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Trial record 2 of 7 for:    Etirinotecan Pegol

Phase II Etirinotecan Pegol in Refractory Brain Metastases & Advanced Lung Cancer / Metastatic Breast Cancer

This study is currently recruiting participants.
See Contacts and Locations
Verified June 2017 by Joel Neal, Stanford University
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Joel Neal, Stanford University Identifier:
First received: December 4, 2014
Last updated: June 24, 2017
Last verified: June 2017
This phase II trial studies how well pegylated irinotecan NKTR 102 works in treating patients with non-small cell lung cancer, small cell lung cancer, or breast cancer that has spread to the brain and does not respond to treatment. Pegylated irinotecan NKTR 102 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Condition Intervention Phase
Extensive Stage Small Cell Lung Cancer Recurrent Non-small Cell Lung Cancer Recurrent Small Cell Lung Cancer Stage IV Non-small Cell Lung Cancer Tumors Metastatic to Brain Metastatic Breast Cancer Drug: pegylated irinotecan NKTR 102 Other: laboratory biomarker analysis Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II Study of Etirinotecan Pegol (NKTR 102) in Patients With Refractory Brain Metastases and Advanced Lung Cancer or Metastatic Breast Cancer (MBC)

Resource links provided by NLM:

Further study details as provided by Joel Neal, Stanford University:

Primary Outcome Measures:
  • CNS disease control rate (number of patients with stable disease + partial response + complete response divided by the total number of evaluable patients) (Cohort A and C) [ Time Frame: At 12 weeks ]
    The proportion, its 95% confidence interval using the Exact method, and p value of rejection the null hypothesis (5%) will be summarized.

Secondary Outcome Measures:
  • Overall disease control rate (Cohort A and C) [ Time Frame: At 12 weeks ]
  • Overall response rate (Cohort A and C) [ Time Frame: At 12 weeks ]
  • Systemic (non-CNS) disease control rate (Cohort A and C) [ Time Frame: At 12 weeks ]
  • Systemic (non-CNS) response rate (Cohort A and C) [ Time Frame: At 12 weeks ]
  • Progression-free survival (PFS) (Cohort A and C) [ Time Frame: Date of first dose of pegylated irinotecan NKTR 102 to date of disease progression, assessed up to 2 years ]
    Will be described using Kaplan Meier estimates. The PFS probability at 12 weeks will be estimated with an 80% power and 95% confidence intervals (80% in accord with the planned alpha level, 95% for comparability with other studies, confidence intervals based on the Grenwood formula for the variance of a survival probability).

  • Overall survival (Cohort A and C) [ Time Frame: Date of pathologic diagnosis to date of death, assessed up to 2 years ]
    Will be described using Kaplan Meier estimates.

  • CNS and/or systemic disease control (Cohort B) [ Time Frame: At 12 weeks ]
  • Incidence of toxicity, graded according to National Cancer Institute Common Terminology for Adverse Events version 4.0 (Cohorts A, B, and C) [ Time Frame: Up to 2 years ]
    Toxicity will be tabulated by organ system and grade.

Estimated Enrollment: 15
Study Start Date: January 2015
Estimated Study Completion Date: January 2019
Estimated Primary Completion Date: January 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Treatment (pegylated irinotecan NKTR 102)
Patients receive pegylated irinotecan NKTR 102 IV over 90 minutes every 21 days in the absence of disease progression or unacceptable toxicity.
Drug: pegylated irinotecan NKTR 102
Given IV
Other Names:
  • NKTR 102
  • PEG-irinotecan
Other: laboratory biomarker analysis
Correlative studies

Detailed Description:

Primary Objective:

For cohort A and Cohort C, to determine the CNS disease control rate (number of patients with stable disease or partial response or complete response / total number of treated patients) at 12 weeks following treatment with etirinotecan pegol in patients with advanced NSCLC or with MBC with refractory brain metastases

Secondary Objectives:

Cohorts A and C:

  • To measure the overall disease control rate and response rate for patients receiving study therapy
  • To measure the systemic (non CNS) disease control rate and response rate for patients receiving study therapy
  • To observe the progression free survival of the study population
  • To observe the overall survival of the study population

Cohort B:

- To observe CNS and systemic disease control in SCLC

Cohorts A, B and C:

- To determine the safety profile of etirinotecan pegol (NKTR 102)


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • At least 18 years of age.
  • Life expectancy of 3 months or longer.
  • ECOG performance status of 0, 1, or 2.

Advanced or refractory cancer, consisting of

  • Metastatic breast cancer (mBC) for which single-agent cytotoxic chemotherapy is indicated. OR
  • Histologically-proven metastatic lung cancer:

    • Non-small cell lung cancer (NSCLC) as Stage IV disease or recurrent metastatic disease (per lung cancer TNM classification system, 7th ed) (Cohort A) OR
    • Small cell lung cancer (SCLC) as extensive stage or recurrent metastatic disease (cohort B), including tumors with mixed small cell and non-small cell elements.

Prior chemotherapy (at least one of the following):

  • At least one line of prior systemic chemotherapy
  • At least one line of prior targeted treatment for metastatic disease Adjuvant systemic chemotherapy within prior 6 months Prior treatment for mBC must have included taxane-based regimen

Prior chemotherapy, including other investigational therapy, has been completed prior to initiation of study treatment, according to the following:

  • ≥ 2 weeks if immediately preceding treatment was chemotherapy/targeted therapy administered on a daily or weekly schedule
  • ≥ 3 weeks if immediately preceding treatment was chemotherapy/targeted therapy administered every 2 weeks
  • ≥ 4 weeks if immediately preceding treatment was chemotherapy/targeted therapy administered every 3 weeks Previously received at least one CNS directed treatment (such as surgery or radiation) OR not be eligible for CNS stereotactic radiosurgery Measurable CNS disease, either previously untreated (not counting systemic therapy), or progressed following previous radiation treatment. Lesions that have progressed after prior radiosurgery should not be selected as measurable disease if they are suspected of being radionecrosis.

