An Observational Study to Learn More About Treatment With Regorafenib in People With Advanced Gastrointestinal Stromal Tumors in the United States

Real-World Regorafenib Use Among Advanced Gastrointestinal Stromal Tumors

This is an observational study in which data already collected from people with advanced gastrointestinal stromal tumors are studied.

In this observational study data are collected from participants who have private insurance or Medicare and who had started regorafenib treatment.

Gastrointestinal stromal tumor (GIST) is a type of cancer that most commonly originates from the stomach or small intestine. Advanced means that the cancer has spread to other parts of the body.

The study drug, regorafenib, is already approved for doctors to prescribe to people with GIST. Regorafenib works by blocking certain proteins that cause the growth of cancer cells. Regorafenib is recommended as the third choice of treatment for patients after imatinib and sunitinib have stopped working or have caused side effects that are too severe to continue the treatment. In addition, it is also the recommended first choice of treatment in people with GIST who had low levels of protein called succinate dehydrogenase (SDH) protein. This condition is called SDHdeficient GIST. However, doctors might sometimes give it in a different order. To better understand the treatment patterns with regorafenib for GIST, more knowledge is needed about its use in the real world.

The participants in this study had started treatment with regorafenib as part of their regular care from their doctors.

The main purpose of this study is to learn more about the use of regorafenib treatment among people with advanced GIST who have private insurance or Medicare in the United States. To do this, researchers will collect information on:

Duration of treatment with regorafenib (also known as duration of therapy)

The length of time it took for participants to switch to another GIST treatment after starting regorafenib (also known as time to next therapy)

The data will come from the participants' information stored in a database, called Merative MarketScan for people in the United States. Data collected will be from April 2002 to September 2023.

Researchers will track the data of people with GIST who started regorafenib and will follow them for at least 28 days.

In this study, only available data are collected. No visits or tests are required as part of this study.

Study Overview

• Status: Not yet recruiting
• Conditions: Advanced Gastrointestinal Stromal Tumor
• Intervention / Treatment: Drug: Regorafenib (Stivarga, BAY73-4506)

• Study Type: Observational
• Enrollment (Estimated): 136

Contacts and Locations

• Study Contact: Name: Bayer Clinical Trials Contact; Phone Number: (+)1-888-84 22937; Email: clinical-trials-contact@bayer.com

Study Locations

• United States
  • New Jersey
    • Whippany, New Jersey, United States, 07981
      • Bayer

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

The study population will consist of adult GIST patients with evidence of regorafenib treatment initiation among commercially insured or Medicare patients in the Merative MarketScan database.

Description

Inclusion Criteria:

• Patients will be included if they:

  1. Have evidence of ≥1 pharmacy claim for regorafenib during the identification period from 1 October 2015, through 30 November 2022 The first prescription claim date for regorafenib during the identification period will be designated as the index date
  2. Have evidence of ≥1 medical claim with GIST diagnosis codes defined by International Classification of Diseases, 10th Revision, Clinical Modification (ICD-10-CM) diagnosis codes any time during the baseline period (establish a GIST diagnosis prior to regorafenib initiation- primary analysis)
  3. Have evidence of ≥1 medical claim with GIST diagnosis codes defined by International Classification of Diseases, 10th Revision, Clinical Modification (ICD-10-CM) diagnosis codes any time during the patient identification period (capture all regorafenib patients with GIST diagnosis in the database- sensitivity analysis)
  4. Have ≥12 months of continuous health plan enrollment prior to the index date, not including the index date

  5. Have ≥28 days of continuous health plan enrollment after the index date, including the index date

     Exclusion Criteria:

• Patients will be excluded if they:

  1. Are aged <18 years as of the index date

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Advanced Gastro-intestinal soft-tissue tumors (GIST) patients; Adult GIST patients with evidence of regorafenib treatment initiation among commercially insured or Medicare patients in the Merative MarketScan database.	Drug: Regorafenib (Stivarga, BAY73-4506); Retrospective analysis.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Duration of Therapy (DOT) on regorafenib	Retrospective analysis from 1 April 2002 to 30 September 2023
Time to Next Therapy (TTNT) therapy after regorafenib	Retrospective analysis from 1 April 2002 to 30 September 2023

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of the guideline-listed medications other than regorafenib	Imatinib (Gleevec); Sunitinib (Sutent); Dasatinib (Sprycel); Avapritinib (Ayvakit); Larotrectinib (Vitrakvi); Entrectinib (Rozlytrek); Ripretinib (Qinlock); Dabrafenib (Tafinlar); Trametinib (Mekinist); Cabozantinib (Cabometyx); Nilotinib (Tasigna); Pazopanib (Votrient); Sorafenib (Nexavar); Everolimus in combination with tyrosine kinase inhibitors (Afinitor, Zortress); Ponatinib (Iclusig); Binimetinib (Mektovi)	Retrospective analysis from 1 April 2002 to 30 September 2023
Descriptive analysis of baseline demographic		Retrospective analysis from 1 April 2002 to 30 September 2023
Descriptive analysis of clinical characteristics		Retrospective analysis from 1 April 2002 to 30 September 2023
Number of patients with evidence of any metastasis on or after the index date during the follow-up period.		Retrospective analysis from 1 April 2002 to 30 September 2023
Number of patients with evidence of surgery on or after the index date during the follow-up period using Current Procedural Terminology (CPT) codes and International Classification of Disease 9/10 Procedure codes (ICD-PC).		Retrospective analysis from 1 April 2002 to 30 September 2023

Collaborators and Investigators

• Sponsor: Bayer

Publications and helpful links

Helpful Links

• Click here for access to information about Bayer's transparency standards and Bayer studies.

Study record dates

Study Major Dates

• Study Start (Estimated): March 20, 2024
• Primary Completion (Estimated): April 28, 2024
• Study Completion (Estimated): April 28, 2024

Study Registration Dates

• First Submitted: March 14, 2024
• First Submitted That Met QC Criteria: March 14, 2024
• First Posted (Actual): March 20, 2024

Study Record Updates

• Last Update Posted (Actual): March 20, 2024
• Last Update Submitted That Met QC Criteria: March 14, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Gastrointestinal Neoplasms; Digestive System Neoplasms; Gastrointestinal Diseases; Neoplasms, Connective Tissue; Gastrointestinal Stromal Tumors
• Other Study ID Numbers: 22533

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014. Interested researchers can use www.vivli.org to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the member section of the portal.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No