JAKoMo: A Prospective, Two-arm, Non-interventional Study of JAKAVI® (Ruxolitinib) in Patients With Myelofibrosis
Study Details
Study Description
Brief Summary
This is a prospective, two-arm, non-interventional study of JAKAVI® (Ruxolitinib) in patients with myelofibrosis
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
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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.
Study Design
Arms and Interventions
| Arm | 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:
|
| Pretreated patients Patients pretreated for at least 8 weeks with one or more JAK-inhibitors (Jakavi® or otherwise) |
Other: Jakavi
Prospective observational study. There is no treatment allocation. Patients administered Jakavi by prescription and administered according to the SmPC.
Other Names:
|
Outcome Measures
Primary Outcome Measures
- Safety and tolerability [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 [Up to 36 months]
Measured by palpation/sonography/CT/MRI
- Eastern Cooperative Oncology Group (ECOG) performance status [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 [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) [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) [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 [Up to 36 months]
Overall survival for JAK inhibitor naive and pretreated patients
- Initial dosing and dose modifications [Up to 36 months]
Initial dosing and dose modifications to be collected
- Number of patients with treatment interruptions [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 [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 [Up to 36 months]
Number of patients with concomitant medications prescribed for myelofibrosis therapy and for the management of side effects to be collected
Eligibility Criteria
Criteria
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:
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Novartis Investigative Site | Aachen | Germany | 52074 |
Sponsors and Collaborators
- Novartis Pharmaceuticals
Investigators
- Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- CINC424ADE05