The following measurement criteria are required, as visualized by contrast-enhanced MRI with slice thickness of ≤ 1.5 mm, unless absence of contrast or thicker slices is specifically authorized by Protocol Director. Measurements do not include tumor edema.

  • At least one CNS tumor measuring ≥ 10 mm in longest diameter, OR
  • At least one CNS tumor measuring 5-9 mm in longest diameter, plus one or two additional CNS tumors measuring ≥ 3 mm in longest diameter, for which the sum of the longest diameters is ≥ 10 mm. Additional tumors are not exclusionary.

Adequate organ function as evidenced by:

  • Absolute neutrophil count (ANC) ≥ 1.5 x 10e9/L without G-CSF (filgrastim, pegfilgrastim, or equivalent) support within 7 days
  • Hemoglobin (Hgb) ≥ 9.0 g/dL (90 g/L) without blood transfusion within 7 days
  • Platelet count ≥ 100 x 10e9/L without platelet transfusion within 7 days
  • Bilirubin ≤ 1.5 X upper limit of normal (ULN), except for patients with documented history of Gilbert's disease who may have DIRECT bilirubin ≤ 1.5 X ULN
  • Alanine aminotransferase (ALT) ≤ 2.5 X ULN, except ≤ 5 X ULN for patients with liver metastases
  • Aspartate aminotransferase (AST) ≤ 2.5 X ULN, except ≤ 5 X ULN for patients with liver metastases
  • Serum creatinine ≤ 1.5 X ULN; or calculated creatinine clearance ≥ 50 mL/min (using Cockcroft-Gault formula), or measured creatinine clearance ≥ 50 mL/min.

Exclusion Criteria:

  • Previous treatment with a camptothecin derivative (eg., irinotecan, topotecan, and investigational agents including but not limited to exatecan, rubitecan, gimatecan, karenitecan, SN38 investigational agents, EZN 2208, SN 2310, and AR 67) is not allowed
  • Patients may not have a known history of leptomeningeal disease, as diagnosed by positive CSF cytology, unless prospective permission for enrollment is granted from the sponsor and the PI
  • Patients may not have had major surgery or radiotherapy (therapeutic and/or palliative) within 14 days prior to initiation of study treatment, including CNS-directed radiation therapy. Minor procedures, such as tumor biopsy, thoracentesis, or intravenous catheter placement are allowed with no waiting period
  • Patients may not have the following co morbid disease or concurrent illness:

    • Chronic or acute gastrointestinal (GI) disorders resulting in diarrhea of any severity grade; patients may not use chronic anti-diarrheal supportive care (more than 3 days/week) to control diarrhea in the 28 days prior to first dose of investigational drug. (exception: anti-diarrheal medications used to control symptoms from a medication that will be discontinued prior to study are allowed with a 7 day washout before study therapy, for example loperamide for erlotinib-associated diarrhea)
    • Known cirrhosis, defined as Child Pugh class A or higher liver disease
    • Other active malignancy, except for non melanoma skin cancer and carcinoma in situ (of the cervix or bladder)
    • Any other severe/uncontrolled inter current illness or significant co morbid conditions that in the opinion of the investigator would impair study participation or cooperation
  • Patients may not have a known allergy or hypersensitivity to any of the components of the investigational therapy, including polyethylene glycol (PEG) or topoisomerase inhibitors
  • Patients may not be receiving the following medications at the time of first dose of investigational drug:

    • Pharmacotherapy for known hepatitis B or C, tuberculosis, or human immunodeficiency virus (HIV)
    • Any of the following enzyme inducing anti epileptic medications (EIAEDs): phenytoin, carbamazepine, oxcarbazepine, phenobarbital
    • Other chemotherapy, hormonal therapy, immunotherapy, other investigational agents, or biologic agents for the treatment of cancer except for bisphosphonates or denosumab
  • Pregnant or nursing patients will be excluded from the study
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT02312622

Contact: Sophie Bertrand 650-723-4467

United States, California
Stanford University, School of Medicine Recruiting
Stanford, California, United States, 94305
Contact: Sophie Bertrand    650-723-4467   
Principal Investigator: Joel W. Neal         
Sponsors and Collaborators
Joel Neal
National Cancer Institute (NCI)
Principal Investigator: Joel Neal Stanford University Hospitals and Clinics
  More Information

Responsible Party: Joel Neal, ASSISTANT PROFESSOR OF MEDICINE, Stanford University Identifier: NCT02312622     History of Changes
Other Study ID Numbers: LUN0067
NCI-2014-02101 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
LUN0067 ( Other Identifier: Stanford University Hospitals and Clinics )
P30CA124435 ( U.S. NIH Grant/Contract )
IRB - 30982 ( Other Identifier: Stanford University IRB )
Study First Received: December 4, 2014
Last Updated: June 24, 2017

Additional relevant MeSH terms:
Breast Neoplasms
Lung Neoplasms
Carcinoma, Non-Small-Cell Lung
Small Cell Lung Carcinoma
Neoplasms by Site
Breast Diseases
Skin Diseases
Respiratory Tract Neoplasms
Thoracic Neoplasms
Lung Diseases
Respiratory Tract Diseases
Carcinoma, Bronchogenic
Bronchial Neoplasms
Antineoplastic Agents, Phytogenic
Antineoplastic Agents
Topoisomerase I Inhibitors
Topoisomerase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action processed this record on September 21, 2017