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

NCT02494570 | Completed Phase 2 Results DMC FDA Drug

• 3 other identifiers: PEC-001FD005749AMPECT
• Study Type: interventional
• Target: 34
• Locations: 1 country
• Sites: 9

Brief Summary

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).

Conditions

Malignant PEComa

Keywords

malignant PEComa, perivascular epithelioid cell tumors

Outcome Measures

Primary Outcomes (1)

• Objective Overall Response Rate (ORR)

  Best ORR was assessed by Independent Radiology Review using Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 Criteria. Best overall response was the best response recorded from the start of the study treatment until the end of treatment taking into account the requirement for confirmation. Per RECIST v1.1, best overall response assignment depended on the findings of both target and non-target disease and also took into consideration the appearance of new lesions. Overall response rate was defined as the percentage of patients who achieve a confirmed PR or CR per RECIST 1.1. At each timepoint, objective tumor response for target lesions were assessed as such: Complete Response (CR), disappearance of all target lesions, and pathological lymph nodes must have a reduction \<10 mm; Partial Response (PR), ≥30% decrease in the sum of the longest diameter of target lesions.

  through study completion (up to 72 months)

Secondary Outcomes (4)

• Duration of Response

  From Initial response until tumor progression, through study completion (up to 72 months)

• Progression-free Survival Rate at 6 Months

  6 months

• Progression-free Survival (Median)

  from start of treatment to first documented disease progression, through study completion (up to 72 months)

• Overall Survival

  From start of treatment to date of death (of any cause), through study completion (up to 72 months)

Study Arms (1)

nab-Sirolimus

EXPERIMENTAL

Patients with malignant PEComa received nab-sirolimus on Days 1 and 8 of every 21-day cycle, as a 30-minute IV infusion.

Drug: nab-Sirolimus

Interventions

nab-Sirolimus DRUG

Patients received nab-sirolimus at 100 mg/m2 on Days 1 and 8 of a 21-day cycle by intravenous infusion over 30 minutes.

Also known as: ABI-009, Fyarro, nab-rapamycin

nab-Sirolimus

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Aadi Bioscience, Inc.: lead

Study Sites (9)

Stanford University Medical Center

Palo Alto, California, 94304, United States

Location

Sarcoma Oncology Research Center

Santa Monica, California, 90403, United States

Location

Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Washington University School of Medicine - Siteman Cancer Center

St Louis, Missouri, 63110, United States

Location

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

Duke University Medical Center - Duke Cancer Center

Durham, North Carolina, 27710, United States

Location

University of Texas, MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

University of Washington - Seattle Cancer Care Alliance

Seattle, Washington, 98109, United States

Location

Related Publications (3)

• Wagner AJ, Ravi V, Riedel RF, Ganjoo K, Van Tine BA, Chugh R, Cranmer L, Gordon EM, Hornick JL, Du H, Polson KM, Yalamanchili S, DeBoer C, Ding L, Schmid AN, Navarro WH, Kwiatkowski DJ, Dickson MA. A plain language summary of the AMPECT study: nab-sirolimus for advanced malignant perivascular epithelioid cell tumors. Future Oncol. 2026 Jan;22(1):1-13. doi: 10.1080/14796694.2025.2574811. Epub 2025 Dec 22.

  PMID: 41424265 DERIVED

• Wagner AJ, Ravi V, Riedel RF, Ganjoo K, Van Tine BA, Chugh R, Cranmer L, Gordon EM, Hornick JL, Du H, Ding L, Schmid AN, Navarro WH, Kwiatkowski DJ, Dickson MA. Phase II Trial of nab-Sirolimus in Patients With Advanced Malignant Perivascular Epithelioid Cell Tumors (AMPECT): Long-Term Efficacy and Safety Update. J Clin Oncol. 2024 May 1;42(13):1472-1476. doi: 10.1200/JCO.23.02266. Epub 2024 Mar 1.

  PMID: 38427923 DERIVED

• 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.

  PMID: 34637337 DERIVED

MeSH Terms

Conditions

Perivascular Epithelioid Cell Neoplasms

Condition Hierarchy (Ancestors)

Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms

Results Point of Contact

• Title: Medical Information
• Organization: Aadi Bioscience

MedInfo@aadibio.com

1-888-246-2234

Study Officials

• Willis Navarro, MD

  Aadi Bioscience

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: July 8, 2015
• First Posted: July 10, 2015
• Study Start: October 1, 2015
• Primary Completion: November 1, 2021
• Study Completion: April 1, 2022
• Last Updated: April 7, 2023
• Results First Posted: April 7, 2023

Record last verified: 2023-03

Locations

US (9)