<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/NCT03817515</url>
  </required_header>
  <id_info>
    <org_study_id>PEX-002</org_study_id>
    <nct_id>NCT03817515</nct_id>
  </id_info>
  <brief_title>Expanded Access for ABI-009 in Patients With Advanced PEComa and Patients With a Malignancy With Relevant Genetic Mutations or mTOR Pathway Activation</brief_title>
  <official_title>Expanded Access for an Intermediate-size Population for ABI-009 (Sirolimus Albumin-bound Nanoparticles for Injectable Suspension) in Patients With Advanced Perivascular Epithelioid Cell Tumors (PEComa) and Patients With a Malignancy With Relevant Genetic Mutations or mTOR Pathway Activation</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Aadi Bioscience, Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Aadi Bioscience, Inc.</source>
  <brief_summary>
    <textblock>
      Expanded Access for an Intermediate-size Population for ABI-009 (Sirolimus Albumin-bound&#xD;
      Nanoparticles for Injectable Suspension) in Patients with Advanced Perivascular Epithelioid&#xD;
      Cell Tumors (PEComa) and Patients with a Malignancy with Relevant Genetic Mutations or mTOR&#xD;
      Pathway Activation&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Expanded Access for an Intermediate-size Population for ABI-009 (Sirolimus Albumin-bound&#xD;
      Nanoparticles for Injectable Suspension) in Patients with Advanced Perivascular Epithelioid&#xD;
      Cell Tumors (PEComa) and Patients with a Malignancy with Relevant Genetic Mutations or mTOR&#xD;
      Pathway Activation&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Approved for marketing</overall_status>
  <study_type>Expanded Access</study_type>
  <has_expanded_access>No</has_expanded_access>
  <expanded_access_info>
    <expanded_access_type_intermediate>Yes</expanded_access_type_intermediate>
  </expanded_access_info>
  <condition>PEComa, Malignant</condition>
  <condition>TSC1</condition>
  <condition>TSC2</condition>
  <condition>mTOR Pathway Abberation</condition>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>ABI-009</intervention_name>
    <description>Sirolimus Albumin-bound Nanoparticles</description>
    <other_name>nab-sirolimus, nab-rapamycin, albumin-bound sirolimus, Sirolimus Albumin-bound Nanoparticles</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
        A patient will be eligible for inclusion in this expanded access program only if all of the&#xD;
        following criteria are met:&#xD;
&#xD;
          1. a) Diagnosis of advanced (locally advanced and inoperable or metastatic) malignant&#xD;
             PEComa confirmed by pathology and immunohistochemistry, whether or not previously&#xD;
             exposed to an mTOR inhibitor, or b) diagnosis of any other malignancy with activation&#xD;
             in any mTOR pathway component as identified by immunohistochemistry or an identified&#xD;
             relevant rare genetic mutation in mTOR pathway genes, including but not limited to&#xD;
             TSC1, TSC2, PIK3CA, PTEN, for which there are no FDA-approved treatments or no other&#xD;
             comparable or satisfactory alternative therapies available whether or not they been&#xD;
             previously exposed to a mTOR inhibitor.&#xD;
&#xD;
          2. 18 years or older, with Eastern Cooperative Oncology Group (ECOG) performance status&#xD;
             0, 1, or 2.&#xD;
&#xD;
          3. Acceptable blood chemistry levels at screening (obtained ≤14 days prior to enrollment&#xD;
             (local laboratory) including:&#xD;
&#xD;
               1. total bilirubin ≤1.5 x upper limit of normal (ULN)&#xD;
&#xD;
               2. AST ≤2.5 x ULN (≤5 x ULN if attributable to liver metastases)&#xD;
&#xD;
               3. serum creatinine ≤1.5 x ULN&#xD;
&#xD;
          4. Adequate biological parameters as demonstrated by the following blood counts at&#xD;
             screening (obtained ≤14 days prior to enrollment, local laboratory):&#xD;
&#xD;
               1. Absolute neutrophil count (ANC) ≥1.5 × 109/L;&#xD;
&#xD;
               2. Platelet count ≥100,000/mm3 (100 × 109/L);&#xD;
&#xD;
               3. Hemoglobin ≥8 g/dL.&#xD;
&#xD;
