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

NCT05044026 | Completed

• 1 other identifier: CINC424ADE05
• Study Type: observational
• Target: 1,012
• Locations: 1 country
• Sites: 1

Brief Summary

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

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

Outcome Measures

Primary Outcomes (12)

• Safety and tolerability

  Evaluation of all occurring adverse events, serious adverse events and serious and non-serious adverse drug reactions

  Up to 36 months

• Spleen size (or volume) reduction

  Spleen size (or volume) reduction was measured by palpation

  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

• Change in the number of patients with constitutional symptoms

  Number of patients with change in constitutional symptoms was collected

  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

• 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

Study Arms (2)

Arm A: JAK inhibitor naive

JAK-inhibitor-naive patients, treatment start with ruxolitinib less than 14 days prior to the baseline visit

Other: Jakavi

Arm B: Pretreated patients

Patients pretreated with a JAK-inhibitor for more than 14 days prior to the baseline visit

Other: Jakavi

Interventions

Jakavi OTHER

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

Also known as: Ruxolitinib

Arm A: JAK inhibitor naive; Arm B: Pretreated patients

Eligibility Criteria

• Age: 18 Years - 120 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (1)

Novartis Investigative Site

Aachen, 52074, Germany

Location

Related Publications (2)

• Koschmieder S, Isfort S, Schulte C, Jacobasch L, Geer T, Reiser M, Koenigsmann M, Heinrich B, Wehmeyer J, von der Heyde E, Tesch H, Groschl B, Bachhuber P, Grosser S, Koehler M, Pahl HL. Final Results From a Large, Non-Interventional, Phase 4 Study of Ruxolitinib for the Treatment of Myelofibrosis in Clinical Routine. Eur J Haematol. 2025 Oct;115(4):380-390. doi: 10.1111/ejh.70005. Epub 2025 Jul 6.

  PMID: 40619737 DERIVED

• Koschmieder S, Isfort S, Schulte C, Jacobasch L, Geer T, Reiser M, Koenigsmann M, Heinrich B, Wehmeyer J, von der Heyde E, Tesch H, Groschl B, Bachhuber P, Grosser S, Pahl HL. Real-world analysis of ruxolitinib in myelofibrosis: interim results focusing on patients who were naive to JAK inhibitor therapy treated within the JAKoMo non-interventional, phase IV trial. Ann Hematol. 2023 Dec;102(12):3383-3399. doi: 10.1007/s00277-023-05458-1. Epub 2023 Oct 4.

  PMID: 37792065 DERIVED

Related Links

• Results for CINC424ADE05 that is getting linked from the Novartis Clinical Trials Website

MeSH Terms

Conditions

Primary Myelofibrosis

Interventions

ruxolitinib

Condition Hierarchy (Ancestors)

Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 6, 2021
• First Posted: September 14, 2021
• Study Start: September 20, 2012
• Primary Completion: September 19, 2022
• Study Completion: September 19, 2022
• Last Updated: September 21, 2023

Record last verified: 2023-09

Data Sharing

• IPD Sharing: Will not share

Locations

DE (1)