Evaluation of HU-resistance in Adult Patients With Polycythemia Vera Who Meet PV-AIM Predictors

Phase 4

Recruiting

• Conditions: Polycythemia Vera
• Interventions: Drug: Hydroxyurea

• Registration Number: NCT05853458
• Lead Sponsor: Novartis Pharmaceuticals

Brief Summary

The purpose of this study is to confirm the predictive factors for hydroxyurea (HU) failure (hemoglobin (HGB) \<15.5 g/dL (9.62 mmol/L) and red blood cell distribution width (RDW) ≥17%) identified by machine learning in the polycythemia vera advanced integrated model (PV-AIM) project in the real-life setting.

Detailed Description

The study consists of three periods: Screening period, treatment period (observation for HU-resistance/intolerance) and follow-up (FU) period.

Eligible participants will enter the treatment period (observation period for HU-resistance/intolerance) and start receiving the de novo HU treatment. The maximum treatment duration for each participant in the study will be up to 15 months.

This study will be conducted in a total of 300 adult PV patients and approximately at 30 to 40 sites in Germany. If necessary, the study will be extended to other countries to achieve the target population.

Study Timeline

• Study First Submitted: 2023-05-02
• Study First Submitted QC: 2023-05-02
• Study First Posted: 2023-05-10
• Study Start: 2023-07-28
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-11
• Last Update Posted: 2025-04-16
• Primary Completion: 2026-06-01
• Study Completion: 2027-01-27

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 300

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Hydroxyurea (HU)	Hydroxyurea	Participants will be treated with HU capsules, orally taken, for a maximum duration of 15 months.

Primary Outcome Measures

Name	Time	Method
Proportion of PV patients with HU-resistance/intolerance within 6-9 months after start of de novo HU- treatment in presence of the PV-AIM HU-resistance predictors at the start of HU treatment.	From 6 to 9 months after start of de novo HU-treatment	Proportion of PV participants with HU resistance/intolerance within 6-9 months after start of de novo HU treatment in presence of the PV-AIM HU resistance predictors at the start of HU treatment. The proportion will be assessed calculating the rate together with the respective 95% confidence interval (CI).

Secondary Outcome Measures

Name	Time	Method
Proportion of PV patients who meet the PV-AIM HU-resistance predictors before start of HU-treatment	Baseline	Proportion of PV patients who meet the PV-AIM HU-resistance predictors before start of HU-treatment. The proportion will be assessed calculating the rate together with the respective 95% confidence interval (CI).
Proportion of patients developing HU resistance/intolerance at any time within the maximum treatment period of 15 months	Up to 15 months	Proportion of patients developing HU resistance/intolerance at any time within the maximum treatment period of 15 months. The proportion will be assessed calculating the rate together with the respective 95% confidence interval (CI).
Proportion of "non-switchers"	Up to 15 months	For all patients who develop HU resistance/intolerance according to modified European LeukemiaNet (ELN) criteria at any time during the maximum treatment period of 15 months, the proportion of "non-switchers" (i.e., patients remaining on HU despite they meet the HU-resistance/intolerance criteria) will be assessed. The rate will be calculated together with the respective 95% confidence interval (CI).
Timepoint of therapy switch (after confirmation of HU resistance/intolerance)	Up to 15 months	For all patients who develop HU resistance/intolerance according to modified ELN criteria at any time during the maximum treatment period of 15 months, the timepoint of therapy switch (after confirmation of HU resistance/intolerance) will be assessed.
Reasons for therapy switch / non-switch	Up to 15 months	For all patients who develop HU resistance/intolerance according to modified ELN criteria at any time during the maximum treatment period of 15 months, the reasons for therapy switch or non-switch will be summarized
Therapies applied during follow-up period	Up to 3 months after treatment discontinuation	For all patients who develop HU resistance/intolerance according to modified ELN criteria at any time during the maximum treatment period of 15 months, the therapies applied during the follow-up period will be summarized

Trial Locations

Locations (1)

Novartis Investigative Site; 🇩🇪 Wuerselen, Germany