NCT05548062

Brief Summary

This is a prospective observational study that will enroll patients with high-risk Polycythemia Vera (PV) with at least one Thromboembolic Event (TE) after diagnosis or up to 2 years prior to diagnosis. This is a non-randomized study, and to ensure a sufficient number of patients in both cohorts, enrollment in each cohort will be terminated once the target of 150 patients has been reached.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
242

participants targeted

Target at P75+ for all trials

Timeline
16mo left

Started Mar 2023

Longer than P75 for all trials

Geographic Reach
1 country

35 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress71%
Mar 2023Aug 2027

First Submitted

Initial submission to the registry

September 16, 2022

Completed
5 days until next milestone

First Posted

Study publicly available on registry

September 21, 2022

Completed
5 months until next milestone

Study Start

First participant enrolled

March 2, 2023

Completed
4.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 31, 2027

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 31, 2027

Last Updated

September 3, 2025

Status Verified

September 1, 2025

Enrollment Period

4.5 years

First QC Date

September 16, 2022

Last Update Submit

September 2, 2025

Conditions

Keywords

polycythemia verahigh-risk polycythemia veraPVRuxolitinibNISItalyTEpredictive factorshydroxyurea

Outcome Measures

Primary Outcomes (8)

  • Percentage of abnormalities presented in patients with Thromboembolic Events (TEs) during the follow up

    Demographics, blood pressure, laboratory blood tests, and use of antiplatelets and/or anticoagulants are predictors of TE occurrence, a univariate Cox regression model for repeated events will be applied for each predictor considering all the TEs occurring within 12 months and the time from baseline to each occurrence. Predictors found statistically significant at the 5% level will then be considered in a multivariate Cox regression model for repeated events.

    Up to 36 months

  • Blood pressure

    Blood pressure is going to be collected

    Up to month 36

  • Number of patients with abnormal Body Mass Index (BMI)

    Number of patients with abnormal Body Mass Index (BMI) will be collected

    Up to month 36

  • Number of patients with abnormal weight

    Number of patients with abnormal weight will be collected

    Up to month 36

  • Number of patients with abnormal Neutrophil (NEP) count

    Number of patients with abnormal Neutrophil (NEP) count will be collected

    Up to 36 months

  • Number of patients with abnormal White blood Count (WBC)

    Number of patients with abnormal White blood Count (WBC) will be collected

    Up to 36 months

  • Number of patients with abnormal Lymphocytes (LYP) count

    Number of patients with abnormal Lymphocytes (LYP) count will be collected

    Up to 36 months

  • Number of participants using antiplatelets and/or anticoagulants on the incidence of TEs

    Number of participants using antiplatelets and/or anticoagulants on the incidence of Thromboembolic Events (TEs) will be collected

    Up to 36 months

Secondary Outcomes (4)

  • Synergistic combinations of predictive factors

    Up to 36 months

  • Yearly incidence of TEs

    36 months

  • Incidence of arterial and venous TEs in the hydroxyurea and ruxolitinib cohorts

    36 months

  • Incidence and severity of adverse events

    36 months

Study Arms (2)

Hydroxyurea

Patients being treated with hydroxyurea at enrollment and for at least 18 months prior to enrollment. Patients may switch to ruxolitinib treatment during the study in case of inadequate response or intolerance.

Other: Hydroxyurea

Ruxolitinib

Patients on treatment with ruxolitinib who started treatment up to 18 months prior to enrollment.

Other: Ruxolitinib

Interventions

Prospective observational study. There is no treatment allocation. Patients prescribed with Hydroxyurea are eligible to enroll into this study.

Hydroxyurea

Prospective observational study. There is no treatment allocation. Patients prescribed with Ruxolitinib are eligible to enroll into this study.

