotyping confirms either PIK3CA or TEK mutations, known to be pathogenic&#xD;
&#xD;
          4. Agrees to use contraception if of childbearing potential&#xD;
&#xD;
          5. Be willing and able to comply with the protocol and be available for the entire study&#xD;
&#xD;
          6. Be at least 18 to 60 years of age&#xD;
&#xD;
          7. Lesion must be amenable to defining a contiguous study treatment area of 140 cm2&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Lesion to be treated is on the face or involves mucosa&#xD;
&#xD;
          2. Presence of ulcerations on the target-treatment lesion&#xD;
&#xD;
          3. Known systemic hypersensitivity to the VT30 drug substance, its inactive ingredients,&#xD;
             or the vehicle&#xD;
&#xD;
          4. Uncontrolled diabetes mellitus&#xD;
&#xD;
          5. Hyperlipidemia that is poorly controlled on current treatment&#xD;
&#xD;
          6. Pregnant or nursing, planning to become pregnant, or planning to father a child during&#xD;
             the study&#xD;
&#xD;
          7. History of malignancy except successfully treated nonmetastatic cutaneous squamous&#xD;
             cell or basal cell carcinoma and/or localized carcinoma in situ of the cervix&#xD;
&#xD;
          8. Major surgery within 8 weeks of Screening, or a surgical, laser or other procedure&#xD;
             involving the target lesion within 8 weeks of Screening, or planned to occur during&#xD;
             the study&#xD;
&#xD;
          9. Any other medical or personal condition that, in the opinion of the Investigator, may&#xD;
             potentially compromise the safety or compliance of the subject, or may preclude the&#xD;
             subject's successful completion of the clinical study&#xD;
&#xD;
         10. Medically significant infection (eg, cellulitis or abscess, or a systemic infection)&#xD;
             within 8 weeks of Screening&#xD;
&#xD;
         11. Ongoing therapy with another topical treatment or any medication that inhibits PI3K,&#xD;
             Akt pathway, or the mTOR pathway, or in the opinion of the Investigator, the subject&#xD;
             requires systemic therapy for their vascular malformation condition&#xD;
&#xD;
         12. Use of a biologic or systemic immunosuppressive agent within 3 months of Screening&#xD;
&#xD;
         13. Systemic use of corticosteroids, within 30 days of Screening&#xD;
&#xD;
         14. Treatment with a small molecule investigational product within 30 days of Screening,&#xD;
             or with any investigational biologic products within 3 months of Screening&#xD;
&#xD;
         15. Positive for hepatitis C antibody, hepatitis B surface antigen, hepatitis B core&#xD;
             antibody, or human immunodeficiency virus&#xD;
&#xD;
         16. Alanine transaminase or aspartate transaminase laboratory values in excess of 1.5X the&#xD;
             upper limit of normal at Screening&#xD;
&#xD;
         17. Hemoglobin A1c is &gt;8%&#xD;
&#xD;
         18. Any other clinically significant laboratory or testing abnormality that, in the&#xD;
             opinion of the Investigator, might confound the study, interfere with the subject's&#xD;
             ability to complete the study, or represent a meaningful safety risk upon study&#xD;
             enrollment&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>60 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Michael Henderson</last_name>
    <role>Study Director</role>
    <affiliation>Venthera, Inc., a BridgeBio company</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Phoenix Children's Hospital</name>
      <address>
        <city>Phoenix</city>
        <state>Arizona</state>
        <zip>85016</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Arkansas Children's Hospital/UAMS</name>
      <address>
        <city>Little Rock</city>
        <state>Arkansas</state>
        <zip>72202</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Stanford University Medical Center</name>
      <address>
        <city>Palo Alto</city>
        <state>California</state>
        <zip>94304</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Dermatology Cosmetic Laser Medical Associates of La Jolla</name>
      <address>
        <city>San Diego</city>
        <state>California</state>
        <zip>92121</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital of Colorado</name>
      <address>
        <city>Aurora</city>
        <state>Colorado</state>
        <zip>80045</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Indiana University Health (Riley Children's Hospital and University Hospital)</name>
      <address>
        <city>Indianapolis</city>
        <state>Indiana</state>
        <zip>46202</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Mayo Clinic</name>
      <address>
        <city>Rochester</city>
        <state>Minnesota</state>
        <zip>55905</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Duke University</name>
      <address>
        <city>Raleigh</city>
        <state>North Carolina</state>
        <zip>27513</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Cincinnatti Children's Hospital</name>
      <address>
        <city>Cincinnati</city>
        <state>Ohio</state>
        <zip>45229</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University Hospitals- Cleveland Medical Center</name>
      <address>
        <city>Cleveland</city>
        <state>Ohio</state>
        <zip>44106</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital of Philadelphia</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Texas Children's Hospital</name>
      <address>
        <city>Houston</city>
        <state>Texas</state>
        <zip>77030</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Texas Dermatology and Laser Specialists</name>
      <address>
        <city>San Antonio</city>
        <state>Texas</state>
        <zip>78218</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Virginia Department of Dermatology</name>
      <address>
        <city>Charlottesville</city>
        <state>Virginia</state>
        <zip>44106</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Wisconsin-Madison</name>
      <address>
        <city>Madison</city>
        <state>Wisconsin</state>
        <zip>53715</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>August 2022</verification_date>
  <study_first_submitted>May 11, 2020</study_first_submitted>
  <study_first_submitted_qc>May 26, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 1, 2020</study_first_posted>
  <last_update_submitted>August 1, 2022</last_update_submitted>
  <last_update_submitted_qc>August 1, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">August 4, 2022</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>Lymphangioma</mesh_term>
    <mesh_term>Lymphatic Abnormalities</mesh_term>
    <mesh_term>Congenital Abnormalities</mesh_term>
  </condition_browse>
  <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/NCT04409145
org study id: VT30-101
nct id: NCT04409145
lead sponsor: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
VT30-101 is a 2-part first-in-human trial of topically administered VT30 to subjects with
      cutaneous venous malformations, lymphatic malformations, or mixed venolymphatic malformations
      associated with PIK3CA or TEK mutations.

