RecruitingPHASE2INTERVENTIONAL

An Experimental Study of Belzutifan Impact on Catecholamine Metabolism

This study is testing a medication called Belzutifan in people who have a rare type of growth or tumour known as pheochromocytoma or paraganglioma (PPGL). These conditions can cause your body to make too much adrenaline and noradrenaline. The main goal is to find out if Belzutifan changes how these important chemicals are produced and used by your body. Twelve patients will take a 120mg dose of Belzutifan every day for five days. Researchers will measure specific substances in their blood before, during, and after taking the medication. This will help us understand the drug's impact on these chemical processes. This small study aims to gather initial information for a condition that affects only a small number of people.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

Cambridge University Hospitals NHS Foundation Trust

Enrolment target

Start

27 Apr 2026

Estimated completion

01 Dec 2027

Pheochromocytoma and Paraganglioma (PPGL)Pheochromocytoma Malignant Pheochromocytoma, Metastatic Pheochromocytoma/Paraganglioma Pheochromocytoma

What is this study about?

This study is an early-stage project looking into a new medication called Belzutifan for people diagnosed with a rare condition known as pheochromocytoma or paraganglioma (PPGL). These conditions are linked to growths or tumours, usually in or near the adrenal glands, that can cause your body to produce too much of chemicals like adrenaline and noradrenaline. These chemicals are important for how your body responds to stress, but too much can cause problems.

The main purpose of this study is to see if Belzutifan affects how these adrenaline-like chemicals are made and processed in the body. Researchers will be tracking a specific marker in the blood called tyrosine, along with adrenaline-related substances, to understand what changes might happen when someone takes Belzutifan. This is a very specific type of study, and it helps scientists learn more about how new treatments might work for very rare diseases.

Because this is a rare condition (only about 100-150 new cases each year in England), this is a small study involving just 12 patients. The results from this pilot study will give doctors and researchers valuable information. This information could be used to design larger studies in the future, helping to improve our understanding and treatment of PPGL.

Key takeaways

This study is for adults with a rare condition called pheochromocytoma or paraganglioma.
It aims to see how a drug called Belzutifan affects adrenaline-like chemicals in the body.
Participation involves taking Belzutifan for 5 days and attending clinic visits over 14 days.
Blood tests will be taken regularly to measure key chemicals.
This is a small, early-stage study to gather initial information for a rare disease.
You can stop participating at any time.

Who may be eligible?

To join this study, you must be an adult aged 18 or older. You need to have a confirmed diagnosis of pheochromocytoma or paraganglioma, which means your blood or urine tests show high levels of specific chemicals related to these conditions. If you are a woman who could become pregnant, you'll need a negative pregnancy test before starting the medication, as a safety measure. You must also be able to understand the study and willingly agree to take part.

There are some reasons why you couldn't join. For example, if you have severe heart problems, need regular oxygen, have another type of cancer, or have very low red blood cell counts, you wouldn't be eligible. Also, if you're taking certain medications that might react badly with Belzutifan, or if you have HIV, Hepatitis B, or Hepatitis C, you unfortunately cannot participate.

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 18 years old or older?
Do you have a confirmed diagnosis of pheochromocytoma or paraganglioma with high blood/urine levels?
Are you able to agree to take part after understanding the study information?
Are you free from severe heart problems or other serious medical conditions mentioned by the study team?
Are you not currently taking specific medications that interfere with the study drug?
If you are a woman who could become pregnant, are you able to have a negative pregnancy test?

Answer every question to see your result.

What does participation involve?

If you decide to take part, the study will last a total of 14 days. During this time, you will take 120mg of Belzutifan by mouth every day for five days. You'll have several visits to the study centre, especially at the beginning. You will come in daily for the first seven days, and then again on day 10 and day 14. During these visits, the study doctor will review how you're feeling, and blood samples will be taken. These blood samples will help us measure the chemicals we are interested in. All these steps help the research team understand how Belzutifan affects your body.

Potential risks and benefits

Taking part in this study may offer some potential benefits by helping researchers learn more about your condition and how Belzutifan might work. While this study is designed to be safe, there are potential risks, as with any medication, which the study team will explain fully. These might include side effects from Belzutifan or discomfort from blood tests. You will have a doctor overseeing your care throughout the study. Remember, joining is entirely your choice, and you are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

Cambridge University Hospitals NHS Foundation Trust
Verified postcode
Cambridge, United Kingdom· Recruiting

Common questions

What is pheochromocytoma/paraganglioma?

These are rare, usually non-cancerous growths that can lead to your body making too much adrenaline-like chemicals, which can cause symptoms like high blood pressure.

What does 'catecholamine metabolism' mean?

It refers to how your body produces, uses, and breaks down important chemicals like adrenaline and noradrenaline.

Why am I taking Belzutifan for only five days?

This is a small pilot study, and the researchers want to see the immediate effects of the drug over a short period to gather initial information.

Will I get 'better' from this study?

This is an experimental study designed to understand the drug's effects, not primarily to treat your condition. However, the information gained may help future treatments.

What will the blood tests show?

The blood tests will measure specific chemicals (tyrosine and metanephrines) that are involved in the production and breakdown of adrenaline-like substances in your body.

How to find out more

August Palma Senior Research Nurse - Endocrinology

august.palma@nhs.net 01223 217 848 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.