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Intensity-Modulated Proton Beam Therapy or Intensity-Modulated Photon Therapy in Treating Patients With Stage III-IVB Oropharyngeal Cancer

Phase 3
Active, not recruiting
Conditions
Stage III Oropharyngeal Squamous Cell Carcinoma AJCC v7
Stage IVA Oropharyngeal Squamous Cell Carcinoma AJCC v7
Stage IVB Oropharyngeal Squamous Cell Carcinoma AJCC v7
Interventions
Radiation: Intensity-Modulated Radiation Therapy
Other: Laboratory Biomarker Analysis
Radiation: Proton Beam Radiation Therapy
Radiation: Photon Beam Radiation Therapy
Other: Quality-of-Life Assessment
Registration Number
NCT01893307
Lead Sponsor
M.D. Anderson Cancer Center
Brief Summary

This randomized phase III trial studies the side effects and how well intensity-modulated proton beam therapy works and compares it to intensity-modulated photon therapy in treating patients with stage III-IVB oropharyngeal cancer. Radiation therapy uses high-energy x-rays, protons, and other types of radiation to kill tumor cells and shrink tumors. It is not yet known whether intensity-modulated proton beam therapy is more effective than intensity-modulated photon therapy in treating oropharyngeal cancer.

Detailed Description

PRIMARY OBJECTIVES:

To compare the progression-free survival (PFS) between concurrent chemo-radiation strategies with IMRT and IMPT following the treatment of oropharyngeal tumors.

SECONDARY OBJECTIVES:

1. To assess and compare overall survival between IMRT and IMPT along with estimating disease-related outcomes such as: \[2-year progression-free survival, patterns of failure, 2-year overall survival, 2-year distant metastasis free survival, and second primary cancers\]

2. To assess acute and chronic/late side effects, and to compare the rates of Grade 3-5 toxicity between IMRT and IMPT following the treatment of oropharyngeal tumors.

3. To assess Patient Reported Outcome (PRO) measures of symptoms using MD Anderson Symptom Inventory (MDASI), MD Anderson Dysphagia Inventory (MDADI), FACT-HN, Xerostomia and Health Questionnaire (EQ-5D-3L), Work status (WPAI: SHP)

4. To assess Physician Reported Toxicity using Common Terminology Criteria for Adverse Events (CTCAE)-4.0

5. To evaluate and compare Quality-Adjusted-Life-Years (QALY) between IMPT and IMRT;

6. To perform Cost-benefit economic analysis of treatment;

7. To determine whether specific molecular profiles are associated with overall or progression-free survival;

8. To investigate associations between changes in blood biomarkers, or HPV-specific cellular immune responses, or HPV ctDNA (measured at baseline and three months and at each follow-up visit for up to 10 years) with overall or progression-free survival;

9. To bank peripheral blood at time of enrollment, weeks 2, 4, and 6 during treatment and at each follow up visits for up to 10 years to explore the ability of circulating markers to predict outcome;

10. To bank head and neck tissues to explore the ability of tissue-based markers to predict outcome;

11. To bank peripheral blood and tissues for future interrogations; Finally, all additional secondary endpoints

EXPLORATORY OBJECTIVE:

I. To assess potential differences between patients on study and patients who were considered eligible for randomized, were randomized to a treatment arm, but were denied insurance coverage for the treatment arm she/he was randomized to; or may have dropped out of the study for other reasons after being randomized. These patients will compromise Group 3: consisting of patients randomized to Protons but not treated and Group 4: consisting of patients randomized to IMRT but not treated at the designated institution. Furthermore, these patients will only be followed for recurrence and survival.(Phase III)

OUTLINE: Patients are randomized to 1 of 2 treatment arms.

ARM I: Patients undergo IMRT once daily (QD) five days a week for approximately 6.5 weeks.

ARM II: Patients undergo IMPT QD five days a week for approximately 6.5 weeks.

After completion of study treatment, patients are followed up every 3 months for 1 year, every 4 months for 1 year, and then every 6 months for 5 years.

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
440
Inclusion Criteria
  • Histologically documented squamous cell carcinoma of the oropharynx (American Joint Committee on Cancer [AJCC] version [v]7 stage III-IV A,B)
  • Tumor tissue (primary or cervical metastasis) available for human papilloma virus (HPV) and/or p16 (in situ hybridization [ISH], immunohistochemistry [IHC] or genotyping testing); if you do not have enough leftover tumor tissue available, you will have a tumor biopsy for tumor marker testing
  • Eastern Cooperative Oncology Group (ECOG) performance status = 0, 1, or 2
  • Negative pregnancy test for women of child bearing potential
  • Concurrent chemotherapy
  • Bilateral neck radiation
Exclusion Criteria
  • Previous radiation treatment for head and neck mucosal primary cancers within the past 5 years (i.e. oropharynx, nasopharynx, hypopharynx, larynx, and oral cavity)

  • Pregnant or breast-feeding females

  • Clinically significant uncontrolled major cardiac, respiratory, renal, hepatic, gastrointestinal or hematologic disease but not limited to:

    • Symptomatic congestive heart failure, unstable angina, or cardiac dysrhythmia not controlled by pacer device
    • Myocardial infarction within 3 months of registration
  • Distant metastases (stage IV C, any T, any N and M1)

  • Previous surgical resection or neck dissection for oropharyngeal cancer, administered with therapeutic intent

