FUS1-nanoparticles and Erlotinib in Stage IV Lung Cancer

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT01455389
Recruitment Status : Recruiting
First Posted : October 20, 2011
Last Update Posted : March 16, 2017
Information provided by (Responsible Party):
Genprex, Inc.

Brief Summary:

The goal of phase 1 of this clinical research study is to find the highest dose of DOTAP:Chol-fus1 that can be safely given in combination with Tarceva (erlotinib hydrochloride) to patients with NSCLC.

The goal of phase 2 of this clinical research study is to learn if the combination of DOTAP:Chol-fus1 and erlotinib hydrochloride can help to control NSCLC.

The safety of this drug combination will also be studied in both phases.

DOTAP:Chol-fus1 is a drug that helps transfer a gene called fus1 into cancer cells. Researchers think that cells without this gene may be involved in the development of lung cancer tumors. They want to find out if replacing the gene in these cells may keep the tissue from forming cancer cells.

Erlotinib hydrochloride is designed to block a protein on tumor cells that may control tumor growth and survival. This may stop tumors from growing.

Condition or disease Intervention/treatment Phase
Lung Cancer Drug: DOTAP:Chol-fus1 Drug: Erlotinib Drug: Dexamethasone Drug: Diphenhydramine Phase 1 Phase 2

  Show Detailed Description

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 57 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase I/II Clinical Trial Combining FUS1-nanoparticles and Erlotinib in Stage IV Lung Cancer
Study Start Date : February 2014
Estimated Primary Completion Date : March 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Lung Cancer

Arm Intervention/treatment
Experimental: DOTAP + Erlotinib
DOTAP:Chol-fus1 0.045 mg/kg by vein over 25-35 minutes on day 1 of each 21 day cycle; and Erlotinib 100 mg by mouth daily for each 21 day cycle.
Drug: DOTAP:Chol-fus1
Starting Dose 0.045 mg/kg by vein on day 1 of each 21 day cycle; Phase II is Maximum Tolerated Dose from Phase I.
Other Name: DOTAP:Cholesterol-Fus1 Liposome Complex

Drug: Erlotinib
Starting Dose 100 mg by mouth each day of a 21 day cycle (except for first week of Cycle 1, if enrolled in Phase II delayed-schedule group). Phase II is Maximum Tolerated Dose from Phase I.
Other Names:
  • Erlotinib Hydrochloride
  • OSI-774
  • Tarceva

Drug: Dexamethasone
8 mg orally 24 and 12 hours and 20 mg by vein 30 minutes before DOTAP:Chol-fus1 treatment followed by 8 mg orally at 12, 24 and 36 hours after treatment (total number doses = 5).
Other Name: Decadron

Drug: Diphenhydramine
50 mg by mouth or by vein 30 minutes prior to treatment with DOTAP:Chol FUS1
Other Names:
  • Benadryl
  • Benylin Cough Syrup [OTC]

Primary Outcome Measures :
  1. Maximum Tolerated Dose (MTD) Level for Drug Treatment Combination [ Time Frame: First 21 day cycle ]
    MTD defined as dose level at which less than 2 participants experience dose-limiting toxicity (DLT). Toxicity graded according to National Cancer Institute (NCI) Common Toxicity Criteria (CTC) Version 4. DLT will be grade > 3 toxicity occurring during the first cycle of therapy (i.e., within the first 3 weeks).

Secondary Outcome Measures :
  1. Response Rate [ Time Frame: After two, 21 day cycles ]
    Responses determined by RECIST criteria. Responses will include only complete response (CR) + partial response (PR). Participants considered as non-responders when tumor progression by RECIST is observed. Measurable disease is defined as tumor masses with identifiable diameters measurable in two dimensions by computed tomography. Best overall response is best response designation recorded from the start of treatment until disease progression. Complete and partial responses have to be confirmed by two evaluations of the disease taken at least four weeks apart.

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

Inclusion Criteria:

