Momelotinib in Transfusion-Dependent Adults With Primary Myelofibrosis (PMF) or Post-polycythemia Vera or Post-essential Thrombocythemia Myelofibrosis (Post-PV/ET MF)
Study Details
Study Description
Brief Summary
This study will evaluate the transfusion independence response rate in transfusion-dependent adults with myelofibrosis after treatment with momelotinib (MMB).
Condition or Disease | Intervention/Treatment | Phase |
---|---|---|
Phase 2 |
Study Design
Arms and Interventions
Arm | Intervention/Treatment |
---|---|
Experimental: Momelotinib MMB for 24 weeks (± 7 days) |
Drug: MMB
Momelotinib (MMB) tablet administered orally once daily
Other Names:
|
Outcome Measures
Primary Outcome Measures
- Transfusion Independence Response by Week 24 [From baseline to 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.
Secondary Outcome Measures
- Transfusion Response Rate by Week 24 [From baseline to 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.
- Splenic Response Rate at Week 24 [Measured at Week 24]
The percentage of subjects who achieved a ≥ 35% reduction in spleen volume from baseline as measured by MRI at Week 24.
- Response Rate in Total Symptom Score (TSS) at Week 24 [Measured 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.
- Change in Markers of Iron Metabolism and Anemia - Change From Baseline in Hepcidin Daily Change [At baseline, Day 1, Weeks 2, 4, 8, 12, 16, 20 and 24]
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.
- Change in Markers of Iron Metabolism and Anemia - Trough Hepcidin [At baseline, Day 1, Weeks 2, 4, 8, 12, 16, 20 and 24]
Median hepcidin at trough was assessed predose at each study visit.
- Change in Markers of Iron Metabolism and Anemia - Serum Iron [At Weeks 2, 4, 8, 12, 16, 20 and 24]
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).
- Change in Markers of Iron Metabolism and Anemia - Hemoglobin [At Weeks 2, 4, 8, 12, 16, 20 and 24]
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).
- Change in Markers of Iron Metabolism and Anemia - Total Iron Binding Capacity [At Weeks 2, 4, 8, 12, 16, 20 and 24]
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).
- Change in Markers of Iron Metabolism and Anemia - Reticulocytes [At Weeks 2, 4, 8, 12, 16, 20 and 24]
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).
- Change in Markers of Iron Metabolism and Anemia - Reticulocytes/Erythrocytes% [At Weeks 2, 4, 8, 12, 16, 20 and 24]
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).
- Change in Markers of Iron Metabolism and Anemia - Erythropoietin [At Weeks 8 and 20]
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).
- Change in Markers of Iron Metabolism and Anemia - Erythrocytes [At Weeks 2, 4, 8, 12, 16, 20 and 24]
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).
- Change in Markers of Iron Metabolism and Anemia - Hematocrit [At Weeks 2, 4, 8, 12, 16, 20 and 24]
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).
- Change in Markers of Iron Metabolism and Anemia - Ferritin [At Weeks 2, 4, 8, 12, 16, 20 and 24]
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).
- Change in Markers of Iron Metabolism and Anemia - Soluble Transferrin Receptor [At Weeks 2, 4, 8, 12, 16, 20 and 24]
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).
- Change in Markers of Iron Metabolism and Anemia - Transferrin Saturation [At Weeks 2, 4, 8, 12, 16, 20 and 24]
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).
- Change in Markers of Iron Metabolism and Anemia - Unsaturated Iron Binding Capacity [At Weeks 2, 4, 8, 12, 16, 20 and 24]
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).
- Change in Markers of Iron Metabolism and Anemia - Platelets [At Weeks 2, 4, 8, 12, 16, 20 and 24]
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).
- Change in Markers of Iron Metabolism and Anemia - Leukocytes [At Weeks 2, 4, 8, 12, 16, 20 and 24]
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).
- Change in Markers of Iron Metabolism and Anemia - Blasts [At Weeks 2 and 4]
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).
- Change in Liver Iron Content [Measured at Week 24]
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).
- Change in Pharmacodynamics Biomarker - pSTAT3 [On Day 1 and at Weeks 4 and 24]
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).
- Change in Pharmacodynamics Biomarker - pSTAT3/tSTAT3 Ratio [On Day 1 and at Weeks 4 and 24]
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).
- Change in Inflammatory Markers - C-Reactive Protein (CRP) [At Weeks 2, 12 and 24]
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).