      Part 1 is a 4-week treatment, open-label, 4-sequence, escalating repeat-application cohort
      study, with intra-subject and inter-cohort dose escalation.

      Part 2 is a 12-week treatment, randomized, placebo-controlled, double-blind, safety and
      exploratory efficacy study. Part 2 will be initiated only after the successful completion of
      Part 1 with results that demonstrate the general safety and tolerability of topically applied
      VT30. Up to 12 subjects who complete Part 1 may be enrolled into Part 2 of the study.

      The primary objective is to evaluate the safety and tolerability of VT30. The study will also
      determine the dose and regimen of VT30 to be carried into Part 2 of the protocol. Other aims
      include documenting plasma drug levels of VT30 and VT10 and, on an exploratory basis,
      examining pharmacologic target engagement and change in potential efficacy readouts.
VT30-101 is designed as a Phase 1/2, first-in-human study of topically administered VT30 to
      subjects with cutaneous venous malformations (VMs), lymphatic malformations (LMs), or mixed
      venolymphatic malformations (VLMs) associated with phosphatidylinositol 3-kinase catalytic
      alpha polypeptide (PIK3CA) or tyrosine receptor kinase (TEK) mutations. Capillary involvement
      and/or extension of the lesion into subcutaneous tissues is permitted.

      The study will occur in 2 parts, and in both study parts, subjects will participate in a
      Screening Period (up to 6 weeks) before beginning the indicated Treatment Period.

      Part 1 will be an open-label, 4-sequence, escalating repeat-application study comprised of up
      to 4 cohorts (3 subjects per cohort, with 3 up to 6 in Cohort 4, or the final Part 1 cohort).
      In each cohort, subjects will be given topical VT30 for a 4-week Treatment Period. Subjects
      will begin treatment with the designated dose on Day 1. After 2 weeks, the Investigator will
      examine the treated surface area and determine if the formulation is tolerated such that the
      subject may apply the next dose strength of VT30 gel for the remaining duration of the
      Treatment Period.

      Specifically, the following gel dose strengths (concentrations) are planned for Cohorts 1
      through 3 in Part 1:

        -  Cohort 1: initiate dosing with 0.12% (w/w) gel and progress to 0.6% (w/w) gel for the
           final 2 weeks of the Part 1 Treatment Period (if the lower dose is tolerated)

        -  Cohort 2: initiate dosing with 0.6% (w/w) gel and progress to 1.2% (w/w) gel for the
           final 2 weeks of the Part 1 Treatment Period (if the lower dose is tolerated)

        -  Cohort 3: initiate dosing with 1.2% (w/w) gel and progress to 2.3% (w/w) gel for the
           final 2 weeks of the Part 1 Treatment Period (if the lower dose is tolerated)

      A Safety Review Committee (SRC), with sponsor, investigator and independent medical
      representation, will provide oversight and guidance for study conduct. After 3 subjects have
      been dosed for at least 21 days in any of the first three Part 1 cohorts, the SRC may request
      additional data, approve initiation of the subsequent cohort, or mandate that additional
      subjects to be enrolled at either the higher or lower dose within the cohort.

