Resectable CholangiocarcinomaPhase 2NCT03579771
Gemcitabine, Cisplatin, and Nab-Paclitaxel Before Surgery in Patients With High-Risk Liver Bile Duct Cancer
A Phase 2 study testing Cisplatin, Gemcitabine and Nab-paclitaxel in people with Resectable Cholangiocarcinoma and Stage IB Intrahepatic Cholangiocarcinoma AJCC v8, run by Emory University at 7 sites in United States.
Trial summary
This phase II trial studies how well gemcitabine, cisplatin, and nab-paclitaxel work before surgery in treating participants with high-risk bile duct cancer in the liver (intrahepatic cholangiocarcinoma). Read more about this study
- Who is it for?
- Ages 19 years and older
- Conditions the study is for
- Resectable Cholangiocarcinoma, Stage IB Intrahepatic Cholangiocarcinoma AJCC v8, Stage II Intrahepatic Cholangiocarcinoma AJCC v8
- What is being tested?
- Cisplatin, Gemcitabine and Nab-paclitaxel
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 7 sitesUnited States
- Study dates
- 26 Sep 2018to 16 Sep 2023
- 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 19 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of intrahepatic cholangiocarcinoma.
- High-quality cross-sectional imaging by computerized tomography (CT) or magnetic resonant imaging (MRI) performed within 6 weeks prior to enrollment and showed a resectable, but high-risk, intrahepatic cholangiocarcinoma (IHCCA) confined to the liver, bile duct, and/or regional lymph nodes. Tumors will be considered high-risk if the high-quality, contrast-enhanced CT and/or MRI +/- positron emission tomography (PET) scan showed: (must meet at least one of the criteria below)
- T-stage ≥ Ib (Ib-IV)
- Solitary lesion > 5 cm
- Multifocal tumors or satellite lesions present confined to the same lobe of the liver as the dominant lesion but still technically resectable
- Presence of major vascular invasion but still technically resectable
Show all 21Show fewer
- Suspicious or involved regional lymph nodes (N1)
- No distant extrahepatic disease (M0)
- Able to give informed consent.
- Able to adhere to study visit schedule and other protocol requirements.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0-1.
- Absolute neutrophil count (ANC) ≥ 1,500 cells/μL
- Platelet count ≥ 100,000 cells/μL
- Hemoglobin ≥ 9 g/dL
- Serum total bilirubin ≤ 1.5 x upper limit of normal (ULN)
- Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5 x ULN
- Albumin ≥ 3 g/dL
- Creatinine ≤ 1.5 x ULN
- Non-pregnant and non-lactating.
- Women of child-bearing potential (defined as a sexually mature woman who [1] has not undergone hysterectomy [the surgical removal of the uterus] or bilateral oophorectomy [the surgical removal of both ovaries] or (2) has not been naturally postmenopausal for at least 24 consecutive months [i.e., has had menses at any time during the preceding 24 consecutive months]) must commit to true abstinence from heterosexual contact or agree to use, and be able to comply with, effective contraception without interruption for 28 days prior to starting gemcitabine/cisplatin/nab-paclitaxel (including dose interruptions) until treatment with gemcitabine/cisplatin/nab-paclitaxel is complete.
- Male subjects must practice true abstinence or agree to use a condom during sexual contact with a female of childbearing potential or a pregnant female while on treatment (including during dose interruptions) with gemcitabine/cisplatin/nab-paclitaxel and for 6 months following gemcitabine/cisplatin/nab-paclitaxel discontinuation, even if he has undergone a successful vasectomy.
Who cannot join
- Peripheral neuropathy of grade 2 or greater by Common Terminology Criteria for Adverse Events (CTCAE) 4.0. In CTCAE version 4.0 grade 2 sensory neuropathy is defined as "moderate symptoms; limiting instrumental activities of daily living (ADLs)".
- Concurrent severe and/or uncontrolled medical conditions which could compromise participation in the study such as unstable angina, myocardial infarction within 6 months, unstable symptomatic arrhythmia, symptomatic congestive heart failure, uncontrolled diabetes, serious active, uncontrolled infection after inadequate biliary drainage if tumor obstructing bile duct, or psychiatric illness/social situations.
- Pregnancy (positive pregnancy test) or lactation.
- Known central nervous system (CNS) disease, except for treated brain metastasis. Treated brain metastases are defined as having no evidence of progression or hemorrhage after treatment and no ongoing requirement for dexamethasone, as ascertained by clinical examination and brain imaging (MRI or CT) during the screening period. Anticonvulsants (stable dose) are allowed. Treatment for brain metastases may include whole brain radiotherapy (WBRT), radiosurgery (RS; Gamma Knife, linear accelerator [LINAC], or equivalent) or a combination as deemed appropriate by the treating physician. Patients with CNS metastases treated by neurosurgical resection or brain biopsy performed within 3 months prior to day 1 will be excluded.
