Expanded Access for ABI-009 in Patients With Advanced 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 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 Overview

• Status: Approved for marketing
• Conditions: TSC1; TSC2; PEComa, Malignant; mTOR Pathway Abberation
• Intervention / Treatment: Drug: ABI-009

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

• Study Type: Expanded Access
• Expanded Access Type: Intermediate-size Population

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 85 years (Adult, Older Adult)
• Accepts Healthy Volunteers: N/A
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Collaborators and Investigators

• Sponsor: Aadi Bioscience, Inc.

Study record dates

Study Registration Dates

• First Submitted: January 22, 2019
• First Submitted That Met QC Criteria: January 22, 2019
• First Posted (Actual): January 25, 2019

Study Record Updates

• Last Update Posted (Actual): March 16, 2022
• Last Update Submitted That Met QC Criteria: March 2, 2022
• Last Verified: March 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Perivascular Epithelioid Cell Neoplasms; Physiological Effects of Drugs; Anti-Infective Agents; Antineoplastic Agents; Immunosuppressive Agents; Immunologic Factors; Anti-Bacterial Agents; Antibiotics, Antineoplastic; Antifungal Agents; Sirolimus
• Other Study ID Numbers: PEX-002