Active not recruitingPHASE3INTERVENTIONAL

A Phase 3 Study of Rusfertide in Patients With Polycythemia Vera

This study is for people in the UK who have polycythemia vera (PV). We are testing a new medicine called rusfertide to see if it can help control the condition and reduce the need for regular blood draws (phlebotomy). PV can make your blood too thick, leading to symptoms and health risks. This study will compare rusfertide with a dummy medicine (placebo) alongside your usual PV treatment. We want to find out if rusfertide is safe and effective in keeping your blood thickness (haematocrit) stable and improving how you feel. Participants will be in the study for about 32 weeks with either rusfertide or placebo, then all will receive rusfertide for a longer period, followed by safety checks.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Protagonist Therapeutics, Inc.

Enrolment target

293

Start

01 Apr 2022

Estimated completion

01 Jun 2027

Polycythemia Vera

What is this study about?

Polycythemia vera (PV) is a long-term condition where your body makes too many red blood cells. This makes your blood thicker than it should be, which can lead to various symptoms like headaches, dizziness, itching, and tiredness. It can also increase your risk of blood clots. A common way to manage PV is by removing blood regularly, a procedure called phlebotomy, to thin your blood. Some people also take other medicines to help control the condition.

This study is looking at a new medicine called rusfertide. We want to find out if adding rusfertide to your current PV treatment can help control your blood thickness better and reduce how often you need phlebotomy. We also want to see if it helps improve your symptoms. About 250 people with PV who need regular phlebotomy will take part. Participants will be randomly assigned to either receive rusfertide or a dummy medicine (placebo) in addition to their current PV treatment for about 32 weeks.

After this initial period, all participants who complete the first part of the study will then receive rusfertide for a much longer time, about 124 weeks (that's over two years). Throughout the study, we will carefully check your health, blood tests, and how you are feeling to understand the effects of rusfertide. After you finish taking the study medicine, we will contact you a couple of times to make sure you are still doing well.

Key takeaways

This study is testing a new medicine called rusfertide for polycythemia vera (PV).
It aims to reduce the need for frequent blood draws (phlebotomy) and improve PV symptoms.
The study involves comparing rusfertide with a dummy medicine (placebo) first, alongside your usual care.
Participation includes regular clinic visits, blood tests, and follow-up for a total of over three years.
You might receive rusfertide or placebo in the first phase, but all participants will eventually receive rusfertide.
Your decision to join or leave the study won't affect your regular medical care.

Who may be eligible?

To join this study, you need to be at least 18 years old and have a confirmed diagnosis of polycythemia vera (PV) according to specific medical guidelines. A key requirement is that your PV has recently needed frequent blood removal (phlebotomy) to control your blood thickness, specifically at least three times in the last six months or five times in the last year.

Your blood test results, including your red blood cell count, white blood cell count, and platelet count, need to be within certain ranges just before starting the study. If you are already taking other medicines for your PV, these treatments need to have been stable for a while. If you manage your PV with phlebotomy alone, you should not have taken other PV-specific medicines within 2 to 6 months before the study starts.

There are also some reasons why you might not be able to join. For example, if you have other serious health problems, have had recent blood clots, or have active bleeding. You also can't join if you’ve had certain types of cancer in the last five years (unless fully treated and localised, like some skin or prostate cancers), or if you’ve taken certain specific PV medicines within the last seven months.

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?
Have you been diagnosed with polycythemia vera (PV) by a doctor?
Have you needed blood removed (phlebotomy) often recently for your PV (at least 3 times in 6 months or 5 times in 1 year)?
Are your current PV medicines (if any) at a stable dose, or have you stopped other PV medicines for at least 2-6 months if only on phlebotomy?
Do you not have any serious recent blood clots, active bleeding, or certain types of cancer in the last 5 years?

Answer every question to see your result.

What does participation involve?

If you decide to take part, the study will last for a long time. For the first 32 weeks, you will receive either the study medicine (rusfertide) or a dummy medicine (placebo), in addition to your usual PV treatment. This phase is 'double-blind', meaning neither you nor your study doctor will know which one you are receiving. During this time, you will have regular visits to the clinic for check-ups, blood tests, and to get your study medicine.

After these 32 weeks, if you successfully complete this part of the study, you will then receive rusfertide for a much longer period – about 124 weeks (over two years). This ensures everyone gets a chance to take the active medicine. Throughout these phases, you will continue to have regular clinic visits to monitor your health, blood counts, and any side effects. After your last dose of rusfertide, the study team will contact you by phone around 6 months and 12 months later to check on your well-being. The total duration of active participation where you are taking medication and having clinic visits will be approximately 156 weeks (32 + 124).

Potential risks and benefits

Taking part in any medical study carries potential benefits and risks. A potential benefit of this study is that rusfertide might help control your PV better, potentially reducing the need for frequent phlebotomy and improving your symptoms. However, it's also possible that rusfertide may not work for you, or you might receive the placebo during the first part of the study. Like all medicines, rusfertide could have side effects, which the study team will monitor closely. You will be provided with detailed information about potential risks. Remember, your participation is completely voluntary; you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (184)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Infirmary Cancer Care
Verified postcode
Mobile, United States
Palo Verde Hematology-Oncology
Verified postcode
Glendale, United States
City of Hope Medical Center, Duarte
Verified postcode
Duarte, United States
California Cancer Associates for Research and Excellence - Fresno
Verified postcode
Fresno, United States
Marin Cancer Care
Verified postcode
Greenbrae, United States
City of Hope Medical Center, Lenner
Verified postcode
Irvine, United States
University of California, San Diego (UCSD) - Moores Cancer Center
Verified postcode
La Jolla, United States
University of California, Los Angeles (UCLA) - Medical Center
Verified postcode
Los Angeles, United States
Stanford University
Verified postcode
Palo Alto, United States
Pomona Valley Hospital Medical Center
Verified postcode
Pomona, United States
Sharp Memorial Hospital
Verified postcode
San Diego, United States
University of California, San Francisco
Verified postcode
San Francisco, United States

Common questions

What is polycythemia vera (PV)?

PV is a blood disorder where your body makes too many red blood cells, making your blood thicker than normal.

What is rusfertide?

Rusfertide is an investigational medicine being tested to see if it can help manage PV by controlling blood thickness.

What is a 'placebo'?

A placebo is a dummy medicine that looks like the real thing but has no active ingredients. It helps us compare the effects of the new medicine.

How long will I be in the study?

The study involves an initial 32-week period where you might get rusfertide or placebo, followed by over two years where all participants receive rusfertide, plus some follow-up calls.

Will I still take my regular PV medication?

Yes, rusfertide or placebo will be added to your current PV treatment. You will continue with your ongoing therapy.