Hepatocellular CarcinomaPhase 2NCT02232633
A Study of BBI503 in Adult Patients With Advanced Hepatobiliary Cancer
A Phase 2 study testing BBI503 in people with Hepatocellular Carcinoma and Cholangiocarcinoma, run by Sumitomo Pharma America, Inc. at 3 sites in Canada.
Trial summary
This is an open label, multi-center, phase II study of BBI503 administered to adult patients with advanced hepatobiliary cancer who have exhausted all currently approved standard anti-cancer treatment options. BBI503 will be administered orally, daily, in continuous 28-day cycles at a dose of 300 mg once daily. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hepatocellular Carcinoma, Cholangiocarcinoma
- What is being tested?
- BBI503
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 45 participants
- Where
- 3 sitesCanada
- Study dates
- Feb 2015to 14 Dec 2017
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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
- Signed written informed consent must be obtained and documented according to International Conference on Harmonisation (ICH) - Good Clinical Practice (GCP), the local regulatory requirements, and permission to use private health information in accordance with the Health Insurance Portability and Accountability Act (HIPPA) prior to study-specific screening procedures
- Histologically or cytologically confirmed hepatocellular carcinoma or cholangiocarcinoma, that is metastatic, unresectable, or recurrent; and for which no currently approved, standard anti-cancer treatment option is available. Patients must have received standard of care treatment prior to enrollment.
- ≥ 18 years of age
- Measurable disease as defined by Response Evaluation Criteria in Solid Tumors (RECIST) 1.1
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1
- Male or female patients of child-producing potential must agree to use contraception or avoidance of pregnancy measures during the study and for 30 days after the last BBI503 dose
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- Females of childbearing potential must have a negative serum pregnancy test
- Aspartate transaminase (AST) and Alanine transaminase (ALT) ≤ 5.0x the upper limit of normal (ULN)
- Hemoglobin > 8.0 g/dL
- Total bilirubin ≤ 2.5 x ULN
- Creatinine ≤ 1.5 x ULN or creatinine clearance > 50 mL/min according to the Cockcroft-Gault estimation.
- Absolute neutrophil count ≥ 1.5 x 10^9/L
- Platelets ≥ 60 x 10^9/L
- Life expectancy ≥ 3 months
- A patient with hepatocellular carcinoma (HCC) which has arisen out of any medical context must also meet the following criteria:
- Must not be a candidate for potentially curative resection
- Must be Child-Pugh class A or B7 (i.e., in order to be eligible, the total Child-Pugh score for a patient must be ≤ 7)
- Must have received prior treatment with sorafenib; and have had either disease progression during treatment or have had documented intolerance to sorafenib such that further treatment with sorafenib is not possible.
- Patients with uncontrolled massive ascites or presence of hepatic encephalopathy within four (4) weeks of first dose are excluded
- A patient with confirmed cholangiocarcinoma of any type must also meet the following criteria:
- Must have disease which is not amenable to surgical, radiation, or combined modality therapy with curative intent
- Must have received prior treatment with gemcitabine, either alone or in combination with a platinum agent. Patients who are not eligible for gemcitabine must have received an alternate first-line systemic chemotherapy regimen
Who cannot join
- Anti-cancer chemotherapy, radiotherapy, immunotherapy, or investigational agents within 7 days of first dose of BBI503. Patients may begin BBI503 on a date determined by the investigator and medical monitor for the sponsor provided there is a minimum of 7 days since last receiving anti-cancer treatment, and that all prior treatment-related adverse events (AEs) have resolved or have been deemed irreversible.
- Major surgery within 4 weeks prior to first dose (requiring general anesthesia and/or inpatient hospitalization for recovery).
- Any known symptomatic or untreated brain metastases requiring increase of steroid dose within 2 weeks prior to starting on study. Patients with treated brain metastases must be stable for 4 weeks after completion of that treatment. Patients must have no clinical symptoms from brain metastases and must be either off steroids or on a stable dose of steroids for at least 2 weeks prior to protocol enrollment. Patients with known leptomeningeal metastases are excluded, even if treated.
