Cancer of the Bile DuctPhase 2NCT02115542
Single Agent Regorafenib in Refractory Advanced Biliary Cancers
A Phase 2 study testing Regorafenib in people with Cancer of the Bile Duct, run by H. Lee Moffitt Cancer Center and Research Institute at 3 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cancer of the Bile Duct
- What is being tested?
- Regorafenib
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 39 participants
- Where
- 3 sitesUnited States
- Study dates
- 5 Jun 2014to 20 Sep 2021
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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)
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- 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
Who cannot join
- 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
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- 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
- Renal failure requiring hemo-or peritoneal dialysis
- Patients with seizure disorder requiring medication
- Persistent proteinuria >/= Grade 3 NCI-CTCAE v4.0 (> 3.5 g/24 hours, measured by urine protein:creatinine ratio on a random urine sample)
- Interstitial lung disease with ongoing signs and symptoms at the time of informed consent
- Pleural effusion or ascites that causes respiratory compromise (≥ NCI-CTCAE version 4.0 Grade 2 dyspnea)
- History of organ allograft (including corneal transplant)
- Known or suspected allergy or hypersensitivity to any of the study drugs, study drug classes, or excipients of the formulations given during the course of this trial
- Any malabsorption condition
- Women who are pregnant or breast-feeding
- Any condition which, in the investigator's opinion, makes the potential participant unsuitable for trial participation
- Substance abuse, medical, psychological or social conditions that may interfere with participation in the study or evaluation of the study results
Trial locations and contacts
3 sites in 1 country.
United States3 sites
UNC Lineberger Comprehensive Cancer Center
Chapel Hill, North Carolina, 27514
VCU Massey Cancer Center
Richmond, Virginia, 23298
H. Lee Moffitt Cancer Center and Research Institute
Tampa, Florida, 33612
About this study
Official title: Multi Institutional Phase II Trial of Single Agent Regorafenib in Refractory Advanced Biliary Cancers
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
- Being tested
- Regorafenib
- 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
- Medical subject headings
- Bile Duct Neoplasms, Gallbladder Neoplasms, Carcinoma, Neoplasm Metastasis, Bile Duct Diseases, Gallbladder Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RegorafenibDrug
Also called Stivarga, BAY73-4506
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Regorafenib |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: at 6 month follow-up
Secondary outcomes (2)
Disease Control Response (DCR)
DCR defined as Complete Response (CR) + Partial Response (PR)+ Stable Disease (SD). CR: Complete disappearance of all target and non-target lesions (with the exception of lymph nodes mentioned below); No new lesions. PR: Applies only to patients with at least one measurable lesion; Greater than or equal to 30% decrease under baseline of the sum of appropriate diameters of all target measurable lesions; No unequivocal progression of nonmeasurable disease; No new lesions. SD: Does not qualify for CR, PR, Progression or Symptomatic Deterioration.
Measured: Post 6 months follow-up, up to 13 months from on treatment per participant
Progression Free Survival (PFS)
PFS is defined as the duration of time from start of treatment to time of progression or death, whichever comes first.
Progression - One or more of the following must occur: 20% increase in the sum of appropriate diameters of target measurable lesions over smallest sum observed (over baseline if no decrease during therapy) using the same techniques as baseline, as well as an absolute increase of at least 0.5 cm. Unequivocal progression of non-measurable disease in the opinion of the treating physician (an explanation must be provided). Appearance of any new lesion/site.
Measured: Post 6 months follow-up, up to 13 months from on treatment per participant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Apr 2014 (estimated)
- Study start5 Jun 2014
- Primary completion10 Dec 2018
- Study completion20 Sep 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Cancer of the Bile DuctStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 39 participants (actual)
Sponsor and investigators
- Lead sponsor
- H. Lee Moffitt Cancer Center and Research Institute
- Sponsor type
- University, hospital or other
- Collaborators
- Bayer
- Responsible party
- Sponsor
- Investigators
- Richard Kim, M.D., Principal investigator, H. Lee Moffitt Cancer Center and Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02115542
- Sponsor's protocol ID
- MCC-17651
- Status last verified
- Sep 2021
- First submitted
- 11 Apr 2014
- Last updated
- 12 Oct 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Oct 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multi Institutional Phase II Trial of Single Agent Regorafenib in Refractory Advanced Biliary Cancers
- ClinicalTrials.gov ID
- NCT02115542
- Acronym / sponsor ID
- MCC-17651
- 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
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 39 participants (actual)
- Lead sponsor
- H. Lee Moffitt Cancer Center and Research InstituteUniversity, hospital or other
- Collaborators
- Bayer
- Study start
- 5 Jun 2014 (actual)
- Primary completion
- 10 Dec 2018 (actual)
- Study completion
- 20 Sep 2021 (actual)
- First posted
- 16 Apr 2014 (estimated)
- Results
- Posted 13 Jan 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Regorafenib Monotherapy | Experimental | Drug: RegorafenibRegorafenib 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- 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.
Key exclusion criteria (26 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RegorafenibAlso: Stivarga, BAY73-4506Four 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. | Drug | Regorafenib Monotherapy |
Primary outcome
Overall Survival (OS) at 6 Months
Time frame: at 6 month follow-up
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.
2 secondary outcomes
Disease Control Response (DCR)
Time frame: Post 6 months follow-up, up to 13 months from on treatment per participant
Progression Free Survival (PFS)
Time frame: Post 6 months follow-up, up to 13 months from on treatment per participant
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Oct 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Moffitt Cancer Center Clinical Trials websiteLink provided by the sponsor.
Related trials for Cancer of the Bile Duct
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Active, not recruitingPhase 2
Testing the Combination of Pevonedistat With Chemotherapy for Bile Duct Cancer of the Liver
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Richard Kim, M.D. principal investigator
H. Lee Moffitt Cancer Center and Research Institute
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Regorafenib in people with Cancer of the Bile Duct, run by H. Lee Moffitt Cancer Center and Research Institute at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 26 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 Oct 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 13 Jan 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Survival (OS) at 6 Months (time frame: at 6 month follow-up). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Oct 2021.