Ruxolitinib in Myelofibrosis Patients in Lombardy, Italy

May 18, 2019 updated by: Margherita Maffioli

Observational, Retrospective and Prospective Study on the Use of Ruxolitinib in Myelofibrosis Patients in Lombardy, Italy

The RUXOREL-MF observational study includes patients with primary and post-essential thrombocythemia/post-polycythemia vera myelofibrosis (MF) being treated with the oral JAK1-/JAK2-inhibitor ruxolitinib in a "real world" setting. Patients are treated according to current indications in Italy (i.e., primary and secondary MF patients with intermediate-1, intermediate-2, and high risk IPSS (International Prognostic Scoring System) scores and symptomatic splenomegaly and/or systemic symptoms). Patients are treated at facilities pertaining to the regional Hematology Network of Lombardy (Rete Ematologica Lombarda) in Italy. Efficacy data, data related to infectious and vascular events, data related to second primary malignancies, data regarding disease progression/transformation, and molecular information in relationship to ruxolitinib treatment will be collected and analyzed.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Anticipated)

620

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
      • Bergamo, Italy
        • Recruiting
        • ASST Papa Giovanni XXIII
        • Contact:
          • Maria Chiara Finazzi, MD
      • Brescia, Italy
        • Recruiting
        • U.O. Ematologia, ASST Spedali Civili
        • Contact:
          • Mariella D'Adda, MD
      • Brescia, Italy
        • Recruiting
        • U.S.D. Trapianti di Midollo Osseo, ASST Spedali Civili
        • Contact:
          • Nicola Polverelli, MD
      • Lecco, Italy
        • Recruiting
        • U.O.C. di Ematologia Clinica, ASST Lecco
        • Contact:
          • Michela Anghileri, MD
      • Milan, Italy
        • Recruiting
        • U.O. Ematologia, ASST Fatebenefratelli-Sacco
        • Contact:
          • Maria Cristina Carraro, MD
      • Milan, Italy
        • Recruiting
        • U.O. Ematologia, Fondazione IRCCS Istituto Nazionale Tumori
        • Contact:
          • Francesco Spina, MD
      • Milan, Italy
        • Recruiting
        • U.O. Ematologia, Grande Ospedale Metropolitano Niguarda
        • Contact:
          • Marianna Caramella, MD
      • Milan, Italy
        • Recruiting
        • U.O. Ematologia, Humanicas Cancer Center
        • Contact:
          • Marianna Rossi, MD
      • Milan, Italy
        • Recruiting
        • U.O. Ematologia, Ospedale San Raffaele
        • Contact:
          • Simona Malato, MD
      • Milan, Italy
        • Recruiting
        • U.O. Oncoematologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
        • Contact:
          • Alessandra Iurlo, MD
      • Monza, Italy
        • Recruiting
        • Clinica Ematologica, Ospedale San Gerardo
        • Contact:
          • Elena Elli, MD
      • Varese, Italy, 21100
        • Recruiting
        • Ospedale di Circolo, ASST Sette Laghi
        • Contact:
          • Margherita Maffioli, MD

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

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Patients with primary myelofibrosis diagnosis according to the WHO (World Health Organization) 2016 classification or post-essential thrombocythemia/post-polycythemia vera myelofibrosis diagnosis according to the IWG-MRT (International Working Group for Myelofibrosis Research and Treatment) 2008 classification, with an intermediate-1, intermediate-2, or high risk score according to the IPSS (International Prognostic Scoring System), treated with ruxolitinib in accordance with current indications in Italy and pertaining to centers of the regional Hematology Network of Lombardy (Rete Ematologica Lombarda), Italy.

