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Comparing Ruxolitinib to Hydroxycarbamide or Interferon for High Risk Polycythemia Vera

A Phase III, Randomised, Open-label, Multicenter International Trial Comparing Ruxolitinib With Either HydRoxycarbamIDe or Interferon Alpha as First Line ThErapy for High Risk Polycythemia Vera

Phase 3 Interventional University of Birmingham · NCT04116502

This study is testing whether ruxolitinib works better than hydroxycarbamide or interferon for people with high-risk polycythemia vera.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment586 (estimated)
Ages18 Years and up
SexAll
SponsorUniversity of Birmingham Academic / other
Drugs / interventionsruxolitinib
Locations47 sites (Aberdeen and 46 other locations)
Trial IDNCT04116502 on ClinicalTrials.gov

What this trial studies

This phase III, randomized-controlled trial aims to evaluate the efficacy of ruxolitinib compared to best available therapy, which includes hydroxycarbamide or interferon alpha, in patients with high-risk polycythemia vera. Participants will be randomly assigned to receive either ruxolitinib or the chosen best available therapy, with no cross-over allowed between treatment arms. The trial will be conducted across multiple international centers, ensuring a diverse patient population. The primary goal is to determine which treatment provides better outcomes for patients at high risk of complications from their condition.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older with a recent diagnosis of high-risk polycythemia vera.

Not a fit: Patients with polycythemia vera diagnosed more than 15 years ago or those without a JAK-2 mutation may not benefit from this study.

Why it matters

Potential benefit: If successful, this trial could provide a more effective first-line treatment option for patients with high-risk polycythemia vera.

How similar studies have performed: Other studies have shown promising results with ruxolitinib in similar patient populations, indicating potential for success in this approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
Population:

High risk PV defined as WBC \>11 x 10\^9/l\* AND at least ONE of the following

* Age \>60 years
* Prior thrombosis or haemorrhage
* Platelet count \>1000 x 10\^9/l\*
* Hypertension or diabetes requiring pharmacological therapy (\*At any time since diagnosis)

Inclusion Criteria:

1. Patient ≥18 years of age
2. Diagnosis of PV meeting the WHO criteria within the past 15 years
3. Meets criteria of high risk\* PV (see above for specific population)
4. Patients must have a screening haemoglobin of \>8g/dl
5. Patients may have received antiplatelet agents and venesection
6. Patients may have received ONE cytoreductive therapy for PV less than 10 years (BUT they should not be resistant or intolerant to that therapy)
7. Able to provide written informed consent

Exclusion Criteria:

1. Diagnosis of PV \> 15 years previously
2. Absence of JAK-2 mutation
3. Patients with any contraindications to any of the investigational medical products
4. Treatment with \>1 cytoreductive therapy OR a cytoreductive treatment duration exceeding 10 years OR resistance/intolerance to that therapy
5. Active infection including Human Immunodeficiency Virus (HIV), hepatitis B, hepatitis C, autoimmune hepatitis, Tuberculosis
6. Pregnant or lactating patients (Women of childbearing potential must have a negative urine or blood Human Chorionic Gonadotropin pregnancy test prior to trial entry)
7. Patients with lactose allergies, hypersensitivities, or rare hereditary problems, of galactose intolerance, total lactase deficiency or glucose- galactose malabsorption
8. Patients with uncontrolled neuropsychiatric disorders
9. Patients with uncontrolled cutaneous cancers
10. Patients and partners not prepared to adopt highly effective contraception measures (if sexually active) whilst on treatment and for at least 6 months after completion of study medication
11. ECOG Performance Status Score ≥ 3
12. Uncontrolled rapid or paroxysmal atrial fibrillation, uncontrolled or unstable angina, recent (within the last 6 months) myocardial infarction or acute coronary syndrome or any clinically significant cardiac disease \> NYHA ( New York Heart Association) Class II
13. Patients who have transformed to myelofibrosis
14. Previous treatment with ruxolitinib
15. Previous (within the last 12 months) or current platelet count \<100 x 109/L or neutrophil count \< 1 x 109/L not due to therapy
16. Inadequate liver function as defined by ALT/AST \>2.0 x ULN
17. Inadequate renal function as defined by eGFR \< 30 mls/min
18. Unable to give informed consent

    Additional Exclusion Criteria for France Only
19. All women of childbearing potential (as per Appendix 8 definition)
20. No affiliation with the French healthcare system
21. Persons under psychiatric care that would impede understanding of informed consent and optimal treatment and follow-up
22. Adults subject to a legal protection measure (guardianship, curatorship and safeguard of justice)
23. Patients deprived of their liberty by a judicial or administrative decision

Where this trial is running

Aberdeen and 46 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Polycythemia Vera
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.