Ruxolitinib

Eligibility Criteria

Age18 Years - 99 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

The study will involve patients in Italy with high-risk Polycythemia Vera

You may qualify if:

  • Signed informed consent must be obtained prior to participation in the study.
  • Age ≥18 years.
  • Diagnosis of PV according to WHO 2008 or WHO 2016 and high-risk stratification according to European LeukemiaNet (ELN) classification.
  • At least one TE after diagnosis or up to 2 years prior to diagnosis.
  • Patients on treatment with hydroxyurea at enrollment and for at least 18 months prior to enrollment or those on treatment with ruxolitinib who started treatment up to 18 months before enrollment.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (35)

Novartis Investigative Site

Alessandria, AL, 15121, Italy

Location

Novartis Investigative Site

Ancona, AN, 60126, Italy

Location

Novartis Investigative Site

Bari, BA, 70124, Italy

Location

Novartis Investigative Site

Bologna, BO, 40138, Italy

Location

Novartis Investigative Site

Como, CO, 22100, Italy

Location

Novartis Investigative Site

Cosenza, CS, 87100, Italy

Location

Novartis Investigative Site

Catania, CT, 95123, Italy

Location

Novartis Investigative Site

Florence, FI, 50134, Italy

Location

Novartis Investigative Site

Genova, GE, 16132, Italy

Location

Novartis Investigative Site

Lecce, LE, 73100, Italy

Location

Novartis Investigative Site

Tricase, LE, 73039, Italy

Location

Novartis Investigative Site

Monza, MB, 20900, Italy

Location

Novartis Investigative Site

Milan, MI, 20122, Italy

Location

Novartis Investigative Site

Palermo, PA, 90127, Italy

Location

Novartis Investigative Site

Palermo, PA, 90146, Italy

Location

Novartis Investigative Site

Piacenza, PC, 29100, Italy

Location

Novartis Investigative Site

Padua, PD, 35100, Italy

Location

Novartis Investigative Site

Padua, PD, 35128, Italy

Location

Novartis Investigative Site

Pisa, PI, 56124, Italy

Location

Novartis Investigative Site

Parma, PR, 43126, Italy

Location

Novartis Investigative Site

Reggio Calabria, RC, 89100, Italy

Location

Novartis Investigative Site

Reggio Emilia, RE, 42123, Italy

Location

Novartis Investigative Site

Roma, RM, 00161, Italy

Location

Novartis Investigative Site

Roma, RM, 00168, Italy

Location

Novartis Investigative Site

Roma, RM, 00189, Italy

Location

Novartis Investigative Site

Pagani, SA, 84016, Italy

Location

Novartis Investigative Site

Orbassano, TO, 10043, Italy

Location

Novartis Investigative Site

Torino, TO, 10126, Italy

Location

Novartis Investigative Site

Terni, TR, 05100, Italy

Location

Novartis Investigative Site

Varese, VA, 21100, Italy

Location

Novartis Investigative Site

Vicenza, VI, 36100, Italy

Location

Novartis Investigative Site

Verona, VR, 37134, Italy

Location

Novartis Investigative Site

Viterbo, VT, 01033, Italy

Location

Novartis Investigative Site

Napoli, 80131, Italy

Location

Novartis Investigative Site

Napoli, 80132, Italy

Location

MeSH Terms

Conditions

Polycythemia Vera

Interventions

Hydroxyurearuxolitinib

Condition Hierarchy (Ancestors)

Bone Marrow NeoplasmsHematologic NeoplasmsNeoplasms by SiteNeoplasmsBone Marrow DiseasesHematologic DiseasesHemic and Lymphatic DiseasesMyeloproliferative Disorders

Intervention Hierarchy (Ancestors)

UreaAmidesOrganic Chemicals

Study Officials

  • Novartis Pharmaceuticals

    Novartis Pharmaceuticals

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 16, 2022

First Posted

September 21, 2022

Study Start

March 2, 2023

Primary Completion (Estimated)

August 31, 2027

Study Completion (Estimated)

August 31, 2027

Last Updated

September 3, 2025

Record last verified: 2025-09

Data Sharing

IPD Sharing
Will not share

Locations