- Previous (within the past 5 years) or concurrent presence of other cancer, except non-melanoma skin cancer and in situ carcinomas.
- History of allergy or hypersensitivity to any of the study drugs.
- Current abuse of alcohol or illicit drugs.
- Inability or unwillingness to sign the informed consent form.
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
Emory University Hospital Midtown
Atlanta, Georgia, 30308
Emory University Hospital/Winship Cancer Institute
Atlanta, Georgia, 30322
Emory Saint Joseph's Hospital
Atlanta, Georgia, 30342
MD Anderson Cancer Center
Houston, Texas, 77030
Oregon Health and Science University
Portland, Oregon, 97239
Mayo Clinic
Rochester, Minnesota, 55905
Benaroya Research Institute at Virginia Mason
Seattle, Washington, 98101
About this study
Official title: A Single-Arm Feasibility Study of Gemcitabine, Cisplatin, and Nab-Paclitaxel as Neoadjuvant Therapy for Resectable Oncologically High-Risk Intrahepatic Cholangiocarcinoma
This phase II trial studies how well gemcitabine, cisplatin, and nab-paclitaxel work before surgery in treating participants with high-risk bile duct cancer in the liver (intrahepatic cholangiocarcinoma). Drugs used in chemotherapy, such as nab-paclitaxel, cisplatin, and gemcitabine, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving combination chemotherapy before surgery may make the tumor smaller and reduce the amount of normal tissue that needs to be removed.
Read the detailed description
PRIMARY OBJECTIVE:
To assess the feasibility of therapeutic approach that includes neoadjuvant chemotherapy including gemcitabine hydrochloride (gemcitabine), cisplatin, and nab-paclitaxel for high-risk but technically resectable intrahepatic cholangiocarcinoma and is completed with surgical resection.
SECONDARY OBJECTIVES:
- To assess the radiological response rate to neoadjuvant systemic chemotherapy according to the Response Evaluation Criteria in Solid Tumors (RECIST).
II. To determine the R0 resection rate.
III. To determine patient recurrence-free survival (RFS).
IV. To identify patient overall survival (OS) rate.
OUTLINE:
Participants receive nab-paclitaxel intravenously (IV) over 30 minutes, cisplatin IV over 60 minutes, and gemcitabine IV over 30 minutes on days 1 and 8. Treatment repeats every 21 days for up to 4 courses in the absence of disease progression or unacceptable toxicity. Participants with stable disease (SD), partial response (PR), or complete response (CR) then undergo standard of care hepatectomy with portal lymphadenectomy.
After completion of study treatment, participants are followed up every 4 months for 3 years.
- Conditions
- Resectable Cholangiocarcinoma, Stage IB Intrahepatic Cholangiocarcinoma AJCC v8, Stage II Intrahepatic Cholangiocarcinoma AJCC v8, Stage III Intrahepatic Cholangiocarcinoma AJCC v8, Stage IIIA Intrahepatic Cholangiocarcinoma AJCC v8, Stage IIIB Intrahepatic Cholangiocarcinoma AJCC v8
- Being tested
- Cisplatin, Gemcitabine, Nab-paclitaxel
- Medical subject headings
- Cholangiocarcinoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CisplatinDrug
Also called CDDP, Cis-diamminedichloridoplatinum, Cismaplat, Cisplatinum
Given IV
GemcitabineDrug
Also called dFdCyd, Difluorodeoxycytidine hydrochloride, Gemcitabine hydrochloride, Gemzar
Given IV
Nab-paclitaxelDrug
Also called ABI-007, Abraxane, Nanoparticle albumin-bound paclitaxel
Given IV
| Study group | Role | What they receive |
|---|---|---|
| Gemcitabine, cisplatin, nab-paclitaxel | Experimental | Participants receive nab-paclitaxel IV over 30 minutes, cisplatin IV over 60 minutes, and gemcitabine IV over 30 minutes on days 1 and 8. Treatment repeats every 21 days for up to 4 courses in the absence of disease progression or unacceptable toxicity. Participants with stable disease (SD), partial response (PR), or complete response (CR) then undergo standard of care hepatectomy with portal lymphadenectomy. Receives: Drug: Cisplatin; Drug: Gemcitabine; Drug: Nab-paclitaxel |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Who Completed All Preoperative and Operative Therapy
Completion of all therapy rate will be recorded.
