Myelofibrosis and Essential Thrombocythemia Observational Study (MOST)

Sponsor

Incyte Corporation (Industry)

Overall Status

Completed

CT.gov ID

NCT02953704

Collaborator

(none)

1,469

Enrollment

116

Locations

62.9

Actual Duration (Months)

12.7

Patients Per Site

0.2

Patients Per Site Per Month

Study Details

Study Description

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).

Condition or Disease	Intervention/Treatment	Phase
MPN (Myeloproliferative Neoplasms)

Study Design

Study Type:

Observational

Actual Enrollment :

1469 participants

Observational Model:

Cohort

Time Perspective:

Prospective

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

Actual Study Start Date :

Dec 31, 2016

Actual Primary Completion Date :

Mar 29, 2022

Actual Study Completion Date :

Mar 29, 2022

Arms and Interventions

Arm	Intervention/Treatment
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.

Outcome Measures

Primary Outcome Measures

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 Outcome Measures

Description of patterns of treatment, therapies, and clinical management [Approximately every 3-6 months through end of study, up to at least 36 months]
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 comorbidities associated with disease and progression [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 the rate and time to leukemic transformation [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 reasons for patient ineligibility based on Dynamic International Prognostic Scoring System (DIPSS) during screening (MF patients only) [At screening]
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.]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.

Contacts and Locations

Locations

	City	State	Country
1	Huntsville	Alabama	United States
2	Tempe	Arizona	United States
3	Tucson	Arizona	United States
4	Little Rock	Arkansas	United States
5	Berkeley	California	United States
6	Highland	California	United States
7	Long Beach	California	United States
8	Oceanside	California	United States
9	Palo Alto	California	United States
10	Santa Rosa	California	United States
11	Aurora	Colorado	United States
12	Colorado Springs	Colorado	United States
13	Fort Collins	Colorado	United States
14	Middletown	Connecticut	United States
15	Washington	District of Columbia	United States
16	Aventura	Florida	United States
17	Boynton Beach	Florida	United States
18	Fort Lauderdale	Florida	United States
19	Jacksonville	Florida	United States
20	Pembroke Pines	Florida	United States
21	Tampa	Florida	United States
22	Winter Haven	Florida	United States
23	Columbus	Georgia	United States
24	Marietta	Georgia	United States
25	Thomasville	Georgia	United States
26	Boise	Idaho	United States
27	Chicago	Illinois	United States
28	Elk Grove Village	Illinois	United States
29	Harvey	Illinois	United States
30	Maywood	Illinois	United States
31	Normal	Illinois	United States
32	River Forest	Illinois	United States
33	Skokie	Illinois	United States
34	Anderson	Indiana	United States
35	Avon	Indiana	United States
36	Indianapolis	Indiana	United States
37	Terre Haute	Indiana	United States
38	Ames	Iowa	United States
39	Topeka	Kansas	United States
40	Westwood	Kansas	United States
41	Danville	Kentucky	United States
42	Mount Sterling	Kentucky	United States
43	Paducah	Kentucky	United States
44	Alexandria	Louisiana	United States
45	Covington	Louisiana	United States
46	Marrero	Louisiana	United States
47	Rockport	Maine	United States
48	Baltimore	Maryland	United States
49	Bethesda	Maryland	United States
50	Columbia	Maryland	United States
51	Boston	Massachusetts	United States
52	Worcester	Massachusetts	United States
53	Lansing	Michigan	United States
54	Jackson	Minnesota	United States
55	Lansing	Minnesota	United States
56	Bolivar	Missouri	United States
57	Cape Girardeau	Missouri	United States
58	Jefferson City	Missouri	United States
59	Saint Louis	Missouri	United States
60	Billings	Montana	United States
61	Kalispell	Montana	United States
62	Grand Island	Nebraska	United States
63	Omaha	Nebraska	United States
64	Las Vegas	Nevada	United States
65	Lebanon	New Hampshire	United States
66	Brick	New Jersey	United States
67	East Orange	New Jersey	United States
68	Florham Park	New Jersey	United States
69	Morristown	New Jersey	United States
70	Paramus	New Jersey	United States
71	Albany	New York	United States
72	Brooklyn	New York	United States
73	East Setauket	New York	United States
74	Elmira	New York	United States
75	New York	New York	United States
76	Rochester	New York	United States
77	Staten Island	New York	United States
78	Boone	North Carolina	United States
79	Charlotte	North Carolina	United States
80	Durham	North Carolina	United States
81	Greenville	North Carolina	United States
82	Hendersonville	North Carolina	United States
83	Canton	Ohio	United States
84	Cincinnati	Ohio	United States
85	Cleveland	Ohio	United States
86	Toledo	Ohio	United States
87	Tualatin	Oregon	United States
88	Bethlehem	Pennsylvania	United States
89	Danville	Pennsylvania	United States
90	Dunmore	Pennsylvania	United States
91	Gettysburg	Pennsylvania	United States
92	Pittsburgh	Pennsylvania	United States
93	Willow Grove	Pennsylvania	United States
94	Lancaster	South Carolina	United States
95	Spartanburg	South Carolina	United States
96	Watertown	South Dakota	United States
97	Jackson	Tennessee	United States
98	Amarillo	Texas	United States
99	Arlington	Texas	United States
100	Beaumont	Texas	United States
101	Bryan	Texas	United States
102	Dallas	Texas	United States
103	Houston	Texas	United States
104	McAllen	Texas	United States
105	McKinney	Texas	United States
106	Mesquite	Texas	United States
107	Midland	Texas	United States
108	San Antonio	Texas	United States
109	Wichita Falls	Texas	United States
110	Bristol	Virginia	United States
111	Seattle	Washington	United States
112	Yakima	Washington	United States
113	Martinsburg	West Virginia	United States
114	Milwaukee	Wisconsin	United States
115	Bayamon		Puerto Rico
116	Ponce		Puerto Rico