A Randomized Phase II Study of Systemic Chemotherapy With or Without HAI FUDR/Dexamethasone in Patients With Unresectable Intrahepatic Cholangiocarcinoma

Plain English Summary

Gemcitabine and Oxaliplatin Chemotherapy With or Without a Floxuridine and Dexamethasone Pump in People With Cholangiocarcinoma That Cannot Be Removed With Surgery is a Phase 2 clinical trial sponsored by Memorial Sloan Kettering Cancer Center studying Intrahepatic Cholangiocarcinoma. This trial tests a new way to treat a specific type of liver cancer (cholangiocarcinoma) that cannot be surgically removed. It is for patients with this type of liver cancer who have not received prior chemotherapy for it. Participation involves receiving standard chemotherapy with or without a special pump that delivers chemotherapy directly to the liver. The main alternative is standard chemotherapy alone, as this study compares a new approach to the current standard of care. The trial aims to enroll 164 participants.

Official Summary

This study will compare the safety and effects of HAI floxuridine and dexamethasone combined with the standard chemotherapy drugs gemcitabine and oxaliplatin (GemOx) with those of GemOx alone in people with untreated cholangiocarcinoma that cannot be removed with surgery. The researchers want to find out whether the study treatment works better than the standard chemotherapy to delay progression of disease. For the study treatment to be considered better than the standard treatment, the study treatment should increase the time until progression of disease by an average of 3 months, compared with the usual approach.

Who Can Participate

Here is what you need to know about eligibility for this trial. Patients aged 18 or older with a confirmed diagnosis of intrahepatic cholangiocarcinoma. Patients must have cancer that has spread within the liver or to nearby lymph nodes but cannot be removed by surgery. Patients should have measurable disease and be healthy enough for surgery to place a chemotherapy pump. Individuals with certain liver conditions (like hepatitis or cirrhosis) may be eligible if their liver function is good. Patients with widespread cancer outside the liver or abdomen are not eligible. This trial is studying Intrahepatic Cholangiocarcinoma, so participants generally need a confirmed diagnosis. The trial is currently accepting new participants.

What They're Measuring

The primary outcome measures how long patients live without their cancer getting worse, with the goal of showing that the new treatment extends this time by at least 3 months compared to standard chem The specific primary outcome measures are: assess progression-free survival (PFS) (2 years). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.

About This Phase

This trial is in Phase 2, which tests whether the treatment actually works against the target condition. Phase 2 trials involve 100-300 patients and continue to monitor safety while evaluating effectiveness. This phase often tests different dosages to find the optimal amount. About 33% of Phase 2 drugs advance to Phase 3. If successful, the treatment will move to large-scale Phase 3 trials needed for FDA approval.

Why This Trial Matters

This trial addresses a critical need for better treatments for unresectable intrahepatic cholangiocarcinoma, aiming to improve outcomes where surgical options are limited. Phase 2 success would typically lead to larger Phase 3 trials needed for regulatory approval. This research targets Intrahepatic Cholangiocarcinoma, where improved treatment options are needed.

Investor Insight

This trial targets a rare but aggressive cancer, and a successful outcome could lead to a new standard of care, potentially impacting a significant market for advanced liver cancer therapies. Phase 2 trials have approximately a 15-20% chance of eventually gaining FDA approval.

Is This Trial Right for Me?

Ask your doctor if this trial is right for you, especially regarding the chemotherapy pump and potential side effects. Participation involves regular chemotherapy infusions and potentially a surgical procedure to implant a pump for direct liver chemotherapy delivery. You will need to attend regular appointments for treatment, monitoring, and follow-up scans. This trial is currently recruiting participants. The trial is being conducted at 11 sites. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.

AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.

