Myelofibrosis and Essential Thrombocythemia Observational Study (MOST)

• ClinicalTrials.gov Identifier: NCT02953704
• Recruitment Status: Completed
• First Posted: November 3, 2016
• Last Update Posted: May 13, 2022
• Sponsor: Incyte Corporation
• Information provided by (Responsible Party): Incyte Corporation

Tracking Information

• First Submitted Date: November 1, 2016
• First Posted Date: November 3, 2016
• Last Update Posted Date: May 13, 2022
• Actual Study Start Date: December 31, 2016
• Actual Primary Completion Date: March 29, 2022 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: November 1, 2016): 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 ]
• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: November 1, 2016)

• Description of patterns of treatment, therapies, and clinical management [ Time Frame: Approximately every 3-6 months through end of study, up to at least 36 months ]
• Description of disease progression over time [ Time Frame: 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 [ Time Frame: Approximately every 3-6 months through end of study, up to at least 36 months ]
• Description of the comorbidities associated with disease and progression [ Time Frame: 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) [ Time Frame: Approximately every 3-6 months through end of study, up to at least 36 months ]
• Description of the rate and time to leukemic transformation [ Time Frame: 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 [ Time Frame: 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) [ Time Frame: At screening ]
• Description of time to first disease-related intervention or first progression event during the period of observation (MF patients only) [ Time Frame: Baseline to end of study, up to 36 months. ]

• 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: 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
• 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).
• Detailed Description: Not Provided
• Study Type: Observational
• Study Design: Observational Model: Cohort; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• Sampling Method: Non-Probability Sample
• Study Population: Subjects will be enrolled from up to 250 community and academic centers across the United States.
• Condition: MPN (Myeloproliferative Neoplasms)
• Intervention: Not Provided

Study Groups/Cohorts

• Myelofibrosis Cohort
  Patients will be categorized as low-risk using Dynamic International Prognostic Scoring System (DIPSS) risk OR intermediate-1 risk by DIPSS by reason of age alone.
• Essential Thrombocythemia Cohort
  Patients will be age ≥ 60 years OR have history of thromboembolic events OR currently receiving ET-directed therapy.

• Publications *: 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.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: May 11, 2022): 1469
• Original Estimated Enrollment (submitted: November 1, 2016): 1500
• Actual Study Completion Date: March 29, 2022
• Actual Primary Completion Date: March 29, 2022 (Final data collection date for primary outcome measure)

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Puerto Rico, United States

Administrative Information

• NCT Number: NCT02953704
• Other Study ID Numbers: INCB-MA-MF-401
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided

IPD Sharing Statement

• Plan to Share IPD: Undecided

• Responsible Party: Incyte Corporation
• Study Sponsor: Incyte Corporation
• Collaborators: Not Provided

Investigators

• Study Director: Robyn M. Scherber, MD, MPH; Incyte Corporation

• PRS Account: Incyte Corporation
• Verification Date: May 2022