Active not recruitingPHASE3INTERVENTIONAL

Study of Efficacy and Safety of Dabrafenib in Combination With Trametinib in Previously Treated Patients With Metastatic, Radio-active Iodine Refractory BRAF V600E Mutation Positive Differentiated Thyroid Cancer

This study is for adults with a type of thyroid cancer called Differentiated Thyroid Cancer (DTC). Specifically, it's for those whose cancer has a particular gene change (BRAF V600E), has stopped responding to radioactive iodine treatment, and has grown despite previous treatments aimed at blood vessel growth (VEGFR-targeted therapies). Researchers want to see if a combination of two drugs, dabrafenib and trametinib, is safe and effective compared to a dummy pill (placebo). About 150 patients will take part worldwide. The aim is to find better ways to treat this specific type of advanced thyroid cancer.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Novartis Pharmaceuticals

Enrolment target

153

Start

15 Nov 2021

Estimated completion

17 May 2027

Differentiated Thyroid Cancer (DTC)

Results

Results from this study

Posted May 2026

Results have been published for this study.

Primary outcome

Progression Free Survival (PFS)

Progression Free Survival (PFS) was defined as the time from the date of randomization to the date of the first documented progression according to RECIST 1.1 based on Blinded Independent Review Committee (BIRC) assessment, or death due to any cause. The primary analysis was conducted after all patients had either completed a minimum of 16 weeks of treatment or discontinued earlier, and following the occurrence of approximately 95 PFS events (Data cut-off date for the primary analysis was 22 January 2025).

Full results on the registry

What is this study about?

This study is looking at a new treatment approach for a specific type of thyroid cancer called Differentiated Thyroid Cancer (DTC). This particular type of cancer is identified by a special gene change called BRAF V600E. Patients joining this study will have had previous treatments, specifically radioactive iodine, which is a common treatment for thyroid cancer, but their cancer has stopped responding to it. They will also have tried one or two other treatments that target how blood vessels grow in the tumour, but the cancer has continued to progress.

The main goal of the study is to test if a combination of two drugs, dabrafenib and trametinib, can help shrink or stop the growth of this advanced thyroid cancer. These drugs are designed to target the BRAF V600E gene change, which is thought to be driving the cancer's growth. We want to see how well these drugs work and if they are safe for patients.

About 150 people globally will take part in this study. Some patients will receive the active drugs, while others will receive a dummy pill (placebo). This helps researchers fairly compare the new treatment. If patients on the dummy pill find their cancer is still growing, they might then be offered the active drug combination. This study is important because it could lead to new and more effective treatments for people living with this challenging type of advanced thyroid cancer.

Key takeaways

This study is for advanced Differentiated Thyroid Cancer (DTC) with a specific gene change.
It tests two new drugs (dabrafenib and trametinib) against dummy pills (placebo).
Eligible patients must have tried radioactive iodine and other specific treatments without success.
Participation involves regular clinic visits for checks and scans.
Patients on dummy pills might switch to active drugs if their cancer progresses.
The study aims to find better treatments for this challenging cancer.

Who may be eligible?

To be considered for this study, you need to be an adult (18 or older) with a confirmed diagnosis of advanced or widespread Differentiated Thyroid Cancer. Your cancer must have stopped responding to radioactive iodine treatment, and tests on your tumour must show a specific gene change called BRAF V600E. You should also have tried one or two types of treatment that target blood vessel growth, but your cancer has continued to progress. You also need to be fit enough for the study, as assessed by a doctor.

There are also some reasons why you might not be able to join. For example, if you have other types of thyroid cancer like anaplastic or medullary thyroid cancer, or if you've already taken similar types of drugs that target BRAF or MEK, you wouldn't be eligible. Also, recent treatments, like chemotherapy or radiation, or certain eye conditions like a history of blood clots in your eye, might prevent you from joining. This is to ensure the safety of all participants.

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 an adult (18 or older)?
Do you have advanced Differentiated Thyroid Cancer (DTC)?
Has your cancer stopped responding to radioactive iodine?
Do genetic tests show a 'BRAF V600E' change in your cancer?
Have you tried 1 or 2 other specific treatments for your cancer that target blood vessel growth?
Are you able to attend regular clinic appointments?

Answer every question to see your result.

What does participation involve?

If you join this study, you'll be assigned by chance (like flipping a coin) to one of two groups. Two out of every three people will receive the new drug combination (dabrafenib plus trametinib), and one out of every three will receive dummy pills (placeebos). Neither you nor your doctor will know which treatment you are getting.

You will take the study medication regularly. You'll also have regular visits to the clinic for various assessments, which will include blood tests, physical exams, and scans to see how your cancer is responding. These assessments help doctors check your health and how well the treatment is working. If you are in the placebo group and your cancer gets worse, you might have the option to switch to receive the active study drugs.

Treatment will continue as long as it's helping and you're not having serious side effects. Even after you stop the treatment, doctors will keep in touch to check on your health and any effects of the study over time. The study will involve around 150 patients in total.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. For potential benefits, you might receive a new treatment that could help your cancer, which might be more effective than other options. You'll also be closely monitored by a medical team. However, there's no guarantee that the new drugs will work for you, and like all medications, they can cause side effects. These could range from mild to more serious, and your study team will explain these to you in detail. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (41)

Northwestern University Med School
Verified postcode
Chicago, United States
Massachusetts General Hospital
Verified postcode
Boston, United States
Novartis Investigative Site
City only
CABA, Argentina
Novartis Investigative Site
City only
Rio de Janiero, Brazil
Novartis Investigative Site
Verified postcode
Blumenau, Brazil
Novartis Investigative Site
Verified postcode
São Paulo, Brazil
Novartis Investigative Site
Verified postcode
Edmonton, Canada
Novartis Investigative Site
Verified postcode
London, Canada
Novartis Investigative Site
Verified postcode
Fuzhou, China
Novartis Investigative Site
Verified postcode
Zhengzhou, China
Novartis Investigative Site
Verified postcode
Wuhan, China
Novartis Investigative Site
Verified postcode
Changsha, China

Common questions

What is Differentiated Thyroid Cancer (DTC)?

DTC is the most common type of thyroid cancer, usually growing slowly. This study focuses on DTC that has spread or is advanced.

What does 'BRAF V600E mutation' mean?

It's a specific change in a gene that can make cancer cells grow and multiply. This study targets treatments for cancers with this particular change.

What does 'radioactive iodine (RAI) refractory' mean?

This means the cancer has stopped responding to radioactive iodine treatment, which is a common treatment for thyroid cancer.

Will I know if I'm getting the real drugs or a dummy pill?

No, neither you nor your doctor will know. This is called 'double-blind' and helps ensure the study results are fair and unbiased.

What if the dummy pills don't work?

If your cancer gets worse while you're on the dummy pills, your study doctor may be able to offer you the active drug combination.