Trial of Nab-sirolimus in Patients With Well-differentiated Neuroendocrine Tumors (NETs) of the Gastrointestinal Tract, Lung, or Pancreas Who Have Not Received Prior Treatment With mTOR Inhibitors

NCT05997056 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: NET-202
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 4

Brief Summary

A Phase 2 multi-center, open-label, single arm study of nab-sirolimus in patients with well-differentiated neuroendocrine tumors (NETs) of the gastrointestinal tract, lung, or pancreas who have not received prior treatment with mTOR inhibitors

Conditions

Neuroendocrine Tumors; NET; Pancreatic Neuroendocrine Tumor; Gastrointestinal Neuroendocrine Tumor; Pulmonary Neuroendocrine Tumor

Keywords

FYARRO; nab-sirolimus; ABI-009; Neuroendocrine Tumors; NET; Pancreatic Neuroendocrine Tumor; Gastrointestinal Neuroendocrine Tumor; Pulmonary Neuroendocrine Tumor

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants With Objective Response Rate

  Objective Response Rate (ORR) is defined as the proportion of patients with best overall response (BOR) of confirmed partial response (PR) or complete response (CR) from the time of study treatment initiation until progression of disease (PD) as determined by the Investigator using Response Evaluation Criteria in Solid Tumors (RECIST) v1.1. Progression (PD) is defined per RECIST v1.1 as at least a 20% increase in the sum of diameters of target lesions and an absolute increase of at least 5 mm, or unequivocal progression of non-target lesions, or the appearance of one or more new lesions.

  From the start of study treatment to radiographically confirmed disease progression (PD) per RECIST v1.1, assessed at screening, every 6 weeks (±7 days) for the first 12 weeks, and every 12 weeks (±7 days) thereafter, up to 22 months.

Secondary Outcomes (6)

• Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Related Adverse Events.

  From first dose of study treatment through end of treatment (estimated up to 18 months) plus safety follow-up of 30 days after the last dose.

• Duration of Response (DOR)

  From first documented response (CR or PR) to radiographically confirmed disease progression (PD) per RECIST v1.1 or death from any cause, assessed at screening, every 6 weeks for the first 12 weeks, and every 12 weeks thereafter, up to 22 months

• Disease Control Rate (DCR)

  From the start of study treatment to radiographically confirmed disease progression (PD) per RECIST v1.1, assessed at screening, every 6 weeks for the first 12 weeks, and every 12 weeks thereafter, up to 22 months

• Time to Response (TTR)

  From the first dose of study treatment to the initial documentation of complete response (CR) or partial response (PR) per RECIST v1.1 , assessed at screening, every 6 weeks for the first 12 weeks, and every 12 weeks thereafter, up to 22 months

• Progression-free Survival（PFS）

  From first dose of study treatment until the date of first documented disease progression or death from any cause, whichever occurs first, assessed every 6 weeks for the first 12 weeks and every 12 weeks thereafter, up to approximately 21 months

Study Arms (1)

neuroendocrine tumors

EXPERIMENTAL

Patients with well-differentiated neuroendocrine tumors of the gastrointestinal tract, lung, or pancreas.

Drug: nab-sirolimus

Interventions

nab-sirolimus DRUG

Prospective phase 2 single arm, open-label, multi-institutional study to determine the efficacy and safety prospective of nab-sirolimus administered by IV infusion

Also known as: ABI-009

neuroendocrine tumors

Eligibility Criteria

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

You may qualify if:

• Patients with functional or non-functional, well-differentiated, locally advanced unresectable or metastatic NETs of the GI tract, lung, or pancreas who have received 2 or less prior lines of therapy excluding somatostatin analogs
• Patients with functional NETs may enroll if:
• the patient has been on a stable dose of an somatostatin analogs for ≥12 weeks and
• the patient has experienced disease progression while on stable somatostatin analogs dose
• Patients must have 1 or more measurable target lesions by RECIST v1.1
• Age: 18 years or older
• Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1 or Karnofsky Performance Status (KPS) ≥80
• Adequate liver function:
• Total bilirubin ≤1.5 × upper limit of normal (ULN) (unless due to Gilbert's syndrome or attributable to liver metastases, then ≤3 × ULN)
• Aspartate aminotransferase (AST) and alanine transaminase (ALT) ≤2.5 × ULN (≤5 × ULN if attributable to liver metastases)
• Adequate renal function: creatinine clearance ≥30 mL/min, Cockcroft-Gault creatinine clearance = ((140-age) × weight\[kg\]) / (72 × serum creatinine \[mL/min\]) × 0.85, if female.
• Adequate hematologic parameters:
• Absolute neutrophil count (ANC) ≥1.0 × 10\^9/L (growth factor support allowed)
• Platelet count ≥100,000/mm\^3 (100 × 10\^9/L) (transfusion and/or growth factor support allowed)
• Hemoglobin ≥8.0 g/dL (transfusion and/or growth factor support allowed)

