Real-world Ruxolitinib Experience in PV

Not yet recruiting

• Conditions: Polycythemia Vera

• Registration Number: NCT06251102
• Lead Sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto

Brief Summary

This is a multicentric, observational, retro-prospective study in adult PV patients - resistant or intolerant to hydroxyurea - who are going to receive or have already initiated treatment with ruxolitinib according to the approved local label. Enrolment will last 9 months after the first enrolled patient. Patients will be observed for a minimum of 3 months, in order to evaluate the primary endpoint for all patients.

Detailed Description

This is a multicentric, observational, retro-prospective study in adult population who have been diagnosed with polycythemia vera according to the 2022 (WHO or ICC) criteria, who are resistant or intolerant to hydroxyurea and who are going to be prescribed or have already initiated treatment with ruxolitinib according to the approved local label. Patients who started treatment with ruxolitinib - according to clinical practice - will be enrolled. Enrolment will last 9 months after the first enrolled patient. Patients will be observed for a minimum of 3 months, in order to evaluate the primary endpoint for all patients.

Study Timeline

• Study First Submitted: 2024-02-01
• Study First Submitted QC: 2024-02-01
• Study First Posted: 2024-02-09
• Status Verified: 2024-05
• Last Update Submitted: 2024-05-08
• Last Update Posted: 2024-05-09
• Study Start: 2024-07
• Primary Completion: 2025-07
• Study Completion: 2025-07

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 153

Inclusion Criteria

1. Patients aged ≥ 18 years of age
2. Subjects must be diagnosed with PV according to the 2022 World Health Organization (WHO) or International Consensus Classification (ICC) criteria
3. Subjects must have a treatment history for PV that meets the definition of resistance or intolerance to hydroxyurea (HU) in accordance with the indications of the Italian Medicines Agency
4. Patients already on ruxolitinib treatment (retrospective cohort) at the start date of the study or patients who will start ruxolitinib (prospective cohort) during the study enrollment
5. Signed informed consent

Exclusion Criteria

1. Different diagnosis from PV [eg. other chronic myeloproliferative neoplasia such as essential thrombocythemia, myelofibrosis; or of congenital erythrocytosis or secondary erythrocytosis]

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
effectiveness of ruxolitinib	at 3 months	Proportion of patients who reach the target hct \<45% within 3 months in the absence of phlebotomy