A Prospective, Non-interventional Study of JAKAVI® (Ruxolitinib) Treatment in Patients With Polycythemia Vera (PAVE)

A Prospective, Non-interventional Study of JAKAVI® (Ruxolitinib) Treatment in Patients With Polycythemia Vera (PAVE)

Jakavi® therapy for polycythemia vera (PV) has so far been studied exclusively in clinical trials and at selected clinical trial centres. This observational study is intended to document the therapy of PV in daily practice with a broad patient population and a geographically representative selection of German centres (both hospitals and practices). The prospective mapping of daily practice reality is thus the main goal of this project.

Study Overview

• Status: Completed
• Conditions: Polycythemia Vera
• Intervention / Treatment: Other: Jakavi

Detailed Description

To achieve meaningful results in accordance with the study objective and to obtain long-term data from daily clinical practice in a real-world setting, the observation period under Jakavi® therapy is specified as 36 months.

• Study Type: Observational
• Enrollment (Actual): 467

Contacts and Locations

Study Locations

• Germany
  • Altoetting, Germany, 84503
    • Novartis Investigative Site
  • Augsburg, Germany, 86150
    • Novartis Investigative Site
  • Bad Homburg vor der Höhe, Germany, 61348
    • Novartis Investigative Site
  • Bad Soden, Germany, 65812
    • Novartis Investigative Site
  • Berlin, Germany, 12351
    • Novartis Investigative Site
  • Berlin, Germany, 13357
    • Novartis Investigative Site
  • Bielefeld, Germany, 33604
    • Novartis Investigative Site
  • Bottrop, Germany, 46236
    • Novartis Investigative Site
  • Chemnitz, Germany, 09113
    • Novartis Investigative Site
  • Dortmund, Germany, 44263
    • Novartis Investigative Site
  • Dresden, Germany, 01307
    • Novartis Investigative Site
  • Dueren, Germany, 52353
    • Novartis Investigative Site
  • Duisburg, Germany, 47166
    • Novartis Investigative Site
  • Erfurt, Germany, 99085
    • Novartis Investigative Site
  • Essen, Germany, 45147
    • Novartis Investigative Site
  • Essen, Germany, 45136
    • Novartis Investigative Site
  • Fuerth, Germany, 90766
    • Novartis Investigative Site
  • Gera, Germany, 07548
    • Novartis Investigative Site
  • Goslar, Germany, 38642
    • Novartis Investigative Site
  • Halberstadt, Germany, 38820
    • Novartis Investigative Site
  • Halle, Germany, 06110
    • Novartis Investigative Site
  • Hamburg, Germany, 20259
    • Novartis Investigative Site
  • Hamburg, Germany, 22081
    • Novartis Investigative Site
  • Hameln, Germany, 31785
    • Novartis Investigative Site
  • Hamm, Germany, 59063
    • Novartis Investigative Site
  • Hamm, Germany, 59065
    • Novartis Investigative Site
  • Hannover, Germany, 30161
    • Novartis Investigative Site
  • Hannover, Germany, 30170
    • Novartis Investigative Site
  • Heidelberg, Germany, 69115
    • Novartis Investigative Site
  • Heilbronn, Germany, 74072
    • Novartis Investigative Site
  • Hildesheim, Germany, 31134
    • Novartis Investigative Site
  • Hildesheim, Germany, 31135
    • Novartis Investigative Site
  • Hof, Germany, 95028
    • Novartis Investigative Site
  • Idar-Oberstein, Germany, 55743
    • Novartis Investigative Site
  • Kaiserslautern, Germany, 67655
    • Novartis Investigative Site
  • Koblenz, Germany, 56068
    • Novartis Investigative Site
  • Koeln, Germany, 50671
    • Novartis Investigative Site
  • Koeln, Germany, 51103
    • Novartis Investigative Site
  • Kronach, Germany, 96317
    • Novartis Investigative Site
  • Leipzig, Germany, 04289
    • Novartis Investigative Site
  • Lemgo, Germany, 32657
    • Novartis Investigative Site
  • Loerrach, Germany, 79539
    • Novartis Investigative Site
  • Luedenscheid, Germany, 58507
    • Novartis Investigative Site
  • Lutherstadt Wittenberg, Germany, 06886
    • Novartis Investigative Site
  • Magdeburg, Germany, 39104
    • Novartis Investigative Site
  • Marburg, Germany, 35037
    • Novartis Investigative Site
  • Mayen, Germany, 56727
    • Novartis Investigative Site
