Active not recruitingPHASE3INTERVENTIONAL

Durvalumab or Placebo in Combination With Gemcitabine/Cisplatin in Patients With 1st Line Advanced Biliary Tract Cancer (TOPAZ-1)

This study, named TOPAZ-1, is actively investigating a new medication called Durvalumab for individuals diagnosed with advanced bile duct or gallbladder cancer. These cancers are collectively known as Biliary Tract Cancers. The study aims to determine if adding Durvalumab to standard chemotherapy (Gemcitabine and Cisplatin) can improve patient outcomes. This is a large, international study where some patients will receive Durvalumab plus chemotherapy, while others will receive a placebo (a dummy drug) plus chemotherapy. This setup helps researchers fairly compare the new treatment's effectiveness. It's designed for people who have advanced cancer that hasn't been treated before, or cancer that has returned more than six months after previous treatment. The goal is to find better ways to treat these cancers.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

AstraZeneca

Enrolment target

810

Start

16 Apr 2019

Estimated completion

16 May 2027

Biliary Tract Neoplasms

Results

Results from this study

Posted April 2023

Results have been published for this study.

Primary outcome

Overall Survival (OS)

Overall Survival (OS) was defined as the time from the date of randomization until death due to any cause. Any patient not known to have died at the time of analysis was censored based on the last recorded date on which the patient was known to be alive. Median OS was calculated using the Kaplan-Meier technique. The second interim analysis was pre-specified after approximately 397 OS events occurred in both arms (59% maturity).

Full results on the registry

What is this study about?

This study, called TOPAZ-1, is focused on finding better treatments for a type of cancer known as biliary tract cancer. This includes cancers of the bile ducts (cholangiocarcinoma) and the gallbladder. When these cancers are advanced, meaning they have spread or can't be removed with surgery, doctors are always looking for new and more effective ways to help patients.

The current standard treatment for these advanced cancers often involves chemotherapy, specifically a combination of two drugs called Gemcitabine and Cisplatin. This study is testing if adding a new drug, Durvalumab, to this standard chemotherapy can make the treatment even more effective. Durvalumab is a type of immunotherapy, which works by helping your own immune system fight the cancer.

By comparing Durvalumab plus chemotherapy to a placebo (a harmless dummy drug) plus chemotherapy, researchers can get a clear picture of whether Durvalumab truly adds benefit. This is a 'Phase 3' study, which means it's one of the final steps in testing a new treatment before it might become widely available. The information gathered from studies like TOPAZ-1 is crucial for improving care and outcomes for patients with these challenging cancers.

Key takeaways

This study is for advanced bile duct and gallbladder cancer.
It tests a new drug, Durvalumab, with standard chemotherapy.
Participants will be assigned to a treatment or placebo group.
Regular hospital visits and health monitoring are required.
You can stop participating at any time.
The study helps find better treatments for these cancers.

Who may be eligible?

This study is looking for adults aged 18 and over who have advanced bile duct or gallbladder cancer. Your cancer must have been confirmed by a biopsy (where a small piece of tissue is taken for examination).

You might be suitable if your cancer cannot be removed by surgery or has spread to other parts of your body, and it hasn't been treated before. You could also be eligible if your cancer has come back more than six months after you had surgery to remove it, or more than six months after you finished any previous chemotherapy or radiation treatment.

There are also some reasons you wouldn't be able to join. For example, if you've had another type of cancer in the past, or if the cancer has spread to your brain or spinal cord. You also need to be generally well enough to take part, as assessed by your doctor, and not have had any major surgery recently (within the last 28 days).

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Is my cancer bile duct or gallbladder cancer that is advanced or has spread?
Has my cancer not been treated before, or has it come back more than 6 months after my last treatment?
Am I generally well enough to take part in medical treatment?
Have I not had any major surgery in the last month?
Do I not have another type of cancer or cancer that has spread to my brain or spinal cord?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will be randomly assigned to one of two groups. One group will receive Durvalumab along with the standard chemotherapy (Gemcitabine and Cisplatin), and the other group will receive a placebo (which looks like Durvalumab but contains no active drug) along with the same standard chemotherapy. You, your family, and your study doctor will not know which group you are in.

You will receive your treatment, which involves infusions, on a regular schedule. This will mean regular visits to the clinic for your treatments, blood tests, and check-ups to monitor your health and how the treatment is working. These check-ups will continue for a period after treatment finishes. The total duration of your participation, including follow-up, will be explained in detail by the study team.

Potential risks and benefits

Taking part in a clinical trial offers potential benefits, such as accessing a new treatment that might be more effective than standard care, and contributing to medical knowledge that can help future patients. However, there are also potential risks, including side effects from the study drugs, which can vary from mild to severe, and the possibility that the new treatment may not work for you. You will be closely monitored by a medical team and have the right to withdraw from the study at any time, for any reason, without it affecting your future medical care.

Locations (126)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Los Angeles, United States
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Orange, United States
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Washington D.C., United States
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Fort Myers, United States
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St. Petersburg, United States
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Westwood, United States
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Louisville, United States
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Burlington, United States
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St Louis, United States
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Chapel Hill, United States
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Portland, United States
Research Site
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Philadelphia, United States

Common questions

What is Durvalumab?

Durvalumab is a type of immunotherapy designed to help your body's immune system fight cancer cells.

What is a 'placebo'?

A placebo is a substance that looks exactly like the study drug but contains no active medicine. It's used for comparison.

What kind of cancer is this study for?

This study is for advanced bile duct cancer (cholangiocarcinoma) and gallbladder cancer.

Will I know if I'm getting the new drug or the placebo?

No, neither you nor your doctor will know whether you are receiving Durvalumab or the placebo; this is called 'double-blind'.

Can I leave the study at any time?

Yes, you have the right to withdraw from the study at any point without it affecting your medical care.