Measured: Up to 12 weeks after study start
Number of Participants With Adverse Events
Will be monitored using method of Thall, Simon and Estey, and will be tabulated by the maximum reported Common Terminology Criteria for Adverse Events (CTCAE) grade.
Measured: Up to 3 years after study start
Secondary outcomes (3)
Radiological Response Rate Defined as the Percentage of Patients Who Will Have Complete Response (CR), Partial Response (PR) or Stable Disease (SD) After the Neoadjuvant Therapy
Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), >=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR.
Measured: Up to 12 weeks after study start
Recurrence-free Survival (RFS)
RFS is defined as the time between the date of surgery and the date of disease recurrence or death, whichever occurred first. If a patient did not have an event (i.e. disease recurrence or death) by the time of final analysis, patient will be censored at the last disease evaluation time.
Measured: From the date of surgery up to 3 years
Number of Participants With Overall Survival
OS is defined as the time from date of neoadjuvant treatment start to the date of death from any cause or to the date of last follow-up if patients are alive. If a patient is alive by the time of final analysis, the patient will be censored at the last follow-up date.
Measured: From date of neoadjuvant treatment start up to 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jul 2018
- Study start26 Sep 2018
- Primary completion16 Sep 2023
- Study completion16 Sep 2023
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 Resectable CholangiocarcinomaStudies of the same condition, open and under way.
- More trials run by Emory UniversityThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Collaborators
- Celgene, National Institutes of Health (NIH), National Cancer Institute (NCI)
- Responsible party
- Principal investigator, Shishir Kumar Maithel, Emory University
- Investigators
- Shishir Maithel, MD, Principal investigator, Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03579771
- Sponsor's protocol ID
- IRB00104175
- Other IDs
- NCI-2018-00998, EU4339-18, P30CA138292
- Status last verified
- Jul 2025
- First submitted
- 26 Jun 2018
- Last updated
- 15 Aug 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-Arm Feasibility Study of Gemcitabine, Cisplatin, and Nab-Paclitaxel as Neoadjuvant Therapy for Resectable Oncologically High-Risk Intrahepatic Cholangiocarcinoma
- ClinicalTrials.gov ID
- NCT03579771
- Acronym / sponsor ID
- IRB00104175
- Conditions
- Resectable Cholangiocarcinoma, Stage IB Intrahepatic Cholangiocarcinoma AJCC v8, Stage II Intrahepatic Cholangiocarcinoma AJCC v8, Stage III Intrahepatic Cholangiocarcinoma AJCC v8, Stage IIIA Intrahepatic Cholangiocarcinoma AJCC v8, Stage IIIB Intrahepatic Cholangiocarcinoma AJCC v8
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Collaborators
- Celgene, National Institutes of Health (NIH), National Cancer Institute (NCI)
- Study start
- 26 Sep 2018 (actual)
- Primary completion
- 16 Sep 2023 (actual)
- Study completion
- 16 Sep 2023 (actual)
- First posted
- 9 Jul 2018 (actual)
- Results
- Posted 15 Aug 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This phase II trial studies how well gemcitabine, cisplatin, and nab-paclitaxel work before surgery in treating participants with high-risk bile duct cancer in the liver (intrahepatic cholangiocarcinoma). Drugs used in chemotherapy, such as nab-paclitaxel, cisplatin, and gemcitabine, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving combination chemotherapy before surgery may make the tumor smaller and reduce the amount of normal tissue that needs to be removed.
Detailed description
PRIMARY OBJECTIVE:
To assess the feasibility of therapeutic approach that includes neoadjuvant chemotherapy including gemcitabine hydrochloride (gemcitabine), cisplatin, and nab-paclitaxel for high-risk but technically resectable intrahepatic cholangiocarcinoma and is completed with surgical resection.
SECONDARY OBJECTIVES:
- To assess the radiological response rate to neoadjuvant systemic chemotherapy according to the Response Evaluation Criteria in Solid Tumors (RECIST).
II. To determine the R0 resection rate.
III. To determine patient recurrence-free survival (RFS).
IV. To identify patient overall survival (OS) rate.
OUTLINE:
Participants receive nab-paclitaxel intravenously (IV) over 30 minutes, cisplatin IV over 60 minutes, and gemcitabine IV over 30 minutes on days 1 and 8. Treatment repeats every 21 days for up to 4 courses in the absence of disease progression or unacceptable toxicity. Participants with stable disease (SD), partial response (PR), or complete response (CR) then undergo standard of care hepatectomy with portal lymphadenectomy.