Description

Inclusion Criteria:

  • Age >= 18 years
  • Diagnosis of primary myelofibrosis diagnosis according to the WHO 2016 classification or post-essential thrombocythemia/post-polycythemia vera myelofibrosis according to the IWG-MRT 2008 classification
  • Patients with an intermediate-1, intermediate-2, or high risk score according to the IPSS (International Prognostic Scoring System)
  • Patients treated with ruxolitinib in accordance with current indications in Italy
  • Patients eligible or ineligible to hematopoietic stem cell transplant or who have already undergone a hematopoietic stem cell transplant

Exclusion Criteria:

  • Diagnoses other than primary myelofibrosis or post-essential thrombocythemia/post-polycythemia vera myelofibrosis
  • Patients treated with ruxolitinib having a platelet count at treatment initiation <50 x10^9/L
  • Patients treated with ruxolitinib for conditions other than primary myelofibrosis or post-essential thrombocythemia/post-polycythemia vera myelofibrosis

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

  • Observational Models: Cohort
  • Time Perspectives: Other

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Rate of infectious events after ruxolitinib exposure in myelofibrosis patients
Time Frame: Through study completion, an average of 1 year
Through study completion, an average of 1 year
Rate of vascular events after ruxolitinib exposure in myelofibrosis patients
Time Frame: Through study completion, an average of 1 year
Through study completion, an average of 1 year

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Spleen response rate
Time Frame: At 3 and 6 months from ruxolitinib start
At 3 and 6 months from ruxolitinib start
Rate of primary secondary malignancies
Time Frame: Through study completion, an average of 1 year
Through study completion, an average of 1 year
Acute myeloid leukemia transformation rate
Time Frame: Through study completion, an average of 1 year
Through study completion, an average of 1 year
Rate of infectious events according to driver mutational status (i.e., mutations of JAK2, CALR, or MPL)
Time Frame: Through study completion, an average of 1 year
Association of rate of infectious events with driver mutational status
Through study completion, an average of 1 year
Rate of vascular events according to driver mutational status (i.e., mutations of JAK2, CALR, or MPL)
Time Frame: Through study completion, an average of 1 year
Association of rate of vascular events with driver mutational status
Through study completion, an average of 1 year
Spleen response rate according to driver mutational status (i.e., mutations of JAK2, CALR, or MPL)
Time Frame: Through study completion, an average of 1 year
Association of spleen response rate with driver mutational status
Through study completion, an average of 1 year
Rate of primary secondary malignancies according to driver mutational status (i.e., mutations of JAK2, CALR, or MPL)
Time Frame: Through study completion, an average of 1 year
Association of rate of primary secondary malignancies with driver mutational status
Through study completion, an average of 1 year
Acute myeloid leukemia transformation rate according to driver mutational status (i.e., mutations of JAK2, CALR, or MPL)
Time Frame: Through study completion, an average of 1 year
Association of acute myeloid leukemia transformation rate with driver mutational status
Through study completion, an average of 1 year
Rate of infectious events according to the presence of additional mutations
Time Frame: Through study completion, an average of 1 year
Association of rate of infectious events with the presence of additional mutations
Through study completion, an average of 1 year
Rate of vascular events according to the presence of additional mutations
Time Frame: Through study completion, an average of 1 year
Association of rate of vascular events with the presence of additional mutations
Through study completion, an average of 1 year
Spleen response rate according to the presence of additional mutations
Time Frame: Through study completion, an average of 1 year
Association of spleen response rate with the presence of additional mutations
Through study completion, an average of 1 year
Rate of primary secondary malignancies according to the presence of additional mutations
Time Frame: Through study completion, an average of 1 year
Association of rate of primary secondary malignancies with the presence of additional mutations
Through study completion, an average of 1 year
Acute myeloid leukemia transformation rate according to the presence of additional mutations
Time Frame: Through study completion, an average of 1 year
Association of acute myeloid leukemia transformation rate with the presence of additional mutations
Through study completion, an average of 1 year
Evaluation of overall survival after ruxolitinib start and, if applicable, discontinuation
Time Frame: Through study completion, an average of 1 year
Through study completion, an average of 1 year

Collaborators and Investigators

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

Sponsor

Margherita Maffioli

Investigators

  • Principal Investigator: Francesco Passamonti, MD, Ospedale di Circolo ASST Sette Laghi, Università dell'Insubria, Varese, Italy

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

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)

April 11, 2017

Primary Completion (Anticipated)

December 31, 2021

Study Completion (Anticipated)

December 31, 2021

Study Registration Dates

First Submitted

April 7, 2019

First Submitted That Met QC Criteria

May 18, 2019

First Posted (Actual)

May 22, 2019

Study Record Updates

Last Update Posted (Actual)

May 22, 2019

Last Update Submitted That Met QC Criteria

May 18, 2019

Last Verified

May 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • RUXOREL-MF

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

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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