This study will evaluate the transfusion independence response rate in transfusion-dependent adults with myelofibrosis after treatment with momelotinib (MMB).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
41
Momelotinib (MMB) tablet administered orally once daily
Unnamed facility
Phoenix, Arizona, United States
Unnamed facility
Los Angeles, California, United States
Unnamed facility
Orange, California, United States
Unnamed facility
Transfusion Independence Response by Week 24
The percentage of subjects who became transfusion independent for ≥ 12 weeks at any time on study. A subject was considered transfusion independent on study if no RBC transfusion occurred in any 12-week period during the 24-week treatment period.
Time frame: From baseline to Week 24
Transfusion Response Rate by Week 24
The percentage of subjects who became transfusion independent for ≥ 8 weeks, defined as no RBC transfusions for at least an 8-week period at any time on study.
Time frame: From baseline to Week 24
Splenic Response Rate at Week 24
The percentage of subjects who achieved a ≥ 35% reduction in spleen volume from baseline as measured by MRI at Week 24.
Time frame: Measured at Week 24
Response Rate in Total Symptom Score (TSS) at Week 24
The percentage of subjects achieving a ≥ 50% reduction from baseline in TSS at Week 24, as measured by the modified Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPNSAF TSS) diary. Total symptom score was assessed using the modified MPN-SAF TSS Version 2, an 8-item questionnaire developed to assess symptom burden and quality of life in patients with MPN. The modified MPN-SAF TSS contained 8 questions, 7 of which were summed to generate the score (the included questions related to tiredness, early satiety, abdominal discomfort, night sweats, itching, bone pain, and pain under the ribs on the left side). Each question is scored on a scale of 0-10, where higher numbers indicate more severe symptoms. For this study, the TSS scale ranges from 0 to 70. The questionnaire was completed daily on an electronic diary device.
Time frame: Measured at Week 24
Change in Markers of Iron Metabolism and Anemia - Change From Baseline in Hepcidin Daily Change
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Jacksonville, Florida, United States
Unnamed facility
Baltimore, Maryland, United States
Unnamed facility
Ann Arbor, Michigan, United States
Unnamed facility
St Louis, Missouri, United States
Unnamed facility
New York, New York, United States
Unnamed facility
The Bronx, New York, United States
Unnamed facility
Durham, North Carolina, United States
...and 3 more locations
Hepcidin daily change (in nM) was calculated as the predose value subtracted from the 6 hours postdose value at each study visit. Daily hepcidin change at the baseline visit was the difference between 2 values obtained 6 hours apart. No momelotinib was administered on that day.
Time frame: At baseline, Day 1, Weeks 2, 4, 8, 12, 16, 20 and 24
Change in Markers of Iron Metabolism and Anemia - Trough Hepcidin
Median hepcidin at trough was assessed predose at each study visit.
Time frame: At baseline, Day 1, Weeks 2, 4, 8, 12, 16, 20 and 24
Change in Markers of Iron Metabolism and Anemia - Serum Iron
Percent change from baseline in serum iron, where the baseline value is defined as the last value from the baseline period prior to or on the date of first dose of momelotinib administration (Baseline visit).
Time frame: At Weeks 2, 4, 8, 12, 16, 20 and 24
Change in Markers of Iron Metabolism and Anemia - Hemoglobin
Percent change from baseline in hemoglobin, where the baseline value is defined as the last value from the baseline period prior to or on the date of first dose of momelotinib administration (Baseline visit).
Time frame: At Weeks 2, 4, 8, 12, 16, 20 and 24
Change in Markers of Iron Metabolism and Anemia - Total Iron Binding Capacity
Percent change from baseline in total iron binding capacity, where the baseline value is defined as the last value from the baseline period prior to or on the date of first dose of momelotinib administration (Baseline visit).
Time frame: At Weeks 2, 4, 8, 12, 16, 20 and 24
Change in Markers of Iron Metabolism and Anemia - Reticulocytes
Percent change from baseline in reticulocytes, where the baseline value is defined as the last value from the baseline period prior to or on the date of first dose of momelotinib administration (Baseline visit).
Time frame: At Weeks 2, 4, 8, 12, 16, 20 and 24
Change in Markers of Iron Metabolism and Anemia - Reticulocytes/Erythrocytes%
Percent change from baseline in reticulocytes/erythrocytes%, where the baseline value is defined as the last value from the baseline period prior to or on the date of first dose of momelotinib administration (Baseline visit).
