A Prospective, Two-arm, Non-interventional Study of JAKAVI® (Ruxolitinib) in Patients With Myelofibrosis

Overview

This is a prospective, two-arm, non-interventional study of JAKAVI® (Ruxolitinib) in patients with myelofibrosis

Full Title of Study: “A Prospective, Two-arm, Non Interventional Study of JAKAVI® (Ruxolitinib) in Patients With Myelofibrosis”

Study Type

  • Study Type: Observational
  • Study Design
    • Time Perspective: Prospective
  • Study Primary Completion Date: September 30, 2022

Detailed Description

The purpose of this NIS is to gather data from the daily clinical practice of the Jakavi®-treatment in a broad patient population. In order to evaluate the direct effect of Jakavi®, only JAK inhibitor naive patients will be documented in the first study arm; patients pretreated with JAK inhibitors will be documented in the second study arm to evaluate the long-term efficacy of Jakavi® in this subpopulation. The documentation of all patients is carried out prospectively and begins after the baseline visit. The medical decision on which therapeutic and diagnostic measures to take is made solely by the responsible physician. The observational period per patient is 36 months. The visit schedule after the baseline visit is set by the responsible physician according to standard clinical care, the clinical condition of the respective patients and the SmPC.

Interventions

  • Other: Jakavi
    • Prospective observational study. There is no treatment allocation. Patients administered Jakavi by prescription and administered according to the SmPC.

Arms, Groups and Cohorts

  • JAK inhibitor naive
    • JAK-inhibitor-naive patients
  • Pretreated patients
    • Patients pretreated for at least 8 weeks with one or more JAK-inhibitors (Jakavi® or otherwise)

Clinical Trial Outcome Measures

Primary Measures

  • Safety and tolerability
    • Time Frame: Up to 36 months
    • Evaluation of all occurring adverse events, serious adverse events and serious and non-serious adverse drug reactions
  • Spleen size (or volume) reduction
    • Time Frame: Up to 36 months
    • Measured by palpation/sonography/CT/MRI
  • Eastern Cooperative Oncology Group (ECOG) performance status
    • Time Frame: Up to 36 months
    • 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).
  • Number of patients with change in constitutional symptoms
    • Time Frame: Up to 36 months
    • Number of patients with change in constitutional symptoms to be collected
  • Assessment of the Quality of Life (QoL) – Myeloproliferative Neoplasm – Symptom Assessment Form (MPN-SAF)
    • Time Frame: Baseline, month 1, month 3, month 6, month 12, month 24 and month 36
    • The MPN-SAF questionnaire contains important questions that cover MF-specific symptoms whose analysis is part of the standard of care. 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.
  • Assessment of the Quality of Life (QoL) – Short Form-36 (SF-36)
    • Time Frame: Baseline month 6, month 12, month 24 and month 36
    • This 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.
  • Overall survival
    • Time Frame: Up to 36 months
    • Overall survival for JAK inhibitor naive and pretreated patients
  • Initial dosing and dose modifications
    • Time Frame: Up to 36 months
    • Initial dosing and dose modifications to be collected
  • Number of patients with treatment interruptions
    • Time Frame: Up to 36 months
    • Number of patients with treatment interruptions to be collected
  • Number of patients with co-morbidities, transfusion dependency, iron overload and transformation into acute leukemia
    • Time Frame: Up to 36 months
    • Number of patients with co-morbidities, transfusion dependency, iron overload and transformation into acute leukemia to be collected
  • Number of patients with concomitant medications
    • Time Frame: Up to 36 months
    • Number of patients with concomitant medications prescribed for myelofibrosis therapy and for the management of side effects to be collected

Participating in This Clinical Trial

Inclusion Criteria

  • Male and female patients with Primary Myelofibrosis (PMF), post-Polycythemia Vera-Myelofibrosis (PPV-MF), or post-Essential Thrombocythemia-Myelofibrosis (post-ET-MF), for whom Jakavi® therapy is indicated. – Patients that were informed about all aspects of this NIS and provided written informed consent. Exclusion Criteria:

-

Gender Eligibility: All

Minimum Age: 18 Years

Maximum Age: 120 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Novartis Pharmaceuticals
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Novartis Pharmaceuticals, Study Director, Novartis Pharmaceuticals

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