      After 9 subjects in Cohorts 1 through 3 (3 subjects per cohort) have completed the 4-week
      Treatment Period, the SRC will determine if additional subjects should be assigned to Cohort
      4 (or the final Part 1 cohort) to receive the MTD (maximum tolerated dose) or MFD (maximum
      feasible dose) strength of VT30. Subjects in Cohort 4 will receive the designated dose
      strength and regimen of VT30 for a full 4 weeks.

      Following the completion of Cohort 4 in Part 1, the SRC will determine whether to authorize
      initiation of Part 2 and will confirm the dose level and regimen to be administered in Part
      2.

      Part 2 will be a randomized, placebo-controlled, double-blind study containing 36 subjects
      assigned in a 2:1 ratio to receive either VT30 or placebo. Up to 12 subjects who complete
      Part 1 may enroll in Part 2, provided they meet all Part 2-specific inclusion criteria with
      no applicable exclusions.

      After the first 12 subjects in Part 2 have completed 4 weeks of treatment, the SRC will
      conduct a review of blinded safety data to confirm no revisions or changes are needed to the
      protocol. Subsequent reviews may also be conducted to ensure continued acceptable safety and
      tolerability.

      After subjects complete their designated Treatment Period (in Part 1 or Part 2), they will
      participate in a 4-week post treatment Follow-up Period.
allocation: N/A
intervention model: Single Group Assignment
intervention model description: Part 1: Open-label, 4-sequence, escalating repeat-application cohort study, with intra-subject and inter-cohort dose escalation to determine safety and tolerability, and maximum feasible dose/maximum tolerable dose Part 2: Randomized, placebo-controlled, double-blind, safety and exploratory efficacy study
primary purpose: Treatment
masking: None (Open Label)
masking description: Part 1: Open label Part 2: Double blind
measure: Evaluation of safety and tolerability
time frame: From pre-treatment to 4 weeks of treatment
description: Composite of adverse events and changes in physical exam findings, vital signs, lab tests, and electrocardiogram evaluations
measure: Maximum feasible dose / maximum tolerable dose
time frame: From pre-treatment to 4 weeks
description: The MTD or MFD strength will be determined based on results from Part 1, and will inform the dose strength to be used in Part 2
measure: Tissue and serum drug levels
time frame: From pre-treatment to 4 weeks
description: Plasma levels of VT30 and VT10 will be assessed following topical administration of VT30 to determine level of systemic exposure
measure: Maximum tissue concentration of study drug
time frame: From pre-treatment to 4 weeks
description: As assessed by treated lesion tissue levels of phosphoproteins, as an indicator of local target engagement
measure: Changes in Pain
time frame: From pre-treatment to 4 weeks
description: Assessed by subjects' self-reporting their pain, related to the treated lesion, on a Numerical Rating Scale
measure: Changes in lesion
time frame: From pre-treatment to 4 weeks
description: Assessed by change in appearance of the treated lesion
measure: Changes in management of lesion bleeding, oozing, or discharge
time frame: From pre-treatment to 4 weeks
description: Assessed by subject-reported difficulty managing bleeding, oozing, or discharge from the treated lesion
arm group label: VT30
arm group type: Experimental
description: VT30 is a PI3K-inhibitor prodrug, formulated as a topical gel and dispensed from a metered dose pump; administration is once or twice daily, applied to target-treatment area(s) on the skin. One pump action dispenses 250 µL of gel, intended to treat an area of 140 cm2.
intervention type: Drug
intervention name: VT30
description: VT30 gel is intended as a topical treatment of cutaneous VMs, LMs, or VLMs that driven by inappropriate PI3K activation. In the skin, VT30 is rapidly metabolized to VT10, an active drug form, and is intended to sufficiently permeate the stratum corneum and achieve target engagement. It is expected that VT30 will lead to amelioration of the signs and symptoms of cutaneous VMs, LMs and/or VLMs.
arm group label: VT30
criteria: 
gender: All
minimum age: 18 Years
maximum age: 60 Years
healthy volunteers: No
last name: Michael Henderson
role: Study Director
affiliation: Venthera, Inc., a BridgeBio company
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country: United States
responsible party type: Sponsor
mesh term: Lymphangioma
mesh term: Lymphatic Abnormalities
mesh term: Congenital Abnormalities
sharing ipd: No