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GastrinomaPhase 2NCT02259725

Regorafenib in Treating Patients With Advanced or Metastatic Neuroendocrine Tumors

A Phase 2 study testing regorafenib and laboratory biomarker analysis in people with Gastrinoma and Glucagonoma, run by University of Southern California at 5 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This phase II trial studies regorafenib in treating patients with neuroendocrine tumors that have spread from the primary site (place where it started) to other places in the body. Regorafenib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Gastrinoma, Glucagonoma, Insulinoma
What is being tested?
regorafenib and laboratory biomarker analysis
Could I receive a placebo?
No, everyone receives an active treatment
How many people
3 participants
Where
5 sitesUnited States
Study dates
16 Aug 2016to 20 Aug 2020
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

  • Advanced metastatic, progressing carcinoid or pancreatic islet cell cancers
  • No prior targeted treatment (tx) or anti-angiogenic therapy; patients may have received one line of prior therapy with octreotide, locoregional therapy; continuation of concurrent octreotide is allowed; patients will be maintained on octreotide (sandostatin) for the duration of their treatment
  • Life expectancy of at least 12 weeks (3 months)
  • Subjects must be able to understand and be willing to sign the written informed consent form (ICF); a signed ICF must be appropriately obtained prior to the conduct of any trial-specific procedure
  • All acute toxic effects of any prior treatment have resolved to National Cancer Institute-Common Terminology Criteria for Adverse Events (NCI-CTCAE) version (v)4.0 grade 1 or less at the time of signing the informed consent form (ICF); exceptions to this include alopecia
  • Total bilirubin =< 1.5 x the upper limits of normal (ULN)
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  • Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) =< 2.5 x ULN (=< 5 x ULN for subjects with liver involvement of their cancer)
  • Alkaline phosphastase limit =< 2.5 x ULN (=< 5 x ULN for subjects with liver involvement of their cancer)
  • Lipase =< 1.5 x the ULN
  • Amylase =< 1.5 x the ULN
  • Serum creatinine =< 1.5 x the ULN
  • International normalized ratio (INR)/ partial thromboplastin time (PTT) < 1.5 x ULN; (subjects who are treated with an agent such as warfarin or heparin will be allowed to participate provided that no prior evidence of underlying abnormality in coagulation parameters exists; close monitoring [day 5 of cycle 1 and day 1 of each cycle] is mandatory) will be performed until INR/PTT is stable based on a measurement that is pre-dose as defined by the local standard of care)
  • Platelet count >= 100,000 /mm^3
  • Hemoglobin (Hb) >= 9 g/dL
  • Absolute neutrophil count (ANC) >= 1,500/mm^3; blood transfusion to meet the inclusion criteria will not be allowed
  • Glomerular filtration rate (GFR) >= 30 ml/min/1.73 m^2 according to the Modified Diet in Renal Disease (MDRD) abbreviated formula
  • Women of childbearing potential must have a negative serum pregnancy test performed within 7 days prior to the start of study drug; post-menopausal women (defined as no menses for at least 1 year) and surgically sterilized women are not required to undergo a pregnancy test; the definition of adequate contraception will be based on the judgement of the investigator
  • Subjects (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; the definition of adequate contraception will be based on the judgment of the principal investigator or a designated associate
  • Subject must be able to swallow and retain oral medication
  • Southwest Oncology Group (SWOG) performance status 0-1
  • Patients must have measurable disease

Who cannot join

  • Previous assignment to treatment during this study; subjects permanently withdrawn from study participation will not be allowed to re-enter study
  • 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 including:
  • Congestive heart failure - New York Heart Association (NYHA) > class II
  • Active coronary artery disease
  • Cardiac arrhythmias requiring anti-arrhythmic therapy other than beta blockers or digoxin
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  • Unstable angina (anginal symptoms at rest), new-onset angina within 3 months before randomization, or myocardial infarction within 6 months before randomization
  • 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
  • Subjects 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 start of study treatment
  • Subjects with any previously untreated or concurrent cancer that is distinct in primary site or histology from carcinoid or pancreatic islet cancer except cervical cancer in-situ, treated basal cell carcinoma, or superficial bladder tumor; subjects surviving a cancer that was curatively treated and without evidence of disease for more than 3 years before randomization 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)
  • Patients with pheochromocytoma
  • 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
  • Presence of a non-healing wound, non-healing ulcer, or bone fracture
  • Renal failure requiring hemo-or peritoneal dialysis
  • Dehydration grade >= 1 NCI-CTCAE v4.0
  • Patients with seizure disorder requiring medication
  • Persistent proteinuria >= grade 3 NCI-CTCAE v4.0 (> 3.5 g/24 hrs, 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 subject unsuitable for trial participation
  • Substance abuse, medical, psychological or social conditions that may interfere with the subject's participation in the study or evaluation of the study results
  • Patients with known brain metastases should be excluded from this clinical trial
  • History of allergic reactions attributed to compounds of similar chemical or biologic composition to regorafenib or other agents used in study
  • Uncontrolled, intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
  • Excluded therapies and medications, previous and concomitant
  • Concurrent anti-cancer therapy (chemotherapy, radiation therapy, surgery, immunotherapy, biologic therapy, or tumor embolization) other than study treatment (regorafenib)
  • Prior use of regorafenib
  • Concurrent use of chemotherapy, radiotherapy or another investigational drug or device therapy (i.e., outside of study treatment) during, or within 4 weeks of trial entry (signing of the ICF)
  • Major surgical procedure, open biopsy, or significant traumatic injury within 28 days before start of study medication
  • Use of any herbal remedy (e.g. St. John's wort [Hypericum perforatum])

Trial locations and contacts

5 sites in 1 country.

