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CompletedNCT03579771Updated Aug 15, 2025Results posted

Gemcitabine, Cisplatin, and Nab-Paclitaxel Before Surgery in Patients With High-Risk Liver Bile Duct Cancer

A Phase 2 interventional study of Cisplatin and Gemcitabine in Resectable Cholangiocarcinoma, Stage IB Intrahepatic Cholangiocarcinoma AJCC v8 and Stage II Intrahepatic Cholangiocarcinoma AJCC v8, sponsored by Emory University. Completed at 7 sites in United States. Open to participants aged 19 Years and older. Per ClinicalTrials.gov, last updated 2025-08-15.

Sponsored by Emory University · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
30
Allocation
Not applicable
Ages
19 Years and older
Sex
All
01

Study 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). 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:

I. 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.

02

Conditions studied

  • 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

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03

In context

Cholangiocarcinoma

914 studies on the registry are indexed under Cholangiocarcinoma; 286 are open to participants now.

This study's enrollment of 30 is below the median of 50 across 687 interventional studies indexed under Cholangiocarcinoma.

Browse Cholangiocarcinoma studies →

Lead sponsor

Emory University is the lead sponsor of 1,386 studies on the registry; 236 are open to participants now.

Of its 229 completed or terminated interventional studies of FDA-regulated products, 174 (76%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
19 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • 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)

    1. T-stage ≥ Ib (Ib-IV)
    2. Solitary lesion > 5 cm
    3. Multifocal tumors or satellite lesions present confined to the same lobe of the liver as the dominant lesion but still technically resectable
    4. Presence of major vascular invasion but still technically resectable
    5. 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.

Exclusion criteria

Exclusion Criteria:

  • 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.
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
30 participants (actual)

Study arms

  • Experimental
    Gemcitabine, cisplatin, nab-paclitaxel

    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.

    Drug: Cisplatin · Drug: Gemcitabine · Drug: Nab-paclitaxel

Interventions

  • DrugCisplatin

    Given IV

    Also known as: CDDP, Cis-diamminedichloridoplatinum, Cismaplat, Cisplatinum, Neoplatin, Platamin, Platinol

  • DrugGemcitabine

    Given IV

    Also known as: dFdCyd, Difluorodeoxycytidine hydrochloride, Gemcitabine hydrochloride, Gemzar

  • DrugNab-paclitaxel

    Given IV

    Also known as: ABI-007, Abraxane, Nanoparticle albumin-bound paclitaxel

06

What researchers measure

Primary outcomes

  1. Number of Participants Who Completed All Preoperative and Operative Therapy

    Completion of all therapy rate will be recorded.

    Time frame: Up to 12 weeks after study start

  2. 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.

    Time frame: Up to 3 years after study start

Secondary outcomes

  1. 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.

    Time frame: Up to 12 weeks after study start

  2. 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.

    Time frame: From the date of surgery up to 3 years

  3. 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.

    Time frame: From date of neoadjuvant treatment start up to 3 years

07

Results

Posted Aug 15, 2025

Participant flow

Participant flow — Overall Study
MilestoneGemcitabine, Cisplatin, Nab-paclitaxel
Started30
Completed22
Not completed8

Outcome measures

PrimaryNumber of Participants Who Completed All Preoperative and Operative Therapy

Completion of all therapy rate will be recorded.

Time frame:
Up to 12 weeks after study start
Reported as:
Count of participants · Participants
Number of Participants Who Completed All Preoperative and Operative Therapy
ParticipantsGemcitabine, Cisplatin, Nab-paclitaxel
Number of Participants Who Completed All Preoperative and Operative Therapy22
PrimaryNumber 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.

Time frame:
Up to 3 years after study start
Reported as:
Number · participants
Number of Participants With Adverse Events
participantsGemcitabine, Cisplatin, Nab-paclitaxel
Any adverse event26
Blood and lymphatic system disorders13
Ear and labyrinth disorders2
Endocrine disorders1
Gastrointestinal disorders11
General disorders and administration site conditions11
Investigations15
Metabolism and nutrition disorders4
Nervous system disorders9
Renal and urinary disorders1
Respiratory, thoracic and mediastinal disorders4
Skin and subcutaneous tissue disorders13
Vascular disorders1
SecondaryRadiological 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.

Time frame:
Up to 12 weeks after study start
Reported as:
Count of participants · Participants
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
ParticipantsGemcitabine, Cisplatin, Nab-paclitaxel
PD3
PR7
SD20
SecondaryRecurrence-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.