  • Memmingen, Germany, 87700
    • Novartis Investigative Site
  • Minden, Germany, 32429
    • Novartis Investigative Site
  • Moers, Germany, 47441
    • Novartis Investigative Site
  • Muelheim, Germany, 45468
    • Novartis Investigative Site
  • Muenster, Germany, 48149
    • Novartis Investigative Site
  • Naunhof, Germany, 04683
    • Novartis Investigative Site
  • Nordhorn, Germany, 48527
    • Novartis Investigative Site
  • Nuernberg, Germany, 90419
    • Novartis Investigative Site
  • Nuernberg, Germany, 90403
    • Novartis Investigative Site
  • Nuernberg, Germany, 90449
    • Novartis Investigative Site
  • Offenburg, Germany, 77654
    • Novartis Investigative Site
  • Oldenburg, Germany, 26121
    • Novartis Investigative Site
  • Passau, Germany, 94036
    • Novartis Investigative Site
  • Porta Westfalica, Germany, 32457
    • Novartis Investigative Site
  • Potsdam, Germany, 14467
    • Novartis Investigative Site
  • Rostock, Germany, 18057
    • Novartis Investigative Site
  • Rostock, Germany, 18059
    • Novartis Investigative Site
  • Ruesselsheim, Germany, 65428
    • Novartis Investigative Site
  • Schorndorf, Germany, 73614
    • Novartis Investigative Site
  • Schöneck, Germany, 08621
    • Novartis Investigative Site
  • Stolberg, Germany, 52222
    • Novartis Investigative Site
  • Stuttgart, Germany, 70174
    • Novartis Investigative Site
  • Stuttgart, Germany, 70178
    • Novartis Investigative Site
  • Westerstede, Germany, 26655
    • Novartis Investigative Site
  • Wiesbaden, Germany, 65189
    • Novartis Investigative Site
  • Wolfsburg, Germany, 38440
    • Novartis Investigative Site
  • Wuerselen, Germany, 52146
    • Novartis Investigative Site
  • Wuerzburg, Germany, 97080
    • Novartis Investigative Site
  • Baden Wuerttemberg
    • Mannheim, Baden Wuerttemberg, Germany, 68305
      • Novartis Investigative Site
    • Reutlingen, Baden Wuerttemberg, Germany, 72764
      • Novartis Investigative Site
    • Winnenden, Baden Wuerttemberg, Germany, 71364
      • Novartis Investigative Site
  • Baden-Wuerttemberg
    • Heidenheim, Baden-Wuerttemberg, Germany, 89518
      • Novartis Investigative Site
  • Bavaria
    • Augsburg, Bavaria, Germany, 86152
      • Novartis Investigative Site
    • Herrsching, Bavaria, Germany, 82211
      • Novartis Investigative Site
    • Muenchen, Bavaria, Germany, 80639
      • Novartis Investigative Site
  • Bayern
    • Aschaffenburg, Bayern, Germany, 63739
      • Novartis Investigative Site
    • Donauwoerth, Bayern, Germany, 86609
      • Novartis Investigative Site
    • Erlangen, Bayern, Germany, 91052
      • Novartis Investigative Site
    • Muenchen, Bayern, Germany, 81241
      • Novartis Investigative Site
  • Brandenburg
    • Frankfurt, Brandenburg, Germany, 15236
      • Novartis Investigative Site
  • Bvaria
    • Landshut, Bvaria, Germany, 84036
      • Novartis Investigative Site
  • Lower Saxony
    • Goettingen, Lower Saxony, Germany, 37073
      • Novartis Investigative Site
    • Twistringen, Lower Saxony, Germany, 27239
      • Novartis Investigative Site
  • Niedersachsen
    • Brake, Niedersachsen, Germany, 26919
      • Novartis Investigative Site
  • North Rhine-Westphalia
    • Duisburg, North Rhine-Westphalia, Germany, 47166
      • Novartis Investigative Site
  • Northrhine Westfalia
    • Bad Salzuflen, Northrhine Westfalia, Germany, 32105
      • Novartis Investigative Site
    • Iserlohn, Northrhine Westfalia, Germany, 58644
      • Novartis Investigative Site
  • Sachsen
    • Dresden, Sachsen, Germany, 01127
      • Novartis Investigative Site
    • Pirna, Sachsen, Germany, 01796
      • Novartis Investigative Site
  • Saxony
    • Bautzen, Saxony, Germany, 02625
      • Novartis Investigative Site
  • Schleswig-Holstein
    • Luebeck, Schleswig-Holstein, Germany, 23563
      • Novartis Investigative Site
  • Thueringen
    • Erfurt, Thueringen, Germany, 99084
      • Novartis Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Adult male and female patients with PV for whom Jakavi® therapy is indicated.

