Clinical trial NCT02953704: Myelofibrosis and Essential Thrombocythemia Observational Study (MOST)
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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: MPN (Myeloproliferative Neoplasms)
Keywords: Myelofibrosis
essential thrombocythemia
clinical characteristics
disease burden
patient-reported outcomes
Lead sponsor: Incyte Corporation
Sponsor class: INDUSTRY
First posted: 2016-11-03
Enrollment: 1469
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Subjects will be enrolled from up to 250 community and academic centers across the United States.

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).


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.

Primary outcomes:
measure: Description of the clinical characteristics and evolution of disease burden in essential thrombocythemia (ET) and myelofibrosis (MF) patients
timeFrame: Approximately every 6 months through end of study, up to approximately 36 months
Secondary outcomes:
measure: Description of patterns of treatment, therapies, and clinical management
timeFrame: Approximately every 3-6 months through end of study, up to at least 36 months
measure: Description of disease progression over time
timeFrame: Approximately every 3-6 months through end of study, up to at least 36 months
measure: Describe hematocrit, hemoglobin, white blood cell (WBC) count, and platelet counts over time
timeFrame: Approximately every 3-6 months through end of study, up to at least 36 months
measure: Description of the comorbidities associated with disease and progression
timeFrame: Approximately every 3-6 months through end of study, up to at least 36 months
measure: Description of changes in patient-reported symptoms and quality of life (QOL)
timeFrame: Approximately every 3-6 months through end of study, up to at least 36 months
measure: Description of the rate and time to leukemic transformation
timeFrame: Approximately every 3-6 months through end of study, up to at least 36 months
measure: Description of rate of all-cause mortality and aggregate causes of mortality
timeFrame: Approximately every 3-6 months through end of study, up to at least 36 months
measure: Description of reasons for patient ineligibility based on Dynamic International Prognostic Scoring System (DIPSS) during screening (MF patients only)
timeFrame: At screening
measure: Description of time to first disease-related intervention or first progression event during the period of observation (MF patients only)
timeFrame: Baseline to end of study, up to 36 months.
Other outcomes:
Locations:
city: Huntsville
country: United States
state: Alabama
city: Tempe
country: United States
state: Arizona
city: Tucson
country: United States
state: Arizona
city: Little Rock
country: United States
state: Arkansas
city: Berkeley
country: United States
state: California
city: Highland
country: United States
state: California
city: Long Beach
country: United States
state: California
city: Oceanside
country: United States
state: California
city: Palo Alto
country: United States
state: California
city: Santa Rosa
country: United States
state: California
city: Aurora
country: United States
state: Colorado
city: Colorado Springs
country: United States
state: Colorado
city: Fort Collins
country: United States
state: Colorado
city: Middletown
country: United States
state: Connecticut
city: Washington
country: United States
state: District of Columbia
city: Aventura
country: United States
state: Florida
city: Boynton Beach
country: United States
state: Florida
city: Fort Lauderdale
country: United States
state: Florida
city: Jacksonville
country: United States
state: Florida
city: Pembroke Pines
country: United States
state: Florida
city: Tampa
country: United States
state: Florida
city: Winter Haven
country: United States
state: Florida
city: Columbus
country: United States
state: Georgia
city: Marietta
country: United States
state: Georgia
city: Thomasville
country: United States
state: Georgia
city: Boise
country: United States
state: Idaho
city: Chicago
country: United States
state: Illinois
city: Elk Grove Village
country: United States
state: Illinois
city: Harvey
country: United States
state: Illinois
city: Maywood
country: United States
state: Illinois
city: Normal
country: United States
state: Illinois
city: River Forest
country: United States
state: Illinois
city: Skokie
country: United States
state: Illinois
city: Anderson
country: United States
state: Indiana
city: Avon
country: United States
state: Indiana
city: Indianapolis
country: United States
state: Indiana
city: Terre Haute
country: United States
state: Indiana
city: Ames
country: United States
state: Iowa
city: Topeka
country: United States
state: Kansas
city: Westwood
country: United States
state: Kansas
city: Danville
country: United States
state: Kentucky
city: Mount Sterling
