Clinical trial NCT05548062: Study to Identify and Describe Predictive Factors for Thromboembolic Events in Patients With High-risk Polycythemia Vera
Official title: Prospective Observational Study to Identify and Describe Predictive Factors for Thromboembolic Events in Patients With High-risk Polycythemia Vera
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Polycythemia Vera
Keywords: polycythemia vera
high-risk polycythemia vera
PV
Ruxolitinib
NIS
Italy
TE
predictive factors
hydroxyurea
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2022-09-21
Enrollment: 242
Sex: ALL
Minimum age (years): 18
Maximum age (years): 99
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study will involve patients in Italy with high-risk Polycythemia Vera

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.

Detailed description: All patients are already on treatment with hydroxyurea or ruxolitinib at enrollment as per clinical practice and independently of their participation in this study. In addition, the follow-up visits and the evaluation procedures required in the study protocol correspond to current clinical practice. According to local regulations related to observational studies, assessments such as blood tests are justified by the purpose and rationale of the study (i.e., the identification of possible predictive factors of TEs) and are considered current clinical practice. Data related to other procedures will be collected only if such procedures are performed as per clinical practice but are not required otherwise.

Patients in both cohorts will be followed for 3 years after enrollment and will have visits at Months 6, 12, 18, 24, 30 and 36. A time window of ± 1 month is permitted for all visits.

Eligibility criteria: Inclusion Criteria:

1. Signed informed consent must be obtained prior to participation in the study.
2. Age ≥18 years.
3. Diagnosis of PV according to WHO 2008 or WHO 2016 and high-risk stratification according to European LeukemiaNet (ELN) classification.
4. At least one TE after diagnosis or up to 2 years prior to diagnosis.
5. 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.

Primary outcomes:
measure: Percentage of abnormalities presented in patients with Thromboembolic Events (TEs) during the follow up
timeFrame: Up to 36 months
measure: Blood pressure
timeFrame: Up to month 36
measure: Number of patients with abnormal Body Mass Index (BMI)
timeFrame: Up to month 36
measure: Number of patients with abnormal weight
timeFrame: Up to month 36
measure: Number of patients with abnormal Neutrophil (NEP) count
timeFrame: Up to 36 months
measure: Number of patients with abnormal White blood Count (WBC)
timeFrame: Up to 36 months
measure: Number of patients with abnormal Lymphocytes (LYP) count
timeFrame: Up to 36 months
measure: Number of participants using antiplatelets and/or anticoagulants on the incidence of TEs
timeFrame: Up to 36 months
Secondary outcomes:
measure: Synergistic combinations of predictive factors
timeFrame: Up to 36 months
measure: Yearly incidence of TEs
timeFrame: 36 months
measure: Incidence of arterial and venous TEs in the hydroxyurea and ruxolitinib cohorts
timeFrame: 36 months
measure: Incidence and severity of adverse events
timeFrame: 36 months
Other outcomes:
Locations:
city: Alessandria
country: Italy
facility: Novartis Investigative Site
state: AL
city: Ancona
country: Italy
facility: Novartis Investigative Site
state: AN
city: Bari
country: Italy
facility: Novartis Investigative Site
state: BA
city: Bologna
country: Italy
facility: Novartis Investigative Site
state: BO
city: Como
country: Italy
facility: Novartis Investigative Site
state: CO
city: Cosenza
country: Italy
facility: Novartis Investigative Site
state: CS
city: Catania
country: Italy
facility: Novartis Investigative Site
state: CT
city: Firenze
country: Italy
facility: Novartis Investigative Site
state: FI
city: Genova
country: Italy
facility: Novartis Investigative Site
state: GE
city: Lecce
country: Italy
facility: Novartis Investigative Site
state: LE
city: Tricase
country: Italy
facility: Novartis Investigative Site
state: LE
city: Monza
country: Italy
facility: Novartis Investigative Site
state: MB
city: Milano
country: Italy
facility: Novartis Investigative Site
state: MI
city: Palermo
country: Italy
facility: Novartis Investigative Site
state: PA
city: Palermo
country: Italy
facility: Novartis Investigative Site
state: PA
city: Piacenza
country: Italy
facility: Novartis Investigative Site
state: PC
city: Padova
country: Italy
facility: Novartis Investigative Site
state: PD
city: Padova
country: Italy
facility: Novartis Investigative Site
state: PD
city: Pisa
country: Italy
facility: Novartis Investigative Site
state: PI
city: Parma
country: Italy
facility: Novartis Investigative Site
state: PR
city: Reggio Calabria
country: Italy
facility: Novartis Investigative Site
state: RC
city: Reggio Emilia
country: Italy
facility: Novartis Investigative Site
state: RE
city: Roma
country: Italy
facility: Novartis Investigative Site
state: RM
city: Roma
country: Italy
facility: Novartis Investigative Site
state: RM
city: Roma
country: Italy
facility: Novartis Investigative Site
state: RM
city: Pagani
country: Italy
facility: Novartis Investigative Site
state: SA
city: Orbassano
country: Italy
facility: Novartis Investigative Site
state: TO
city: Torino
country: Italy
facility: Novartis Investigative Site
state: TO
city: Terni
country: Italy
facility: Novartis Investigative Site
state: TR
city: Varese
country: Italy
facility: Novartis Investigative Site
state: VA
city: Vicenza
country: Italy
facility: Novartis Investigative Site
state: VI
city: Verona
country: Italy
facility: Novartis Investigative Site
state: VR
city: Viterbo
country: Italy
facility: Novartis Investigative Site
state: VT
city: Napoli
country: Italy
facility: Novartis Investigative Site
city: Napoli
country: Italy
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: