Biliary Tract CancerPhase 1/2NCT04203160

Gemcitabine and Cisplatin With or Without CPI-613 as First Line Therapy for Patients With Advanced Unresectable Biliary Tract Cancer (BilT-04)

A Phase 1/2 study testing CPI 613, Gemcitabine and Cisplatin in people with Biliary Tract Cancer, run by University of Michigan Rogel Cancer Center at 10 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

The purpose of this research study is to determine the safety and efficacy of CPI-613 (devimistat) in the treatment of advanced biliary tract cancer when used in combination with standard of care chemotherapy (gemcitabine plus cisplatin) compared to gemcitabine plus cisplatin alone. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Biliary Tract Cancer
What is being tested?: CPI 613, Gemcitabine and Cisplatin
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 75 participants
Where: 10 sitesUnited States
Study dates: 23 Jun 2020to 29 Apr 2024
Phase: Phase 1/2
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

Patients must have a pathologically or cytologically confirmed carcinoma (except neuroendocrine) of the biliary tract (intra-hepatic, extra-hepatic (hilar, distal) or gallbladder) that is not eligible for curative resection, transplantation, or ablative therapies. Tumors of mixed cholangiocarcinoma/hepatocellular carcinoma histology are excluded.
Patients may not have received prior systemic treatment (chemotherapy or targeted therapy) for advanced BTC. Prior peri-operative chemotherapy is permitted provided it was completed > 6 months from enrollment.
Patients may have received prior radiation, chemoembolization, radioembolization or other local ablative therapies or hepatic resection if completed ≥ 4 weeks prior to enrollment AND if patient has recovered to ≤ grade 1 toxicity. Extrahepatic palliative radiation is permitted if completed ≥ 2 weeks prior to enrollment AND if patient has recovered to ≤ grade 1 toxicity
Patients must have radiographically measurable disease (as per RECISTv1.1) in at least one site not previously treated with radiation or liver directed therapy (including bland, chemo- or radio-embolization, or ablation) either within the liver or in a metastatic site.
Must be ≥ 18 years of age.
Must have an ECOG performance status of 0-1.

Show all 22Show fewer

Ability to understand and willingness to sign IRB-approved informed consent.
Willing to provide archived tissue, if available, from a previous diagnostic biopsy or surgery.
Must be able to tolerate CT and/or MRI with contrast.
Adequate organ function (per protocol) obtained ≤ 2 weeks prior to enrollment.
Women of child-bearing potential and men must agree to use 2 methods of adequate contraception (hormonal plus barrier or 2 barrier forms) OR abstinence prior to study entry, for the duration of study participation and for 6 months (for men and women) following completion of study therapy.
Exclusions:
Prior history of brain metastasis (unless previously treated, asymptomatic and stable for at least 3 months) or organ transplant.
Underwent a major surgical procedure < 4 weeks prior to enrollment.
Active second malignancy other than in situ cancer or localized prostate cancer (Gleason score <8). Patients with history of other malignancy are eligible provided primary treatment of that cancer was completed > 1 year prior to enrollment and the patient is free of clinical or radiologic evidence of recurrent or progressive malignancy.
Ongoing active, uncontrolled infection (must be afebrile for > 48 hours off antibiotics) .
Psychiatric illness, other significant medical illness, or social situation which, in the investigator's opinion, would limit compliance or ability to comply with study requirements.
Pregnancy or breastfeeding. (Women must not be pregnant or breastfeeding since study drugs may harm the fetus or child. All females of childbearing potential [not surgically sterilized and between menarche and 1 year post menopause] must have a negative screening pregnancy test.)
Active heart disease including symptomatic heart failure (NYHA class 3 or 4), unstable angina pectoris, uncontrolled cardiac arrhythmia or interstitial lung disease.
Prisoners or subjects who are involuntarily incarcerated, or compulsorily detained for treatment of either a psychiatric or physical (e.g. infectious disease) illness would be excluded.
Prolonged QTcF interval >480 msec.
Known hypersensitivity to cisplatin, gemcitabine or CPI-613, or its inactive components.

Who cannot join

None listed.

Trial locations and contacts

10 sites in 1 country.

