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

Completed · Phase 2

Conditions studied: Malignant PEComa

In brief

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).

Key facts

Study ID: NCT02494570
Run by: Aadi Bioscience, Inc.
People needed: 34
Starts: 2015-10-01
Expected to finish: 2022-04-01
Last updated by the study team: 2023-04-07

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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 option.
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.
Patients must have one or more measurable target lesions by CT scan or MRI. Measurable disease by RECIST v1.1.
Patients must not have been previously treated with an mTOR inhibitor.
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.
Eligible patients, 18 years or older, with Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1.
Patients must have the following blood chemistry levels at screening (obtained
≤14 days prior to enrollment (local laboratory):
total bilirubin ≤1.5 x upper limit of normal (ULN) mg/dl
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 ≥9 g/dL.
Serum triglyceride <300 mg/dL; serum 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 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.
Life expectancy of >3 months, as determined by the investigator.
Ability to understand and sign informed consent.
Willingness and ability to comply with scheduled visits, laboratory tests, and other study procedures.

You may not qualify if…

A patient will not be eligible for inclusion in this study if any of the following criteria apply:
Patients with lymphangioleiomyomatosis (LAM) are excluded.
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.
Active gastrointestinal bleeding, if transfusion dependent.
Pre-existing thyroid abnormality is allowed provided thyroid function can be controlled with medication.
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).
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 protocol.
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).
Uncontrolled diabetes mellitus as defined by HbA1c >8% despite adequate therapy.
Unstable coronary artery disease or myocardial infarction during preceding 6 months.
Receiving any concomitant antitumor therapy.
Patients with history of interstitial lung disease and/or pneumonitis, or pulmonary hypertension.
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.
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.

Where it is running

Stanford University Medical Center — Palo Alto, California, United States
Sarcoma Oncology Research Center — Santa Monica, California, United States
Dana-Farber Cancer Institute — Boston, Massachusetts, United States
University of Michigan — Ann Arbor, Michigan, United States
Washington University School of Medicine - Siteman Cancer Center — St Louis, Missouri, United States
Memorial Sloan Kettering Cancer Center — New York, New York, United States
Duke University Medical Center - Duke Cancer Center — Durham, North Carolina, United States
University of Texas, MD Anderson Cancer Center — Houston, Texas, United States
University of Washington - Seattle Cancer Care Alliance — Seattle, Washington, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.