Study Design

• Study Type: INTERVENTIONAL
• Allocation: RANDOMIZED
• Model: CROSSOVER
• Masking: NONE
• Enrollment: 164 participants

Interventions

• DRUG: Gemcitabine — See arm for details.
• DRUG: Oxaliplatin — See arm for details.
• DRUG: Dexamethasone — See arm for details.
• DRUG: Floxuridine (FUDR) — See arm for details.
• DEVICE: Implanted Medical Device — Implanted hepatic arterial infusion pump by surgical oncology, to deliver HAI therapy

Primary Outcomes

• assess progression-free survival (PFS) (2 years)

Secondary Outcomes

• Overall survival (OS) (2 years)

Full Eligibility Criteria

Inclusion Criteria:

* Age ≥18 years
* ECOG 0-1
* Histologically confirmed intrahepatic cholangiocarcinoma (also variously reported as peripheral cholangiocarcinoma, cholangiolar carcinoma or cholangiocellular carcinoma) (IHC). Confirmation of the diagnosis at MSKCC or at the enrolling institution must be obtained prior to randomization.
* Clinical or radiographic evidence of metastatic disease confined to the liver. Note: presence of regional (porta hepatis) lymph node metastases will be allowed, provided they are amenable to resection. (Note: If peritoneal or other extrahepatic disease is found at time of pump placement, the pump will not be implanted. The patient will be removed from study, deemed nonevaluable and will not count toward the overall study accrual.)
* Radiographically measurable disease. Measurable disease is defined as disease that can be assessed with 2-dimensional measurements on a cross-sectional imaging. Minimum lesion size is 2 cm in greatest diameter as per RECIST criteria.
* Disease must be considered unresectable at the time of preoperative evaluation.\*
* Considered candidate for general anesthesia, abdominal exploration and hepatic artery pump placement.
* Patients with chronic hepatitis and/or cirrhosis are eligible, but must be Child-Pugh class A.
* WBC ≥ 2,000/mcL , ANC ≥ 1000/mcL
* Platelet count ≥ 75,000/mcL
* Creatinine ≤ 1.8 mg/dL
* Total bilirubin \< 1.5 mg/dL
* Hgb \> 7 g/dL The % involvement of the liver will be determined by radiologists after review of imaging

Exclusion Criteria:

* Presence of distant metastatic disease. Patients will undergo radiographic evaluation to exclude the possibility of distant metastatic disease. For patients who have undergone pre- or post-operative biopsies that definitively diagnose IHC, the diagnostic studies may be modified at the discretion of the MSKCC Principal Investigator. Clinical or radiographic evidence of metastatic disease to regional lymph nodes will be allowed, provided it is amenable to resection.
* Patients previously treated with systemic chemotherapy for IHC will be non-eligible.
* Prior treatment with FUDR.
* Prior external beam radiation therapy to the liver.
* Prior ablative therapy to the liver.
* Diagnosis of sclerosing cholangitis.
* Clinical evidence or portal hypertension (ascites, gastroesophageal varices, or portal vein thrombosis; surgically related ascites does not exclude the patient).
* Active infection within one week prior to HAI placement.
* Pregnant or lactating women.
* History of other malignancy within the past 3 years except with early stage/localized cancer that was surgically resected or radiation treatment that would yield the same result as surgery within the past 3 years.
* Life expectancy \<12 weeks.
* Inability to comply with study and/or follow-up procedures.
* History of peripheral neuropathy. There is no exclusion of patients based on sex, ethnicity or race. For these reasons, the study results are expected to be generalizable to the Medicare beneficiary population.

Trial Locations

• National Heart, Lung, and Blood Institute (NIH) (Data Collection Only), Bethesda, Maryland, United States
• Washington University (Data Collection Only), St Louis, Missouri, United States
• Memorial Sloan Kettering Basking Ridge - Limited Protocol Activities, Basking Ridge, New Jersey, United States
• Memorial Sloan Kettering Monmouth - Limited Protocol Activities, Middletown, New Jersey, United States
• Memorial Sloan Kettering Bergen - Limited Protocol Activities, Montvale, New Jersey, United States
• Memorial Sloan Kettering Commack - Limited Protocol Activities, Commack, New York, United States
• Memorial Sloan Kettering Westchester - Limited Protocol Activities, Harrison, New York, United States
• Memorial Sloan Kettering Cancer Center, New York, New York, United States
• Memorial Sloan Kettering Nassau - Limited Protocol Activities, Uniondale, New York, United States
• Duke University (Data Collection Only), Durham, North Carolina, United States
• ...and 1 more locations

Frequently Asked Questions

Related Conditions

• Cholangiocarcinoma
• Bile Duct Cancer
• Liver Cancer
• Hepatocellular Carcinoma
• Gastrointestinal Cancers
• Oncology
• Metastatic Cancer
• Unresectable Tumors
• Chemotherapy
• Targeted Therapy

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