Eligibility Criteria
Criteria
Key Inclusion Criteria:
-
Diagnosis of PMF or Post PV/ET-MF
-
Requires myelofibrosis therapy, in the opinion of the investigator
-
High risk OR intermediate-2 risk defined by dynamic international prognostic scoring system (DIPSS) OR intermediate-1 risk defined by DIPSS and associated with symptomatic splenomegaly and/or hepatomegaly
-
Transfusion dependent at baseline, defined as ≥ 4 U red blood cell (RBC) transfusion in the 8 weeks prior to first dose of MMB
-
Acceptable organ function as evidenced by the following:
-
Platelet Count ≥ 50 x 10^9/L
-
Aspartate aminotransferase (AST/SGOT) and alanine aminotransferase (ALT/SGPT) ≤ 3 x upper limit of normal (ULN) or AST or ALT ≤ 5 x ULN if liver is involved by disease process as judged by the investigator
-
Serum creatinine ≤ 2.0 mg/dL or calculated creatinine clearance of ≥ 60 mL/min
-
Direct bilirubin ≤ 2.0 x ULN
-
Life expectancy of > 24 weeks
-
Males and females of childbearing potential who engage in heterosexual intercourse must agree to use protocol specified method(s) of contraception
-
Lactating females must agree to discontinue nursing before MMB administration
-
Able to understand and willing to sign the informed consent form
Key Exclusion Criteria:
-
Prior splenectomy
-
Splenic irradiation within 3 months prior to the first dose of MMB
-
Prior treatment with MMB
-
Known positive status of human immunodeficiency virus (HIV)
-
Chronic active or acute viral hepatitis A, B, or C infection (testing required for hepatitis B and C), or hepatitis B or C carrier
-
Use of strong cytochrome P450 3A4 (CYP3A4) inducer within 2 weeks prior to the first dose of MMB
-
Uncontrolled intercurrent illness per protocol
-
Treatment with a Janus kinase (JAK) inhibitor within 21 days of the planned first dose of MMB
-
Presence of peripheral neuropathy ≥ Common Terminology Criteria for Adverse Events (CTCAE) Grade 2
-
Unwilling or unable to undergo a MRI per requirements in the study protocol
-
Unwilling to consent to genomics sampling
Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Contacts and Locations
Locations
Site | City | State | Country | Postal Code | |
---|---|---|---|---|---|
1 | Phoenix | Arizona | United States | ||
2 | Los Angeles | California | United States | ||
3 | Orange | California | United States | ||
4 | Jacksonville | Florida | United States | ||
5 | Baltimore | Maryland | United States | ||
6 | Ann Arbor | Michigan | United States | ||
7 | Saint Louis | Missouri | United States | ||
8 | Bronx | New York | United States | ||
9 | New York | New York | United States | ||
10 | Durham | North Carolina | United States | ||
11 | Cleveland | Ohio | United States | ||
12 | Houston | Texas | United States | ||
13 | Toronto | Ontario | Canada |
Sponsors and Collaborators
- Sierra Oncology, Inc.
Investigators
- Study Director: Gilead Study Director, Gilead Sciences
Study Documents (Full-Text)
More Information
Publications
None provided.- GS-US-352-1672
Study Results
Participant Flow
Recruitment Details | This study enrolled transfusion-dependent myelofibrosis patients at oncology centers in the United States and Canada. Following completion of the 24-week study period, patients who were benefiting had the option to continue maintenance momelotinib treatment in an open-label extension study (NCT02124746). |
---|---|
Pre-assignment Detail |
Arm/Group Title | Momelotinib (MMB) |
---|---|
Arm/Group Description | Subjects received oral MMB at a starting dose of 200 mg once daily for 24 weeks (± 7 days) on study. |
Period Title: Overall Study | |
STARTED | 41 |
COMPLETED | 25 |
NOT COMPLETED | 16 |
Baseline Characteristics
Arm/Group Title | Transfusion Independence Responders | Transfusion Independence Non-Responders | Total |
---|---|---|---|
Arm/Group Description | Subjects who became transfusion independent by Week 24 | Subjects who did not become transfusion independent by Week 24 | Total of all reporting groups |
Overall Participants | 14 | 27 | 41 |
Age (years) [Mean (Standard Deviation) ] | |||
Mean (Standard Deviation) [years] |
65
(10.0)
|
72
(7.5)
|
70
(9.0)
|
Sex: Female, Male (Count of Participants) | |||
Female |
6
42.9%
|
9
33.3%
|
15
36.6%
|
Male |
8
57.1%
|
18
66.7%
|
26
63.4%
|
Race/Ethnicity, Customized (Count of Participants) | |||
Black |
1
7.1%
|
2
7.4%
|
3
7.3%
|
White |
11
78.6%
|
25
92.6%
|
36
87.8%
|
Not Permitted |
1
7.1%
|
0
0%
|
1
2.4%
|
Other |
1
7.1%
|
0
0%
|
1
2.4%
|
Spleen Volume (cm^3) [Mean (Standard Deviation) ] | |||
Mean (Standard Deviation) [cm^3] |
2132.2
(1439.5)
|
2018.1
(1248.5)
|
2057.1
(1299.8)
|
Total Symptom Score (units on a scale) [Mean (Standard Deviation) ] | |||
Mean (Standard Deviation) [units on a scale] |
19.00
(16.14)
|
21.73
(13.97)
|
20.73
(14.65)
|
RBC Units Transfused within 8 weeks prior to Enrollment (Units of red blood cell) [Mean (Standard Deviation) ] | |||
Mean (Standard Deviation) [Units of red blood cell] |
5
(1.9)
|
7
(2.2)
|
6
(2.3)
|
Hepcidin (nM) [Median (Inter-Quartile Range) ] | |||
Median (Inter-Quartile Range) [nM] |
22.0
|
42.4
|
36.6
|
Serum iron (ug/dL) [Median (Inter-Quartile Range) ] | |||
Median (Inter-Quartile Range) [ug/dL] |
96.0
|
127.0
|
119.0
|
Hemoglobin (g/dL) [Median (Inter-Quartile Range) ] | |||
Median (Inter-Quartile Range) [g/dL] |
8.5
|
7.8
|
8.0
|
Total iron binding capacity (ug/dL) [Median (Inter-Quartile Range) ] | |||
Median (Inter-Quartile Range) [ug/dL] |
214.5
|
201.0
|
205.0
|
Reticulocytes (cells*10^6/uL) [Median (Inter-Quartile Range) ] | |||
Median (Inter-Quartile Range) [cells*10^6/uL] |
0.096
|
0.067
|
0.071
|
Reticulocytes/erythrocytes (%) [Median (Inter-Quartile Range) ] | |||
Median (Inter-Quartile Range) [%] |
3.5
|
2.2
|
2.4
|
Erythropoietin (mIU/mL) [Median (Inter-Quartile Range) ] | |||
Median (Inter-Quartile Range) [mIU/mL] |
238.7
|
88.1
|
109.8
|
Erythrocytes (cells*10^6/uL) [Median (Inter-Quartile Range) ] | |||
Median (Inter-Quartile Range) [cells*10^6/uL] |
3.0
|
2.8
|
2.9
|
Hematocrit (%) [Median (Inter-Quartile Range) ] | |||
Median (Inter-Quartile Range) [%] |
27.5
|
24.0
|
25.0
|
Ferritin (ng/mL) [Median (Inter-Quartile Range) ] | |||
Median (Inter-Quartile Range) [ng/mL] |
605.9
|
1259.4
|
1008.9
|
Soluble transferrin receptor (mg/L) [Median (Inter-Quartile Range) ] | |||
Median (Inter-Quartile Range) [mg/L] |
2.5
|
1.3
|
1.6
|
Transferrin saturation (%) [Median (Inter-Quartile Range) ] | |||
Median (Inter-Quartile Range) [%] |
43.0
|
56.5
|
53.0
|
Unsaturated iron binding capacity (ug/dL) [Median (Inter-Quartile Range) ] | |||
Median (Inter-Quartile Range) [ug/dL] |
113.5
|
81.0
|
92.0
|
Platelets (cells*10^3/uL) [Median (Inter-Quartile Range) ] | |||
Median (Inter-Quartile Range) [cells*10^3/uL] |
190.5
|
134.0
|
146.0
|
Leukocytes (cells*10^3/uL) [Median (Inter-Quartile Range) ] | |||
Median (Inter-Quartile Range) [cells*10^3/uL] |
6.4
|
8.5
|
6.4
|
Blasts (%) [Median (Inter-Quartile Range) ] | |||
Median (Inter-Quartile Range) [%] |
0
|
2.0
|
2.0
|
Liver Iron Content (mg/g) [Median (Inter-Quartile Range) ] | |||
Median (Inter-Quartile Range) [mg/g] |
2.4
|
4.2
|
3.5
|
%pSTAT Stimulated CD3+/4+ T cell (%) [Median (Inter-Quartile Range) ] | |||
Median (Inter-Quartile Range) [%] |
85.1
|
77.8
|
79.8
|
%pSTAT %tSTAT Stimulated CD3+/4+ T cell Ratio (ratio) [Median (Inter-Quartile Range) ] | |||
Median (Inter-Quartile Range) [ratio] |
0.9
|
0.8
|
0.9
|
C-Reactive Protein (mg/dL) [Median (Inter-Quartile Range) ] | |||
Median (Inter-Quartile Range) [mg/dL] |
0.7
|
1.8
|
1.2
|
Outcome Measures
Title | Transfusion Independence Response by Week 24 |
---|---|
Description | 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 |
Outcome Measure Data
Analysis Population Description |
---|
Assessed in the Safety Analysis Set, which includes all subjects who received ≥ 1 dose of momelotinib. Note that 6 subjects discontinued prior to Week 12 and could not be evaluated over the minimum period required for assessment of the endpoint (12 weeks). |
Arm/Group Title | Total |
---|---|
Arm/Group Description | All enrolled subjects |
Measure Participants | 41 |
Number (90% Confidence Interval) [percentage of subjects] |
34.1
|
Title | Transfusion Response Rate by Week 24 |
---|---|
Description | 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 |
Outcome Measure Data
Analysis Population Description |
---|
Assessed in the Safety Analysis Set, which includes all subjects who received ≥ 1 dose of momelotinib. Note that 3 subjects discontinued prior to Week 8 and could not be evaluated over the minimum period required for assessment of the endpoint (8 weeks). |
Arm/Group Title | Total |
---|---|
Arm/Group Description | All enrolled subjects |
Measure Participants | 41 |
Number (90% Confidence Interval) [percentage of subjects] |
39.0
|
Title | Splenic Response Rate at Week 24 |
---|---|
Description | 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 |
Outcome Measure Data
Analysis Population Description |
---|
Assessed in the Safety Analysis Set, which includes all subjects who received ≥ 1 dose of momelotinib. Note that spleen volume at Week 24 was not available for 15 subjects. |
Arm/Group Title | Total |
---|---|
Arm/Group Description | All enrolled subjects |
Measure Participants | 41 |
Number (90% Confidence Interval) [percentage of subjects] |
12.2
|
Title | Response Rate in Total Symptom Score (TSS) at Week 24 |
---|---|
Description | 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 |
Outcome Measure Data
Analysis Population Description |
---|
There were 3 subjects with missing TSS at baseline who were excluded from the analysis. Note that of the 38 subjects evaluated, 17 were missing Week 24 TSS assessments. |
Arm/Group Title | Total |
---|---|
Arm/Group Description | All enrolled subjects |
Measure Participants | 38 |
Number (90% Confidence Interval) [percentage of subjects] |
15.8
|
Title | Change in Markers of Iron Metabolism and Anemia - Change From Baseline in Hepcidin Daily Change |
---|---|
Description | 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 |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Transfusion Independence Responders | Transfusion Independence Non-Responders | Total |
---|---|---|---|
Arm/Group Description | Subjects who became transfusion independent by Week 24 | Subjects who did not become transfusion independent by Week 24 | All enrolled subjects |
Measure Participants | 14 | 27 | 41 |
Baseline |
-1.7
|
0.1
|
0.0
|
Day 1 |
-4.8
|
-11.2
|
-8.6
|
Week 2 |
-4.9
|
-6.7
|
-6.3
|
Week 4 |
-2.6
|
-7.4
|
-5.4
|
Week 8 |
-2.9
|
-5.2
|
-4.4
|
Week 12 |
-1.4
|
-5.7
|
-4.1
|
Week 16 |
-0.4
|
-4.3
|
-2.7
|
Week 20 |
0.0
|
-7.8
|
-5.3
|
Week 24 |
-1.0
|
-1.8
|
-1.4
|
Title | Change in Markers of Iron Metabolism and Anemia - Trough Hepcidin |
---|---|
Description | 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 |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Transfusion Independence Responders | Transfusion Independence Non-Responders | Total |
---|---|---|---|
Arm/Group Description | Subjects who became transfusion independent by Week 24 | Subjects who did not become transfusion independent by Week 24 | All enrolled subjects |
Measure Participants | 14 | 27 | 41 |
Baseline |
22.0
|
42.4
|
36.6
|
Day 1 |
19.7
|
43.7
|
36.5
|
Week 2 |
25.1
|
52.1
|
38.4
|
Week 4 |
19.8
|
46.4
|
37.6
|
Week 8 |
16.6
|
51.7
|
35.5
|
Week 12 |
21.3
|
44.7
|
30.4
|
Week 16 |
16.9
|
51.9
|
34.5
|
Week 20 |
13.4
|
44.2
|
27.5
|
Week 24 |
15.5
|
51.5
|
28.4
|
Title | Change in Markers of Iron Metabolism and Anemia - Serum Iron |
---|---|
Description | 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 |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Transfusion Independence Responders | Transfusion Independence Non-Responders | Total |
---|---|---|---|
Arm/Group Description | Subjects who became transfusion independent by Week 24 | Subjects who did not become transfusion independent by Week 24 | All enrolled subjects |
Measure Participants | 14 | 27 | 41 |
Week 2 |
4.5
|
5.9
|
5.9
|
Week 4 |
39.8
|
5.7
|
13.3
|
Week 8 |
9.3
|
5.4
|
6.4
|
Week 12 |
3.0
|
14.3
|
12.8
|
Week 16 |
-15.5
|
16.5
|
8.9
|
Week 20 |
-11.1
|
9.9
|
1.9
|
Week 24 |
-8.1
|
-5.1
|
-6.6
|
Title | Change in Markers of Iron Metabolism and Anemia - Hemoglobin |
---|---|
Description | 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 |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Transfusion Independence Responders | Transfusion Independence Non-Responders | Total |
---|---|---|---|
Arm/Group Description | Subjects who became transfusion independent by Week 24 | Subjects who did not become transfusion independent by Week 24 | All enrolled subjects |
