A Phase 2 Study of ABI-009 in Patients With Advanced Malignant PEComa

Study Description

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Brief Summary:

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

Condition or disease	Intervention/treatment	Phase
PEComa	Drug: ABI-009	Phase 2

Study Design

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Study Type :	Interventional (Clinical Trial)
Estimated Enrollment :	30 participants
Intervention Model:	Single Group Assignment
Masking:	None (Open Label)
Primary Purpose:	Treatment
Official Title:	A Phase 2 Multi-center Investigation of Efficacy of ABI-009 (Nab-rapamycin) in Patients With Advanced Malignant Perivascular Epithelioid Cell Tumors (PEComa)
Study Start Date :	October 2015
Estimated Primary Completion Date :	September 2018
Estimated Study Completion Date :	September 2020

Arms and Interventions

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Arm	Intervention/treatment
Experimental: ABI-009	Drug: ABI-009

Outcome Measures

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Primary Outcome Measures :

1. objective response rate [ Time Frame: up to 32 months ]

Secondary Outcome Measures :

1. Duration of response [ Time Frame: From Initial response until tumor progression, up to 32 months ]
2. Progression free rate at 6 months [ Time Frame: 6 months ]
3. Progression free survival [ Time Frame: from start of treatment to first documented disease progression, up to 32 months ]
4. Overall survival [ Time Frame: From start of treatment to date of death (of any cause), up to 32 months ]
5. Number of participants with Adverse events [ Time Frame: up to 32 months ]

Eligibility Criteria

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Ages Eligible for Study:	18 Years and older (Adult, Older Adult)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

Criteria

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 option.
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 apply:

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 protocol.

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 hypertension.
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.

Contacts and Locations

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Contacts

Contact: Berta Grigorian		bgrigorian@aadibio.com

Locations

United States, California
Stanford University Medical Center	Recruiting
Palo Alto, California, United States, 94304
Contact: Marti Lohman 650-714-6582 mlohman@stanford.edu
Sarcoma Oncology Research Center	Recruiting
Santa Monica, California, United States, 90403
Contact: Victoria Chua 310-552-9999 vchua@sarcomaoncology.com
United States, Massachusetts
Dana-Farber Cancer Institute	Recruiting
Boston, Massachusetts, United States, 02215
Contact: Sarcoma Research Nursing 617-632-5204
United States, Michigan
University of Michigan	Recruiting
Ann Arbor, Michigan, United States, 48109
Contact: Rikki Leavitt 734-936-2712 leavitar@med.umich.edu
United States, Missouri
Washington University School of Medicine - Siteman Cancer Center	Recruiting
Saint Louis, Missouri, United States, 63110
Contact: Ashley Bolden 314-747-8064 abolden@dom.wustl.edu
United States, New York
Memorial Sloan Kettering Cancer Center	Recruiting
New York, New York, United States, 10065
Contact: Tim Barz 646-888-1399 barzt@mskcc.org
United States, North Carolina
Duke University Medical Center - Duke Cancer Center	Recruiting
Durham, North Carolina, United States, 27710
Contact: Colleen Piechocki 919-681-1883 Colleen.piechocki@duke.edu
United States, Texas
University of Texas, MD Anderson Cancer Center	Recruiting
Houston, Texas, United States, 77030
Contact: Aaron Reckeweg 713-794-4274 asreckew@mdanderson.org
United States, Washington
University of Washington - Seattle Cancer Care Alliance	Recruiting
Seattle, Washington, United States, 98109
Contact: Roxanne Moore 206-288-6425 romoore@seattlecca.org

Sponsors and Collaborators

Aadi, LLC

More Information

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Responsible Party:	Aadi, LLC
ClinicalTrials.gov Identifier:	NCT02494570 History of Changes
Other Study ID Numbers:	PEC001; FD005749 ( Other Grant/Funding Number: FDA-OOPD )
First Posted:	July 10, 2015
Last Update Posted:	February 6, 2018
Last Verified:	February 2018

Additional relevant MeSH terms:

Perivascular Epithelioid Cell Neoplasms; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms