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Ruxolitinib in Myelofibrosis Patients in Lombardy, Italy

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ClinicalTrials.gov Identifier: NCT03959371
Recruitment Status : Recruiting
First Posted : May 22, 2019
Last Update Posted : May 22, 2019
Sponsor:
Information provided by (Responsible Party):
Margherita Maffioli, Ospedale di Circolo - Fondazione Macchi

Brief Summary:
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.

Condition or disease Intervention/treatment
Myelofibrosis Drug: Ruxolitinib

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Study Type : Observational
Estimated Enrollment : 620 participants
Observational Model: Cohort
Time Perspective: Other
Official Title: Observational, Retrospective and Prospective Study on the Use of Ruxolitinib in Myelofibrosis Patients in Lombardy, Italy
Actual Study Start Date : April 11, 2017
Estimated Primary Completion Date : December 31, 2021
Estimated Study Completion Date : December 31, 2021



Intervention Details:
  • Drug: Ruxolitinib
    Observational study including patients with myelofibrosis being treated with ruxolitinib in a "real world" setting. Patients are treated according to current indications.


Primary Outcome Measures :
  1. Rate of infectious events after ruxolitinib exposure in myelofibrosis patients [ Time Frame: Through study completion, an average of 1 year ]
  2. Rate of vascular events after ruxolitinib exposure in myelofibrosis patients [ Time Frame: Through study completion, an average of 1 year ]

Secondary Outcome Measures :
  1. Spleen response rate [ Time Frame: At 3 and 6 months from ruxolitinib start ]
  2. Rate of primary secondary malignancies [ Time Frame: Through study completion, an average of 1 year ]
  3. Acute myeloid leukemia transformation rate [ Time Frame: Through study completion, an average of 1 year ]
  4. 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

  5. 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

  6. 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

  7. 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

  8. 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

  9. 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

  10. 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

  11. 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

  12. 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

  13. 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

  14. Evaluation of overall survival after ruxolitinib start and, if applicable, discontinuation [ Time Frame: Through study completion, an average of 1 year ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03959371


Contacts
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Contact: Margherita Maffioli, MD +39-0332-278281 margherita.maffioli@asst-settelaghi.it
Contact: Francesco Passamonti, MD +39-0332-393648 francesco.passamonti@asst-settelaghi.it

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

Publications:

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Responsible Party: Margherita Maffioli, Principal Investigator, Ospedale di Circolo - Fondazione Macchi
ClinicalTrials.gov Identifier: NCT03959371     History of Changes
Other Study ID Numbers: RUXOREL-MF
First Posted: May 22, 2019    Key Record Dates
Last Update Posted: May 22, 2019
Last Verified: May 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Margherita Maffioli, Ospedale di Circolo - Fondazione Macchi:
Myelofibrosis
Ruxolitinib
Real world data

Additional relevant MeSH terms:
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Primary Myelofibrosis
Myeloproliferative Disorders
Bone Marrow Diseases
Hematologic Diseases