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Comparison of ruxolitinib versus hydroxycarbamide or interferon alfa as first treatment for high-risk polycythemia vera patients

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on Polycythemia Vera (PV), a rare blood disorder where the body produces too many red blood cells. The research compares three different treatments: ruxolitinib (also known as Jakavi), hydroxycarbamide, and peginterferon alfa-2a (also known as Pegasys) as initial treatments for high-risk Polycythemia Vera patients.

The purpose of the study is to determine which treatment is most effective at preventing serious complications in patients with high-risk Polycythemia Vera. The study will monitor patients for major blood clots, severe bleeding, disease progression to other blood disorders such as Myelodysplastic Syndrome, Acute Myeloid Leukemia, or Post-PV Myelofibrosis, and overall survival.

Patients will be randomly assigned to receive either ruxolitinib tablets taken by mouth, or one of two comparison treatments: hydroxycarbamide capsules taken by mouth, or peginterferon alfa-2a given as an injection under the skin. The treatment and monitoring period will continue for up to 96 months, during which patients will have regular check-ups to assess their response to treatment and monitor for any side effects.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial assignment to treatment group
You will be randomly assigned to receive one of the following treatments:
- Ruxolitinib (Jakavi) tablets taken by mouth
- Hydroxycarbamide (Hydroxycarbamide medac) capsules taken by mouth
- Peginterferon alfa-2a (Pegasys) injection under the skin
Step 2
Treatment period
The study will continue until April 30, 2030
Regular monitoring will include checks for:
- Blood cell counts
- Blood clots
- Bleeding events
- General health status
Your doctor may perform blood draws to check your response to treatment
Step 3
Health assessments
Throughout the study, you will have regular assessments of:
- Symptoms related to your condition
- Quality of life
- Side effects from treatment
- Spleen size (if enlarged before treatment)
- Need for blood draws (venesection) to control blood thickness
Step 4
Safety monitoring
Regular checks will be performed to monitor:
- Heart function
- Blood pressure
- Development of any new medical conditions
Any side effects or health changes should be reported to the study doctor
Step 5
Follow-up period
After completing treatment, you will continue to be monitored for:
- Long-term health outcomes
- Disease progression
- Any delayed effects of treatment

Who can join the trial?

10 criteria

Must be at least 18 years old
Must have been diagnosed with Polycythaemia Vera (PV) within the last 15 years, meeting World Health Organization criteria
check
Must be classified as high-risk PV, which means having an elevated white blood cell count above 11 x 109/l AND at least one of these conditions:
- Age over 60 years
- Previous blood clots or major bleeding related to the disease
- Platelet count higher than 1000 x 109/l
- High blood pressure or diabetes that requires medication
- Must have a hemoglobin level above 8g/dl during screening (hemoglobin is a protein that carries oxygen in blood)
- Can be currently taking blood-thinning medications and receiving blood removal treatments (venesection)
- Can have received no more than one type of cell-reducing therapy for less than 10 years, and must not have shown resistance or intolerance to that therapy
- Must be able to provide written informed consent to participate in the study

Who cannot join the trial?

14 criteria

Age below 18 years old
Prior treatment with ruxolitinib or other JAK inhibitors (medications that affect certain proteins in blood cells)
Active or uncontrolled bleeding disorders
Severe heart problems or uncontrolled high blood pressure
Significant liver disease or abnormal liver function tests
Severe kidney problems or impaired kidney function
Active, uncontrolled infections
History of other cancers in the past 3 years (except for successfully treated skin cancer or early-stage cancer)
Pregnant or breastfeeding women
Mental conditions that could interfere with following study procedures
Participation in other clinical trials within 30 days before starting this study
Known allergic reactions to the study medications
Unstable medical conditions that could affect study participation
Use of medications that could interact with the study drug

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Verified
Assistance Publique Hopitaux De Marseille
Marseille, France
Verified
Assistance Publique Hopitaux De Paris
Paris, France
Responsive
CHRU De Nancy
Vandoeuvre Les Nancy, France

Trial locations

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Investigated drugs

Ruxolitinib
is a medication that helps control blood cell production by blocking specific proteins in the body. It is used to treat blood disorders, including polycythemia vera, a condition where the body produces too many red blood cells.
Hydroxycarbamide
(also known as hydroxyurea) is a medication that slows down the production of blood cells in the bone marrow. It is commonly used to treat blood disorders and helps reduce the risk of complications in patients with polycythemia vera.
Interferon Alpha
is a protein-based medication that helps regulate blood cell production and supports the immune system. It is used to treat various blood disorders, including polycythemia vera, by helping to control the excessive production of blood cells.

What is already known about the treatment

Ruxolitinib
An oral medication taken in tablet form that belongs to the class of JAK inhibitors, specifically targeting JAK1 and JAK2 enzymes. This medication is used in the treatment of Polycythemia Vera (PV), a blood disorder where the body produces too many red blood cells, and works by blocking specific cellular signals that contribute to blood cell overproduction. It is currently approved as a treatment option for patients with PV who have had an inadequate response to or are intolerant of other therapies.
Hydroxycarbamide
An oral chemotherapy medication, also known as hydroxyurea, that is taken in capsule form and works by slowing down the production of blood cells in the bone marrow. This medication has been a standard treatment for Polycythemia Vera for many years, helping to reduce the risk of blood clots and manage excessive blood cell production. It belongs to the antimetabolite class of drugs and works by interfering with DNA synthesis in rapidly dividing cells.
Interferon Alpha
A protein-based medication administered via subcutaneous injection that mimics the body's natural immune response. It is used in the treatment of Polycythemia Vera to help regulate blood cell production and has shown effectiveness in controlling the disease while also potentially reducing the risk of disease progression. This medication belongs to the biological response modifier class and works by enhancing the body's immune system response while also directly affecting blood cell production in the bone marrow.

Investigated diseases

Polycythemia Vera (PV)
A rare blood disorder where the bone marrow produces too many red blood cells, white blood cells, and platelets. The excess blood cells cause the blood to become thicker than normal, which slows down blood flow. This condition develops slowly and can cause the spleen to become enlarged. PV is typically caused by a genetic mutation in the JAK2 gene. The condition may progress to other blood disorders over time.
Myelodysplastic Syndrome (MDS)
A group of blood disorders where the bone marrow fails to produce enough healthy blood cells. The blood cells that are produced are abnormal in shape and function. The condition causes the bone marrow to make fewer and fewer healthy blood cells over time.
Acute Myeloid Leukemia (AML)
A blood cancer that starts in the bone marrow, where immature blood cells develop abnormally and accumulate rapidly. These abnormal cells interfere with the production of normal blood cells. The condition develops quickly, usually over days or weeks.
Post-PV Myelofibrosis (PPV-MF)
A condition that can develop in some people with Polycythemia Vera, where the bone marrow is gradually replaced by fibrous tissue. This leads to decreased production of blood cells. The condition causes the bone marrow to become scarred and less able to produce blood cells.