City, postcode or hospital

10 sites in 1 country

United States10 sites

University of Michigan Rogel Cancer Center
Ann Arbor, Michigan, 48109
Northwestern University -- Lurie Comprehensive Cancer Center
Chicago, Illinois, 60611
University Hospitals - Seidman Cancer Center
Cleveland, Ohio, 44106
University of Texas Southwestern -- Simmons Comprehensive Cancer Center
Dallas, Texas, 75390
University of Wisconsin - Carbone Cancer Center
Madison, Wisconsin, 53705
Atlantic Health System
Morristown, New Jersey, 07960
Vanderbilt-Ingram Cancer Center
Nashville, Tennessee, 37232
Allegheny Health Network
Pittsburgh, Pennsylvania, 15224
Fred Hutch/University of Washington Cancer Consortium
Seattle, Washington, 98109
University of Arizona Cancer Center
Tucson, Arizona, 85724

Other trials in United States

About this study

Official title: A Multi-Center Randomized Phase IB/II Study of Gemcitabine and Cisplatin With or Without CPI-613 as First Line Therapy for Patients With Advanced Unresectable Biliary Tract Cancer (BilT-04)

This research study has two parts:

In the phase 1 portion of this study, patients will receive a combination of CPI-613 and standard of care chemotherapy. Dose levels of CPI-613 will be adjusted to find the best dose, which will be the recommended phase 2 dose level.

In the phase 2 portion of this study, patients will be randomized into two arms. Patients in Arm A will receive the combination of the recommended dose level of CPI-613 and standard of care chemotherapy. Patients in Arm B will receive standard of care chemotherapy.

At the end of the study, researchers will compare the health outcomes of the patients that received CPI-613 + standard care to the outcomes of patients that received only standard care.

Conditions: Biliary Tract Cancer
Being tested: CPI 613, Gemcitabine, Cisplatin
Keywords: Cholangiocarcinoma, Gallbladder cancer
Medical subject headings: Biliary Tract Neoplasms, Cholangiocarcinoma, Gallbladder Neoplasms

What participants receive

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

CPI 613Drug
Also called Devimistat®
Given intravenously
GemcitabineDrug
Also called Gemzar®
Given intravenously
CisplatinDrug
Also called CDDP, Platinol®, NSC-119875
Given intravenously

Study groups in NCT04203160
Study group	Role	What they receive
Phase 1 and Phase 2, Arm A (investigational)	Experimental	On Day 1 and Day 8 of each 3-week cycle, patients will receive CPI-613 + gemcitabine and cisplatin. Patients may continue gemcitabine, cisplatin and CPI-613 for up to 2 years in absence of disease progression or unacceptable toxicity. Receives: Drug: CPI 613; Drug: Gemcitabine; Drug: Cisplatin
Phase 2, Arm B (standard of care)	Active comparator	On Day 1 and Day 8 of each 3-week cycle, patients will receive gemcitabine and cisplatin. Patients may continue gemcitabine and cisplatin for up to 2 years in absence of disease progression or unacceptable toxicity. Receives: Drug: Gemcitabine; Drug: Cisplatin

What is being measured

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

Phase 1: Incidence of Dose-limiting Toxicity
Dose limiting toxicities will determine the maximum tolerated dose (MTD)/recommended Phase 2 dose (RP2D) of combination therapy with CPI-613 + gemcitabine and cisplatin. Assessed using the NCI CTCAE v5.0
Measured: Up to day 22
Phase 2: Overall Response Rate (ORR)
Objective response assessment will be determined by review of CT or MR scans of the chest, abdomen and pelvis. ORR (Partial Response + Complete Response) will be assessed per Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1, during active study treatment. All enrolled patients who receive at least 1 cycle of therapy and have their disease re-evaluated will be considered evaluable for response. (Note: Patients who exhibit objective disease progression prior to the end of cycle 1 will also be considered evaluable.)
Measured: Until last dose of study treatment (up to 2 years)

Secondary outcomes (3)

