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

• ClinicalTrials.gov Identifier: NCT02494570
• Recruitment Status: Completed
• First Posted: July 10, 2015
• Last Update Posted: May 3, 2022
• Sponsor: Aadi Bioscience, Inc.
• Information provided by (Responsible Party): Aadi Bioscience, Inc.

Tracking Information

• First Submitted Date: July 8, 2015
• First Posted Date: July 10, 2015
• Last Update Posted Date: May 3, 2022
• Study Start Date: October 2015
• Actual Primary Completion Date: November 2021 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: July 9, 2015): objective response rate [ Time Frame: up to 32 months ]
• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (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: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Phase 2 Study of ABI-009 in Patients With Advanced Malignant 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)

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: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: PEComa
• Intervention: Drug: ABI-009

Study Arms

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.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: February 12, 2019): 34
• Original Estimated Enrollment (submitted: July 9, 2015): 30
• Actual Study Completion Date: April 2022
• Actual Primary Completion Date: November 2021 (Final data collection date for primary outcome measure)

Eligibility 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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT02494570
• Other Study ID Numbers: 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: Not Provided
• Responsible Party: Aadi Bioscience, Inc.
• Study Sponsor: Aadi Bioscience, Inc.
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Aadi Bioscience, Inc.
• Verification Date: April 2022