Time frame: At Weeks 2, 4, 8, 12, 16, 20 and 24
Change in Markers of Iron Metabolism and Anemia - Erythropoietin
Percent change in erythropoietin at Weeks 8 and 20. The baseline erythropoietin value is defined as the last value from the baseline period prior to or on the date of first dose of momelotinib administration (Baseline visit).
Time frame: At Weeks 8 and 20
Change in Markers of Iron Metabolism and Anemia - Erythrocytes
Percent change from baseline in erythrocytes, where the baseline value is defined as the last value from the baseline period prior to or on the date of first dose of momelotinib administration (Baseline visit).
Time frame: At Weeks 2, 4, 8, 12, 16, 20 and 24
Change in Markers of Iron Metabolism and Anemia - Hematocrit
Percent change from baseline in hematocrit, where the baseline value is defined as the last value from the baseline period prior to or on the date of first dose of momelotinib administration (Baseline visit).
Time frame: At Weeks 2, 4, 8, 12, 16, 20 and 24
Change in Markers of Iron Metabolism and Anemia - Ferritin
Percent change from baseline in ferritin, where the baseline value is defined as the last value from the baseline period prior to or on the date of first dose of momelotinib administration (Baseline visit).
Time frame: At Weeks 2, 4, 8, 12, 16, 20 and 24
Change in Markers of Iron Metabolism and Anemia - Soluble Transferrin Receptor
Percent change from baseline in soluble transferrin receptor, where the baseline value is defined as the last value from the baseline period prior to or on the date of first dose of momelotinib administration (Baseline visit).
Time frame: At Weeks 2, 4, 8, 12, 16, 20 and 24
Change in Markers of Iron Metabolism and Anemia - Transferrin Saturation
Percent change from baseline in transferrin saturation, where the baseline value is defined as the last value from the baseline period prior to or on the date of first dose of momelotinib administration (Baseline visit).
Time frame: At Weeks 2, 4, 8, 12, 16, 20 and 24
Change in Markers of Iron Metabolism and Anemia - Unsaturated Iron Binding Capacity
Percent change from baseline in unsaturated iron binding capacity, where the baseline value is defined as the last value from the baseline period prior to or on the date of first dose of momelotinib administration (Baseline visit).
Time frame: At Weeks 2, 4, 8, 12, 16, 20 and 24
Change in Markers of Iron Metabolism and Anemia - Platelets
Percent change from baseline in platelets, where the baseline value is defined as the last value from the baseline period prior to or on the date of first dose of momelotinib administration (Baseline visit).
Time frame: At Weeks 2, 4, 8, 12, 16, 20 and 24
Change in Markers of Iron Metabolism and Anemia - Leukocytes
Percent change from baseline in leukocytes, where the baseline value is defined as the last value from the baseline period prior to or on the date of first dose of momelotinib administration (Baseline visit).
Time frame: At Weeks 2, 4, 8, 12, 16, 20 and 24
Change in Markers of Iron Metabolism and Anemia - Blasts
Change from baseline in % blasts at Weeks 2 and 4. The baseline % blasts value is defined as the last value from the baseline period prior to or on the date of first dose of momelotinib administration (Baseline visit).
Time frame: At Weeks 2 and 4
Change in Liver Iron Content
Percent change from baseline in liver iron content assessed by MRI. The baseline value is defined as the last value from the baseline period prior to or on the date of first dose of momelotinib administration (Baseline visit).
Time frame: Measured at Week 24
Change in Pharmacodynamics Biomarker - pSTAT3
Percent change in %pSTAT stimulated CD3+/4+ T cell at Day 1 (postdose), Week 4 and Week 24. The baseline value is defined as the last predose value from the baseline period prior to or on the date of first dose of momelotinib administration (Day 1 predose).
Time frame: On Day 1 and at Weeks 4 and 24
Change in Pharmacodynamics Biomarker - pSTAT3/tSTAT3 Ratio
Percent change in %pSTAT/%tSTAT Stimulated CD3+/4+ T cell ratio at Day 1 (postdose), Week 4 and Week 24. The baseline value is defined as the last predose value from the baseline period prior to or on the date of first dose of momelotinib administration (Day 1 predose).
Time frame: On Day 1 and at Weeks 4 and 24
Change in Inflammatory Markers - C-Reactive Protein (CRP)
Percent change in C-reactive protein at Weeks 2, 12 and 24. The baseline C-reactive protein value is defined as the last value from the baseline period prior to or on the date of first dose of momelotinib administration (Baseline visit).
Time frame: At Weeks 2, 12 and 24