Median Progression Free Survival (PFS)
PFS will be determined from date of first study treatment until the date of radiological or clinical progression (leading to withdrawal from the study) or date of last disease evaluation (for patients without progression). It will be calculated using the product-limit method of Kaplan and Meier. All patients that receive at least one dose of study treatment will be considered evaluable.
Measured: Until last dose of study treatment (up to 2 years)
Median Overall Survival (OS)
From date of first study treatment until date of last disease evaluation or until death from any cause. Using the product-limit method of Kaplan and Meier.
Measured: Up to 3 years after enrollment
Incidence of Toxicities
To evaluate the safety of CPI-613 in combination with gemcitabine and cisplatin in this patient population, assessed using the Common Toxicity Criteria for Adverse Events (CTCAE) v5.0. All patients that receive at least one dose of study treatment will be considered evaluable. Number of patients experiencing the maximum graded toxicity (Grade 2 or higher).
Measured: Up to 100 days after last dose of study treatment

Timeline

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

23 Jun 202029 Apr 2024

First posted18 Dec 2019
Study start23 Jun 2020
Primary completion16 Apr 2024
Study completion29 Apr 2024

Why it stopped: Loss of funding

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 Biliary Tract CancerStudies of the same condition, open and under way.
More trials run by University of Michigan Rogel Cancer CenterThe sponsor's other registered studies.
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 1/2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Sequential
Model description: Phase II: 2:1 Randomization with Bayesian Design Control Arm
Masking: None (open label)
Enrolment: 75 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04203160
Sponsor's protocol ID: UMCC 2019.116
Other IDs: HUM00170490
Status last verified: Dec 2025
First submitted: 16 Dec 2019
Last updated: 31 Dec 2025
Results posted: Yes, on ClinicalTrials.gov
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Active, not recruitingPhase 2/3
A Study of CTX-009 in Combination With Paclitaxel in Adult Patients With Unresectable Advanced, Metastatic or Recurrent Biliary Tract Cancers (COMPANION-002)
Biliary Tract Cancer, Cholangiocarcinoma
RecruitingPhase 2
At-Home Cancer Directed Therapy Versus in Clinic for the Treatment of Patients With Advanced Cancer
Advanced Anal Carcinoma, Advanced Biliary Tract Carcinoma
RecruitingObservational study
Tissue Procurement and Natural History Study of Patients With Malignant Mesothelioma
Thymoma, Stomach Neoplasms
Active, not recruitingPhase 2
Durvalumab and Tremelimumab in Combination With Propranolol and Chemotherapy for Treatment of Advanced Hepatopancreabiliary Tumors (BLOCKED)
Pancreatic Cancer, Hepatocellular Cancer
Not yet recruitingPhase 3
Hepatic Arterial Infusion Chemotherapy Versus Capecitabine as Adjuvant Therapy for Resected Biliary Tract Cancer
Biliary Tract Cancer, Cholangiocarcinoma

All Biliary Tract Cancer trials

Study at a glance

From the registry record, last updated by the sponsor on 31 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Multi-Center Randomized Phase IB/II Study of Gemcitabine and Cisplatin With or Without CPI-613 as First Line Therapy for Patients With Advanced Unresectable Biliary Tract Cancer (BilT-04)
ClinicalTrials.gov ID: NCT04203160
Acronym / sponsor ID: UMCC 2019.116
Conditions: Biliary Tract Cancer
Keywords: Cholangiocarcinoma, Gallbladder cancer
Phase: Phase 1/2
Study type: Interventional
Recruitment status: TerminatedReason stopped: Loss of funding
Enrolment: 75 participants (actual)
Lead sponsor: University of Michigan Rogel Cancer CenterUniversity, hospital or other
Study start: 23 Jun 2020 (actual)
Primary completion: 16 Apr 2024 (actual)
Study completion: 29 Apr 2024 (actual)
First posted: 18 Dec 2019 (actual)
Results: Posted 31 Dec 2025

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

This research study has two parts:

At the end of the study, researchers will compare the health outcomes of the patients that received CPI-613 + standard care to the outcomes of patients that received only standard care.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Sequential
Masking: None (open label)
Number of arms: 2
Model description: Phase II: 2:1 Randomization with Bayesian Design Control Arm

