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出境医 / 临床实验 / Ruxolitinib in Myelofibrosis Patients in Lombardy, Italy
Ruxolitinib in Myelofibrosis Patients in Lombardy, Italy
Study Description
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 |
Study Design
| 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 |
Arms and Interventions
Outcome Measures
Primary Outcome Measures :
- Rate of infectious events after ruxolitinib exposure in myelofibrosis patients [ Time Frame: 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 ]
Secondary Outcome Measures :
- Spleen response rate [ Time Frame: At 3 and 6 months from ruxolitinib start ]
- Rate of primary secondary malignancies [ Time Frame: Through study completion, an average of 1 year ]
- Acute myeloid leukemia transformation rate [ Time Frame: 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- Evaluation of overall survival after ruxolitinib start and, if applicable, discontinuation [ Time Frame: Through study completion, an average of 1 year ]
Eligibility Criteria
Contacts and Locations
| Tracking Information | |||||||||
|---|---|---|---|---|---|---|---|---|---|
| First Submitted Date | April 7, 2019 | ||||||||
| First Posted Date | May 22, 2019 | ||||||||
| Last Update Posted Date | May 22, 2019 | ||||||||
| Actual Study Start Date | April 11, 2017 | ||||||||
| Estimated Primary Completion Date | December 31, 2021 (Final data collection date for primary outcome measure) | ||||||||
| Current Primary Outcome Measures |
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| Original Primary Outcome Measures | Same as current | ||||||||
| Change History | No Changes Posted | ||||||||
| Current Secondary Outcome Measures |
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| Original Secondary Outcome Measures | Same as current | ||||||||
| Current Other Pre-specified Outcome Measures | Not Provided | ||||||||
| Original Other Pre-specified Outcome Measures | Not Provided | ||||||||
| Descriptive Information | |||||||||
| Brief Title | Ruxolitinib in Myelofibrosis Patients in Lombardy, Italy | ||||||||
| Official Title | Observational, Retrospective and Prospective Study on the Use of Ruxolitinib in Myelofibrosis Patients in Lombardy, Italy | ||||||||
| 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. | ||||||||
| Detailed Description | Not Provided | ||||||||
| Study Type | Observational | ||||||||
| Study Design | Observational Model: Cohort Time Perspective: Other |
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| Target Follow-Up Duration | Not Provided | ||||||||
| Biospecimen | Not Provided | ||||||||
| 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. | ||||||||
| Condition | Myelofibrosis | ||||||||
| Intervention | Drug: Ruxolitinib
Observational study including patients with myelofibrosis being treated with ruxolitinib in a "real world" setting. Patients are treated according to current indications.
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| Study Groups/Cohorts | Not Provided | ||||||||
| Publications * |
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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| Recruitment Information | |||||||||
| Recruitment Status | Recruiting | ||||||||
| Estimated Enrollment |
620 | ||||||||
| Original Estimated Enrollment | Same as current | ||||||||
| Estimated Study Completion Date | December 31, 2021 | ||||||||
| Estimated Primary Completion Date | December 31, 2021 (Final data collection date for primary outcome measure) | ||||||||
| Eligibility Criteria |
Inclusion Criteria:
Exclusion Criteria:
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| Sex/Gender |
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| Ages | 18 Years and older (Adult, Older Adult) | ||||||||
| Accepts Healthy Volunteers | No | ||||||||
| Contacts |
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| Listed Location Countries | Italy | ||||||||
| Removed Location Countries | |||||||||
| Administrative Information | |||||||||
| NCT Number | NCT03959371 | ||||||||
| Other Study ID Numbers | RUXOREL-MF | ||||||||
| Has Data Monitoring Committee | No | ||||||||
| U.S. FDA-regulated Product | Not Provided | ||||||||
| IPD Sharing Statement |
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| Responsible Party | Margherita Maffioli, Ospedale di Circolo - Fondazione Macchi | ||||||||
| Study Sponsor | Margherita Maffioli | ||||||||
| Collaborators | Not Provided | ||||||||
| Investigators |
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| PRS Account | Ospedale di Circolo - Fondazione Macchi | ||||||||
| Verification Date | May 2019 | ||||||||
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