Time frame:
From the date of surgery up to 3 years
Reported as:
Median · months
Recurrence-free Survival (RFS)
monthsGemcitabine, Cisplatin, Nab-paclitaxel
Recurrence-free Survival (RFS)23.7 (17.1 to 37.1)
SecondaryNumber 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.

Time frame:
From date of neoadjuvant treatment start up to 3 years
Reported as:
Count of participants · Participants
Number of Participants With Overall Survival
ParticipantsGemcitabine, Cisplatin, Nab-paclitaxel
Number of Participants With Overall Survival30

Adverse events

Collected over 3 years. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Gemcitabine, Cisplatin, Nab-paclitaxel0/30 (0%)14/30 (46.7%)30/30 (100%)
Most frequent serious events
Most frequent serious events
EventGemcitabine, Cisplatin, Nab-paclitaxel
Neutrophil count decreasedInvestigations5/30
DiarrheaGastrointestinal disorders2/30
AnemiaBlood and lymphatic system disorders1/30
Alanine aminotransferase increasedInvestigations1/30
White blood cell decreasedInvestigations1/30
Febrile neutropeniaBlood and lymphatic system disorders1/30
ColitisGastrointestinal disorders1/30
DehydrationGeneral disorders1/30
HypophosphatemiaInvestigations1/30
LeukocytosisBlood and lymphatic system disorders1/30
Most frequent other events
Showing 10 of 94
Most frequent other events
EventGemcitabine, Cisplatin, Nab-paclitaxel
FatigueGeneral disorders20/30
Abdominal painGastrointestinal disorders13/30
AnemiaBlood and lymphatic system disorders13/30
DiarrheaGastrointestinal disorders13/30
HyperglycemiaEndocrine disorders12/30
Neutrophil count decreasedBlood and lymphatic system disorders12/30
NauseaGastrointestinal disorders11/30
AlopeciaSkin and subcutaneous tissue disorders10/30
Alanine aminotransferase increasedInvestigations9/30
Alkaline phosphatase increasedInvestigations9/30

Baseline characteristics

Age, Continuous
Age, Continuous(years)Gemcitabine, Cisplatin, Nab-paclitaxel
Mean61.17 ± 9.57
Sex: Female, Male
Sex: Female, Male(Participants)Gemcitabine, Cisplatin, Nab-paclitaxel
Female12
Male18
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Gemcitabine, Cisplatin, Nab-paclitaxel
Hispanic or Latino3
Not Hispanic or Latino27
Unknown or Not Reported0
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Gemcitabine, Cisplatin, Nab-paclitaxel
American Indian or Alaska Native0
Asian3
Native Hawaiian or Other Pacific Islander0
Black or African American0
White20
More than one race0
Unknown or Not Reported7
Region of Enrollment
Region of Enrollment(Participants)Gemcitabine, Cisplatin, Nab-paclitaxel
Benaroya Research Institute at Virginia Mason3
Emory University11
MD Anderson12
Mayo Clinic-Rochester4
08

Study locations

7 sites
  • Emory University Hospital Midtown
    Atlanta, Georgia 30308, United States
  • Emory University Hospital/Winship Cancer Institute
    Atlanta, Georgia 30322, United States
  • Emory Saint Joseph's Hospital
    Atlanta, Georgia 30342, United States
  • Mayo Clinic
    Rochester, Minnesota 55905, United States
  • Oregon Health and Science University
    Portland, Oregon 97239, United States
  • MD Anderson Cancer Center
    Houston, Texas 77030, United States
  • Benaroya Research Institute at Virginia Mason
    Seattle, Washington 98101, United States
09

References and documents

Study documents

  • Protocol and statistical analysis plan · Jul 27, 2023
  • Informed consent form · May 19, 2021

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: No

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Aug 15, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT03579771
Lead sponsor
Emory University
Collaborators
Celgene, National Institutes of Health (NIH), National Cancer Institute (NCI)
Responsible party
Shishir Kumar Maithel (Principal Investigator, Emory University) — Principal investigator
First posted
Jul 9, 2018
Start date
Sep 26, 2018
Primary completion
Sep 16, 2023
Completion
Sep 16, 2023
Results posted
Aug 15, 2025
Last update
Aug 15, 2025

Study contacts

Shishir Maithel, MD
principal investigator · Emory University

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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