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S1314, Co-expression Extrapolation (COXEN) Program to Predict Chemotherapy Response in Patients With Bladder Cancer

Registration Number
NCT02177695
Lead Sponsor
SWOG Cancer Research Network
Brief Summary

The primary focus of this study is to see if looking at tumor biomarkers using a program called coexpression extrapolation or "COXEN" may predict a patient's response to chemotherapy before surgery.

Detailed Description

The COXEN program will not select a patient's therapy, but the type of chemotherapy that he/she will receive will be randomly decided. The patient's response to chemotherapy will be used to test the usefulness of the COXEN program, which is the main goal of this trial. Other potential tests to predict a patient's response to chemotherapy will also be evaluated. In this study, the patient may receive the treatment in Arm 1 (gemcitabine and cisplatin) or the treatment in Arm 2 \[methotrexate, vinblastine, doxorubicin, cisplatin, and filgrastim (or pegfilgrastim)\]. There will be about 230 people taking part in this study (115 in each arm).

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
237
Inclusion Criteria
  • Histologically proven bladder cancer (pure small cell carcinoma, pure adenocarcinoma, and pure squamous cell carcinoma histologies are excluded).
  • Stage cT2-T4a N0 M0 disease.
  • Documented muscle invasive disease with at least one of the following: disease measuring at least 10 mm on cross-sectional imaging OR the presence of tumor-associated hydronephrosis.
  • Staging scans with abdominal/pelvic CT or MRI scan and CT scan or x-ray of the chest within 56 days prior to registration. If alkaline phosphatase is above the treating institution's upper limit of normal (ULN), presence of suspicious bone pain, or if other clinical suspicion, a whole body bone scan is required within 56 days prior to registration.
  • Performance status = 0 or 1
  • 18 years of age or older
  • Must have tumor tissue from transurethral resection of the bladder tumor (TURBT) available for submission that is sufficient for COXEN testing and must agree to submission of 20 (10 micron) slides plus 2 (5 micron) slides from the start and end of the 20 slides for a total of 22 unstained slides.
  • Must agree to collection of tissue (if residual disease is present), urine, and whole blood.
  • Must agree to participate in the translational medicine studies outlined in the protocol
Exclusion Criteria
  • Prior systemic cytotoxic chemotherapy or systemic anthracycline
  • Peripheral neuropathy >/= Grade 2
  • Class III/IV heart failure or known left ventricular ejection fraction (LVEF) < 50%
  • Clinically relevant hearing impairment > Grade 2
  • Renal function, calculated creatinine clearance < 60 mL/min
  • Hepatic function, total bilirubin > 1.5 x institutional upper limit of normal (IULN) (or > 2.5 x IULN with Gilbert's disease); AST & ALT > 2 X IULN
  • Hematologic function, absolute neutrophil count (ANC) < 1,500/mcL, hemoglobin < 9 g/dL, and platelets < 100,000/mcL
  • Hypersensitivity to cisplatin, gemcitabine, doxorubicin, vinblastine, methotrexate, or filgrastim/pegfilgrastim
  • Incidence of or uncontrolled medical illness (e.g. active cardiac symptoms, active systemic infection, etc.)
  • Pregnant or nursing females
  • No other prior malignancy is allowed except for the following: adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, adequately treated Stage I or II cancer from which the patient is currently in complete remission, or any other cancer from which the patient has been disease free for five years. However, patients with localized prostate cancer who are being followed by an active surveillance program are eligible.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Dose Dense MVACCisplatinMethotrexate, 30 mg/m2, IV, Day 1, q 14 days x 4 cycles Vinblastine, 3 mg/m2, IV, Day 1 or 2, q 14 days x 4 cycles Doxorubicin, 30 mg/m2, IV, Day 1 or 2, q 14 days x 4 cycles Cisplatin, 70 mg/m2, IV, Day 1 or 2, q 14 days x 4 cycles Filgrastim, 5 mcg/kg, SubQ/IV, Days 3-7, q 14 days x 4 cycles
Gemcitabine & CisplatinGemcitabineGemcitabine, 1000 mg/m2, IV, Days 1\&8, q 21 days x 4 cycles Cisplatin, 70 mg/m2, IV, Day 1, q 21 days x 4 cycles
Gemcitabine & CisplatinCisplatinGemcitabine, 1000 mg/m2, IV, Days 1\&8, q 21 days x 4 cycles Cisplatin, 70 mg/m2, IV, Day 1, q 21 days x 4 cycles
Dose Dense MVACDoxorubicinMethotrexate, 30 mg/m2, IV, Day 1, q 14 days x 4 cycles Vinblastine, 3 mg/m2, IV, Day 1 or 2, q 14 days x 4 cycles Doxorubicin, 30 mg/m2, IV, Day 1 or 2, q 14 days x 4 cycles Cisplatin, 70 mg/m2, IV, Day 1 or 2, q 14 days x 4 cycles Filgrastim, 5 mcg/kg, SubQ/IV, Days 3-7, q 14 days x 4 cycles
Dose Dense MVACMethotrexateMethotrexate, 30 mg/m2, IV, Day 1, q 14 days x 4 cycles Vinblastine, 3 mg/m2, IV, Day 1 or 2, q 14 days x 4 cycles Doxorubicin, 30 mg/m2, IV, Day 1 or 2, q 14 days x 4 cycles Cisplatin, 70 mg/m2, IV, Day 1 or 2, q 14 days x 4 cycles Filgrastim, 5 mcg/kg, SubQ/IV, Days 3-7, q 14 days x 4 cycles
Dose Dense MVACVinblastineMethotrexate, 30 mg/m2, IV, Day 1, q 14 days x 4 cycles Vinblastine, 3 mg/m2, IV, Day 1 or 2, q 14 days x 4 cycles Doxorubicin, 30 mg/m2, IV, Day 1 or 2, q 14 days x 4 cycles Cisplatin, 70 mg/m2, IV, Day 1 or 2, q 14 days x 4 cycles Filgrastim, 5 mcg/kg, SubQ/IV, Days 3-7, q 14 days x 4 cycles
Dose Dense MVACFilgrastimMethotrexate, 30 mg/m2, IV, Day 1, q 14 days x 4 cycles Vinblastine, 3 mg/m2, IV, Day 1 or 2, q 14 days x 4 cycles Doxorubicin, 30 mg/m2, IV, Day 1 or 2, q 14 days x 4 cycles Cisplatin, 70 mg/m2, IV, Day 1 or 2, q 14 days x 4 cycles Filgrastim, 5 mcg/kg, SubQ/IV, Days 3-7, q 14 days x 4 cycles
Primary Outcome Measures
NameTimeMethod
Assessment of Whether the Treatment-specific COXEN Score is Prognostic of ≤ pT1 Rateup to 5 years post-registration

