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

• ClinicalTrials.gov Identifier: NCT02494570
• Recruitment Status: Active, not recruiting
• First Posted: July 10, 2015
• Last Update Posted: December 10, 2019
• Sponsor: Aadi, LLC
• Information provided by (Responsible Party): Aadi, LLC

Study Description

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

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

Expanded Access : An investigational treatment associated with this study is available outside the clinical trial.   More info ...

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 34 participants
• Allocation: N/A
• 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-sirolimus) in Patients With Advanced Malignant Perivascular Epithelioid Cell Tumors (PEComa)
• Study Start Date: October 2015
• Estimated Primary Completion Date: September 2020
• Estimated Study Completion Date: September 2021

Arms and Interventions

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

Outcome Measures

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

• 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

Locations

United States, California

Stanford University Medical Center

Palo Alto, California, United States, 94304

Sarcoma Oncology Research Center

Santa Monica, California, United States, 90403

United States, Massachusetts

Dana-Farber Cancer Institute

Boston, Massachusetts, United States, 02215

United States, Michigan

University of Michigan

Ann Arbor, Michigan, United States, 48109

United States, Missouri

Washington University School of Medicine - Siteman Cancer Center

Saint Louis, Missouri, United States, 63110

United States, New York

Memorial Sloan Kettering Cancer Center

New York, New York, United States, 10065

United States, North Carolina

Duke University Medical Center - Duke Cancer Center

Durham, North Carolina, United States, 27710

United States, Texas

University of Texas, MD Anderson Cancer Center

Houston, Texas, United States, 77030

United States, Washington

University of Washington - Seattle Cancer Care Alliance

Seattle, Washington, United States, 98109

Sponsors and Collaborators

Aadi, LLC

More Information

• Responsible Party: Aadi, LLC
• ClinicalTrials.gov Identifier: NCT02494570
• Other Study ID Numbers: PEC001; FD005749 ( Other Grant/Funding Number: FDA-OOPD ); AMPECT ( Other Identifier: Aadi )
• First Posted: July 10, 2015
• Last Update Posted: December 10, 2019
• Last Verified: December 2019

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

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