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Trial record 1 of 1 for:    NCT02494570
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A Phase 2 Study of ABI-009 in Patients With Advanced Malignant PEComa (AMPECT)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02494570
Recruitment Status : Completed
First Posted : July 10, 2015
Last Update Posted : May 3, 2022
Information provided by (Responsible Party):
Aadi Bioscience, Inc.

Tracking Information
First Submitted Date  ICMJE July 8, 2015
First Posted Date  ICMJE July 10, 2015
Last Update Posted Date May 3, 2022
Study Start Date  ICMJE October 2015
Actual Primary Completion Date November 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 9, 2015)
objective response rate [ Time Frame: up to 32 months ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: July 9, 2015)
  • Duration of response [ Time Frame: From Initial response until tumor progression, up to 32 months ]
  • Progression free rate at 6 months [ Time Frame: 6 months ]
  • Progression free survival [ Time Frame: from start of treatment to first documented disease progression, up to 32 months ]
  • Overall survival [ Time Frame: From start of treatment to date of death (of any cause), up to 32 months ]
  • Number of participants with Adverse events [ Time Frame: up to 32 months ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE A Phase 2 Study of ABI-009 in Patients With Advanced Malignant PEComa
Official Title  ICMJE A Phase 2 Multi-center Investigation of Efficacy of ABI-009 (Nab-sirolimus) in Patients With Advanced Malignant Perivascular Epithelioid Cell Tumors (PEComa)
Brief Summary

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

Funding Source - FDA OOPD

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE PEComa
Intervention  ICMJE Drug: ABI-009
Study Arms  ICMJE Experimental: ABI-009
Intervention: Drug: ABI-009
Publications * Wagner AJ, Ravi V, Riedel RF, Ganjoo K, Van Tine BA, Chugh R, Cranmer L, Gordon EM, Hornick JL, Du H, Grigorian B, Schmid AN, Hou S, Harris K, Kwiatkowski DJ, Desai NP, Dickson MA. nab-Sirolimus for Patients With Malignant Perivascular Epithelioid Cell Tumors. J Clin Oncol. 2021 Nov 20;39(33):3660-3670. doi: 10.1200/JCO.21.01728. Epub 2021 Oct 12.

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: February 12, 2019)
Original Estimated Enrollment  ICMJE
 (submitted: July 9, 2015)
Actual Study Completion Date  ICMJE April 2022
Actual Primary Completion Date November 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT02494570
Other Study ID Numbers  ICMJE PEC001
FD005749 ( Other Grant/Funding Number: FDA-OOPD )
AMPECT ( Other Identifier: Aadi )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Aadi Bioscience, Inc.
Study Sponsor  ICMJE Aadi Bioscience, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Aadi Bioscience, Inc.
Verification Date April 2022

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP