Active not recruitingPHASE1, PHASE2INTERVENTIONAL

A Study of Selpercatinib (LOXO-292) in Participants With Advanced Solid Tumors, RET Fusion-Positive Solid Tumors, and Medullary Thyroid Cancer (LIBRETTO-001)

This research study, called LIBRETTO-001, is testing a new medication called selpercatinib (also known as LOXO-292) for people with advanced solid cancers. These cancers include certain types of lung cancer, a specific thyroid cancer, and other tumours that have a genetic change called a RET alteration. The study has two main parts. The first part safely found the right dose of the drug. The second part, which is ongoing, is looking at how safe the drug is, how it moves through the body, and if it helps to stop the cancer from growing or makes tumours shrink. It's for people whose cancer hasn't responded to standard treatments or for whom other treatments aren't suitable.

At a glance

Status

Active not recruiting

Phase

PHASE1, PHASE2

Sponsor

Eli Lilly and Company

Enrolment target

857

Start

02 May 2017

Estimated completion

01 Feb 2027

Non-Small Cell Lung Cancer Medullary Thyroid Cancer Colon Cancer Any Solid Tumor

What is this study about?

This study is investigating a new drug called selpercatinib (you might also see it referred to as LOXO-292). The main aim is to understand more about this medication and if it can help people with advanced cancers. Advanced cancer means the cancer has grown or spread and can't be cured with standard treatments alone. The study is particularly interested in cancers that have a specific genetic change called a 'RET alteration'. This could mean a 'RET fusion' or a 'RET mutation'; think of it as a particular characteristic of the cancer cells that the new drug is designed to target.

The study has two main stages. The first part, called Phase 1, has already been completed. In this phase, researchers carefully tested different doses of selpercatinib to find a dose that was both safe and effective enough to continue testing. This careful process is standard for all new medicines. Now, the study is in Phase 2, where a larger group of patients will receive the selected dose. The focus here is to gather more information on how safe the drug is, how it's handled by the body, and, importantly, whether it shows anti-cancer effects, like shrinking tumours or stopping their growth.

Over 800 patients are expected to take part in this study. It's for people whose cancer hasn't responded to their usual treatments, who can't have those treatments, or who have decided against them. The goal is to see if selpercatinib can offer a new treatment option for these specific advanced cancers, giving hope to patients when other options are limited.

Key takeaways

This study tests a new drug, selpercatinib, for advanced cancers.
It targets specific genetic changes (RET alterations) in cancer cells.
It's for people whose cancer hasn't responded to other treatments or for whom other treatments aren't suitable.
The study aims to check the drug's safety and effectiveness.
Participation involves taking the drug twice daily and regular hospital visits for monitoring.
You can stop participating at any time.

Who may be eligible?

To be considered for this study, you generally need to have an advanced solid cancer that has either gotten worse on other treatments, or for which standard treatments aren't available, suitable, or you've chosen not to have them. Crucially, your cancer should have a specific genetic change called a RET alteration, which doctors can check for in your tumour tissue or blood.

You also need to be well enough to take part, meaning you should have a good general health score (doctors use a scale for this) and your body's systems, like your blood, liver, and kidneys, should be working adequately. You should also be expected to live for at least three more months. The study may accept people as young as 12 years old.

Some parts of the study are currently open for new patients, while others are closed. For instance, if you have a RET fusion-positive solid tumour (not lung or thyroid cancer) and have already had some treatment, or if you haven't had any treatment yet, you might be eligible for the open parts. There are also specific cohorts for rare RET-altered solid tumours. The medical team will be able to tell you if your individual circumstances match any of the currently open groups and if you fully meet all the other requirements.

Quick self-check

Do I have an advanced solid cancer?
Has my cancer gotten worse on previous treatments, or are standard treatments not an option?
Has my doctor confirmed that my cancer has a 'RET alteration'?
Am I generally well enough to take part, with good overall health and organ function?
Am I at least 12 years old?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to join this study, you will be taking a medication called LOXO-292 (selpercatinib) by mouth, twice a day. The dose has already been set at 160 milligrams (mg) twice daily, based on earlier research. You'd have regular visits to the clinic so the study team can monitor your health, check for any side effects, and see how the medication is affecting your cancer. These visits would involve physical exams, blood tests, and scans (like CT or MRI) to measure your tumour size. The study is designed to continue as long as the medication is helping you and you are not experiencing severe side effects. The total duration of your participation could vary greatly depending on your individual response. Throughout the study, you'll be closely supported by the research team.

Potential risks and benefits

Taking part in any clinical trial has potential benefits and risks. A potential benefit of this study is that selpercatinib might help shrink your tumour or slow its growth, especially if standard treatments haven't worked or aren't suitable for you. However, there's no guarantee it will work for everyone, as this is still a research drug. Potential risks include side effects from the medication. While the study has identified a safe dose, you might experience discomfort or other health issues. The research team will carefully monitor you for any side effects and manage them as best as possible. You have the right to withdraw from the study at any time, for any reason, without it affecting your future medical care.

Locations (85)

Mayo Clinic of Scottsdale
Scottsdale, United States
City of Hope National Medical Center
Duarte, United States
UCLA Medical Center
Los Angeles, United States
Hoag Memorial Hospital Presbyterian
Newport Beach, United States
Kaiser Permanente
Oakland, United States
Irvine Medical Center
Orange, United States
University of California - San Diego
San Diego, United States
UCSF Medical Center at Mission Bay
San Francisco, United States
Kaiser Permanente Medical Center
Walnut Creek, United States
Sarah Cannon Research Institute at HealthOne
Denver, United States
Yale Cancer Center
New Haven, United States
Mayo Clinic in Florida
Jacksonville, United States

+73 more sites — see the official record for the full list.

Common questions

What is a RET alteration?

It's a specific genetic change inside cancer cells, like a switch that's stuck 'on.' This change can make the cancer grow, and new drugs like selpercatinib are designed to target it.

What does 'advanced solid tumour' mean?

It means the cancer has grown or spread and can't be completely cured with standard treatments. The study is looking for ways to control this type of cancer.

Will I get a placebo (dummy drug) in this study?

No, this is an 'open-label' study, meaning everyone who takes part and receives the study drug will be given selpercatinib. There is no placebo group.

How long will I be on the study drug?

You'll continue taking the drug as long as it's helping you and you're not experiencing difficult side effects. The length of time varies for each person.

Who is funding this research?

The information provided doesn't explicitly state the funder, but clinical trials like this are usually sponsored by pharmaceutical companies or research institutions.