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

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

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

Study Overview

• Status: Completed
• Conditions: Primary Myelofibrosis; Post-polycythemia Vera Myelofibrosis; Post-essential Thrombocythemia Myelofibrosis
• Intervention / Treatment: Other: Jakavi

Detailed Description

The purpose of this NIS was 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 were documented in the first study arm; patients pretreated with JAK inhibitors were documented in the second study arm to evaluate the long-term efficacy of Jakavi® in this subpopulation.

The documentation of all patients was carried out prospectively and began after the baseline visit. The medical decision on which therapeutic and diagnostic measures to take was made solely by the responsible physician. The observational period per patient was 36 months. The visit schedule after the baseline visit was set by the responsible physician according to standard clinical care, the clinical condition of the respective patients and the SmPC.

• Study Type: Observational
• Enrollment (Actual): 1012

Contacts and Locations

Study Locations

• Germany
  • Aachen, Germany, 52074
    • Novartis Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 120 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Male and female patients with primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF), or post-essential thrombocythemia Myelofibrosis (PET-MF), for whom Jakavi® therapy is indicated.

Description

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:

-

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Arm A: JAK inhibitor naive; JAK-inhibitor-naive patients, treatment start with ruxolitinib less than 14 days prior to the baseline visit	Other: Jakavi; Prospective observational study. There is no treatment allocation. Patients administered Jakavi by prescription and administered according to the SmPC.; Other Names: Ruxolitinib
Arm B: Pretreated patients; Patients pretreated with a JAK-inhibitor for more than 14 days prior to the baseline visit	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
Safety and tolerability	Evaluation of all occurring adverse events, serious adverse events and serious and non-serious adverse drug reactions	Up to 36 months
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
Assessment of the Quality of Life (QoL) - Myeloproliferative Neoplasm - Symptom Assessment Form (MPN-SAF)	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.	Baseline, month 1, month 3, month 6, month 12, month 24 and month 36
Assessment of the Quality of Life (QoL) - Short Form-36 (SF-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.	Baseline month 6, month 12, month 24 and month 36
Overall survival	Overall survival for JAK inhibitor naive and pretreated patients	Up to 36 months
Spleen size (or volume) reduction	Spleen size (or volume) reduction was measured by palpation	Up to 36 months
Change in the number of patients with constitutional symptoms	Number of patients with change in constitutional symptoms was collected	Up to 36 months
Ruxolitinib start and end dose	Ruxolitinib start and end dose was collected	Up to 36 months
Therapy discontinuation and dose adjustments	Number of participants with therapy discontinuation and dose adjustments was collected	Up to 36 months
Number of patients with co-morbidities	Number of patients with co-morbidities was collected	Up to 36 months
Blood transfusion dependency	Number of patients with blood transfusion dependency was collected	Up to 36 months
Number of patients with concomitant medications	Number of patients with concomitant medications prescribed for myelofibrosis therapy and for the management of side effects was collected	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 CINC424ADE05 that is getting linked from the Novartis Clinical Trials Website

Study record dates

Study Major Dates

• Study Start (Actual): September 20, 2012
• Primary Completion (Actual): September 19, 2022
• Study Completion (Actual): September 19, 2022

Study Registration Dates

• First Submitted: September 6, 2021
• First Submitted That Met QC Criteria: September 6, 2021
• First Posted (Actual): September 14, 2021

Study Record Updates

• Last Update Posted (Actual): September 21, 2023
• Last Update Submitted That Met QC Criteria: September 15, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Keywords: Ruxolitinib; Jakavi; PMF; PPV MF; post-essential thrombocythemia myelofibrosis; Primary Myelofibrosis; post-polycythemia vera myelofibrosis; post ET MF
• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Site; Bone Marrow Diseases; Hematologic Diseases; Hemorrhagic Disorders; Myeloproliferative Disorders; Blood Coagulation Disorders; Blood Platelet Disorders; Bone Marrow Neoplasms; Hematologic Neoplasms; Primary Myelofibrosis; Thrombocytosis; Thrombocythemia, Essential; Polycythemia Vera; Polycythemia
• Other Study ID Numbers: CINC424ADE05

Plan for Individual participant data (IPD)

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