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 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: NET-202
• Study Type: interventional
• Target: 21
• 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)

• Efficacy of nab-sirolimus

  Objective Response Rate (ORR), 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 RECIST v1.1

  12 Months

Secondary Outcomes (6)

• Incidence and severity of treatment

  12 Months

• Duration of response

  12 Months

• Disease control rate

  12 Months

• Time to response

  12 Months

• Progression-free survival

  12 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

RECRUITING

Rocky Mountain Cancer Centers

Denver, Colorado, 80218, United States

RECRUITING

Texas Oncology

Dallas, Texas, 75246, United States

RECRUITING

Medical College of Wisconsin Cancer Center

Milwaukee, Wisconsin, 53226, United States

RECRUITING

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

Study Officials

• Willis Navarro, MD

  Aadi Bioscience

  STUDY DIRECTOR

Central Study Contacts

Aadi Bioscience Medical Information

CONTACT

1-888-246-2234; MedInfo@aadibio.com

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: November 7, 2023
• Primary Completion: May 9, 2025
• Study Completion: December 8, 2025
• Last Updated: July 16, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)