The proportion of participants achieving \<=pT1 in both favorable and unfavorable treatment specific-COXEN score categories.

Unit of measure is the number of participants in each category that achieved \<= pT1

____________________________________________________________________________________________________________________ The relationship of dose-dense methotrexate, vinblastin, doxorubicin, and cisplatin (DDMVAC)- and gemcitabine+cisplatin (GC)- specific CO-eXpression ExtrapolatioN (COXEN) scores

This will be done in two ways:

* By assessing whether the treatment-specific COXEN score is prognostic of pT0 rate or ≤ pT1 in this patient population and to assess in a preliminary fashion whether the COXEN score is a predictive factor distinguishing between these two chemotherapy regimens. .

* By evaluating the correlation between the GC- and the DDMVAC-COXEN score.

Correlation Between GC-and ddMVAC-COXEN Scoreup to 5 years post-registration

The Pearson and Spearman correlation coefficients for GC-and ddMVAC-COXEN score were calculated and are reported below.

___________________________________________________________________________________________________________________ The relationship of dose-dense methotrexate, vinblastin, doxorubicin, and cisplatin (DDMVAC)- and gemcitabine+cisplatin (GC)- specific CO-eXpression ExtrapolatioN (COXEN) scores

This will be done in two ways:

* By assessing whether the treatment-specific COXEN score is prognostic of pT0 rate or ≤ pT1 in this patient population and to assess in a preliminary fashion whether the COXEN score is a predictive factor distinguishing between these two chemotherapy regimens. .

* By evaluating the correlation between the GC- and the DDMVAC-COXEN score.

Assessment of Whether the Treatment-specific COXEN Score is Prognostic of pT0 RateUp to 5 years post registration

The proportion of participants achieving pT0 in both favorable and unfavorable treatment specific-COXEN score categories.

Unit of measure is the number of participants in each category that achieved pT0

_____________________________________________________________________________________________________________________

The relationship of dose-dense methotrexate, vinblastin, doxorubicin, and cisplatin (DDMVAC)- and gemcitabine+cisplatin (GC)- specific CO-eXpression ExtrapolatioN (COXEN) scores

This will be done in two ways:

* By assessing whether the treatment-specific COXEN score is prognostic of pT0 rate or ≤ pT1 in this patient population and to assess in a preliminary fashion whether the COXEN score is a predictive factor distinguishing between these two chemotherapy regimens. .