- Pregnant or breastfeeding
- Significant gastrointestinal disorder(s), in the opinion of the treating investigator, (e.g., Crohn's disease, ulcerative colitis, extensive gastric and small intestine resection); such that absorption of oral medications may be impaired.
- Unable or unwilling to swallow BBI503 capsules daily
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- Uncontrolled intercurrent illness including, but not limited to ongoing or active infection, clinically significant non-healing or healing wounds, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, significant pulmonary disease (shortness of breath at rest or mild exertion), uncontrolled infection or psychiatric illness/social situations that would limit compliance with study requirements (e.g. no reliable transportation).
- Subjects with history of another primary cancer, with the exception of: a) curatively resected non-melanoma skin cancer; b) curatively treated cervical carcinoma in situ; or c) other primary solid tumor with no known active disease present in the opinion of the investigator will not affect patient outcome in the setting of current hepatobiliary malignancy.
- Abnormal ECGs which are clinically significant such as QT prolongation - QTc > 480 msec, clinically significant cardiac enlargement or hypertrophy, new bundle branch block, or signs of active ischemia. Patients with evidence of prior infarction who are New York Heart Association (NYHA) functional class II, III, or IV are excluded, as are patients with marked arrhythmia such as Wolff Parkinson White pattern or complete atrioventricular (AV) dissociation.
Trial locations and contacts
3 sites in 1 country.
Canada3 sites
University of Calgary
Calgary, Alberta, T2N 4N2
The Ottawa Hospital Cancer Centre
Ottawa, Ontario, K1H 8L6
Princess Margaret Hospital
Toronto, Ontario
About this study
Official title: A Phase II Clinical Study of BBI503 in Adult Patients With Advanced Hepatobiliary Cancer
This is an open label, multi-center, phase II study of BBI503 administered to adult patients with advanced hepatobiliary cancer who have exhausted all currently approved standard anti-cancer treatment options. BBI503 will be administered orally, daily, in continuous 28-day cycles at a dose of 300 mg once daily. Cycles will be repeated until patients are no longer clinically benefiting from therapy.
Safety, efficacy and tolerability of BBI503 will be assessed for the duration of study treatment.
- Conditions
- Hepatocellular Carcinoma, Cholangiocarcinoma
- Being tested
- BBI503
- Medical subject headings
- Carcinoma, Hepatocellular, Cholangiocarcinoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BBI503Drug
Also called Amcasertib, BBI-503, BB503
| Study group | Role | What they receive |
|---|---|---|
| BBI503 | Experimental | BBI503 will be administered orally, daily, in continuous 28-day cycles at a dose of 300 mg once daily Receives: Drug: BBI503 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Disease Control Rate (DCR)
Defined as the proportion of patients with a documented complete response, partial response, and stable disease (CR + PR + SD) based on RECIST 1.1.
Measured: 8 weeks
Secondary outcomes (5)
Objective response rate (ORR)
Defined as the proportion of patients with a documented complete response and partial response (CR + PR) based on RECIST 1.1.
Measured: 8 weeks
Progression free survival (PFS)
Defined as the time from enrollment to the first objective documentation of disease progression or death due to any cause.
Measured: 24 months
Overall survival (OS)
Defined as the time from enrollment to death due to any cause.
Measured: 24 months
Pharmacodynamics (biomarkers) of BBI503 when tumor biopsy is possible
Measured: baseline, 4 weeks
Number of Patients with Adverse Events
All patients who have received at least one dose of BBI503 will be included in the safety analysis. The incidence of adverse events will be summarized by type of adverse event and severity.
