Clinical trial NCT05044026: A Prospective, Two-arm, Non-interventional Study of JAKAVI® (Ruxolitinib) in Patients With Myelofibrosis
Official title: A Prospective, Two-arm, Non Interventional Study of JAKAVI® (Ruxolitinib) in Patients With Myelofibrosis
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Primary Myelofibrosis
Post-polycythemia Vera Myelofibrosis
Post-essential Thrombocythemia Myelofibrosis
Keywords: Primary Myelofibrosis
PMF
post-polycythemia vera myelofibrosis
PPV MF
post-essential thrombocythemia myelofibrosis
post ET MF
Ruxolitinib
Jakavi
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2021-09-14
Enrollment: 1012
Sex: ALL
Minimum age (years): 18
Maximum age (years): 120
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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

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.

Eligibility 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:

-

Primary outcomes:
measure: Safety and tolerability
timeFrame: Up to 36 months
measure: Spleen size (or volume) reduction
timeFrame: Up to 36 months
measure: Eastern Cooperative Oncology Group (ECOG) performance status
timeFrame: Up to 36 months
measure: Change in the number of patients with constitutional symptoms
timeFrame: Up to 36 months
measure: Assessment of the Quality of Life (QoL) - Myeloproliferative Neoplasm - Symptom Assessment Form (MPN-SAF)
timeFrame: Baseline, month 1, month 3, month 6, month 12, month 24 and month 36
measure: Assessment of the Quality of Life (QoL) - Short Form-36 (SF-36)
timeFrame: Baseline month 6, month 12, month 24 and month 36
measure: Overall survival
timeFrame: Up to 36 months
measure: Ruxolitinib start and end dose
timeFrame: Up to 36 months
measure: Therapy discontinuation and dose adjustments
timeFrame: Up to 36 months
measure: Number of patients with co-morbidities
timeFrame: Up to 36 months
measure: Blood transfusion dependency
timeFrame: Up to 36 months
measure: Number of patients with concomitant medications
timeFrame: Up to 36 months
Secondary outcomes:
Other outcomes:
Locations:
city: Aachen
country: Germany
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: