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
About this trial
This is an expanded access trial for PEComa, Malignant
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.
Sites / Locations
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
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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