RecruitingOBSERVATIONAL

Identification of Factors Associated With Treatment Response in Patients With Polycythemia Vera, Essential Thrombocythemia, and Pre-myelofibrosis.

This study is investigating why some patients with specific blood conditions called polycythemia vera, essential thrombocythemia, and pre-myelofibrosis don't get better with their usual first treatments, like hydroxyurea or pegylated interferon. These treatments are given to help prevent serious problems like blood clots and other complications. We know that if these first treatments don't work, patients might not do as well overall. At the moment, it's hard to tell who these patients will be. Researchers will be looking at things like a person's genetic make-up (using something called next-generation sequencing) and other biological signals at the time they are diagnosed. The aim is to see if these factors can predict how well someone will respond to treatment after 12 months. Finding this out could help doctors offer more suitable treatments earlier, leading to better results for patients.

At a glance

Status

Recruiting

Sponsor

University Hospital, Angers

Enrolment target

120

Start

17 May 2023

Estimated completion

17 Feb 2032

Myeloproliferative Neoplasm

What is this study about?

This study is about understanding how certain blood conditions, called polycythemia vera, essential thrombocythemia, and pre-myelofibrosis, respond to common initial treatments. These conditions are types of myeloproliferative neoplasms, which means they are disorders where your body makes too many blood cells. The main goal of treatment is to prevent serious issues like blood clots and to stop the disease from getting worse.

The usual first medicines for these conditions are called hydroxyurea or pegylated interferon. While many people do well on these, some don't respond at all, or their response fades over time. When this happens, it can lead to poorer health outcomes. Doctors currently don't have clear ways to predict which patients will or won't respond well to these standard treatments. This study aims to change that by looking for clues in your body.

Researchers will be examining specific biological information from patients, such as their genetic profile (using advanced genetic testing called next-generation sequencing) and other markers. They want to see if there's a link between these factors at the time of diagnosis and how well a patient responds to their treatment after a year. By finding these connections, the study hopes to identify patients who might not benefit from standard treatments early on, allowing doctors to consider other options sooner. This move towards 'precision medicine' means treatments can be tailored more effectively to individual patients, potentially leading to better health and well-being.

Key takeaways

Aims to improve treatment for blood disorders.
Investigates why standard treatments don't always work.
Looks at genetic and biological factors for clues.
Could help doctors choose better treatments earlier.
Focuses on more personalised care for patients.

Who may be eligible?

To be part of this study, you need to be an adult who has been diagnosed with polycythemia vera, essential thrombocythemia, or pre-myelofibrosis. You must be starting your very first treatment with either hydroxyurea or pegylated interferon for one of these conditions. It's also important that you are happy to officially agree to take part.

However, you wouldn't be able to join if you've already had treatment for your blood condition in the past. Also, if you have other types of cancer or if your myelofibrosis has become advanced, you wouldn't be eligible. Lastly, if you are undergoing other specific treatments for your condition, such as phlebotomy (blood removal) only, ruxolitinib, anagrelide, or pipobroman, you wouldn't be able to 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 an adult with polycythemia vera, essential thrombocythemia, or pre-myelofibrosis?
Are you about to start your very first treatment with hydroxyurea or pegylated interferon?
Have you NOT had previous treatment for your condition?
Do you NOT have other ongoing cancers or advanced myelofibrosis?
Are you NOT currently taking specific other treatments like ruxolitinib or anagrelide?
Are you willing to give your consent to participate?

Answer every question to see your result.

What does participation involve?

The detailed involvement in this study would typically include providing samples (like blood samples) at the beginning of your treatment, and potentially at follow-up appointments, so researchers can check your genetic and biological markers. You will be starting either hydroxyurea or pegylated interferon as your main treatment, as decided by your doctor based on your condition. The study will then follow your progress for at least 12 months, checking how well your treatment is working. There won't be any new medication or extra medical procedures specifically for the study beyond what your doctor would normally recommend for your condition. Your total participation involves allowing researchers to collect and analyse your data and samples over this time.

Potential risks and benefits

Participating in this study might not offer direct health benefits to you, but the information gained could greatly help future patients by leading to more targeted and effective treatments. All treatments have potential side effects, and you should discuss those of hydroxyurea and pegylated interferon with your doctor. The main 'risk' of the study itself is the time commitment and providing samples, which is generally considered very low risk. You have the right to withdraw from the study at any time without affecting your medical care.

Locations (6)

Chu Angers
Verified postcode
Angers, France· Recruiting
Chu Brest
Verified postcode
Brest, France· Recruiting
Chu Nantes
Verified postcode
Nantes, France· Recruiting
Chu Poitiers
Verified postcode
Poitiers, France· Recruiting
Chu Rennes
Verified postcode
Rennes, France· Recruiting
Chu Tours
Verified postcode
Tours, France· Recruiting

Common questions

What are polycythemia vera, essential thrombocythemia, and pre-myelofibrosis?

These are types of blood disorders where your body makes too many blood cells. They can sometimes lead to problems like blood clots.

What is 'next-generation sequencing'?

This is a fancy way of saying advanced genetic testing. It helps scientists look closely at your genes to find tiny changes that might affect your health or how you respond to medicine.

What does 'precision medicine' mean?

Precision medicine is about giving treatments that are specifically chosen for an individual patient, based on their unique genetic makeup and other biological information, rather than a 'one-size-fits-all' approach.

Will I get a new medication if I join this study?

No, you will receive the standard first treatments (hydroxyurea or pegylated interferon) that your doctor would normally prescribe. The study is about understanding why these treatments work for some and not others.

How long will I be involved in the study?

The study aims to follow how you respond to treatment for at least 12 months, usually by looking at your medical records and samples from regular appointments.

How to find out more

CORENTIN ORVAIN, DOCTOR

corentin.orvain@chu-angers.fr +3302 41 35 44 75 Official trial record

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