Measure Participants | 14 | 27 | 41 |
Week 2 |
12.8
|
9.2
|
9.9
|
Week 4 |
8.3
|
6.3
|
6.9
|
Week 8 |
12.4
|
0.4
|
5.6
|
Week 12 |
15.6
|
4.5
|
7.5
|
Week 16 |
12.3
|
0.6
|
10.2
|
Week 20 |
13.1
|
6.9
|
7.4
|
Week 24 |
10.0
|
5.8
|
7.9
|
Title | Change in Markers of Iron Metabolism and Anemia - Total Iron Binding Capacity |
---|---|
Description | 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 |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Transfusion Independence Responders | Transfusion Independence Non-Responders | Total |
---|---|---|---|
Arm/Group Description | Subjects who became transfusion independent by Week 24 | Subjects who did not become transfusion independent by Week 24 | All enrolled subjects |
Measure Participants | 14 | 27 | 41 |
Week 2 |
4.5
|
2.0
|
2.8
|
Week 4 |
6.7
|
1.5
|
2.2
|
Week 8 |
4.9
|
0.6
|
2.4
|
Week 12 |
6.6
|
2.4
|
6.6
|
Week 16 |
4.5
|
1.5
|
3.6
|
Week 20 |
15.5
|
5.5
|
10.6
|
Week 24 |
7.3
|
0.5
|
5.5
|
Title | Change in Markers of Iron Metabolism and Anemia - Reticulocytes |
---|---|
Description | 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 |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Transfusion Independence Responders | Transfusion Independence Non-Responders | Total |
---|---|---|---|
Arm/Group Description | Subjects who became transfusion independent by Week 24 | Subjects who did not become transfusion independent by Week 24 | All enrolled subjects |
Measure Participants | 14 | 27 | 41 |
Week 2 |
33.6
|
-9.6
|
2.9
|
Week 4 |
11.8
|
5.5
|
7.5
|
Week 8 |
30.0
|
-16.0
|
15.6
|
Week 12 |
15.8
|
-9.5
|
4.8
|
Week 16 |
31.2
|
8.8
|
18.3
|
Week 20 |
34.0
|
7.7
|
16.3
|
Week 24 |
29.9
|
-0.6
|
24.1
|
Title | Change in Markers of Iron Metabolism and Anemia - Reticulocytes/Erythrocytes% |
---|---|
Description | 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 |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Transfusion Independence Responders | Transfusion Independence Non-Responders | Total |
---|---|---|---|
Arm/Group Description | Subjects who became transfusion independent by Week 24 | Subjects who did not become transfusion independent by Week 24 | All enrolled subjects |
Measure Participants | 14 | 27 | 41 |
Week 2 |
30.0
|
-16.3
|
-8.0
|
Week 4 |
8.2
|
1.5
|
4.1
|
Week 8 |
12.4
|
-11.1
|
0.0
|
Week 12 |
3.6
|
0.0
|
0.0
|
Week 16 |
4.4
|
18.7
|
7.8
|
Week 20 |
2.2
|
14.6
|
11.1
|
Week 24 |
2.2
|
-7.8
|
-2.6
|
Title | Change in Markers of Iron Metabolism and Anemia - Erythropoietin |
---|---|
Description | 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 |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Transfusion Independence Responders | Transfusion Independence Non-Responders | Total |
---|---|---|---|
Arm/Group Description | Subjects who became transfusion independent by Week 24 | Subjects who did not become transfusion independent by Week 24 | All enrolled subjects |
Measure Participants | 14 | 27 | 41 |
Week 8 |
-30.8
|
-8.1
|
-24.5
|
Week 20 |
-45.0
|
8.5
|
-4.4
|
Title | Change in Markers of Iron Metabolism and Anemia - Erythrocytes |
---|---|
Description | 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 |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Transfusion Independence Responders | Transfusion Independence Non-Responders | Total |
---|---|---|---|
Arm/Group Description | Subjects who became transfusion independent by Week 24 | Subjects who did not become transfusion independent by Week 24 | All enrolled subjects |
Measure Participants | 14 | 27 | 41 |
Week 2 |
5.2
|
3.4
|
3.4
|
Week 4 |
6.7
|
3.6
|
3.8
|
Week 8 |
3.6
|
0.0
|
3.0
|
Week 12 |
3.1
|
3.6
|
3.4
|
Week 16 |
0.0
|
-6.9
|
-2.9
|
Week 20 |
-3.3
|
-3.4
|
-3.4
|
Week 24 |
-8.0
|
3.3
|
0.0
|
Title | Change in Markers of Iron Metabolism and Anemia - Hematocrit |
---|---|
Description | 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 |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Transfusion Independence Responders | Transfusion Independence Non-Responders | Total |
---|---|---|---|
Arm/Group Description | Subjects who became transfusion independent by Week 24 | Subjects who did not become transfusion independent by Week 24 | All enrolled subjects |
Measure Participants | 14 | 27 | 41 |