          5. Fasting serum triglyceride &lt;300 mg/dL; fasting serum total cholesterol &lt;350 mg/dL.&#xD;
&#xD;
          6. Male or non-pregnant and non-breast feeding female:&#xD;
&#xD;
               -  Females of child-bearing potential must agree to use effective contraception&#xD;
                  without interruption from 28 days prior to starting IP and while on&#xD;
                  investigational medication and have a negative serum pregnancy test (β -hCG)&#xD;
                  result at screening and agree to ongoing pregnancy testing during the course of&#xD;
                  the expanded access protocol, and after the end of treatment. A second form of&#xD;
                  birth control is required even if she has had a tubal ligation.&#xD;
&#xD;
               -  Male patients must practice abstinence or agree to use a condom during sexual&#xD;
                  contact with a pregnant female or a female of childbearing potential while&#xD;
                  participating in the expanded access protocol. A second form of birth control is&#xD;
                  required even if he has undergone a successful vasectomy.&#xD;
&#xD;
          7. Ability to understand and sign informed consent.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
        A patient will not be eligible for inclusion in this protocol if any of the following&#xD;
        criteria apply:&#xD;
&#xD;
          1. Uncontrolled serious medical or psychiatric illness. Patients with a &quot;currently&#xD;
             active&quot; second malignancy other than non-melanoma skin cancers, carcinoma in situ of&#xD;
             the cervix, resected incidental prostate cancer (staged pT2 with Gleason Score ≤ 6 and&#xD;
             postoperative PSA &lt;0.5 ng/mL), or other adequately treated carcinoma-in-situ are&#xD;
             ineligible. Patients are not considered to have a &quot;currently active&quot; malignancy if&#xD;
             they have completed therapy and are free of disease for ≥1 year.&#xD;
&#xD;
          2. In the treating physician's opinion, the potential risks outweigh the potential&#xD;
             benefits of therapy with ABI-009.&#xD;
&#xD;
          3. Prior exposure to ABI-009.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>85 Years</maximum_age>
  </eligibility>
  <verification_date>March 2022</verification_date>
  <study_first_submitted>January 22, 2019</study_first_submitted>
  <study_first_submitted_qc>January 22, 2019</study_first_submitted_qc>
  <study_first_posted type="Actual">January 25, 2019</study_first_posted>
  <last_update_submitted>March 2, 2022</last_update_submitted>
  <last_update_submitted_qc>March 2, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">March 16, 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>Perivascular Epithelioid Cell Neoplasms</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Sirolimus</mesh_term>
    <mesh_term>Temsirolimus</mesh_term>
    <mesh_term>MTOR Inhibitors</mesh_term>
  </intervention_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/NCT03817515
org study id: PEX-002
nct id: NCT03817515
lead sponsor: 
Expanded Access for an Intermediate-size Population for ABI-009 (Sirolimus Albumin-bound
      Nanoparticles for Injectable Suspension) in Patients with Advanced Perivascular Epithelioid
      Cell Tumors (PEComa) and Patients with a Malignancy with Relevant Genetic Mutations or mTOR
      Pathway Activation
Expanded Access for an Intermediate-size Population for ABI-009 (Sirolimus Albumin-bound
      Nanoparticles for Injectable Suspension) in Patients with Advanced Perivascular Epithelioid
      Cell Tumors (PEComa) and Patients with a Malignancy with Relevant Genetic Mutations or mTOR
      Pathway Activation
expanded access type intermediate: Yes
intervention type: Drug
intervention name: ABI-009
description: Sirolimus Albumin-bound Nanoparticles
other name: nab-sirolimus, nab-rapamycin, albumin-bound sirolimus, Sirolimus Albumin-bound Nanoparticles
criteria: 
gender: All
minimum age: 18 Years
maximum age: 85 Years
responsible party type: Sponsor
mesh term: Perivascular Epithelioid Cell Neoplasms
mesh term: Sirolimus
mesh term: Temsirolimus
mesh term: MTOR Inhibitors