Arm	Type	Receives
Phase 1 and Phase 2, Arm A (investigational)	Experimental	Drug: CPI 613, Drug: Gemcitabine, Drug: CisplatinOn Day 1 and Day 8 of each 3-week cycle, patients will receive CPI-613 + gemcitabine and cisplatin. Patients may continue gemcitabine, cisplatin and CPI-613 for up to 2 years in absence of disease progression or unacceptable toxicity.
Phase 2, Arm B (standard of care)	Active comparator	Drug: Gemcitabine, Drug: CisplatinOn Day 1 and Day 8 of each 3-week cycle, patients will receive gemcitabine and cisplatin. Patients may continue gemcitabine and cisplatin for up to 2 years in absence of disease progression or unacceptable toxicity.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (22 listed)

Patients must have a pathologically or cytologically confirmed carcinoma (except neuroendocrine) of the biliary tract (intra-hepatic, extra-hepatic (hilar, distal) or gallbladder) that is not eligible for curative resection, transplantation, or ablative therapies. Tumors of mixed cholangiocarcinoma/hepatocellular carcinoma histology are excluded.
Patients may not have received prior systemic treatment (chemotherapy or targeted therapy) for advanced BTC. Prior peri-operative chemotherapy is permitted provided it was completed > 6 months from enrollment.
Patients may have received prior radiation, chemoembolization, radioembolization or other local ablative therapies or hepatic resection if completed ≥ 4 weeks prior to enrollment AND if patient has recovered to ≤ grade 1 toxicity. Extrahepatic palliative radiation is permitted if completed ≥ 2 weeks prior to enrollment AND if patient has recovered to ≤ grade 1 toxicity
Patients must have radiographically measurable disease (as per RECISTv1.1) in at least one site not previously treated with radiation or liver directed therapy (including bland, chemo- or radio-embolization, or ablation) either within the liver or in a metastatic site.
Must be ≥ 18 years of age.

Key exclusion criteria (0 listed)

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
CPI 613Also: Devimistat®Given intravenously	Drug	Phase 1 and Phase 2, Arm A (investigational)
GemcitabineAlso: Gemzar®Given intravenously	Drug	Phase 1 and Phase 2, Arm A (investigational), Phase 2, Arm B (standard of care)
CisplatinAlso: CDDP, Platinol®, NSC-119875Given intravenously	Drug	Phase 1 and Phase 2, Arm A (investigational), Phase 2, Arm B (standard of care)

Primary outcome

Phase 1: Incidence of Dose-limiting Toxicity
Time frame: Up to day 22
Dose limiting toxicities will determine the maximum tolerated dose (MTD)/recommended Phase 2 dose (RP2D) of combination therapy with CPI-613 + gemcitabine and cisplatin. Assessed using the NCI CTCAE v5.0
Phase 2: Overall Response Rate (ORR)
Time frame: Until last dose of study treatment (up to 2 years)
Objective response assessment will be determined by review of CT or MR scans of the chest, abdomen and pelvis. ORR (Partial Response + Complete Response) will be assessed per Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1, during active study treatment. All enrolled patients who receive at least 1 cycle of therapy and have their disease re-evaluated will be considered evaluable for response. (Note: Patients who exhibit objective disease progression prior to the end of cycle 1 will also be considered evaluable.)

3 secondary outcomes

Median Progression Free Survival (PFS)
Time frame: Until last dose of study treatment (up to 2 years)
Median Overall Survival (OS)
Time frame: Up to 3 years after enrollment
Incidence of Toxicities
Time frame: Up to 100 days after last dose of study treatment

Results and safety

Results posted to the registry on 31 Dec 2025, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Phase 2, Arm A (Investigational)	37	19
Phase 2, Arm B (Standard of Care)	18	6
Phase 1, CPI-613 500 mg/m2	1	0
Phase 1, CPI-613 1000 mg/m2	1	1
Phase 1, CPI-613 1500 mg/m2	2	2
Phase 1, CPI-613 2000 mg/m2	16	10

