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TUSC2-nanoparticles and Erlotinib in Stage IV Lung Cancer

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ClinicalTrials.gov Identifier: NCT01455389
Recruitment Status : Terminated (Trial terminated by sponsor after decision to evaluate combination of quaratusugene ozeplasmid and osimertinib instead of further evaluating quaratusugene ozeplasmid and erlotinib.)
First Posted : October 20, 2011
Last Update Posted : March 2, 2022
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-TUSC2 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-TUSC2 and erlotinib hydrochloride can help to control NSCLC.

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

DOTAP:Chol-TUSC2 (previously FUS1) is a drug that helps transfer a gene called TUSC2 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-TUSC2 Drug: Erlotinib Drug: Dexamethasone Drug: Diphenhydramine Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 25 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase I/II Clinical Trial Combining TUSC2-nanoparticles and Erlotinib in Stage IV Lung Cancer
Actual Study Start Date : February 2014
Actual Primary Completion Date : October 27, 2016
Actual Study Completion Date : September 28, 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Lung Cancer

Arm Intervention/treatment
Experimental: DOTAP + Erlotinib
DOTAP:Chol-TUSC2 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-TUSC2
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-TUSC2 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-TUSC2 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 TUSC2
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. There is no limit to the number or form of prior therapy regimens received, but patients must have received at least one.
  3. All subjects must have tumor specimens adequate for analysis of EGFR mutations or have tumor accessible for pretreatment biopsy, and must consent to post-treatment Guardant ctDNA liquid biopsy. Subjects must have specimens adequate for analysis of EGFR mutations (and other clinically relevant biomarkers)
  4. All subjects with an activating EGFR mutation (exon 19 deletion or exon 21 L858R mutation) are eligible IF they have progressed following treatment with a first, second, or third generation EGFR inhibitor. All subjects who are EGFR negative or have wild-type EGFR or another non-activating mutation are eligible.
  5. ECOG or Zubrod Performance Status ≤1
  6. Age ≥18 years
  7. Subjects must have voluntarily signed an informed consent in accordance with institutional policies.
  8. Female subjects of childbearing potential (non-childbearing is defined as greater than one year post-menopausal or surgically sterilized) must have a Negative serum pregnancy test (serum beta-HCG) ≤7 days of study treatment. 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 (2) Transvaginal Ultrasound (TVUS) scans one (1) week apart along with serial beta-HCG levels two (2) 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.
  9. Subjects are required to agree to practice effective birth control (i.e., abstinence, intrauterine device for female subjects) during the study period.
  10. Subjects must be ≥4 weeks beyond major surgical procedures such as thoracotomy, laparotomy or joint replacement, and must be ≥1.5 weeks 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. Note: placement of pleurex catheter is not considered minor surgery for this study.
  11. ANC >1500/mm3, platelet count >100,000/mm3 ≤14 days of study treatment
  12. PT and PTT <1.25 times the institutional upper limit of normal ≤14 days of study treatment
  13. Adequate renal function documented by serum creatinine of ≤1.5 mg/dl or calculated creatinine clearance >50 ml/min ≤14 days of study treatment
  14. Adequate hepatic function as documented by serum bilirubin <1.5 mg/dl and SGOT and SGPT ≤2.5 X upper limit of normal ≤14 days of study treatment
  15. Subjects 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 start of study treatment. Subjects must be off steroids that were being administered because of brain metastases or related symptoms for ≥2 weeks prior to start of study treatment. Post-treatment imaging ≤2 weeks of informed consent must demonstrate stability or regression of the brain metastases.
  16. Stable cardiac condition with a left ventricular ejection fraction ≥40% ≤28 days of study treatment

Exclusion Criteria:

  1. Subject is female who is pregnant or breast-feeding.
  2. Subject received investigational therapy (i.e., agents that are not FDA-approved), including monoclonal antibodies such as bevacizumab or cetuximab, or has received radiotherapy to the skull, spine, thorax or pelvis within ≤30 days of start of study treatment. Subjects are permitted to have received palliative radiotherapy to an extremity provided ≥14 days have elapsed since completion of radiotherapy, provided the subject received ≤10 radiotherapy fractions and a dose ≤30 Gy to that site, and provided skull, spine, thorax or pelvis were not in the radiotherapy field.
  3. Subject received standard chemotherapy with FDA-approved agents within ≤21 days of start of study treatment.
  4. Subject received a targeted therapy within ≤14 days prior to start of study treatment.
  5. Subject has active systemic viral, bacterial or fungal infection(s) requiring treatment.
  6. Subject has brain metastases (except as allowed in Section 3.2.1). Neurological assessment will be used to determine brain metastases.
  7. Subject has 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. Subject received prior gene therapy or cell therapy.
  9. Subject has history of myocardial infarction or unstable angina ≤6 months of start of study treatment.
  10. Subject is known to be HIV positive.

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 ClinicalTrials.gov identifier (NCT number): NCT01455389

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United States, Texas
UT MD Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
Genprex, Inc.
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Principal Investigator: Charles Lu, MD UT MD Anderson Cancer Center
Additional Information:
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Responsible Party: Genprex, Inc.
ClinicalTrials.gov Identifier: NCT01455389    
Other Study ID Numbers: ONC-002
First Posted: October 20, 2011    Key Record Dates
Last Update Posted: March 2, 2022
Last Verified: February 2022
Keywords provided by Genprex, Inc.:
Lung Cancer
Non-small cell lung cancer
DOTAP:Cholesterol-TUSC2 Liposome Complex
Erlotinib Hydrochloride
Benylin Cough SyrupPharmacokinetic
Additional relevant MeSH terms:
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Lung Neoplasms
Respiratory Tract Neoplasms
Thoracic Neoplasms
Neoplasms by Site
Lung Diseases
Respiratory Tract Diseases
Erlotinib Hydrochloride
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
Protein Kinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Sleep Aids, Pharmaceutical
Hypnotics and Sedatives
Central Nervous System Depressants
Anesthetics, Local