5 sites in 1 country

United States5 sites

  • USC Norris Comprehensive Cancer Center

    Los Angeles, California, 90033

  • Hoag Memorial Hospital Presbyterian

    Newport Beach, California, 92663

  • USC Norris Oncology Hematology-Newport Beach

    Newport Beach, California, 92663

  • Mayo Clinic in Arizona

    Scottsdale, Arizona, 85259

  • Seattle Cancer Care Alliance

    Seattle, Washington, 98109

Other trials in United States

About this study

Official title: Phase II Study of Single Agent Regorafenib in Patients With Advanced/Metastatic Neuroendocrine Tumors

This phase II trial studies regorafenib in treating patients with neuroendocrine tumors that have spread from the primary site (place where it started) to other places in the body. Regorafenib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Read the detailed description

PRIMARY OBJECTIVES:

  1. To assess progression-free survival (PFS) in advanced/metastatic in patients with carcinoid or pancreatic islet cell tumors.

SECONDARY OBJECTIVES:

  1. To assess overall survival and response rate in advanced/metastatic poor prognosis in patients with carcinoid or pancreatic islet cell tumors.

II. To assess the toxicity of patients treated with regorafenib. III. To explore markers of angiogenesis as they relate to outcome in carcinoid and pancreatic islet cell tumors.

OUTLINE:

Patients receive regorafenib orally (PO) once daily (QD) on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up every 3 months.

Being tested
regorafenib, laboratory biomarker analysis
Medical subject headings
Gastrinoma, Glucagonoma, Insulinoma, Carcinoma, Islet Cell, Somatostatinoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • regorafenibDrug

    Also called BAY 73-4506, multikinase inhibitor BAY 73-4506, Stivarga

    Given PO

  • laboratory biomarker analysisOther

    Correlative studies

Study groups in NCT02259725
Study groupRoleWhat they receive
Treatment (regorafenib)ExperimentalPatients receive regorafenib PO QD on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Receives: Drug: regorafenib; Other: laboratory biomarker analysis

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. PFS

    Two parallel Simon's 2-stage phase II trials will be conducted to evaluate the efficacy of regorafenib in patients with advanced carcinoid (cohort A) or pancreatic islet cell tumors (cohort B). Will be summarized as a proportion of patients who are alive and progression-free among all patients in the primary data analysis set. The 95% confidence intervals (CIs) will be calculated using the Wilson method. Will be analyzed using Kaplan-Meier (KM) curves. The median PFS and 95% CIs will be calculated. The probability of 6-month PFS will be estimated from the KM curve too.

    Measured: Time from start of treatment to time of progression or death on study whichever comes first, assessed at 6 months

Secondary outcomes (3)

  1. Tumor Response Rate, Evaluated Using the New International Criteria Proposed by the Response Evaluation Criteria in Solid Tumors (RECIST) Committee

    Will be calculated as a proportion of patients who have either a complete or partial response among all patients in the primary data analysis set. The 95% CIs will be given.

    Measured: Up to 4 years

  2. Overall Survival

    The 95% CIs will be calculated using the Wilson method. Will be analyzed using KM curves.

    Measured: From start of treatment until death due to any cause, assessed up to 4 years

  3. Incidence of Adverse Events, Assessed According to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v4.0

    Toxicity profile will be summarized by attribution: regorafenib-related and all reported, course: course 1 and all courses, type, and grade: grade 1-2, 3-4, and 5.

    Measured: Up to 4 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted8 Oct 2014 (estimated)
  2. Study start16 Aug 2016
  3. Primary completion20 Aug 2020
  4. Study completion20 Aug 2020

Resources

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
3 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02259725
Sponsor's protocol ID
0S-14-3
Other IDs
NCI-2014-01996, HS-14-00583, 0S-14-3, P30CA014089
Status last verified
Nov 2021
First submitted
3 Oct 2014
Last updated
15 Dec 2021
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing regorafenib and laboratory biomarker analysis in people with Gastrinoma and Glucagonoma, run by University of Southern California at 5 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 21 inclusion and 36 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 15 Dec 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 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 15 Dec 2021). 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?

PFS (time frame: Time from start of treatment to time of progression or death on study whichever comes first, assessed at 6 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02259725.

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 15 Dec 2021.

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