Description

Inclusion Criteria:

• Adult male and female patients with PV for whom Jakavi® therapy is indicated according to the European summary of product characteristics
• Patients who have been informed about this NIS and gave written consent

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
JAK inhibitor naive; JAK inhibitor naive patients	Other: Jakavi; Prospective observational study. There is no treatment allocation. Patients administered Jakavi by prescription and administered according to the SmPC.; Other Names: Ruxolitinib
JAK inhibitor pre-treated; JAK inhibitor pre-treated patients (Jakavi® or any other JAK inhibitor for ≥3 months)	Other: Jakavi; Prospective observational study. There is no treatment allocation. Patients administered Jakavi by prescription and administered according to the SmPC.; Other Names: Ruxolitinib

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall survival	Overall survival for JAK inhibitor naive and pretreated patients	Up to 36 months
Quality of Life (QoL) - MPN-SAF TSS; MPN-10	The Myeloproliferative Neoplasm (MPN) Symptom Assessment Form Total Symptom Score (MPN-SAF TSS; MPN-10) questionnaire contains the ten most clinically relevant symptoms reported by patients with MPNs. It includes disease related symptoms each scored from 0 (absent) to 10 (worst imaginable). Total Scores range from 0-100, with higher scores indicating a greater number of symptoms and severity.	Up to 36 months
Dosing	Number of patients by initial dosing and number of patients with dose modifications will be provided	Up to 36 months
Treatment interruptions	Number of patients with treatment interruptions	Up to 36 months
Hematology	Number of patients with changes in different blood count values over time (hematocrit, erythrocytes, thrombocytes, leukocytes)	Up to 36 months
Thromboembolic events	Number of patients with thromboembolic events.	Up to 36 months
Change in spleen size (or volume)	Measured by palpation/sonography/CT/MRI	Up to 36 months
Number of phlebotomies	Total number of phlebotomies	Up to 36 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Eastern Cooperative Oncology Group (ECOG) performance status	The ECOG performance status is a scale used to assess how a patient's disease is progressing, assess how the disease affects the daily living abilities of the patient, and determine appropriate treatment and prognosis. The grade ranges from 0 (fully active, able to carry on all pre-disease performance without restriction) to 5 (dead)	Up to 36 months
Change in constitutional symptoms	Number of patients with change in constitutional symptoms	Up to 36 months
Quality of Life (QoL) - (SF-36)	The Short Form-36 questionnaire consists of questions measuring physical function, physical role limitation, pain, general health, vitality, social function, emotional role limitations, and mental health status. The scores that can be obtained from the scale vary between 0 and 100 and the increase in the scores indicates that the quality of life is high.	Up to 36 months
Bone marrow aspiration and biopsy	Number of Biopsies per visit	Up to 36 months
Molecular examination	Number of performed assessments of JAK2V617F mutation per visist	Up to 36 months

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Publications and helpful links

Helpful Links

• Results for CINC424BDE12 that is getting linked from the Novartis Clinical Trials Website

Study record dates

Study Major Dates

• Study Start (Actual): August 17, 2015
• Primary Completion (Actual): May 3, 2023
• Study Completion (Actual): May 3, 2023

Study Registration Dates

• First Submitted: May 2, 2023
• First Submitted That Met QC Criteria: May 2, 2023
• First Posted (Actual): May 10, 2023

Study Record Updates

• Last Update Posted (Actual): May 6, 2024
• Last Update Submitted That Met QC Criteria: May 2, 2024
• Last Verified: May 1, 2024

More Information

Terms related to this study

• Keywords: polycythemia vera; ruxolitinib; PV; jakavi
• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Site; Bone Marrow Diseases; Hematologic Diseases; Myeloproliferative Disorders; Bone Marrow Neoplasms; Hematologic Neoplasms; Polycythemia Vera; Polycythemia
• Other Study ID Numbers: CINC424BDE12

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO