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Evaluating resistance to hydroxyurea in adults with polycythemia vera

HU-F-AIM - A Prospective, Interventional Study to Evaluate HU-resistance in Polycythemia Vera Patients Who Meet Predictive Parameters Identified in the Machine Learning Project PV-AIM

Phase 4 Interventional Novartis · NCT05853458

This study is testing why some adults with polycythemia vera don’t respond well to the medication hydroxyurea to help improve treatment options.

Quick facts

PhasePhase 4
Study typeInterventional
Enrollment300 (estimated)
Ages18 Years to 99 Years
SexAll
SponsorNovartis Industry-sponsored
Locations33 sites (Heidelberg, Baden Wuerttemberg and 32 other locations)
Trial IDNCT05853458 on ClinicalTrials.gov

What this trial studies

This study aims to confirm predictive factors for hydroxyurea (HU) treatment failure in adult patients with polycythemia vera (PV) using real-life data. Participants will undergo a screening period followed by a treatment period where they will receive HU and be observed for resistance or intolerance. The study will include approximately 300 patients across 30 to 40 sites in Germany, with a potential extension to other countries if necessary. The maximum duration of treatment for each participant is up to 15 months.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18 and older with a confirmed diagnosis of polycythemia vera who meet specific risk criteria.

Not a fit: Patients who have previously received pharmacologic cytoreductive therapy or have had phlebotomy in the last 14 days may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could improve treatment strategies for patients with polycythemia vera by identifying those who are likely to benefit from hydroxyurea.

How similar studies have performed: Other studies have shown success in using predictive models for treatment outcomes in similar patient populations, making this approach promising.

Eligibility criteria

Show full inclusion / exclusion criteria 
Key Inclusion criteria

1. Signed informed consent must be obtained prior to participation in the study
2. Patients ≥18 years
3. Confirmed diagnosis of Polycythemia vera (according to WHO 2008, 2016, or 2022 criteria) (Tefferi and Vardiman 2008, Arber et al 2016, Khoury et al 2022)
4. Eastern Cooperative Oncology Group (ECOG) ≤ 2
5. No previous pharmacologic cytoreductive therapy (including investigational drugs)
6. No phlebotomy in last 14 days
7. HU-eligible

   * High-risk: age ≥ 60 years and/or prior history of thrombosis
   * Low-risk: showing at least one of the defined criteria

     * Signs of disease progression (myeloproliferation):

       * Increase in spleen size or symptomatic splenomegaly
       * Platelet increase to \> 1,000,000/µl
       * WBC increase to \> 15,000/µl or higher
       * Frequent (\> 10 per year) or increasing frequency of phlebotomies
     * Increasing risk of thromboembolism and bleeding:

       * New thromboembolism and/or hemorrhagic complications
       * Microcirculation disorders despite acetyl salicylic acid (ASA) 2x 100 mg/day
       * Restricted feasibility or intolerance of phlebotomies
       * Symptomatic iron deficiency
       * Uncontrolled increase in hematocrit
       * Severe or distressing disease-related symptoms
8. Female participants of childbearing potential should have a negative serum pregnancy test within 72 hours prior to receiving the first dose of study treatment.

Key Exclusion criteria

1. Patients with post-polycythemia vera myelofibrosis (post-PV MF) or accelerated phase/ blast phase myeloproliferative neoplasm acute myeloid leukemia (AP/BP-MPN AML).
2. Patients with a contraindication to HU according to the SmPC (severe bone marrow depression, leukopenia (\< 2.5 x 109 leukocytes/l), thrombocytopenia (\< 100 x 109 platelets/L), severe anemia (\< 10 g/dL HGB).
3. Patients with rare hereditary galactose intolerance, total lactase deficiency or glucose-galactose malabsorption in their past medical history.
4. Active uncontrolled infection that is considered by the Investigator as a reason for exclusion.
5. Active malignancies (except for carcinoma in situ; prostate cancer and breast cancer in remission and - where necessary - ongoing hormonal therapy).
6. Inadequate renal function as demonstrated by Modification of Diet in Renal Disease estimate glomerular filtration rate (MDRDeGFR) \< 30 mL/min/1.73m2 or on dialysis.
7. Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive human chorionic gonadotrophin (hCG) laboratory test.
8. Sexually active males unwilling to use a condom during intercourse while taking study treatment and for at least 3 months after stopping study treatment.
9. HIV patients treated with nucleoside reverse transcriptase inhibitors like didanosine and stavudine.

Other inclusion/exclusion criteria may apply

Where this trial is running

Heidelberg, Baden Wuerttemberg and 32 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 VeraPolycythemia veraHydroxyureaResistanceIntolerancePredictive parameters
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.