Study to Identify and Describe Predictive Factors for Thromboembolic Events in Patients With High-risk Polycythemia Vera

Polycythemia Vera Clinical Trial

— Prospero  

Official title:

Prospective Observational Study to Identify and Describe Predictive Factors for Thromboembolic Events in Patients With High-risk Polycythemia Vera

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05548062
• Other study ID #: CINC424BIT01
• Status: Recruiting
• Start date: March 2, 2023
• Est. completion date: September 30, 2026

Study information

• Verified date: March 2024
• Source: Novartis
• Contact: Novartis Pharmaceuticals
• Phone: +41613241111
• Email: novartis.email[@]novartis.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 300
• Est. completion date: September 30, 2026
• Est. primary completion date: September 30, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 99 Years

Eligibility

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.

Related Conditions & MeSH terms

• Polycythemia
• Polycythemia Vera
• Thromboembolism

Intervention

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

Locations

Country	Name	City	State
Italy	Novartis Investigative Site	Alessandria	AL
Italy	Novartis Investigative Site	Ancona	AN
Italy	Novartis Investigative Site	Bari	BA
Italy	Novartis Investigative Site	Bologna	BO
Italy	Novartis Investigative Site	Catania	CT
Italy	Novartis Investigative Site	Como	CO
Italy	Novartis Investigative Site	Cosenza	CS
Italy	Novartis Investigative Site	Firenze	FI
Italy	Novartis Investigative Site	Genova	GE
Italy	Novartis Investigative Site	Lecce	LE
Italy	Novartis Investigative Site	Milano	MI
Italy	Novartis Investigative Site	Monza	MB
Italy	Novartis Investigative Site	Napoli
Italy	Novartis Investigative Site	Napoli
Italy	Novartis Investigative Site	Orbassano	TO
Italy	Novartis Investigative Site	Padova	PD
Italy	Novartis Investigative Site	Pagani	SA
Italy	Novartis Investigative Site	Palermo	PA
Italy	Novartis Investigative Site	Palermo	PA
Italy	Novartis Investigative Site	Parma	PR
Italy	Novartis Investigative Site	Piacenza	PC
Italy	Novartis Investigative Site	Pisa	PI
Italy	Novartis Investigative Site	Reggio Calabria	RC
Italy	Novartis Investigative Site	Reggio Emilia	RE
Italy	Novartis Investigative Site	Roma	RM
Italy	Novartis Investigative Site	Roma	RM
Italy	Novartis Investigative Site	Roma	RM
Italy	Novartis Investigative Site	Roma	RM
Italy	Novartis Investigative Site	Terni	TR
Italy	Novartis Investigative Site	Torino	TO
Italy	Novartis Investigative Site	Tricase	LE
Italy	Novartis Investigative Site	Varese	VA
Italy	Novartis Investigative Site	Verona	VR
Italy	Novartis Investigative Site	Vicenza	VI
Italy	Novartis Investigative Site	Viterbo	VT

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Country where clinical trial is conducted

Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	Blood pressure	Blood pressure is going to be collected	Up to month 36
Primary	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
Primary	Number of patients with abnormal weight	Number of patients with abnormal weight will be collected	Up to month 36
Primary	Number of patients with abnormal Neutrophil (NEP) count	Number of patients with abnormal Neutrophil (NEP) count will be collected	Up to 36 months
Primary	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
Primary	Number of patients with abnormal Lymphocytes (LYP) count	Number of patients with abnormal Lymphocytes (LYP) count will be collected	Up to 36 months
Primary	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	Synergistic combinations of predictive factors	Synergistic combinations of predictive factors (Red cell Distribution Width, neutrophils, lymphocytes, neutrophil/ lymphocyte ratio and platelets).; Synergy score is defined as the product of the individual significances of variable 1 and 2 (expected) divided by the significance of the two-variable model (observed) To investigate cases of extreme synergy, instances in which two variables split the given cohort into high-risk and low-risk patients are far better than either variable alone. A simple synergy scoring metric to rank variable in terms of synergy (S):S12=(P1*P2)/P12 where P1 and P2 are the maximum possible (Cox regression derived) p-values for variable 1 and variable 2 and P12 was the max. p-value possible from the combination of variables 1 and 1. This synergy is intended to capture a variable that may provide exclusive non-redundant information when attempting to split a cohort based on risk and may provide an added insight into the functional/clinical rationale of a model	Up to 36 months
Secondary	Yearly incidence of TEs	Yearly incidence of Thromboembolic Events (TEs) will be collected	36 months
Secondary	Incidence of arterial and venous TEs in the hydroxyurea and ruxolitinib cohorts	Incidence of arterial and venous TEs in the hydroxyurea and ruxolitinib cohorts will be collected	36 months
Secondary	Incidence and severity of adverse events	Incidence and severity of adverse events according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.	36 months