| Eligibility |
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 (ß -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.
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