A Phase 2 Study of Nab-sirolimus (ABI-009) in Patients With Advanced Malignant PEComa

Not Recruiting

Trial ID: NCT02494570


A phase 2 multi-center investigation of efficacy of nab-sirolimus (formerly known as ABI-009 or nab-rapamycin) in patients with advanced malignant perivascular epithelioid cell tumor (PEComa).

Official Title

A Phase 2 Multi-center Investigation of Efficacy of ABI-009 (Nab-sirolimus) in Patients With Advanced Malignant Perivascular Epithelioid Cell Tumors (PEComa)

Stanford Investigator(s)

Kristen N Ganjoo
Kristen N Ganjoo

Professor of Medicine (Oncology)


Inclusion Criteria:

   1. Patients must have a histologically confirmed diagnosis of malignant PEComa that is
   either metastatic or locally advanced and for which surgery is not a recommended

   2. Patients must have available tumor block along with the corresponding pathology report
   (or approximately 30 unstained slides, with a minimum of 16 slides), and/or fresh
   biopsy to allow retrospective centralized confirmation of malignant PEComa and for
   mTOR pathway analysis and biomarker analysis.

   3. Patients must have one or more measurable target lesions by CT scan or MRI. Measurable
   disease by RECIST v1.1.

   4. Patients must not have been previously treated with an mTOR inhibitor.

   5. Prior treatment (investigational or other), chemotherapy, radiotherapy, surgery, or
   other therapeutic agents (except mTOR inhibitors) is allowed, if completed after 5
   half-lives or ≥28 days prior to enrollment, whichever is shorter.

   6. Eligible patients, 18 years or older, with Eastern Cooperative Oncology Group (ECOG)
   performance status 0 or 1.

   7. Patients must have the following blood chemistry levels at screening (obtained

   ≤14 days prior to enrollment (local laboratory):

      1. total bilirubin ≤1.5 x upper limit of normal (ULN) mg/dl

      2. AST ≤2.5 x ULN (≤5 x ULN if attributable to liver metastases)

      3. serum creatinine ≤1.5 x ULN

   8. 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 ≥9 g/dL.

   9. Serum triglyceride <300 mg/dL; serum cholesterol < 350 mg/dL.

10. 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 study
      medication and have a negative serum pregnancy test (β -hCG) result at screening
      and agree to ongoing pregnancy testing during the course of the study, and after
      the end of study treatment.

      - 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 study, even if he has undergone a successful vasectomy.

11. Life expectancy of >3 months, as determined by the investigator.

12. Ability to understand and sign informed consent.

13. Willingness and ability to comply with scheduled visits, laboratory tests, and other
   study procedures.

Exclusion Criteria:

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

   1. Patients with lymphangioleiomyomatosis (LAM) are excluded.

   2. Known active uncontrolled or symptomatic central nervous system (CNS) metastases. A
   patient with controlled and asymptomatic CNS metastases may participate in this study.
   As such, the patient must have completed any prior treatment for CNS metastases ≥28
   days (including radiotherapy and/or surgery) prior to start of treatment in this study
   and should not be receiving chronic corticosteroid therapy for the CNS metastases.

   3. Active gastrointestinal bleeding, if transfusion dependent.

   4. Pre-existing thyroid abnormality is allowed provided thyroid function can be
   controlled with medication.

   5. 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).

   6. Liver-directed therapy within 2 months of enrollment. Prior treatment with
   radiotherapy (including radio-labeled spheres and/or cyberknife, hepatic arterial
   embolization (with or without chemotherapy) or cyrotherapy/ablation) is allowed if
   these therapies did not affect the areas of measurable disease being used for this

   7. Recent infection requiring systemic anti-infective treatment that was completed

   ≤14 days prior to enrollment (with the exception of uncomplicated urinary tract
   infection or upper respiratory tract infection).

   8. Uncontrolled diabetes mellitus as defined by HbA1c >8% despite adequate therapy.

   9. Unstable coronary artery disease or myocardial infarction during preceding 6 months.

10. Receiving any concomitant antitumor therapy.

11. Patients with history of interstitial lung disease and/or pneumonitis, or pulmonary

12. The use of certain medications and illicit drugs within 5 half-lives or 28 days,
   whichever is shorter prior to the first dose of study drug and for the duration of the
   study will not be allowed.

13. Use of strong inhibitors and inducers of CYP3A4 within the 14 days prior to receiving
   the first dose of ABI-009. Additionally, use of any known CYP3A4 substrates with
   narrow therapeutic window (such as fentanyl, alfentanil, astemizole, cisapride,
   dihydroergotamine, pimozide, quinidine, terfanide) within the 14 days prior to
   receiving the first dose of ABI-009.


drug: nab-Sirolimus

Not Recruiting

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Shohreh Monshipouri

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