AuthorisedPhase I and Phase II (Integrated)- First administration to humansInterventional

Phase 1/2 Study of BLU-451 in Advanced Cancers with EGFR exon20 Insertion Mutations

This clinical trial is looking at a new medicine called BLU-451 for people who have advanced cancers with a particular genetic change, known as EGFR exon20 insertion mutations. The study has two main parts. The first part (Phase 1) is about finding the safest dose of BLU-451 and understanding any side effects. We'll also check how the body handles the medicine. Once a safe dose is found, the second part (Phase 2) will focus on how effective BLU-451 is at treating the cancer, such as by shrinking tumours or stopping them from growing. We'll be closely monitoring patients for any changes in their cancer and overall health throughout the study.

At a glance

Status

Authorised

Phase

Phase I and Phase II (Integrated)- First administration to humans

Sponsor

Blueprint Medicines Corp.

Enrolment target

Start

10 Oct 2023

Advanced cancers with EGFR Exon20 insertions mutations

What is this study about?

This study is a clinical trial exploring a new potential treatment called BLU-451. It's designed for people living with advanced cancers where there's been a specific genetic change, described as an 'EGFR exon20 insertion mutation.' This mutation means the cancer cells have a particular fault that allows them to grow. By targeting this fault, BLU-451 aims to slow down or stop the cancer's progression. This type of research is crucial because it helps scientists discover new ways to fight cancer, especially for those cases where current treatments might not be as effective.

The study is split into two main stages, called phases. In the first phase (Phase 1), the main goal is to find the safest dose of BLU-451. Researchers start with a very low dose and gradually increase it, carefully watching for any side effects and how participants respond. This helps them understand what dose is most appropriate and doesn't cause too many problems. They'll also learn how the body processes the drug.

Once a suitable dose is identified, the study moves into the second phase (Phase 2). Here, more people will receive BLU-451 at the dose found in Phase 1. The main aim of this phase is to see how well the drug actually works against the cancer. This includes checking if tumours shrink, stop growing, or if the cancer goes away. Throughout both phases, safety is a top priority, and medical teams will regularly check participants' health, including vital signs and blood tests, to ensure they are as safe as possible.

Key takeaways

This study is testing a new drug for specific advanced cancers.
It targets a particular genetic change (EGFR exon20 insertion mutation).
The first stage focuses on finding the safest dose of the drug.
The second stage will check how effective the drug is against the cancer.
Participation involves regular medical check-ups and monitoring.

Who may be eligible?

To be considered for this study, you must be at least 18 years old. There's no upper age limit, meaning older adults can also take part.

This study is open to both men and women.

The most important criteria relate to your cancer. You must have an advanced cancer and your medical tests must show a specific genetic change in your cancer cells. This change is called an 'EGFR exon20 insertion mutation'. Your doctor will be able to tell you if your cancer has this specific mutation.

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.

Are you at least 18 years old?
Do you have an advanced cancer?
Has your doctor confirmed your cancer has an 'EGFR exon20 insertion mutation'?
Are you able to attend regular hospital appointments?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will receive the new investigational drug, BLU-451. You'll have regular hospital visits, where doctors and nurses will closely monitor your health. These visits will involve physical examinations, blood tests, heart checks (ECGs), and other safety tests to make sure you are tolerating the drug well. The frequency of visits might be higher at the beginning of the study and then become less frequent over time. The total duration of your participation will depend on how you respond to the treatment and the study's progress, but it could last for several months or longer. You will also have follow-up appointments after you stop taking the study drug to monitor your health.

Potential risks and benefits

While taking part in this study, you might experience some benefits, such as receiving a new treatment that could potentially help your cancer. However, it's important to remember that this is an experimental drug, and its benefits are not guaranteed. There's also a chance of side effects, as with any medication, which could range from mild to more serious. Your medical team will explain all known potential risks in detail. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

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

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Unverified
Spain

Common questions

What is advanced cancer?

Advanced cancer usually means the cancer has spread from where it started to other parts of the body, or it has come back despite previous treatments.

What is an EGFR exon20 insertion mutation?

This is a specific genetic change or 'mistake' found in some cancer cells. It’s like a faulty switch that tells the cancer to grow. This study drug is designed to target this specific fault.

What does 'Phase 1/2' mean?

This means the study combines two steps. Phase 1 is about finding the safest dose and understanding side effects. Phase 2 then looks at how well the drug works against the cancer at that safe dose.

Will I know if the drug is working?

Your medical team will regularly perform tests like scans to monitor your cancer. They will share the results with you and discuss how your cancer is responding to the treatment.

Is BLU-451 available outside of this study?

No, BLU-451 is an experimental drug. It is not currently approved for general use and is only available to patients participating in this clinical trial.