  1. Histologically or cytologically documented non-small cell lung cancer (NSCLC) .
  2. Stage IV NSCLC, or recurrent NSCLC that is not potentially curable by radiotherapy or surgery whether or not they have received prior chemotherapy. There is no limit to the number of prior chemotherapy regimens received.
  3. All patients must have tumor specimens adequate for analysis of EGFR mutations and have tumor accessible to biopsy and must consent to biopsy.
  4. Karnofsky Performance Status of 70% or greater, or Zubrod Performance Status of 1or less.
  5. Age >/= 18 years.
  6. Patients must have voluntarily signed an informed consent in accordance with institutional policies.
  7. Negative serum pregnancy test (serum HCG) within 7 days of study treatment if female and of childbearing potential (non-childbearing is defined as greater than one year post-menopausal surgically sterilized). Since beta-HCG may be falsely elevated as a result of malignancy, women of child-bearing potential who have an elevated serum beta-HCG level are eligible for enrollment if they have two Transvaginal Ultrasound (TVUS) scans one week apart along with serial beta-HCG levels two weeks apart that are inconsistent with pregnancy and a Gynecology consult to ensure that the beta- HCG level was at a value high enough to see pregnancy with TVUS.
  8. Subjects are required to agree to practice effective birth control (i.e. abstinence, intrauterine device for female subjects) during the study period.
  9. Patients must be 4 weeks or greater, beyond major surgical procedures such as thoracotomy, laparotomy or joint replacement, and must be 1.5 weeks or greater, beyond minor surgical procedures such as biopsy of subcutaneous tumors, pleuroscopy, etc, and must not have evidence of wound dehiscence, active wound infection, or comparable major residual complications of the surgery.
  10. ANC > 1500/mm3, plt count > 100,000/mm3
  11. PT and PTT < 1.25 times the institutional upper limit of normal.
  12. Adequate renal function documented by serum creatinine of 1.5 mg/dl or less, or calculated creatinine clearance > 50 ml/min.
  13. Adequate hepatic function as documented by serum bilirubin< 1.5 mg/dl and SGOT and SGPT 1.5 or less x upper limit of normal.
  14. Patients with asymptomatic brain metastases that have been treated are eligible if the following criteria are met: No history of seizures in the preceding 6 months. Definitive treatment must have been completed >/= 4 weeks prior to registration. Subjects must be off steroids that were being administered because of brain metastases or related symptoms for >/= 2 weeks. Post-treatment imaging within 2 weeks of registration must demonstrate stability or regression of the brain metastases.
  15. Stable cardiac condition with a left ventricular ejection fraction of 40% or greater.
  16. FEV1 and corrected DLCO of 35% or > of predicted.
  17. Absence of an activating mutation (Exon 19 deletion or Exon 21 L858R mutation) in the epidermal growth factor receptor (EGFR) in the pre-treatment biopsy of the tumor. Patients with activating EGFR mutations are eligible if they have progressed following treatment with erlotinib. A pretreatment tumor biopsy must be available for analysis. If a biopsy has not been performed prior to entry, then a biopsy will be required.

Exclusion Criteria:

  1. Females who are pregnant or breast-feeding.
  2. "Study entry" is defined as the date of informed consent. Patients who received investigational therapy (agents that are not FDA approved), monoclonal antibody such as bevacizumab or cetuximab, or who received radiotherapy to the skull, spine, thorax or pelvis within 30 days of entry into the protocol. Patients are permitted to have received palliative radiotherapy to an extremity provided at least 14 days has elapsed since completion of therapy, provided the patient received no more than 10 radiotherapy fractions and a dose no higher than 30 Gy to that site, and provided skull, spine, thorax or pelvis were not in the radiotherapy field.
  3. Patients who have received standard chemotherapy with FDA approved agents within 21 days of entry into the protocol.
  4. Patients who have received therapy with an oral tyrosine kinase inhibitor (eg, erlotinib) within 14 days prior to entry into the protocol.
  5. Active systemic viral, bacterial or fungal infections requiring treatment.
  6. Patients with brain metastases (except as allowed in section 4.1.14 of the protocol. Neurological assessment will be used to determine brain metastases.
  7. Patients with serious concurrent illness or psychological, familial, sociological, geographical, or other concomitant conditions that, in the opinion of the investigator, would not permit adequate follow-up and compliance with the study protocol.
  8. Prior gene therapy.
  9. History of myocardial infarction within 6 months or unstable angina within the past 6 months.
  10. Patients known to be HIV positive are ineligible.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01455389

Contact: Charles Lu, MD 713-792-6363

United States, Texas
UT MD Anderson Cancer Center Recruiting
Houston, Texas, United States, 77030
Sponsors and Collaborators
Genprex, Inc.
Principal Investigator: Charles Lu, MD UT MD Anderson Cancer Center

Additional Information:
Responsible Party: Genprex, Inc. Identifier: NCT01455389     History of Changes
Other Study ID Numbers: 2011-0432
First Posted: October 20, 2011    Key Record Dates
Last Update Posted: March 16, 2017
Last Verified: March 2017

Keywords provided by Genprex, Inc.:
Lung Cancer
Non-small cell lung cancer
DOTAP:Cholesterol-Fus1 Liposome Complex
Erlotinib Hydrochloride
Benylin Cough SyrupPharmacokinetic

Additional relevant MeSH terms:
Lung Neoplasms
Respiratory Tract Neoplasms
Thoracic Neoplasms
Neoplasms by Site
Lung Diseases
Respiratory Tract Diseases
Dexamethasone acetate
Erlotinib Hydrochloride
BB 1101
Anti-Inflammatory Agents
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Gastrointestinal Agents
Hormones, Hormone Substitutes, and Hormone Antagonists
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Protease Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Protein Kinase Inhibitors
Anesthetics, Local