Active not recruitingPHASE3INTERVENTIONAL

Ropeginterferon Alfa-2b (P1101) vs. Anagrelide in Essential Thrombocythemia Patients With Hydroxyurea Resistance or Intolerance

This research study is looking at a new treatment, Ropeginterferon Alfa-2b, for people living with a blood disorder called Essential Thrombocythemia (ET). ET causes your body to make too many platelets, increasing the risk of blood clots. This study aims to find out if Ropeginterferon Alfa-2b is more effective and safer than a standard treatment, Anagrelide, for patients whose ET hasn't improved or who have had problems with another common medicine called Hydroxyurea. Participants will be randomly assigned one of the two medicines and will be closely monitored for 12 months to see how they respond. The study will look at various health markers and side effects to understand the full picture of each treatment.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

PharmaEssentia

Enrolment target

174

Start

25 Aug 2020

Estimated completion

31 Aug 2029

Essential Thrombocythemia

What is this study about?

This clinical trial is designed to investigate a new medication called Ropeginterferon Alfa-2b, often shortened to P1101, for people diagnosed with Essential Thrombocythemia (ET). ET is a chronic condition where your bone marrow produces too many platelets, which are tiny blood cells that help stop bleeding. Having too many platelets can lead to serious health problems like blood clots or bleeding episodes. For many patients, the first line of treatment is a drug called Hydroxyurea.

However, some people don't respond well to Hydroxyurea, or they experience unpleasant side effects. This study aims to provide another option for these patients. It compares the new P1101 treatment with Anagrelide, another medication commonly used for ET, to see if P1101 is more effective at controlling platelet levels and has a better safety profile. By participating, you could help researchers understand if P1101 could become a valuable new treatment for ET patients who struggle with their current therapies.

The study is in 'Phase 3,' which means it's a large-scale trial happening after earlier tests have shown promising results. The goal is to confirm how well P1101 works, how safe it is over a longer period, and how it compares to an established treatment. This kind of research is crucial for developing new and better ways to manage long-term conditions like ET, ultimately offering more choices and better quality of life for patients.

Key takeaways

This study is for people with Essential Thrombocythemia (ET) who haven't responded well to Hydroxyurea.
It compares a new medicine (Ropeginterferon Alfa-2b) with a standard one (Anagrelide).
Participation involves regular clinic visits and health checks over 14 months.
You might gain access to a new treatment, but there are also potential side effects.
Your treatment will be chosen randomly, and you can withdraw at any time.

Who may be eligible?

To be considered for this study, you need to be at least 18 years old and have a specific type of ET called 'high-risk ET.' This means you're either over 60 years old and have a particular gene change (JAK2V617-positive), or you've previously had blood clots or bleeding related to your ET.

You must have tried Hydroxyurea for your ET in the past, but it either didn't work well enough (meaning your platelet count was still too high despite taking a normal or maximum dose) or you experienced side effects that made it difficult to continue the treatment. You also shouldn't have previously received Interferon treatment, or if you have, it needs to have been a while ago, and you can't have certain antibodies related to it. Your blood counts, such as platelets, white blood cells, and hemoglobin, need to be within specific ranges at the start of the study.

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 'high-risk' Essential Thrombocythemia (ET)?
Have you tried Hydroxyurea for your ET, but it didn't work well or caused problems?
Are you able to attend regular clinic visits for about 14 months?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you'll first go through a screening period of about one month to make sure you meet all the requirements. If you're eligible, you'll be randomly assigned to receive either the new medicine, Ropeginterferon Alfa-2b, or the existing medicine, Anagrelide. This means neither you nor your doctor will get to choose which treatment you receive. You'll take your assigned medication for about 12 months.

Throughout these 12 months, you'll have regular visits to the clinic for various assessments. These will include blood tests to check your platelet counts, other blood markers, and the levels of the study drug in your system. Doctors will also check your heart with an ECG and sometimes an ECHO, your lungs with an X-ray, and your overall health with physical exams and eye checks. You'll also be asked to fill out questionnaires about your quality of life and how you're feeling. After the 12-month treatment period, there will be a one-month follow-up period to monitor your health. The total time you'd be involved in the study is about 14 months.

Potential risks and benefits

Participating in a clinical trial offers potential benefits, such as gaining access to a new treatment that isn't widely available yet and receiving close medical monitoring for your condition. This study aims to find a more effective and safer option for ET patients who haven't responded well to standard treatments. However, there are also potential risks; you might experience side effects from the study medication, which could be different from or more severe than those from your current treatment. The new treatment might not work for you, or it might not be better than the existing one. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (65)

Washington University School of Medicine - Division of Oncology
Verified postcode
St Louis, United States
MD Anderson Cancer Center
Verified postcode
Houston, United States
University of Utah
Verified postcode
Salt Lake City, United States
St. Paul's Hospital
Verified postcode
Vancouver, Canada
Princess Margaret Hospital
Verified postcode
Toronto, Canada
Jewish General Hospital
Verified postcode
Montreal, Canada
Peking Union Medical College Hospital
Verified postcode
Beijing, China
Peking University People's Hospital
Verified postcode
Beijing, China
The First Affiliated Hospital, Chongqing Medical University
Verified postcode
Chongqing, China
NanFang Hospital of Southern Medical University
Verified postcode
Guangzhou, China
Union Hospital Tongji Medical College Huazhong University of Science and Technolog
Verified postcode
Wuhan, China
Zhongnan Hospital of Wuhan University
Verified postcode
Wuhan, China

Common questions

What is Essential Thrombocythemia (ET)?

ET is a blood disorder where your body makes too many platelets, which can increase your risk of blood clots or bleeding problems.

Why is this study being done?

This study is looking for better treatment options for ET patients whose current medication, Hydroxyurea, isn't working well or is causing difficult side effects.

What does 'randomised' mean in this study?

It means you'll be randomly chosen to receive one of the two study medicines, like flipping a coin, to ensure a fair comparison between them.

How long will I be involved in the study?

The study involves a screening period, 12 months of treatment, and a 1-month follow-up, so about 14 months in total.

Can I stop participating in the study if I want to?

Yes, you can leave the study at any time, for any reason, and it won't affect your future medical care.