Week 2 |
8.9
|
4.0
|
4.5
|
Week 4 |
7.4
|
0.0
|
3.4
|
Week 8 |
11.1
|
-1.7
|
4.0
|
Week 12 |
7.1
|
1.8
|
3.6
|
Week 16 |
5.9
|
-4.2
|
3.8
|
Week 20 |
7.1
|
0.0
|
0.0
|
Week 24 |
8.0
|
2.3
|
4.5
|
Title | Change in Markers of Iron Metabolism and Anemia - Ferritin |
---|---|
Description | 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 |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Transfusion Independence Responders | Transfusion Independence Non-Responders | Total |
---|---|---|---|
Arm/Group Description | Subjects who became transfusion independent by Week 24 | Subjects who did not become transfusion independent by Week 24 | All enrolled subjects |
Measure Participants | 14 | 27 | 41 |
Week 2 |
11.6
|
8.8
|
8.8
|
Week 4 |
19.1
|
8.3
|
13.6
|
Week 8 |
4.0
|
30.4
|
16.7
|
Week 12 |
-0.6
|
26.4
|
11.3
|
Week 16 |
0.1
|
21.6
|
6.7
|
Week 20 |
3.5
|
13.6
|
7.0
|
Week 24 |
-14.2
|
15.4
|
1.4
|
Title | Change in Markers of Iron Metabolism and Anemia - Soluble Transferrin Receptor |
---|---|
Description | 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 |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Transfusion Independence Responders | Transfusion Independence Non-Responders | Total |
---|---|---|---|
Arm/Group Description | Subjects who became transfusion independent by Week 24 | Subjects who did not become transfusion independent by Week 24 | All enrolled subjects |
Measure Participants | 14 | 27 | 41 |
Week 2 |
4.0
|
-4.9
|
-4.8
|
Week 4 |
-1.4
|
8.8
|
0.7
|
Week 8 |
6.7
|
11.0
|
7.4
|
Week 12 |
0.8
|
11.1
|
10.7
|
Week 16 |
-0.1
|
14.9
|
1.3
|
Week 20 |
-1.8
|
-0.7
|
-1.2
|
Week 24 |
-3.5
|
19.7
|
-1.8
|
Title | Change in Markers of Iron Metabolism and Anemia - Transferrin Saturation |
---|---|
Description | 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 |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Transfusion Independence Responders | Transfusion Independence Non-Responders | Total |
---|---|---|---|
Arm/Group Description | Subjects who became transfusion independent by Week 24 | Subjects who did not become transfusion independent by Week 24 | All enrolled subjects |
Measure Participants | 14 | 27 | 41 |
Week 2 |
2.4
|
13.5
|
10.3
|
Week 4 |
36.4
|
9.7
|
10.5
|
Week 8 |
9.5
|
-5.0
|
-3.1
|
Week 12 |
11.9
|
16.4
|
14.1
|
Week 16 |
-7.2
|
-13.5
|
-10.4
|
Week 20 |
-2.5
|
-20.8
|
-13.3
|
Week 24 |
-8.3
|
-20.8
|
-11.9
|
Title | Change in Markers of Iron Metabolism and Anemia - Unsaturated Iron Binding Capacity |
---|---|
Description | 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 |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Transfusion Independence Responders | Transfusion Independence Non-Responders | Total |
---|---|---|---|
Arm/Group Description | Subjects who became transfusion independent by Week 24 | Subjects who did not become transfusion independent by Week 24 | All enrolled subjects |
Measure Participants | 14 | 27 | 41 |
Week 2 |
0.0
|
-5.1
|
0.0
|
Week 4 |
-4.0
|
0.0
|
-1.0
|
Week 8 |
6.4
|
0.0
|
0.0
|
Week 12 |
2.9
|
-18.4
|
-1.6
|
Week 16 |
4.3
|
0.0
|
2.6
|
Week 20 |
16.5
|
5.1
|
14.2
|
Week 24 |
12.0
|
6.9
|
12.0
|
Title | Change in Markers of Iron Metabolism and Anemia - Platelets |
---|---|
Description | 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 |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Transfusion Independence Responders | Transfusion Independence Non-Responders | Total |
---|---|---|---|
Arm/Group Description | Subjects who became transfusion independent by Week 24 | Subjects who did not become transfusion independent by Week 24 | All enrolled subjects |
Measure Participants | 14 | 27 | 41 |
Week 2 |
21.4
|
-24.4
|
-9.5
|
Week 4 |
0.0
|
-29.7
|
-26.3
|
Week 8 |
19.7
|
-33.6
|
-12.0
|
Week 12 |
24.1
|
-27.7
|
-11.4
|
Week 16 |
17.8
|
-26.1
|
-6.2
|
Week 20 |
31.0
|
-36.2
|
-14.3
|
Week 24 |
38.9
|
-22.5
|
-15.3
|
Title | Change in Markers of Iron Metabolism and Anemia - Leukocytes |
---|---|
Description | 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 |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Transfusion Independence Responders | Transfusion Independence Non-Responders | Total |
---|---|---|---|