Primary outcome: Phase 1: Incidence of Dose-limiting Toxicity

Time frame: Up to day 22. Count of participants. Unit: Participants

Measure	Phase 2, Arm A (Investigational) n = 0	Phase 2, Arm B (Standard of Care) n = 0	Phase 1, CPI-613 500 mg/m2 n = 1	Phase 1, CPI-613 1000 mg/m2 n = 1	Phase 1, CPI-613 1500 mg/m2 n = 2	Phase 1, CPI-613 2000 mg/m2 n = 16
Value	0	0	0	0	0	1

Population analysed: only applies to Phase 1 patients

Primary outcome: Phase 2: Overall Response Rate (ORR)

Time frame: Until last dose of study treatment (up to 2 years). Count of participants. Unit: Participants

Measure	Phase 2, Arm A (Investigational) n = 50	Phase 2, Arm B (Standard of Care) n = 13	Phase 1, CPI-613 500 mg/m2 n = 0	Phase 1, CPI-613 1000 mg/m2 n = 0	Phase 1, CPI-613 1500 mg/m2 n = 0	Phase 1, CPI-613 2000 mg/m2 n = 0
Value	15	6	0	0	0	0

Population analysed: Phase 2, Arm A- 37 patients were randomized to Arm A treatment, with 36 patients receiving treatment. Per protocol the 16 patients in the Phase 1 portion of the study were included, for a total of 52 patients in the investigational arm. All 52 subjects were considered for evaluability. Two subjects were found to be unevaluable. Phase 2, Arm B- 18 patients were randomized to Arm B treatment, with 16 patients receiving treatment.Three subjects were found to be unevaluable.

3 secondary outcomes are reported in the full results.

Adverse events

Time frame: All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. Up to 2 years. All-Cause Mortality monitored/assessed up to 3 years. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Phase 2, Arm A (Investigational)	13 of 37 (35.1%)	35 of 37 (94.6%)	25 of 37
Phase 2, Arm B (Standard of Care)	9 of 18 (50%)	15 of 18 (83.3%)	7 of 18
Phase 1, CPI-613 500 mg/m2	0 of 1 (0%)	1 of 1 (100%)	0 of 1
Phase 1, CPI-613 1000 mg/m2	1 of 1 (100%)	1 of 1 (100%)	1 of 1
Phase 1, CPI-613 1500 mg/m2	2 of 2 (100%)	2 of 2 (100%)	2 of 2
Phase 1, CPI-613 2000 mg/m2	11 of 16 (68.8%)	16 of 16 (100%)	12 of 16

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 31 Dec 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: No

Related trials for Biliary Tract Cancer

Active, not recruitingPhase 2/3
A Study of CTX-009 in Combination With Paclitaxel in Adult Patients With Unresectable Advanced, Metastatic or Recurrent Biliary Tract Cancers (COMPANION-002)
Biliary Tract Cancer, Cholangiocarcinoma
RecruitingPhase 2
At-Home Cancer Directed Therapy Versus in Clinic for the Treatment of Patients With Advanced Cancer
Advanced Anal Carcinoma, Advanced Biliary Tract Carcinoma
RecruitingObservational study
Tissue Procurement and Natural History Study of Patients With Malignant Mesothelioma
Thymoma, Stomach Neoplasms
Active, not recruitingPhase 2
Durvalumab and Tremelimumab in Combination With Propranolol and Chemotherapy for Treatment of Advanced Hepatopancreabiliary Tumors (BLOCKED)
Pancreatic Cancer, Hepatocellular Cancer
Not yet recruitingPhase 3
Hepatic Arterial Infusion Chemotherapy Versus Capecitabine as Adjuvant Therapy for Resected Biliary Tract Cancer
Biliary Tract Cancer, Cholangiocarcinoma

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.

Vaibhav Sahai, MBBS, MS principal investigator

University of Michigan Rogel Cancer Center

10 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing CPI 613, Gemcitabine and Cisplatin in people with Biliary Tract Cancer, run by University of Michigan Rogel Cancer Center at 10 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 22 inclusion and 0 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: stopped early, as of the sponsor's last update on 31 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 10 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 combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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 31 Dec 2025). 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?

Phase 1: Incidence of Dose-limiting Toxicity (time frame: Up to day 22). The study lists 2 primary outcomes in all. 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 NCT04203160.

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 31 Dec 2025.