After completion of study treatment, participants are followed up every 4 months for 3 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Gemcitabine, cisplatin, nab-paclitaxel | Experimental | Drug: Cisplatin, Drug: Gemcitabine, Drug: Nab-paclitaxelParticipants receive nab-paclitaxel IV over 30 minutes, cisplatin IV over 60 minutes, and gemcitabine IV over 30 minutes on days 1 and 8. Treatment repeats every 21 days for up to 4 courses in the absence of disease progression or unacceptable toxicity. Participants with stable disease (SD), partial response (PR), or complete response (CR) then undergo standard of care hepatectomy with portal lymphadenectomy. |
Who may qualify
- Ages
- 19 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (21 listed)
- Diagnosis of intrahepatic cholangiocarcinoma.
- High-quality cross-sectional imaging by computerized tomography (CT) or magnetic resonant imaging (MRI) performed within 6 weeks prior to enrollment and showed a resectable, but high-risk, intrahepatic cholangiocarcinoma (IHCCA) confined to the liver, bile duct, and/or regional lymph nodes. Tumors will be considered high-risk if the high-quality, contrast-enhanced CT and/or MRI +/- positron emission tomography (PET) scan showed: (must meet at least one of the criteria below)
- No distant extrahepatic disease (M0)
- Able to give informed consent.
- Able to adhere to study visit schedule and other protocol requirements.
Key exclusion criteria (8 listed)
- Peripheral neuropathy of grade 2 or greater by Common Terminology Criteria for Adverse Events (CTCAE) 4.0. In CTCAE version 4.0 grade 2 sensory neuropathy is defined as "moderate symptoms; limiting instrumental activities of daily living (ADLs)".
- Concurrent severe and/or uncontrolled medical conditions which could compromise participation in the study such as unstable angina, myocardial infarction within 6 months, unstable symptomatic arrhythmia, symptomatic congestive heart failure, uncontrolled diabetes, serious active, uncontrolled infection after inadequate biliary drainage if tumor obstructing bile duct, or psychiatric illness/social situations.
- Pregnancy (positive pregnancy test) or lactation.
- Known central nervous system (CNS) disease, except for treated brain metastasis. Treated brain metastases are defined as having no evidence of progression or hemorrhage after treatment and no ongoing requirement for dexamethasone, as ascertained by clinical examination and brain imaging (MRI or CT) during the screening period. Anticonvulsants (stable dose) are allowed. Treatment for brain metastases may include whole brain radiotherapy (WBRT), radiosurgery (RS; Gamma Knife, linear accelerator [LINAC], or equivalent) or a combination as deemed appropriate by the treating physician. Patients with CNS metastases treated by neurosurgical resection or brain biopsy performed within 3 months prior to day 1 will be excluded.
- Previous (within the past 5 years) or concurrent presence of other cancer, except non-melanoma skin cancer and in situ carcinomas.
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 |
|---|---|---|
| CisplatinAlso: CDDP, Cis-diamminedichloridoplatinum, Cismaplat, CisplatinumGiven IV | Drug | Gemcitabine, cisplatin, nab-paclitaxel |
| GemcitabineAlso: dFdCyd, Difluorodeoxycytidine hydrochloride, Gemcitabine hydrochloride, GemzarGiven IV | Drug | Gemcitabine, cisplatin, nab-paclitaxel |
| Nab-paclitaxelAlso: ABI-007, Abraxane, Nanoparticle albumin-bound paclitaxelGiven IV | Drug | Gemcitabine, cisplatin, nab-paclitaxel |
Primary outcome
Number of Participants Who Completed All Preoperative and Operative Therapy
Time frame: Up to 12 weeks after study start
Completion of all therapy rate will be recorded.
Number of Participants With Adverse Events
Time frame: Up to 3 years after study start
Will be monitored using method of Thall, Simon and Estey, and will be tabulated by the maximum reported Common Terminology Criteria for Adverse Events (CTCAE) grade.
3 secondary outcomes
Radiological Response Rate Defined as the Percentage of Patients Who Will Have Complete Response (CR), Partial Response (PR) or Stable Disease (SD) After the Neoadjuvant Therapy
Time frame: Up to 12 weeks after study start
Recurrence-free Survival (RFS)
Time frame: From the date of surgery up to 3 years
Number of Participants With Overall Survival
Time frame: From date of neoadjuvant treatment start up to 3 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Aug 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Shishir Maithel, MD principal investigator
Emory University
7 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 Cisplatin, Gemcitabine and Nab-paclitaxel in people with Resectable Cholangiocarcinoma and Stage IB Intrahepatic Cholangiocarcinoma AJCC v8, run by Emory University at 7 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 8 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 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 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 Aug 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?
Number of Participants Who Completed All Preoperative and Operative Therapy (time frame: Up to 12 weeks after study start). 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?
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 Aug 2025.