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Sep 2014 (estimated)
- Study startFeb 2015
- Primary completion14 Dec 2017
- Study completion14 Dec 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hepatocellular CarcinomaStudies 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
- 45 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sumitomo Pharma America, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02232633
- Sponsor's protocol ID
- BBI503-205b
- Other IDs
- BBI503-205HCC
- Status last verified
- Nov 2023
- First submitted
- 3 Sep 2014
- Last updated
- 14 Nov 2023
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Clinical Study of BBI503 in Adult Patients With Advanced Hepatobiliary Cancer
- ClinicalTrials.gov ID
- NCT02232633
- Acronym / sponsor ID
- BBI503-205b
- Conditions
- Hepatocellular Carcinoma, Cholangiocarcinoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 45 participants (actual)
- Lead sponsor
- Sumitomo Pharma America, Inc.Industry (pharma, biotech, devices)
- Study start
- Feb 2015
- Primary completion
- 14 Dec 2017 (actual)
- Study completion
- 14 Dec 2017 (actual)
- First posted
- 5 Sep 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an open label, multi-center, phase II study of BBI503 administered to adult patients with advanced hepatobiliary cancer who have exhausted all currently approved standard anti-cancer treatment options. BBI503 will be administered orally, daily, in continuous 28-day cycles at a dose of 300 mg once daily. Cycles will be repeated until patients are no longer clinically benefiting from therapy.
Safety, efficacy and tolerability of BBI503 will be assessed for the duration of study treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| BBI503 | Experimental | Drug: BBI503BBI503 will be administered orally, daily, in continuous 28-day cycles at a dose of 300 mg once daily |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (22 listed)
- Signed written informed consent must be obtained and documented according to International Conference on Harmonisation (ICH) - Good Clinical Practice (GCP), the local regulatory requirements, and permission to use private health information in accordance with the Health Insurance Portability and Accountability Act (HIPPA) prior to study-specific screening procedures
- Histologically or cytologically confirmed hepatocellular carcinoma or cholangiocarcinoma, that is metastatic, unresectable, or recurrent; and for which no currently approved, standard anti-cancer treatment option is available. Patients must have received standard of care treatment prior to enrollment.
- ≥ 18 years of age
- Measurable disease as defined by Response Evaluation Criteria in Solid Tumors (RECIST) 1.1
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1
Key exclusion criteria (9 listed)
- Anti-cancer chemotherapy, radiotherapy, immunotherapy, or investigational agents within 7 days of first dose of BBI503. Patients may begin BBI503 on a date determined by the investigator and medical monitor for the sponsor provided there is a minimum of 7 days since last receiving anti-cancer treatment, and that all prior treatment-related adverse events (AEs) have resolved or have been deemed irreversible.
- Major surgery within 4 weeks prior to first dose (requiring general anesthesia and/or inpatient hospitalization for recovery).
- Any known symptomatic or untreated brain metastases requiring increase of steroid dose within 2 weeks prior to starting on study. Patients with treated brain metastases must be stable for 4 weeks after completion of that treatment. Patients must have no clinical symptoms from brain metastases and must be either off steroids or on a stable dose of steroids for at least 2 weeks prior to protocol enrollment. Patients with known leptomeningeal metastases are excluded, even if treated.
- Pregnant or breastfeeding
- Significant gastrointestinal disorder(s), in the opinion of the treating investigator, (e.g., Crohn's disease, ulcerative colitis, extensive gastric and small intestine resection); such that absorption of oral medications may be impaired.
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 |
|---|---|---|
| BBI503Also: Amcasertib, BBI-503, BB503 | Drug | BBI503 |
Primary outcome
Disease Control Rate (DCR)
Time frame: 8 weeks
Defined as the proportion of patients with a documented complete response, partial response, and stable disease (CR + PR + SD) based on RECIST 1.1.
5 secondary outcomes
Objective response rate (ORR)
Time frame: 8 weeks
Progression free survival (PFS)
Time frame: 24 months
Overall survival (OS)
Time frame: 24 months
Pharmacodynamics (biomarkers) of BBI503 when tumor biopsy is possible
Time frame: baseline, 4 weeks
Number of Patients with Adverse Events
Time frame: 24 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Nov 2023.
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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.
The record lists no contact. Use the official record to reach the sponsor.
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 BBI503 in people with Hepatocellular Carcinoma and Cholangiocarcinoma, run by Sumitomo Pharma America, Inc. at 3 sites in Canada.
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 22 inclusion and 9 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 14 Nov 2023. 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 Canada. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Disease Control Rate (DCR) (time frame: 8 weeks). 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 14 Nov 2023.