Active not recruitingPHASE3INTERVENTIONAL

A Study of Selpercatinib (LY3527723) in Participants With RET-Mutant Medullary Thyroid Cancer

This research study is looking at a new medicine called selpercatinib for people who have medullary thyroid cancer (MTC). This type of cancer has a specific genetic change called a RET mutation, and it either can't be removed by surgery or has spread to other parts of the body. The main goal is to find out if selpercatinib is safer and more effective than standard treatments currently available. Some patients will receive selpercatinib, while others will get a standard treatment. If a patient on standard treatment gets worse, they might have the option to switch to selpercatinib. This study is in its late stages (Phase 3), meaning it's comparing the new drug to established treatments to see if it's a better option for patients.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Loxo Oncology, Inc.

Enrolment target

291

Start

11 Feb 2020

Estimated completion

01 Nov 2027

Medullary Thyroid Cancer

What is this study about?

This study is a clinical trial, which means it's a careful research study to test new medicines. The medicine being tested here is called selpercatinib. It's for people who have a specific type of cancer called medullary thyroid cancer, often shortened to MTC. This particular study is for people whose MTC has a genetic change called a RET mutation, and unfortunately, the cancer can't be completely removed by surgery or has spread to other parts of their body.

The main purpose of this study is to compare selpercatinib with other existing treatments for this type of cancer. The researchers want to see two important things: first, if selpercatinib is safe for people to take, and second, if it works better than the current standard treatments. This means they'll be looking at how well it shrinks or controls the cancer. Finding new, more effective treatments is incredibly important for improving the health and quality of life for people living with cancer.

Because this is a late-stage study (Phase 3), it's a big step towards potentially making selpercatinib available more widely if it proves to be safe and effective. Some participants will receive selpercatinib, while others will receive one of the standard treatments for MTC. This comparison helps researchers understand the benefits and risks of the new drug. Importantly, if a participant who is on a standard treatment finds that their cancer continues to get worse, they might have the chance to switch over and try selpercatinib.

Key takeaways

This study is testing a new drug (selpercatinib) for a specific type of advanced thyroid cancer.
It targets medullary thyroid cancer with a particular gene change (RET mutation).
The study compares selpercatinib to existing standard treatments.
Participants whose cancer gets worse on standard treatment may be able to switch to selpercatinib.
The aim is to find out if selpercatinib is safer and more effective.

Who may be eligible?

To join this study, you generally need to be at least 18 years old. However, in some places, teenagers as young as 12 might be able to take part if local rules allow it. You must have medullary thyroid cancer that has been confirmed by a doctor, and your cancer can't be removed by surgery or has spread to other parts of your body.

It's important that your cancer hasn't been treated with certain other cancer drugs (kinase inhibitors) before. Also, your scans must show that your cancer has grown in the last 14 months. A key requirement is that your cancer cells or blood must show a specific change in a gene called RET.

There are also some health checks you'll need to pass. For example, your liver, kidneys, and blood need to be working well. If you could have children, you'll need to agree to use effective birth control during and for a few months after the study. You also need to be able to swallow capsules. You wouldn't be able to join if you have certain other specific genetic changes in your cancer that could make selpercatinib not work, or if you have severe heart problems or brain or spinal cord cancer symptoms.

Quick self-check

Are you at least 18 years old (or 12-17 if permitted by local rules)?
Have you been diagnosed with medullary thyroid cancer that cannot be removed by surgery or has spread?
Does your cancer have a 'RET mutation'?
Have your scans shown that your cancer has grown in the last 14 months?
Have you NOT had certain other cancer drugs (kinase inhibitors) for your advanced cancer before?
Can you swallow capsules?

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

What does participation involve?

If you decide to take part in this study, you would be randomly assigned to either receive the new drug, selpercatinib, or one of the standard treatments (cabozantinib or vandetanib). This is like flipping a coin for fairness. You would take the study medication regularly, likely as capsules you swallow.

You would have regular visits to the clinic for medical check-ups, blood tests, and scans to monitor your health and how your cancer is responding. These assessments are important to see if the treatment is working and to keep an eye on any side effects. The study will continue as long as the treatment is helping you and you are not experiencing significant side effects. If your cancer gets worse while on a standard treatment, you might have the option to switch over to receive selpercatinib. The total length of your participation would depend on how your body responds to the treatment and your health.

Potential risks and benefits

Taking part in a clinical trial offers potential benefits, such as access to a new treatment before it's widely available, and careful monitoring of your health. While selpercatinib has shown promise, it's also important to be aware of potential risks. All medications can have side effects, and these will be carefully explained to you by the study team. Some of the known side effects from this type of drug can affect the liver, heart, and blood pressure, among others. There's no guarantee the treatment will work for everyone. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (143)

University of Alabama at Birmingham
Birmingham, United States
City of Hope National Medical Center
Duarte, United States
UCLA Hematology/Oncology - Westwood (Building 100)
Los Angeles, United States
University of California Davis (UC Davis) Comprehensive Cancer Center
Sacramento, United States
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Torrance, United States
Emory University
Atlanta, United States
University of Chicago Medical Center
Chicago, United States
Massachusetts General Hospital
Boston, United States
University of Michigan
Ann Arbor, United States
Memorial Sloan Kettering Cancer Center
New York, United States
University of Cincinnati Medical Center
Cincinnati, United States
The James Cancer Hospital and Solove Research Institute at The Ohio State University Comprehensive Cancer Center
Columbus, United States

+131 more sites — see the official record for the full list.

Common questions

What is medullary thyroid cancer (MTC)?

Medullary thyroid cancer is a less common type of thyroid cancer that starts in specific cells in the thyroid gland that make a hormone called calcitonin.

What does 'RET-mutant' mean?

It means your cancer cells have a specific change (mutation) in a gene called RET. This change can make the cancer grow, and drugs like selpercatinib are designed to target it.

What is the difference between selpercatinib, cabozantinib, and vandetanib?

Selpercatinib is the new medicine being tested. Cabozantinib and vandetanib are existing, approved medicines used to treat advanced medullary thyroid cancer. The study compares the new one to these established treatments.

Why is this study called 'Phase 3'?

Phase 3 means it's a late-stage study where the new drug is being compared to existing treatments in a larger group of people. This helps confirm if it's better than what's already available.

Can I choose which treatment I get?

No, treatments are assigned randomly, like drawing lots. This helps ensure the study results are fair and unbiased. However, if you're on a standard treatment and it stops working, you might be able to switch to selpercatinib.