Single Agent Regorafenib in Refractory Advanced Biliary Cancers

NCT02115542 | Completed Phase 2 Results

• 1 other identifier: MCC-17651
• Study Type: interventional
• Target: 39
• Locations: 1 country
• Sites: 3

Brief Summary

The main purpose of this study is to see if regorafenib can help control or decrease cancer size in patients with cancer of the bile duct. Researchers also want to find out if regorafenib is safe and tolerable.

Conditions

Cancer of the Bile Duct

Keywords

biliary; liver; Refractory Advanced Biliary Cancers; gallbladder cancer; carcinoma; intra-hepatic biliary system; extra-hepatic biliary system; gall bladder; unresectable; locally advanced; metastatic disease; Bile Duct Diseases; Gallbladder Diseases; regorafenib; stivarga; Bay 73-4506; multikinase inhibitor

Outcome Measures

Primary Outcomes (1)

• Overall Survival (OS) at 6 Months

  OS will be defined as the time from starting on trial to date of death due to any cause. The final analysis will be conducted after the follow-time of the last patient exceeds 6 months.

  at 6 month follow-up

Secondary Outcomes (2)

• Disease Control Response (DCR)

  Post 6 months follow-up, up to 13 months from on treatment per participant

• Progression Free Survival (PFS)

  Post 6 months follow-up, up to 13 months from on treatment per participant

Study Arms (1)

Regorafenib Monotherapy

EXPERIMENTAL

Regorafenib is administered as monotherapy during the study. 160 mg once daily (QD) will be administered for 3 weeks on /1 week off. One cycle is 28 days.

Drug: Regorafenib

Interventions

Regorafenib DRUG

Four 40 mg regorafenib tables should be taken in the morning with approximately 8 fluid ounces (240 mL) of water after a low-fat (\<30% fat) breakfast.

Also known as: Stivarga, BAY73-4506

Regorafenib Monotherapy

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Histologically or cytologically documented carcinoma primary to the intra- or extra-hepatic biliary system or gall bladder with clinical and/or radiologic evidence of unresectable, locally advanced or metastatic disease. Patients with ampullary carcinoma are not eligible.
• Have failed no more than 2 prior lines of systemic chemotherapy for advanced biliary cancer. Patients who received adjuvant chemotherapy and had evidence of disease recurrence within 6 months of completion of the adjuvant treatment are also eligible. If patient received adjuvant treatment and had disease recurrence after 6 months, they will only be eligible after failing one line of systemic chemotherapy used to treat the disease recurrence.
• Eastern Cooperative Oncology Group (ECOG) Performance Status Assessment of 0 or 1
• Measurable and non-measurable disease will be allowed.
• Must not have been treated with any vascular endothelial growth factor (VEGF) inhibitors. Prior 5-Fluorouracil (5-FU) or capecitabine treatment is allowed only if given as a radiosensitizer concurrently with radiation therapy at least 12 weeks prior to registration or if given as part of any adjuvant therapy regimen \> 6 months prior to study enrollment.
• Life expectancy of at least 12 weeks (3 months)
• For patients who have received prior cryotherapy, radiofrequency ablation, therasphere, ethanol injection, transarterial chemoembolization (TACE) or photodynamic therapy, the following criteria must be met: 28 days have elapsed since that therapy (lesions that have not been treated with local therapy must be present and measureable.
• Able to understand and willing to sign the written informed consent form
• All acute toxic effects of any prior treatment have resolved to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) v4.0 Grade 1 or less at the time of signing the Informed Consent Form (ICF).
• Adequate bone marrow and liver function
• Participants can receive 5-FU or capecitabine.
• Women of childbearing potential must have a negative serum pregnancy test performed within 7 days prior to the start of study drug.
• Men and women of childbearing potential must agree to use adequate contraception beginning at the signing of the ICF until at least 3 months after the last dose of study drug.
• Able to swallow and retain oral medication

You may not qualify if:

• Previous assignment to treatment during this study. Participants permanently withdrawn from study participation will not be allowed to re-enter study.
• Other investigational treatment during or within 21 days before starting study treatment
• Child Pugh B and C
• Uncontrolled hypertension (systolic pressure \>140 mm Hg or diastolic pressure \> 90 mm Hg \[NCI-CTCAE v4.0\] on repeated measurement) despite optimal medical management
• Active or clinically significant cardiac disease
• Evidence or history of bleeding diathesis or coagulopathy
• Any hemorrhage or bleeding event ≥ NCI CTCAE Grade 3 within 4 weeks prior to start of study medication
• Participants with thrombotic, embolic, venous, or arterial events, such as cerebrovascular accident (including transient ischemic attacks) deep vein thrombosis or pulmonary embolism within 6 months of informed consent
• Active malignancy except for nonmelanoma skin cancer or in situ cervical cancer. Potential participants surviving a cancer that was curatively treated and without evidence of disease for more than 3 years before the trial are allowed. All cancer treatments must be completed at least 3 years prior to study entry (i.e., signature date of the informed consent form).
• Potential participants with phaeochromocytoma
• Potential participants with severe hepatic impairment (Child-Pugh Class C)
• Known history of human immunodeficiency virus (HIV) infection or current chronic or active hepatitis B or C infection requiring treatment with antiviral therapy.
• Ongoing infection \> Grade 2 NCI-CTCAE v4.0
• Symptomatic metastatic brain or meningeal tumors
• Presence of a non-healing wound, non-healing ulcer, or bone fracture

Sponsors & Collaborators

• H. Lee Moffitt Cancer Center and Research Institute: lead
• Bayer: collaborator

Study Sites (3)

H. Lee Moffitt Cancer Center and Research Institute

Tampa, Florida, 33612, United States

Location

UNC Lineberger Comprehensive Cancer Center

Chapel Hill, North Carolina, 27514, United States

Location

VCU Massey Cancer Center

Richmond, Virginia, 23298, United States

Location

MeSH Terms

Conditions

Bile Duct Neoplasms; Gallbladder Neoplasms; Carcinoma; Neoplasm Metastasis; Bile Duct Diseases; Gallbladder Diseases

Interventions

regorafenib

Condition Hierarchy (Ancestors)

Biliary Tract Neoplasms; Digestive System Neoplasms; Neoplasms by Site; Neoplasms; Biliary Tract Diseases; Digestive System Diseases; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplastic Processes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Richard Kim, MD
• Organization: Moffitt Cancer Center

Richard.Kim@moffitt.org

813-745-1432

Study Officials

• Richard Kim, M.D.

  H. Lee Moffitt Cancer Center and Research Institute

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 11, 2014
• First Posted: April 16, 2014
• Study Start: June 5, 2014
• Primary Completion: December 10, 2018
• Study Completion: September 20, 2021
• Last Updated: October 12, 2021
• Results First Posted: January 13, 2020

Record last verified: 2021-09

Locations

US (3)