Myelofibrosis and Essential Thrombocythemia Observational Study (MOST)

MPN (Myeloproliferative Neoplasms) Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02953704
• Other study ID #: INCB-MA-MF-401
• Status: Completed
• Start date: December 31, 2016
• Est. completion date: March 29, 2022

Study information

• Verified date: May 2022
• Source: Incyte Corporation
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 1469
• Est. completion date: March 29, 2022
• Est. primary completion date: March 29, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• MPN (Myeloproliferative Neoplasms)
• Myeloproliferative Disorders
• Primary Myelofibrosis
• Thrombocythemia, Essential
• Thrombocytosis

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Incyte Corporation

Countries where clinical trial is conducted

United States,  Puerto Rico, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Description of the clinical characteristics and evolution of disease burden in essential thrombocythemia (ET) and myelofibrosis (MF) patients		Approximately every 6 months through end of study, up to approximately 36 months
Secondary	Description of patterns of treatment, therapies, and clinical management		Approximately every 3-6 months through end of study, up to at least 36 months
Secondary	Description of disease progression over time		Approximately every 3-6 months through end of study, up to at least 36 months
Secondary	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
Secondary	Description of the comorbidities associated with disease and progression		Approximately every 3-6 months through end of study, up to at least 36 months
Secondary	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
Secondary	Description of the rate and time to leukemic transformation		Approximately every 3-6 months through end of study, up to at least 36 months
Secondary	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
Secondary	Description of reasons for patient ineligibility based on Dynamic International Prognostic Scoring System (DIPSS) during screening (MF patients only)		At screening
Secondary	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.