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Expanded Access for ABI-009 in Patients With Advanced PEComa and Patients With a Malignancy With Relevant Genetic Mutations or mTOR Pathway Activation

Primary Purpose

PEComa, Malignant, TSC1, TSC2

Status
Approved for marketing
Phase
Locations
Study Type
Expanded Access
Intervention
ABI-009
Sponsored by
Aadi Bioscience, Inc.
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for PEComa, Malignant

Eligibility Criteria

18 Years - 85 Years (Adult, Older Adult)All Sexes

Inclusion Criteria:

A patient will be eligible for inclusion in this expanded access program only if all of the following criteria are met:

  1. a) Diagnosis of advanced (locally advanced and inoperable or metastatic) malignant PEComa confirmed by pathology and immunohistochemistry, whether or not previously exposed to an mTOR inhibitor, or b) diagnosis of any other malignancy with activation in any mTOR pathway component as identified by immunohistochemistry or an identified relevant rare genetic mutation in mTOR pathway genes, including but not limited to TSC1, TSC2, PIK3CA, PTEN, for which there are no FDA-approved treatments or no other comparable or satisfactory alternative therapies available whether or not they been previously exposed to a mTOR inhibitor.
  2. 18 years or older, with Eastern Cooperative Oncology Group (ECOG) performance status 0, 1, or 2.
  3. Acceptable blood chemistry levels at screening (obtained ≤14 days prior to enrollment (local laboratory) including:

    1. total bilirubin ≤1.5 x upper limit of normal (ULN)
    2. AST ≤2.5 x ULN (≤5 x ULN if attributable to liver metastases)
    3. serum creatinine ≤1.5 x ULN
  4. Adequate biological parameters as demonstrated by the following blood counts at screening (obtained ≤14 days prior to enrollment, local laboratory):

    1. Absolute neutrophil count (ANC) ≥1.5 × 109/L;
    2. Platelet count ≥100,000/mm3 (100 × 109/L);
    3. Hemoglobin ≥8 g/dL.
  5. Fasting serum triglyceride <300 mg/dL; fasting serum total cholesterol <350 mg/dL.
  6. Male or non-pregnant and non-breast feeding female:

    • Females of child-bearing potential must agree to use effective contraception without interruption from 28 days prior to starting IP and while on investigational medication and have a negative serum pregnancy test (β -hCG) result at screening and agree to ongoing pregnancy testing during the course of the expanded access protocol, and after the end of treatment. A second form of birth control is required even if she has had a tubal ligation.
    • Male patients must practice abstinence or agree to use a condom during sexual contact with a pregnant female or a female of childbearing potential while participating in the expanded access protocol. A second form of birth control is required even if he has undergone a successful vasectomy.
  7. Ability to understand and sign informed consent.

Exclusion Criteria:

A patient will not be eligible for inclusion in this protocol if any of the following criteria apply:

  1. Uncontrolled serious medical or psychiatric illness. Patients with a "currently active" second malignancy other than non-melanoma skin cancers, carcinoma in situ of the cervix, resected incidental prostate cancer (staged pT2 with Gleason Score ≤ 6 and postoperative PSA <0.5 ng/mL), or other adequately treated carcinoma-in-situ are ineligible. Patients are not considered to have a "currently active" malignancy if they have completed therapy and are free of disease for ≥1 year.
  2. In the treating physician's opinion, the potential risks outweigh the potential benefits of therapy with ABI-009.
  3. Prior exposure to ABI-009.

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    January 22, 2019
    Last Updated
    March 2, 2022
    Sponsor
    Aadi Bioscience, Inc.
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    1. Study Identification

    Unique Protocol Identification Number
    NCT03817515
    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
    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
    Study Type
    Expanded Access

    2. Study Status

    Record Verification Date
    March 2022
    Overall Recruitment Status
    Approved for marketing
    Study Start Date
    undefined (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    undefined (undefined)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Aadi Bioscience, Inc.

    4. Oversight

    5. Study Description

    Brief Summary
    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
    Detailed Description
    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

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    PEComa, Malignant, TSC1, TSC2, mTOR Pathway Abberation

    7. Study Design

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    ABI-009
    Other Intervention Name(s)
    nab-sirolimus, nab-rapamycin, albumin-bound sirolimus, Sirolimus Albumin-bound Nanoparticles
    Intervention Description
    Sirolimus Albumin-bound Nanoparticles

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Maximum Age & Unit of Time
    85 Years
    Eligibility Criteria
    Inclusion Criteria: A patient will be eligible for inclusion in this expanded access program only if all of the following criteria are met: a) Diagnosis of advanced (locally advanced and inoperable or metastatic) malignant PEComa confirmed by pathology and immunohistochemistry, whether or not previously exposed to an mTOR inhibitor, or b) diagnosis of any other malignancy with activation in any mTOR pathway component as identified by immunohistochemistry or an identified relevant rare genetic mutation in mTOR pathway genes, including but not limited to TSC1, TSC2, PIK3CA, PTEN, for which there are no FDA-approved treatments or no other comparable or satisfactory alternative therapies available whether or not they been previously exposed to a mTOR inhibitor. 18 years or older, with Eastern Cooperative Oncology Group (ECOG) performance status 0, 1, or 2. Acceptable blood chemistry levels at screening (obtained ≤14 days prior to enrollment (local laboratory) including: total bilirubin ≤1.5 x upper limit of normal (ULN) AST ≤2.5 x ULN (≤5 x ULN if attributable to liver metastases) serum creatinine ≤1.5 x ULN Adequate biological parameters as demonstrated by the following blood counts at screening (obtained ≤14 days prior to enrollment, local laboratory): Absolute neutrophil count (ANC) ≥1.5 × 109/L; Platelet count ≥100,000/mm3 (100 × 109/L); Hemoglobin ≥8 g/dL. Fasting serum triglyceride <300 mg/dL; fasting serum total cholesterol <350 mg/dL. Male or non-pregnant and non-breast feeding female: Females of child-bearing potential must agree to use effective contraception without interruption from 28 days prior to starting IP and while on investigational medication and have a negative serum pregnancy test (β -hCG) result at screening and agree to ongoing pregnancy testing during the course of the expanded access protocol, and after the end of treatment. A second form of birth control is required even if she has had a tubal ligation. Male patients must practice abstinence or agree to use a condom during sexual contact with a pregnant female or a female of childbearing potential while participating in the expanded access protocol. A second form of birth control is required even if he has undergone a successful vasectomy. Ability to understand and sign informed consent. Exclusion Criteria: A patient will not be eligible for inclusion in this protocol if any of the following criteria apply: Uncontrolled serious medical or psychiatric illness. Patients with a "currently active" second malignancy other than non-melanoma skin cancers, carcinoma in situ of the cervix, resected incidental prostate cancer (staged pT2 with Gleason Score ≤ 6 and postoperative PSA <0.5 ng/mL), or other adequately treated carcinoma-in-situ are ineligible. Patients are not considered to have a "currently active" malignancy if they have completed therapy and are free of disease for ≥1 year. In the treating physician's opinion, the potential risks outweigh the potential benefits of therapy with ABI-009. Prior exposure to ABI-009.

    12. IPD Sharing Statement

    Learn more about this trial

    Expanded Access for ABI-009 in Patients With Advanced PEComa and Patients With a Malignancy With Relevant Genetic Mutations or mTOR Pathway Activation

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