Prospective Comparisons of Clinical Trial and Real-world Outcomes in Nasopharyngeal Carcinoma

Brief Title

Prospective Comparisons of Clinical Trial and Real-world Outcomes in Nasopharyngeal Carcinoma

Official Title

Prospective Comparisons of Survival Outcomes, Safety Profile, and Probability of Returning to Society Between Three Randomized Controlled Trials and Real-world Evidence in Nasopharyngeal Carcinoma.

Brief Summary

      Study results from randomized controlled trials (RCTs) usually have been found not adequately
      inform practice. A RCT is optimized to determine efficacy, while real-world study is
      conducted in a routine care setting aimed to determine effectiveness. Thus, it is necessary
      to evaluate the pragmatism of clinical trials for a better understanding of the external
      generalizability. Nonetheless, comparative pragmatic features of RCTs and real-world studies
      still lack well elucidation. By capitalizing on a nasopharyngeal carcinoma (NPC)-specific
      big-data, real-world database and individual patient data extracted from three landmark RCTs,
      investigators conducted the direct comparison of NPC cohorts receiving same treatment
      strategy in clinical trial versus real-world settings, and examined the comparative pragmatic
      features and their influences on survival outcomes, safety profile, and the probability of
      returning to society.
    

Detailed Description

      Study results from randomized controlled trials (RCTs) usually have been found not adequately
      inform practice. A RCT is optimized to determine efficacy. Such trials were performed with
      relatively small samples at sites with experienced investigators and highly selected
      participants, they could be overestimating benefits and underestimating harm. Real-world
      study is conducted in a routine care setting aimed to determine effectiveness. Thus, it is
      necessary to evaluate the pragmatism of clinical trials for a better understanding of the
      external generalizability. Nonetheless, comparative pragmatic features of RCTs and real-world
      studies still lack well elucidation. Nasopharyngeal carcinoma (NPC) is a malignant head and
      neck cancer with the highest incidences in endemic regions such as Southern China, where over
      60,600 new cases were diagnosed in 2015 representing 40% of all cases worldwide. Studies
      conducted in China are critical in optimizing clinical decision-making of NPC. In the past
      two decades, the recommendation level of induction chemotherapy (IC) + concurrent
      chemoradiotherapy (CCRT) has been improved evidently from Category 3 to 2A, and CCRT alone
      has long been a stable (Category 2B) and classic treatment option of NPC and therefore
      becomes the most commonly used control group in comparative studies. By capitalizing on a
      NPC-specific big-data, real-world database via a cancer registry in Southern China and
      individual patient data extracted from the three landmark RCTs, investigators conducted the
      direct comparison on IC+CCRT cohort or CCRT cohort of clinical trial versus real-world
      settings, and examined the comparative pragmatic features and their influences on survival
      outcomes, safety profile, and the probability of returning to society, with the aim to
      provide new insight into the optimization of trial design and the translation of study
      evidences into tangible benefits.
    

Study Type

Observational [Patient Registry]

Primary Outcome

Overall survival

Secondary Outcome

 Incidence of radioactive brain injury

Condition

Nasopharyngeal Carcinoma

Intervention

Trial setting

Study Arms / Comparison Groups

 CCRT-randomized clinical trial

Description:  Trial patients receiving CCRT

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Status

Other

Estimated Enrollment

5448

Start Date

June 1, 2019

Completion Date

August 1, 2019

Primary Completion Date

July 1, 2019

Eligibility Criteria

        Inclusion Criteria:

          1. Patients treated in our center from April 2009 to December 2016;

          2. All patients were pathologically diagnosed, non-metastatic nasopharyngeal carcinoma;

          3. Only included cases staged as T3-4N1 & T1-4N2-3 according to the 6th or 7th edition
             American Joint Committee on Cancer / Union for International Cancer Control
             (AJCC/UICC) system;

          4. For trial patients, treatment strategies only limited to concurrent chemoradiotherapy,
             induction chemotherapy combined with concurrent chemoradiotherapy, or concurrent
             chemoradiotherapy combined with adjuvant chemotherapy;

          5. For patients from real-world database, patients are permitted to receive additional
             targeted therapy to standard chemoradiotherapy, as far as it met clinical needs and
             was approved by physicians.

          6. The patient's basic information, prognosis related data, and follow-up data are
             complete.

        Exclusion Criteria:

          1. The clinical stage of T3-4N0 was excluded;

          2. Cases from large real-world databases need to exclude patients who participate in
             clinical trials;
      

Gender

All

Ages

8 Years - 79 Years

Accepts Healthy Volunteers

No

Contacts

Jun Ma, Professor, , 

Location Countries

China

Location Countries

China

NCT ID

NCT04108338

Organization ID

2019-FXY-239

Responsible Party

Principal Investigator

Study Sponsor

Sun Yat-sen University

Study Sponsor

Jun Ma, Professor, Principal Investigator, Sun Yat-sen University

Verification Date

September 2019