Prospective, Longitudinal, Non-Interventional Study of Disease Burden and Treatment of Patients With Low-Risk Myelofibrosis (MF) or High-Risk Essential Thrombocythemia (ET) or ET Patients Receiving ET-Directed Therapy

Incyte Corporation0 sites1,469 target enrollmentDecember 31, 2016

ConditionsMPN (Myeloproliferative Neoplasms)

Overview

Phase: N/A
Intervention: Not specified
Conditions: MPN (Myeloproliferative Neoplasms)
Sponsor: Incyte Corporation
Enrollment: 1469
Primary Endpoint: Description of the clinical characteristics and evolution of disease burden in essential thrombocythemia (ET) and myelofibrosis (MF) patients
Status: Completed
Last Updated: 3 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials Related News

Overview

Brief Summary

The purpose of this prospective, longitudinal, noninterventional study is to describe clinical characteristics, evolution of disease burden, and treatment patterns in patients with select subcategories of essential thrombocythemia (ET) or myelofibrosis (MF).

Registry

clinicaltrials.gov

Start Date

December 31, 2016

End Date

March 29, 2022

Last Updated

3 years ago

Study Type

Observational

Sex

All

Investigators

Sponsor

Incyte Corporation

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•MF cohort: Diagnosis of MF and low-risk using DIPSS risk categorization OR intermediate-1 risk by DIPSS by reason of age alone.
•ET cohort: Diagnosis of ET and age ≥ 60 years OR history of thromboembolic events OR currently receiving ET-directed therapy (eg, hydroxyurea, anagrelide, interferon, busulfan, ruxolitinib, etc).
•Willing and able to provide written informed consent.
•Willing and able to complete patient assessment questionnaires either alone or with minimal assistance from a caregiver and/or trained site personnel.
•Under the supervision of a physician for the current care of MF or ET.

Exclusion Criteria

•Individuals who are participating in blinded investigational drug studies.
•Individuals who are participating in Incyte investigational/interventional drug trials (company- or investigator-sponsored studies) until they have completed the 30-day end of study visit.
•Life expectancy ≤ 6 months.
•Diagnosis of secondary acute myeloid leukemia, myelodysplastic syndrome, chronic myelogenous leukemia, or secondary thrombocytosis.

Outcomes

Primary Outcomes

Description of the clinical characteristics and evolution of disease burden in essential thrombocythemia (ET) and myelofibrosis (MF) patients

Time Frame: Approximately every 6 months through end of study, up to approximately 36 months

Secondary Outcomes

Description of disease progression over time(Approximately every 3-6 months through end of study, up to at least 36 months)
Describe hematocrit, hemoglobin, white blood cell (WBC) count, and platelet counts over time(Approximately every 3-6 months through end of study, up to at least 36 months)
Description of the rate and time to leukemic transformation(Approximately every 3-6 months through end of study, up to at least 36 months)
Description of changes in patient-reported symptoms and quality of life (QOL)(Approximately every 3-6 months through end of study, up to at least 36 months)
Description of reasons for patient ineligibility based on Dynamic International Prognostic Scoring System (DIPSS) during screening (MF patients only)(At screening)
Description of the comorbidities associated with disease and progression(Approximately every 3-6 months through end of study, up to at least 36 months)
Description of rate of all-cause mortality and aggregate causes of mortality(Approximately every 3-6 months through end of study, up to at least 36 months)
Description of time to first disease-related intervention or first progression event during the period of observation (MF patients only)(Baseline to end of study, up to 36 months.)
Description of patterns of treatment, therapies, and clinical management(Approximately every 3-6 months through end of study, up to at least 36 months)