* By evaluating the correlation between the GC- and the DDMVAC-COXEN score.

Assessment of COXEN Score as a Predictive Factor Distinguishing Between GC and ddMVACup to 5 years post-registration

To assess in a hypothesis generating fashion, the ability of COXEN to select for an individual chemotherapy regimen (GC vs DDMVAC)

P-values are reported as a measure of whether COXEN can select between GC/DDMVAC and to determine the significance of interactions between treatment specific COXEN scores and treatment arms in models predicting either pT0 or \<= pT1. Interactions with p-values \> 0.05 are interpreted as not significant.

________ The relationship of dose-dense methotrexate, vinblastin, doxorubicin, and cisplatin (DDMVAC)- and gemcitabine+cisplatin (GC)- specific CO-eXpression ExtrapolatioN (COXEN) scores

This will be done in two ways:

* By assessing whether the treatment-specific COXEN score is prognostic of pT0 rate or ≤ pT1 in this patient population and to assess in a preliminary fashion whether the COXEN score is a predictive factor distinguishing between these two chemotherapy regimens. .

* By evaluating the correlation between the GC- and the DDMVAC-COXEN score.

Secondary Outcome Measures
NameTimeMethod
Pathologic T0 Rate Evaluation: Gemcitabine+Cisplatin (GC) Versus Dose-dense Methotrexate, Vinblastin, Doxorubicin, and Cisplatin (DDMVAC)Up to 5 years post-registration

Pathologic complete response rate at the time of cystectomy following GC or DDMVAC treatment

Number of Participants With Gr 3 Through 5 Adverse Events That Are Related to Study DrugsDuration of treatment and follow up until death or 5 years post registration

Adverse Events (AEs) are reported by CTCAE Version 4.0. Only adverse events that are possibly, probably or definitely related to study drug are reported. Grade 3 is less severe than Grade 5.

Grade 3 - Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care ADL\* (bathing, dressing and undressing, feeding self, using the toilet, taking medications, and not bedridden).

Grade 4 - Life-threatening consequences; urgent intervention indicated. Grade 5 - Death related to AE

\*ADL- Activities of Daily Living

Predictability of the CO-eXpression ExtrapolatioN (COXEN) Score to Direct Which of the Two Regimens the Patient Should Receive: Gemcitabine+Cisplatin (GC) Versus Dose-dense Methotrexate, Vinblastin, Doxorubicin, and Cisplatin (DDMVAC)Up to 5 years post registration

In addition to stratification factors and dichotomous COXEN GEM score, an indicator for treatment arm and the interaction of treatment arm with COXEN GEM score was also included in a logistic regression model. A significant interaction would suggest that the respective COXEN GEM score was able to differentiate whether a patient was more likely to respond to one chemotherapy regimen over another. \*note that this is the same objective at Primary Outcome #3 above - this was erroneously listed twice in the protocol.

Value of Gene Expression Profiling in Predicting Overall Survival (OS)Up to 5 years post registration

5-year OS curve was estimated using the Kaplan-Meier method. Included all COXEN evaluable participants regardless of arm assignment. The report groups were favorable vs. unfavorable COXEN status.

Trial Locations

Locations (566)

University of Alabama at Birmingham Cancer Center

🇺🇸

Birmingham, Alabama, United States

Fairbanks Memorial Hospital

🇺🇸

Fairbanks, Alaska, United States

University of Arizona Cancer Center at Saint Joseph's

🇺🇸

Phoenix, Arizona, United States

University of Arizona Cancer Center-Orange Grove Campus

🇺🇸

Tucson, Arizona, United States

University of Arizona Cancer Center-North Campus

🇺🇸

Tucson, Arizona, United States

The University of Arizona Medical Center-University Campus

🇺🇸

Tucson, Arizona, United States

Yuma Cancer Center

🇺🇸

Yuma, Arizona, United States

Fowler Family Center for Cancer Care

🇺🇸

Jonesboro, Arkansas, United States

AIS Cancer Center at San Joaquin Community Hospital

🇺🇸

Bakersfield, California, United States

City of Hope Comprehensive Cancer Center

🇺🇸

Duarte, California, United States

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University of Alabama at Birmingham Cancer Center
🇺🇸Birmingham, Alabama, United States

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