Clinical trial NCT05557656: A Real World Study of Regogfinib in HCC
Official title: A Real World Study of Regogfinib in the Treatment of Advanced Hepatocellular Carcinoma
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Hepatocellular Carcinoma
Keywords: Hepatocellular Carcinoma
Regorafenib
Lead sponsor: Zhongda Hospital
Sponsor class: OTHER
Collaborators: name: The Central Hospital of Lishui City
name: Anhui Provincial Hospital
name: Zhejiang University
name: Sun Yat-sen University
name: Wuxi People's Hospital
name: Jiangsu Cancer Institute & Hospital
name: ZhuHai Hospital
First posted: 2022-09-28
Enrollment: 800
Sex: ALL
Minimum age (years): 18
Maximum age (years): 90
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with immediate or advanced stage HCC who have been treated with regorafenib from January 2018 to December 2021 following only one prior systemic therapy

Brief summary: This is a non-interventional observational study with the primary objective of assessing the efficacy of regorafenib in patients with immediate or advanced stage HCC.

Detailed description: 1. Study Objectives Primary Objective: To assess the efficacy of regorafenib in patients with immediate or advanced stage HCC Secondary objective: To assess the safety of regorafenib in patients with immediate or advanced stage HCC
2. Study Design This is a multicenter, retrospective, real-world study that will collect data from patients treated with regorafenib at medical centers in China.
3. Data Collection 3.1 Screening Period

The study procedures to be performed at screening include:

1. . Determine eligible subjects according to the inclusion/exclusion criteria and document the results.
2. . Collect demographic characteristics (eg, age, sex, weight, BMI, and ECOG score);
3. . Collect past medical history;
4. . Collect primary tumor site;
5. . Collect information on prior treatments: all treatments received since diagnosis of advanced HCC, including each treatment regimen (including first-line therapy), start and end date, duration, best response;
6. . Collect information on liver function and ECOG score prior to the first dose of regorafenib;
7. . Collect results of laboratory tests performed prior to and after the first dose of regorafenib, including haematology, biochemistry and genetic testing (if any);
8. . Collect imaging data: the last imaging procedure performed prior to the first dose of regorafenib, such as CT, PET-CT, MRI, and whole-body bone scan (with at least 1 measurable lesion); and information related to metastases (such as metastases to lymph node, bone and/or lung).

3.2 Data Review Period of Regorafenib Treatment

The following information obtained during each cycle of regorafenib will be collected retrospectively:

1. . Liver function and ECOG score obtained prior to each dose (ie., the first day of each cycle) of regorafenib;
2. . Initial dose of regorafenib;
3. . Timing and method of dose adjustment, and adjusted doses;
4. . Final daily dose of regorafenib;
5. . Best response, time to best response, and number of treatment cycles;
6. . Imaging findings (including metastatic sites) related to response evaluation;
7. . Hematology laboratory tests (related to the underlying disease, tumor progression, and ADR);
8. . Any adverse reaction and corresponding CTCAE grade;
9. . Interrupted or discontinued treatment;
10. . The most recent laboratory tests (or alternative doctor's advice and tests) performed prior to the first day of each treatment cycle, including but not limit to hematology and biochemistry.

3.3 Progression and Survival Data Collection Period

The following information obtained during each cycle of regorafenib will be collected retrospectively:

1. . Time to progression
2. . Pattern of progression
3. . Subsequent treatment regimen, dose, number of cycles, efficacy;
4. . Survival status, time of death or last follow-up.

4. Study Drug Generic name: Regorafenib Tablets Trade name: Stivarga® English name: Regorafenib Tablets

Dosage strength: 40 mg tablet Dosage form: Tablets Description: Oval tablets with pale-pink film coat Label dose and mode of administration: Recommended dosage is 160 mg, once daily for the first 21 days of each 28-day cycle. A minimum dose of 80 mg/day and a maximum dose of 160 mg/day are recommended.

Eligibility criteria: Inclusion Criteria:

1. Patients with pathologically or clinically confirmed HCC;
2. Patients with Barcelona Clinic Liver Cancer (BCLC) stage B/C;
3. Patients previously treated with regorafenib for at least one 28-day cycle after only one prior systemic therapy.

Exclusion Criteria:

1. Patients with incomplete information that would impact the assessment of primary endpoint;
2. Patients with medical history of other malignant neoplasms.

Primary outcomes:
measure: Overall survival (OS)
timeFrame: Time from the start of regorafenib treatment to the date of death from any cause or last follow-up，whichever came first, assessed up to 60 months
Secondary outcomes:
measure: Progression-free survival (PFS)
timeFrame: Time from the start of regorafenib treatment to the date of the first documentation of disease progression or death from any cause，whichever came first, assessed up to 60 months.
measure: Time to progression (TTP)
timeFrame: Time from the start of regorafenib treatment to the date of the first documentation of disease progression，whichever came first, assessed up to 60 months
measure: Objective response rate (ORR)
timeFrame: Time from the start of regorafenib treatment to the date of the first documentation of objective response，assessed up to 60 months.
measure: Disease control rate (DCR)
timeFrame: Time from the start of regorafenib treatment to the date of the first documentation of CR or PR or stable，assessed up to 60 months.
measure: Adverse events (AEs)
timeFrame: Time from the start of regorafenib treatment to the date of death or last follow up，whichever came first,assessed up to 60 months.
Other outcomes:
Locations:
Study officials:
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