You may not qualify if:

• Prior treatment with mTOR inhibitors including nab-sirolimus
• Note: Patients who have previously received locoregional or liver-directed therapies (radiofrequency or microwave ablation, transarterial chemoembolization, etc.) are eligible to enroll in the study.
• Patients with functional NETs who are experiencing uncontrolled symptoms attributed to hormones and other vasoactive substances secreted by the tumor
• Patients with inactivating TSC1 or TSC2 alterations (based on tissue or liquid NGS)
• Severe (Grade ≥3) ongoing infection requiring parenteral or oral anti-infective treatment, either ongoing or completed ≤7 days prior to enrollment
• Patients who have any severe and/or uncontrolled medical or psychiatric conditions or other conditions that could affect their participation including:
• Known or suspected brain metastases
• Severe heart disease defined as unstable angina pectoris, NYHA Class III or IV congestive heart failure, myocardial infarction ≤6 months prior to first study treatment, serious uncontrolled cardiac arrhythmia or any other clinically significant cardiac disease.
• Severe lung disease defined as a diffusing capacity for carbon monoxide that is ≤50% of normal predicted value and/or an O2 saturation ≤88% at rest on room air
• (Note: Spirometry and pulmonary function tests are not required to be performed unless clinically indicated.)
• Nonmalignant medical illnesses that are uncontrolled or whose control may be jeopardized by the treatment with the study therapy
• A history of malignancies other than the one under treatment unless the patient is disease-free for more than 5 years from diagnosis. Controlled non-melanoma skin cancers, carcinoma in situ of the cervix, resected incidental prostate cancer, certain low-grade hematologic malignancies (eg, chronic lymphocytic leukemia, follicular lymphoma, etc), or other adequately treated carcinoma in situ may be eligible, after discussion with the medical monitor.
• Uncontrolled hypertension (systolic blood pressure ≥160 mmHg and/or diastolic blood pressure ≥100 mmHg)
• Patients with history of interstitial lung disease and/or pneumonitis, or pulmonary hypertension
• Active Hepatitis B and/or Hepatitis C infection and detectable viral load despite antiviral therapy.

Sponsors & Collaborators

• Aadi Bioscience, Inc.: lead

Study Sites (4)

Hoag Memorial Hospital Presbyterian

Newport Beach, California, 92663, United States

Location

Rocky Mountain Cancer Centers

Denver, Colorado, 80218, United States

Location

Texas Oncology

Dallas, Texas, 75246, United States

Location

Medical College of Wisconsin Cancer Center

Milwaukee, Wisconsin, 53226, United States

Location

MeSH Terms

Conditions

Neuroendocrine Tumors; Adenoma, Islet Cell; Gastro-enteropancreatic neuroendocrine tumor

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Nerve Tissue; Adenoma; Neoplasms, Glandular and Epithelial; Pancreatic Neoplasms; Digestive System Neoplasms; Neoplasms by Site; Endocrine Gland Neoplasms; Digestive System Diseases; Pancreatic Diseases; Endocrine System Diseases

Results Point of Contact

• Title: Leonor Nazareno
• Organization: Aadi Bioscience, Inc.

lnazareno@aadibio.com

(973) 809-2290

Study Officials

• Leonor Nazareno

  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

Study Record Dates

• First Submitted: August 10, 2023
• First Posted: August 18, 2023
• Study Start: October 20, 2023
• Primary Completion: October 3, 2025
• Study Completion: October 3, 2025
• Last Updated: August 25, 2026
• Results First Posted: August 25, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)