Ruxolitinib in Primary Myelofibrosis and Secondary to Essential Thrombocythemia or Polycythemia Vera (RUX-MF)

December 3, 2024 updated by: IRCCS Azienda Ospedaliero-Universitaria di Bologna
The study is observational multicenter retrospective and prospective cohort study of patients with primary or secondary myelofibrosis who have initiated therapy with ruxolitinib, prescribed as part of the normal course of care and completely independent of study participation. The primary purpose is to determine the impact of clinical and laboratory characteristics of myelofibrosis on the prognosis of patients treated with ruxolitinib, understood as long-term survival.

Study Overview

Status

Recruiting

Conditions

Detailed Description

The study is observational multicenter retrospective and prospective cohort study of patients with primary or secondary myelofibrosis who have initiated therapy with ruxolitinib, prescribed as part of the normal course of care and completely independent of study participation. Laboratory tests and histological, cytogenetic, molecular, and radiological investigations performed by the patient and collected for study will be conducted in accordance with clinical practice, independent of the patient's participation in the study. In particular. data on systemic symptoms and splenomegaly will be collected at diagnosis and disease reassessments performed in the context of normal clinical practice. The minimum planned duration of individual patient observation is 3 months and the planned duration of the study is 10 years.

Study Type

Observational

Enrollment (Estimated)

1055

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Italy
    • Calabria
      • Cosenza, Calabria, Italy, 87100
      • Reggio Calabria, Calabria, Italy, 89124
        • Recruiting
        • Grande Ospedale Metropolitano "Bianchi Melacrino Morelli"
        • Contact:
    • Campania
      • Napoli, Campania, Italy, 80131
        • Recruiting
        • Università degli Studi di Napoli Federico II U.O.C. di Ematologia e Trapianti di midollo
        • Contact:
    • Emilia Romagna
      • Bologna, Emilia Romagna, Italy, 40138
        • Recruiting
        • IRCCS Policlinico Sant'Orsola
        • Contact:
    • Emilia-Romagna
      • Ferrara, Emilia-Romagna, Italy, 44124
        • Recruiting
        • Università degli studi di Ferrara - Nuovo Polo Ospedaliero di Cona - A.O.U. Arcispedale S. Anna
        • Contact:
          • Francesco Cavazzini, MD
          • Phone Number: +39 0532236987
          • Email: cvzfnc@unife.it
      • Parma, Emilia-Romagna, Italy, 43126
        • Recruiting
        • Azienda Ospedaliero-Universitaria di Parma
        • Contact:
      • Piacenza, Emilia-Romagna, Italy, 29121
        • Recruiting
        • AUSL di Piacenza - Palazzine Medicine Specialistiche
        • Contact:
      • Ravenna, Emilia-Romagna, Italy, 48121
        • Recruiting
        • Dipartimento Oncoematologico - AUSL della Romagna
        • Contact:
      • Rimini, Emilia-Romagna, Italy, 47923
    • Friuli-Venezia Giulia
      • Udine, Friuli-Venezia Giulia, Italy, 33100
        • Recruiting
        • A.O.U. Integrata di Udine
        • Contact:
    • Lazio
      • Roma, Lazio, Italy, 00161
        • Recruiting
        • A.O.U. Policlinico Umberto I - Università degli Studi di Roma "La Sapienza"
        • Contact:
      • Roma, Lazio, Italy
      • Viterbo, Lazio, Italy, 01100
        • Recruiting
        • Ospedale Belcolle
        • Contact:
    • Liguria
      • Genova, Liguria, Italy, 16132
        • Recruiting
        • IRCCS Azienda Ospedaliera Universitaria "San Martino" - IST
        • Contact:
    • Lombardia
      • Milano, Lombardia, Italy, 20122
        • Recruiting
        • Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico
        • Contact:
      • Monza, Lombardia, Italy, 20900
        • Recruiting
        • Ospedale San Gerardo
        • Contact:
    • Marche
      • Pesaro, Marche, Italy, 61122
    • Piemonte
      • Torino, Piemonte, Italy, 10043
        • Recruiting
        • Ospedale San Luigi Gonzaga - Regione Gonzole
        • Contact:
      • Torino, Piemonte, Italy, 10124
      • Torino, Piemonte, Italy, 10126
      • Torino, Piemonte, Italy, 10128
        • Recruiting
        • A. O. Ordine Mauriziano di Torino
        • Contact:
    • Sardegna
      • Cagliari, Sardegna, Italy, 09131
        • Recruiting
        • Ospedale "A. Businco" - Dipartimento Scienze Mediche e Sanità Pubblica Università degli Studi di Cagliari
        • Contact:
    • Sicilia
      • Catania, Sicilia, Italy, 95124
    • Toscana
      • Siena, Toscana, Italy, 53100
        • Recruiting
        • Policlinico S.Maria alle Scotte
        • Contact:
    • Veneto
      • Padova, Veneto, Italy, 35128
      • Verona, Veneto, Italy, 37134

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

The study is observational multicenter retrospective and prospective cohort will be offered consecutively to any patient with primary or secondary myelofibrosis who has initiated therapy with ruxolitinib, prescribed as part of the normal course of care and completely independent of study participation. The study will be conducted in the study-participating OUs of Hematology, and an estimated total of 1015 patients who initiated therapy with Ruxolitinib between 2008 and the end of the study are expected to participate in this study.

Description

Inclusion Criteria:

  • Age ≥ 18 years
  • Patients diagnosed with Primary Myelofibrosis or secondary to Essential Thrombocythemia/Polycythemia vera who are being treated or have been treated with ruxolitinib therapy in accordance with normal clinical practice.
  • Availability of data on clinical history prior to initiation of Ruxolitinib therapy
  • Obtaining informed consent for data collection and processing

Exclusion Criteria:

  • None

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Long Term Survival
Time Frame: 10 years
To determine the impact of clinical and laboratory characteristics of myelofibrosis on the prognosis of patients treated with ruxolitinib, in terms of long-term survival.
10 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Significance of peripheral blasts
Time Frame: 10 years
To assess correlations between amount of peripheral blasts and response to treatment, leukaemic transformation, drug withdrawal and survival.
10 years
Prognostic value of High Molecular Risk (HMR) mutations.
Time Frame: 10 years
To assess the prognostic value of HMRs (via NGS) in MF patients treated with ruxolitinib and to develop a prognostic model that can be applied to patients before and during ruxolitinib therapy.
10 years
To validate the use of the MTSS score
Time Frame: 10 years
To validate the use of the MTSS score to assess post-ASCT survival of patients with MF
10 years
Incidence of adverse events
Time Frame: 10 years
To assess the incidence of adverse events in patients with myelofibrosis treated with ruxolitinib: thrombosis, haemorrhage, second neoplasms, blastic evolution.
10 years
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Time Frame: 10 years
To assess the toxicity of ruxolitinib therapy in the total cohort
10 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

IRCCS Azienda Ospedaliero-Universitaria di Bologna

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 6, 2022

Primary Completion (Estimated)

May 31, 2032

Study Completion (Estimated)

May 31, 2032

Study Registration Dates

First Submitted

October 5, 2023

First Submitted That Met QC Criteria

July 17, 2024

First Posted (Actual)

July 24, 2024

Study Record Updates

Last Update Posted (Estimated)

December 5, 2024

Last Update Submitted That Met QC Criteria

December 3, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • RUX-MF

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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