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Arm I (IMRT)Intensity-Modulated Radiation TherapyPatients undergo IMRT QD five days a week for approximately 6.5 weeks.
Arm II (IMPT)Intensity-Modulated Radiation TherapyPatients undergo IMPT QD five days a week for approximately 6.5 weeks.
Arm II (IMPT)Quality-of-Life AssessmentPatients undergo IMPT QD five days a week for approximately 6.5 weeks.
Arm II (IMPT)Laboratory Biomarker AnalysisPatients undergo IMPT QD five days a week for approximately 6.5 weeks.
Arm I (IMRT)Quality-of-Life AssessmentPatients undergo IMRT QD five days a week for approximately 6.5 weeks.
Arm II (IMPT)Proton Beam Radiation TherapyPatients undergo IMPT QD five days a week for approximately 6.5 weeks.
Arm I (IMRT)Laboratory Biomarker AnalysisPatients undergo IMRT QD five days a week for approximately 6.5 weeks.
Arm I (IMRT)Photon Beam Radiation TherapyPatients undergo IMRT QD five days a week for approximately 6.5 weeks.
Primary Outcome Measures
NameTimeMethod
Cumulative incidence of late onset grade 3+ toxicity anytime (Phase II)Up to 2 years

Will be graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0. The methods described by Gooley will be used to estimate the cumulative incidence of late onset grade 3+ toxicity by 2 years for each treatment arm with death as a competing risk. The methods of Fine and Gray will be used to model the cumulative incidence of late onset grade 3+ toxicity by 2 years as a function of treatment arm and other potential prognostic factors (e.g., human papillomavirus (HPV)/p16 status, use of induction chemotherapy) considering death as a competing risk. Hazard ratios for the prognostic factors from this model with 95% confidence intervals will be estimated.

Overall survival (OS) (Phase II)Up to 5 years

Stratified by treatment arm and estimated using the product limit estimator of Kaplan and Meier. Cox proportional hazards regression will be used to model OS as a function of potential prognostic factors. Hazard ratios for the prognostic factors from this model will be estimated with 95% confidence intervals.

Overall survival (Phase III)Up to 5 years

Will be summarized at critical time points using the method of Kaplan-Meier. Kaplan-Meier plots will be used to visualize the time-to-event information by treatment arm, and the trial will be monitored based on results from log-rank tests used to compare treatment arms. Furthermore, Cox proportional hazards regression will be used to assess the time-to-event outcomes while adjusting for covariates of interest.

Cumulative incidence of acute grade 3+ toxicity (Phase II)Up to 2 years

Will be graded according to the NCI CTCAE version 4.0. The methods described by Gooley will be used to estimate the cumulative incidence of late onset grade 3+ toxicity by 2 years for each treatment arm with death as a competing risk. The methods of Fine and Gray will be used to model the cumulative incidence of late onset grade 3+ toxicity by 2 years as a function of treatment arm and other potential prognostic factors (e.g., HPV/p16 status, use of induction chemotherapy) considering death as a competing risk. Hazard ratios for the prognostic factors from this model with 95% confidence intervals will be estimated.

Progression-free survival (Phase III)Up to 3 years

Will be summarized at critical time points using the method of Kaplan-Meier. Kaplan-Meier plots will be used to visualize the time-to-event information by treatment arm, and the trial will be monitored based on results from log-rank tests used to compare treatment arms. Furthermore, Cox proportional hazards regression will be used to assess the time-to-event outcomes while adjusting for covariates of interest.

Secondary Outcome Measures
NameTimeMethod
Quality of life (QoL) (Phase II and III)Up to 5 years

QoL assessments will be summarized using the mean score and standard deviation for each time point of interest. Mean response trajectories will be plotted over the time horizon for each QoL instrument administered to patients in order to explore differences between treatment arms along with other patient characteristics of interest. Other analyses such as area under the curve and linear mixed models will be used to make statistical comparisons between treatment arms while adjusting for covariates of interest.

Trial Locations

Locations (22)

University of Pennsylvania/Abramson Cancer Center

🇺🇸

Philadelphia, Pennsylvania, United States

Massachusetts General Hospital Cancer Center

🇺🇸

Boston, Massachusetts, United States

Northwestern Medicine Cancer Center Warrenville

🇺🇸

Warrenville, Illinois, United States

M D Anderson Cancer Center

🇺🇸

Houston, Texas, United States

Inova Schar Cancer Institute

🇺🇸

Fairfax, Virginia, United States

University of Florida Health Science Center - Gainesville

🇺🇸

Gainesville, Florida, United States

Willis-Knighton Medical and Cancer Center

🇺🇸

Shreveport, Louisiana, United States

University of Maryland/Greenebaum Cancer Center

🇺🇸

Baltimore, Maryland, United States

Mayo Clinic in Arizona

🇺🇸

Scottsdale, Arizona, United States

University of Alabama at Birmingham

🇺🇸

Birmingham, Alabama, United States

Miami Cancer Institute

🇺🇸

Miami, Florida, United States

Wayne State University/Karmanos Cancer Institute

🇺🇸

Detroit, Michigan, United States

Mayo Clinic in Rochester

🇺🇸

Rochester, Minnesota, United States

University of Oklahoma Health Sciences Center

🇺🇸

Oklahoma City, Oklahoma, United States

Emory University Hospital/Winship Cancer Institute

🇺🇸

Atlanta, Georgia, United States

Memorial Sloan Kettering Cancer Center

🇺🇸

New York, New York, United States

New York Proton Center

🇺🇸

New York, New York, United States

MD Anderson West Houston

🇺🇸

Houston, Texas, United States

MD Anderson in The Woodlands

🇺🇸

Conroe, Texas, United States

MD Anderson in Sugar Land

🇺🇸

Sugar Land, Texas, United States

MD Anderson League City

🇺🇸

League City, Texas, United States

Fred Hutch/University of Washington Cancer Consortium

🇺🇸

Seattle, Washington, United States

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