country: United States
state: Kentucky
city: Paducah
country: United States
state: Kentucky
city: Alexandria
country: United States
state: Louisiana
city: Covington
country: United States
state: Louisiana
city: Marrero
country: United States
state: Louisiana
city: Rockport
country: United States
state: Maine
city: Baltimore
country: United States
state: Maryland
city: Bethesda
country: United States
state: Maryland
city: Columbia
country: United States
state: Maryland
city: Boston
country: United States
state: Massachusetts
city: Worcester
country: United States
state: Massachusetts
city: Lansing
country: United States
state: Michigan
city: Jackson
country: United States
state: Minnesota
city: Lansing
country: United States
state: Minnesota
city: Bolivar
country: United States
state: Missouri
city: Cape Girardeau
country: United States
state: Missouri
city: Jefferson City
country: United States
state: Missouri
city: Saint Louis
country: United States
state: Missouri
city: Billings
country: United States
state: Montana
city: Kalispell
country: United States
state: Montana
city: Grand Island
country: United States
state: Nebraska
city: Omaha
country: United States
state: Nebraska
city: Las Vegas
country: United States
state: Nevada
city: Lebanon
country: United States
state: New Hampshire
city: Brick
country: United States
state: New Jersey
city: East Orange
country: United States
state: New Jersey
city: Florham Park
country: United States
state: New Jersey
city: Morristown
country: United States
state: New Jersey
city: Paramus
country: United States
state: New Jersey
city: Albany
country: United States
state: New York
city: Brooklyn
country: United States
state: New York
city: East Setauket
country: United States
state: New York
city: Elmira
country: United States
state: New York
city: New York
country: United States
state: New York
city: Rochester
country: United States
state: New York
city: Staten Island
country: United States
state: New York
city: Boone
country: United States
state: North Carolina
city: Charlotte
country: United States
state: North Carolina
city: Durham
country: United States
state: North Carolina
city: Greenville
country: United States
state: North Carolina
city: Hendersonville
country: United States
state: North Carolina
city: Canton
country: United States
state: Ohio
city: Cincinnati
country: United States
state: Ohio
city: Cleveland
country: United States
state: Ohio
city: Toledo
country: United States
state: Ohio
city: Tualatin
country: United States
state: Oregon
city: Bethlehem
country: United States
state: Pennsylvania
city: Danville
country: United States
state: Pennsylvania
city: Dunmore
country: United States
state: Pennsylvania
city: Gettysburg
country: United States
state: Pennsylvania
city: Pittsburgh
country: United States
state: Pennsylvania
city: Willow Grove
country: United States
state: Pennsylvania
city: Lancaster
country: United States
state: South Carolina
city: Spartanburg
country: United States
state: South Carolina
city: Watertown
country: United States
state: South Dakota
city: Jackson
country: United States
state: Tennessee
city: Amarillo
country: United States
state: Texas
city: Arlington
country: United States
state: Texas
city: Beaumont
country: United States
state: Texas
city: Bryan
country: United States
state: Texas
city: Dallas
country: United States
state: Texas
city: Houston
country: United States
state: Texas
city: McAllen
country: United States
state: Texas
city: McKinney
country: United States
state: Texas
city: Mesquite
country: United States
state: Texas
city: Midland
country: United States
state: Texas
city: San Antonio
country: United States
state: Texas
city: Wichita Falls
country: United States
state: Texas
city: Bristol
country: United States
state: Virginia
city: Seattle
country: United States
state: Washington
city: Yakima
country: United States
state: Washington
city: Martinsburg
country: United States
state: West Virginia
city: Milwaukee
country: United States
state: Wisconsin
city: Bayamon
country: Puerto Rico
city: Ponce
country: Puerto Rico
Study officials:
affiliation: Incyte Corporation
name: Robyn M. Scherber, MD, MPH
role: STUDY_DIRECTOR
References:
citation: Gerds AT, Lyons RM, Colucci P, Kalafut P, Paranagama D, Verstovsek S. Disease and Clinical Characteristics of Patients With a Clinical Diagnosis of Myelofibrosis Enrolled in the MOST Study. Clin Lymphoma Myeloma Leuk. 2022 Jul;22(7):e532-e540. doi: 10.1016/j.clml.2022.02.001. Epub 2022 Feb 8.
citation: Yacoub A, Lyons R, Verstovsek S, Shao R, Chu DT, Agrawal A, Sivaraman S, Colucci P, Paranagama D, Mascarenhas J. Disease and Clinical Characteristics of Patients With a Clinical Diagnosis of Essential Thrombocythemia Enrolled in the MOST Study. Clin Lymphoma Myeloma Leuk. 2021 Jul;21(7):461-469. doi: 10.1016/j.clml.2021.02.011. Epub 2021 Mar 1.