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Study Comparing Pemigatinib and Gemcitabine Plus Cisplatin for First-Line Treatment in Patients with Unresectable or Metastatic Cholangiocarcinoma with FGFR2 Rearrangement

Verified siteRegistered drugNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a type of cancer called cholangiocarcinoma, which affects the bile ducts. The study is specifically looking at cases where there is a change in the cancer cells known as FGFR2 rearrangement. The purpose of the study is to compare the effectiveness and safety of a medication called pemigatinib with a combination of two chemotherapy drugs, gemcitabine and cisplatin, in treating this cancer.

Participants in the study will be randomly assigned to receive either pemigatinib or the combination of gemcitabine and cisplatin. Pemigatinib is taken as a tablet by mouth, while gemcitabine and cisplatin are given as infusions, which means they are administered directly into the bloodstream through a vein. The study will last for a period of up to 12 months, during which the participants will receive their assigned treatment and be monitored regularly by the study team.

The study aims to gather information on how well the treatments work in controlling the cancer and their safety profiles. Participants will undergo various assessments, including imaging tests like MRI or CT scans, to evaluate the response of the cancer to the treatment. The study will also collect data on the quality of life of the participants and any side effects they may experience during the treatment period.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, the participant is randomly assigned to one of two treatment groups. This process is called randomization and ensures that each participant has an equal chance of receiving either treatment.

  2. Step 2

    Treatment with pemigatinib

    Participants in the pemigatinib group will take Pemazyre tablets orally. The dosage is determined by the study team and may include 4.5 mg, 9 mg, or 13.5 mg tablets.

    The tablets are taken once daily, following the specific instructions provided by the study team.

  3. Step 3

    Treatment with gemcitabine and cisplatin

    Participants in the gemcitabine and cisplatin group will receive these medications through intravenous infusion.

    Gemcitabine is administered as a powder for solution, and Cisplatin is provided as a solution for infusion.

    The frequency and duration of these infusions are determined by the study protocol and will be explained by the study team.

  4. Step 4

    Monitoring and assessments

    Throughout the study, participants will undergo regular monitoring and assessments to evaluate the effectiveness and safety of the treatment.

    These assessments may include physical examinations, blood tests, imaging studies such as CT or MRI scans, and questionnaires about quality of life.

  5. Step 5

    End of treatment

    The treatment continues until the participant experiences disease progression, unacceptable side effects, or chooses to withdraw from the study.

    After the treatment ends, there will be a follow-up period to monitor the participant's health and any long-term effects of the treatment.

Who can join the trial?

8 criteria

  • Must be able to understand and willing to sign a written consent form for the study.
  • Must be at least 18 years old. If a participant is a minor in Japan, parental consent is required.
  • Must have a confirmed diagnosis of **cholangiocarcinoma** (a type of cancer) that has not been treated before and cannot be removed by surgery or has spread to other parts of the body.
  • Must have a disease that can be measured or evaluated by a CT or MRI scan.
  • Must have an **ECOG performance status** of 0 to 1, which means the participant is fully active or has some symptoms but can still do light work.
  • Must have a documented **FGFR2 rearrangement**, which is a specific change in the genes related to the cancer.
  • Participants from North America, Europe, or other regions must agree to avoid pregnancy or fathering children during the study and for a certain period after the study. This includes using effective birth control methods.
  • Japanese participants must agree to avoid pregnancy or fathering children during the study and for a certain period after the study. This includes using effective birth control methods and not donating sperm.

Who cannot join the trial?

5 criteria

  • Patients who do not have **cholangiocarcinoma**. This is a type of cancer that starts in the bile ducts.
  • Patients who do not have a specific change in their genes called **FGFR2 rearrangement**. This is a change in the genetic material that can affect how cells grow.
  • Patients who are not in the age range specified for the study. The study is looking for participants within certain age groups.
  • Patients who are not able to receive the treatments being tested, which are **pemigatinib** or a combination of **gemcitabine** and **cisplatin**. These are medications used to treat cancer.
  • Patients who are part of a vulnerable population that the study is not designed to include. This means people who might need special protection or care.
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Investigated drugs

  • Pemigatinib

    is a medication being studied for its effectiveness in treating cholangiocarcinoma, a type of cancer that affects the bile ducts. It works by targeting specific genetic changes in the cancer cells, known as FGFR2 rearrangements, which can help slow down or stop the growth of the cancer.

  • Gemcitabine

    is a chemotherapy drug used to treat various types of cancer, including cholangiocarcinoma. It works by interfering with the DNA of cancer cells, preventing them from growing and dividing.

  • Cisplatin

    is another chemotherapy medication that is often used in combination with other drugs like gemcitabine. It helps to kill cancer cells by damaging their DNA, which can lead to the death of these cells.

What is already known about the treatment

  • Pemigatinib

    Pemigatinib is administered orally and is currently being studied in clinical trials for its effectiveness in treating cholangiocarcinoma, a type of bile duct cancer. It is specifically being evaluated for patients with FGFR2 rearrangement, a genetic alteration found in some cancer cells. Pemigatinib works by inhibiting the activity of the FGFR2 protein, which is involved in cell growth and division, thereby slowing down or stopping the growth of cancer cells. It is classified as a targeted therapy, focusing on specific molecular targets associated with cancer.

  • Gemcitabine

    Gemcitabine is given through intravenous infusion and is a well-established chemotherapy drug used in the treatment of various cancers, including cholangiocarcinoma. It works by interfering with the DNA replication process in cancer cells, leading to cell death. Gemcitabine is classified as an antimetabolite, which means it mimics the building blocks of DNA, disrupting the cancer cell's ability to grow and divide.

  • Cisplatin

    Cisplatin is administered intravenously and is a widely used chemotherapy agent for treating different types of cancer, including cholangiocarcinoma. It functions by forming cross-links in DNA, which prevents cancer cells from dividing and leads to cell death. Cisplatin is classified as a platinum-based chemotherapy, known for its ability to damage the DNA of cancer cells, thereby inhibiting their growth.

Investigated diseases

Cholangiocarcinoma – Cholangiocarcinoma is a type of cancer that forms in the bile ducts, which are the tubes that carry bile from the liver to the gallbladder and small intestine. It is a rare disease that can occur in different parts of the bile duct system, either inside the liver (intrahepatic) or outside the liver (extrahepatic). The disease often progresses slowly, with symptoms appearing only in the later stages, which can include jaundice, abdominal pain, and weight loss. As the cancer grows, it can block the bile ducts, leading to a buildup of bile in the liver and causing liver damage. The progression of cholangiocarcinoma can vary, but it typically involves the spread of cancer cells to nearby tissues and organs.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-513513-12-00Protocol codeINCB 54828-302Estimated enrolment242 patientsSponsorIncyte Corp.

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