Arm/Group Description | Subjects who became transfusion independent by Week 24 | Subjects who did not become transfusion independent by Week 24 | All enrolled subjects |
Measure Participants | 14 | 27 | 41 |
Week 2 |
11.6
|
-24.9
|
-14.9
|
Week 4 |
6.8
|
-4.7
|
0.7
|
Week 8 |
34.3
|
-0.9
|
8.9
|
Week 12 |
8.0
|
-15.4
|
5.4
|
Week 16 |
42.0
|
-6.7
|
15.2
|
Week 20 |
44.7
|
-27.5
|
16.3
|
Week 24 |
53.0
|
18.3
|
27.1
|
Title | Change in Markers of Iron Metabolism and Anemia - Blasts |
---|---|
Description | 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 |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Transfusion Independence Responders | Transfusion Independence Non-Responders | Total |
---|---|---|---|
Arm/Group Description | Subjects who became transfusion independent by Week 24 | Subjects who did not become transfusion independent by Week 24 | All enrolled subjects |
Measure Participants | 14 | 27 | 41 |
Week 2 |
0
|
3.0
|
3.0
|
Week 4 |
0
|
1.0
|
1.0
|
Title | Change in Liver Iron Content |
---|---|
Description | 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 |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Transfusion Independence Responders | Transfusion Independence Non-Responders | Total |
---|---|---|---|
Arm/Group Description | Subjects who became transfusion independent by Week 24 | Subjects who did not become transfusion independent by Week 24 | All enrolled subjects |
Measure Participants | 14 | 27 | 41 |
Median (Inter-Quartile Range) [% Change from baseline] |
4.7
|
53.3
|
22.3
|
Title | Change in Pharmacodynamics Biomarker - pSTAT3 |
---|---|
Description | 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 |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Transfusion Independence Responders | Transfusion Independence Non-Responders | Total |
---|---|---|---|
Arm/Group Description | Subjects who became transfusion independent by Week 24 | Subjects who did not become transfusion independent by Week 24 | All enrolled subjects |
Measure Participants | 14 | 27 | 41 |
Day 1 - 2 hours postdose |
-15.8
|
-13.5
|
-14.3
|
Day 1 - 4 hours postdose |
-17.1
|
-10.6
|
-12.8
|
Day 1 - 6 hours postdose |
-9.0
|
-4.3
|
-7.1
|
Week 4 - predose |
-0.9
|
-2.9
|
-2.0
|
Week 4 - 2 hours postdose |
-18.9
|
-20.0
|
-20.0
|
Week 4 - 4 hours postdose |
-19.8
|
-14.1
|
-14.1
|
Week 4 - 6 hours postdose |
-12.1
|
-9.1
|
-9.1
|
Week 24 - predose |
-4.6
|
-0.3
|
-2.8
|
Week 24 - 2 hours postdose |
-31.2
|
-10.7
|
-22.7
|
Week 24 - 4 hours postdose |
-30.6
|
-14.4
|
-22.6
|
Week 24 - 6 hours postdose |
-17.0
|
-10.1
|
-13.4
|
Title | Change in Pharmacodynamics Biomarker - pSTAT3/tSTAT3 Ratio |
---|---|
Description | 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 |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Transfusion Independence Responders | Transfusion Independence Non-Responders | Total |
---|---|---|---|
Arm/Group Description | Subjects who became transfusion independent by Week 24 | Subjects who did not become transfusion independent by Week 24 | All enrolled subjects |
Measure Participants | 14 | 27 | 41 |
Day 1 - 2 hours postdose |
-13.2
|
-15.5
|
-13.4
|
Day 1 - 4 hours postdose |
-9.6
|
-9.7
|
-9.7
|
Day 1 - 6 hours postdose |
-9.1
|
-3.7
|
-4.6
|
Week 4 - predose |
0.5
|
5.0
|
4.8
|
Week 4 - 2 hours postdose |
-14.3
|
-9.1
|
-13.2
|
Week 4 - 4 hours postdose |
3.0
|
-13.4
|
-13.4
|
Week 4 - 6 hours postdose |
12.5
|
-6.1
|
-6.1
|
Week 24 - predose |
-5.7
|
-3.5
|
-4.4
|
Week 24 - 2 hours postdose |
-18.7
|
-7.6
|
-16.2
|
Week 24 - 4 hours postdose |
-21.4
|
-11.5
|
-15.4
|
Week 24 - 6 hours postdose |
-18.4
|
-6.5
|
-11.1
|
Title | Change in Inflammatory Markers - C-Reactive Protein (CRP) |
---|---|
Description | 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 |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Transfusion Independence Responders | Transfusion Independence Non-Responders | Total |
---|---|---|---|
Arm/Group Description | Subjects who became transfusion independent by Week 24 | Subjects who did not become transfusion independent by Week 24 | All enrolled subjects |
Measure Participants | 14 | 27 | 41 |
Week 2 |
-68.7
|
-53.7
|
-55.6
|
Week 12 |
-67.8
|
-43.4
|
-48.9
|
Week 24 |
-59.8
|
-47.1
|
-54.8
|
Adverse Events
