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

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What is this study about?

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.

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

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

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

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

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 Study?

  • 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
  • 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 Study?

  • 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

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Centre Hospitalier Lyon Sud Pierre Benite France
Centre Hospitalier Victor Dupouy Argenteuil Argenteuil France
Centre Hospitalier Universitaire De Bordeaux Bordeaux France
Centre Hospitalier Regional Et Universitaire De Brest Brest France

Other Sites

Site Name City Country Status
Centre Hospitalier De Versailles Le Chesnay-Rocquencourt France
Hôpital Avicenne Bobigny France
Union Mut Gestion Groupe Hosp Mutualiste De Grenoble Grenoble France
Institut De Cancerologie Strasbourg Europe STRASBOURG, Alsace France
Centre Hospitalier Sud Francilien Corbeil Essonnes France
Centre Hospitalier Le Mans Le Mans France
Centre Hospitalier Universitaire De Montpellier Montpellier France
Centre Hospitalier Universitaire De Nantes Nantes France
Clinique Victor Hugo Le Mans France
Hôpital Pontchaillou-CHU Rennes Rennes France
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Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
France France
Recruiting
19.10.2024

Trial locations

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.

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.

Trial ID:
2024-516109-21-00
Protocol code:
RG_16-148
NCT ID:
NCT04116502
Trial Phase:
Therapeutic confirmatory (Phase III)

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