Time Frame | From the first dose of momelotinib until 30 days following the last dose. | |
---|---|---|
Adverse Event Reporting Description | All adverse events summarized were treatment-emergent, defined as any adverse event with an onset date on or after the momelotinib start date and no later than 30 days after permanent discontinuation of momelotinib or any adverse event leading to premature discontinuation of momelotinib. | |
Arm/Group Title | Momelotinib (MMB) | |
Arm/Group Description | Subjects received oral MMB at a starting dose of 200 mg once daily for 24 weeks (± 7 days) on study. | |
All Cause Mortality |
||
Momelotinib (MMB) | ||
Affected / at Risk (%) | # Events | |
Total | 3/41 (7.3%) | |
Serious Adverse Events |
||
Momelotinib (MMB) | ||
Affected / at Risk (%) | # Events | |
Total | 14/41 (34.1%) | |
Blood and lymphatic system disorders | ||
Anemia | 3/41 (7.3%) | |
Neutropenia | 1/41 (2.4%) | |
Cardiac disorders | ||
Atrial fibrillation | 1/41 (2.4%) | |
Cardiac arrest | 1/41 (2.4%) | |
Gastrointestinal disorders | ||
Colitis | 1/41 (2.4%) | |
Small intestinal obstruction | 1/41 (2.4%) | |
General disorders | ||
Asthenia | 1/41 (2.4%) | |
Death | 1/41 (2.4%) | |
Pyrexia | 1/41 (2.4%) | |
Hepatobiliary disorders | ||
Cholecystitis | 1/41 (2.4%) | |
Infections and infestations | ||
Lung infection | 2/41 (4.9%) | |
Pneumonia | 1/41 (2.4%) | |
Sepsis | 1/41 (2.4%) | |
Injury, poisoning and procedural complications | ||
Transfusion reaction | 1/41 (2.4%) | |
Investigations | ||
Hepatic enzyme increased | 1/41 (2.4%) | |
Musculoskeletal and connective tissue disorders | ||
Bone pain | 1/41 (2.4%) | |
Nervous system disorders | ||
Presyncope | 2/41 (4.9%) | |
Dizziness | 1/41 (2.4%) | |
Metabolic encephalopathy | 1/41 (2.4%) | |
Renal and urinary disorders | ||
Acute kidney injury | 3/41 (7.3%) | |
Respiratory, thoracic and mediastinal disorders | ||
Acute respiratory failure | 1/41 (2.4%) | |
Respiratory failure | 1/41 (2.4%) | |
Vascular disorders | ||
Hypotension | 2/41 (4.9%) | |
Other (Not Including Serious) Adverse Events |
||
Momelotinib (MMB) | ||
Affected / at Risk (%) | # Events | |
Total | 36/41 (87.8%) | |
Blood and lymphatic system disorders | ||
Thrombocytopenia | 7/41 (17.1%) | |
Neutropenia | 5/41 (12.2%) | |
Gastrointestinal disorders | ||
Diarrhoea | 10/41 (24.4%) | |
Nausea | 9/41 (22%) | |
Vomiting | 7/41 (17.1%) | |
Abdominal pain | 6/41 (14.6%) | |
Constipation | 3/41 (7.3%) | |
General disorders | ||
Fatigue | 8/41 (19.5%) | |
Chills | 3/41 (7.3%) | |
Infections and infestations | ||
Urinary tract infection | 6/41 (14.6%) | |
Injury, poisoning and procedural complications | ||
Contusion | 5/41 (12.2%) | |
Fall | 4/41 (9.8%) | |
Investigations | ||
Blood creatinine increased | 3/41 (7.3%) | |
Metabolism and nutrition disorders | ||
Decreased appetite | 4/41 (9.8%) | |
Hyperuricaemia | 4/41 (9.8%) | |
Musculoskeletal and connective tissue disorders | ||
Musculoskeletal pain | 3/41 (7.3%) | |
Nervous system disorders | ||
Dizziness | 6/41 (14.6%) | |
Headache | 6/41 (14.6%) | |
Peripheral sensory neuropathy | 3/41 (7.3%) | |
Renal and urinary disorders | ||
Pollakiuria | 3/41 (7.3%) | |
Respiratory, thoracic and mediastinal disorders | ||
Cough | 12/41 (29.3%) | |
Dyspnoea | 5/41 (12.2%) | |
Nasal congestion | 3/41 (7.3%) | |
Skin and subcutaneous tissue disorders | ||
Pruritus | 7/41 (17.1%) | |
Night sweats | 3/41 (7.3%) | |
Vascular disorders | ||
Hypotension | 3/41 (7.3%) |
Limitations/Caveats
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
Most restrictive: Site may not present or publish results of Trial without advance written notice of Sponsor or two years after completion of Trial at all participating sites. Site must submit all proposed publications or presentations to Sponsor for review. Sponsor can review communications prior to public release and embargo trial results communications for a period between 75 and 180 days from submission to Sponsor for review. Sponsor has right to request Site remove Confidential Information.
Results Point of Contact
Name/Title | Dr. Barbara Klencke |
---|---|
Organization | Sierra Oncology, Inc. |
Phone | 1-604-558-6536 |
bklencke@sierraoncology.com |
- GS-US-352-1672