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To evaluate the effects of treatment with ruxolitinib (INCB018424) on spleen volume, symptoms and potential side effects in participants with PMF, PPV-MF and PET-MF who have platelet counts of 50 x 10^9/L to 100 x 10^9/L. It is anticipated that individualized dose optimization from the starting ruxolitinib level of 5 mg bid will be associated with reductions in splenomegaly, MF-associated symptoms and inflammatory cytokine levels.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ruxolitinib 5 mg | Experimental | Participants began administration with 5 mg ruxolitinib twice daily (BID) orally. Beginning at the Week 4 visit, doses of ruxolitinib could be increased in 5 mg once a day (QD) increments every 4 weeks every 4 weeks not to exceed a dose of 25 mg BID. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ruxolitinib | Drug | Ruxolitinib (INCB018424), 5 mg bid |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change From Baseline in Spleen Volume at Week 24 by Final Titrated Dose | Magnetic resonance imaging (MRI) of the upper and lower abdomen and pelvis was performed to assess spleen volumes. Computed tomography (CT) scan was performed if participant was not a candidate for MRI or if MRI was not readily available. MRI was performed with a body coil. Spleen volume was obtained by outlining the circumference of the organ and determining the volume using the validated technique of least squares. The MRI (or CT scan in applicable participants) was performed on the first or second day of the baseline period (ie, Day -7 or Day -6), and the site radiologist sent the scan to the central imaging laboratory that same day. The CT scans were processed by the same central laboratory used for MRIs. | Baseline and Week 24 |
| Percent Change From Baseline in Total Symptom Score (TSS) as Measured by the Modified Myelofibrosis Symptom Assessment Form (MFSAF) V2.0 Diary at Week 24 by Final Titrated Dose | Symptoms of myelofibrosis were assessed using a modified Myelofibrosis Symptom Assessment Form (MFSAF) Version 2.0 diary. Using the diary, patients rated the following symptoms on a scale from 0 (absent) to 10 (worst imaginable): night sweats, itching, abdominal discomfort, pain under ribs on left, feeling of fullness (early satiety), and muscle/bone pain. The total symptom score ranged from 0-60 and was calculated as the sum of the 6 symptom scores. A higher score indicates worse symptoms. | Baseline and Week 24 |
| Percentage of Participants With Treatment-emergent Adverse Events (TEAE) | TEAE was defined as adverse events that began or worsened from baseline after the first administration of the study drug. Participants were analyzed based on the number of subjects who received a dose within the dose group. The percentages for each column are calculated using this N. Participants who had more than 1 event in an AE category (eg, treatment-related TEAE) are counted once at each dose level the event occurred. | Up to Week 156 |
| Percentage of Participants With New Onset Grade 4 Thrombocytopenia Events as Assessed by Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE V4.03) |
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change in Spleen Volume at Week 24 Compared to Baseline | MRI of the upper and lower abdomen and pelvis was performed, to assess spleen volumes. CT scan was performed if participant was not a candidate for MRI, or if MRI was not readily available. MRI was performed with a body coil. Spleen volume was obtained by outlining the circumference of the organ and determining the volume using the validated technique of least squares. The MRI (or CT scan in applicable participants) was performed on the first or second day of the baseline period (ie, Day -7 or Day -6), and the site radiologist sent the scan to the central imaging laboratory that same day. The CT scans were processed by the same central laboratory used for MRIs. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Peter Langmuir, MD | Incyte Corporation | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Birmingham | Alabama | United States | ||||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 34911667 | Derived | Talpaz M, Prchal J, Afrin L, Arcasoy M, Hamburg S, Clark J, Kornacki D, Colucci P, Verstovsek S. Safety and Efficacy of Ruxolitinib in Patients with Myelofibrosis and Low Platelet Counts (50 - 100 x 109/L): Final Analysis of an Open-Label Phase 2 Study. Clin Lymphoma Myeloma Leuk. 2022 May;22(5):336-346. doi: 10.1016/j.clml.2021.10.016. Epub 2021 Nov 2. | |
| 24283202 |
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A total of 66 participants were enrolled in the study. Fifty-five participants completed 24 weeks of treatment (core treatment period), and 23 participants entered the extension phase.
Participants took part in the study at 27 investigative sites in the United States from 15 June 2011 to 19 December 2018.
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| ID | Title | Description |
|---|---|---|
| FG000 | Ruxolitinib 5 Milligram (mg) | Doses may not exceed 10 mg bid except in subjects who continue to meet the above dose escalation criteria, and who have, in addition, a PGIC score of minimally worse, much worse or very much worse while receiving 10 mg bid. Such subjects may continue dose escalation to a maximum dose of 15 mg bid. During the extended treatment phase, doses of ruxolitinib may be increased in 5 mg qd increments up to a dose of 25 mg bid if the subject meets the above dose escalation criteria, or per the investigator's discretion. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Aug 9, 2013 | Dec 13, 2019 |
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Participants with platelet count between 50 and 100 × 10^9/L at the screening and/or baseline visit were enrolled in the study. Thrombocytopenia is defined as a condition with low blood platelet count. Grade 4 thrombocytopenia was platelet count < 25 × 10^9/L. Participants were analyzed based on the number of subjects who received a dose within the dose group. The percentages for each column are calculated using this N. Participants who had more than 1 event in an AE category (eg, treatment-related TEAE) are counted once at each dose level the event occurred. |
| Up to Week 156 |
| Percentage of Participants With New Onset Grade 2 or Higher Hemorrhage as Assessed by CTCAE V4.03 | Hemorrhages were defined as any lower level terms by MedDRA included in the Standardized MedDRA Query (SMQ) for hemorrhage terms. Participants were analyzed based on the number of subjects who received a dose within the dose group. The percentages for each column are calculated using this N. Participants who had more than 1 event in an AE category (eg, treatment-related TEAE) are counted once at each dose level the event occurred. | Up to Week 156 |
| Baseline and Week 24 |
| Percent Change in Total Symptom Score as Measured by the Modified MFSAF V2.0 Diary at Week 24 Compared to Baseline | Symptoms of myelofibrosis were assessed using a modified Myelofibrosis Symptom Assessment Form (MFSAF) Version 2.0 diary. Using the diary, patients rated the following symptoms on a scale from 0 (absent) to 10 (worst imaginable): night sweats, itching, abdominal discomfort, pain under ribs on left, feeling of fullness (early satiety), and muscle/bone pain. The total symptom score ranged from 0-60 and was calculated as the sum of the 6 symptom scores. A higher score indicates worse symptoms.. | Baseline and Week 24 |
| Percentage of Participants With ≥ 35% Reduction in Spleen Volume at Week 24 Compared to Baseline | MRI of the upper and lower abdomen and pelvis was performed, to assess spleen volumes. CT scan was performed if participant is not a candidate for MRI, or if MRI is not readily available. MRI was performed with a body coil. Spleen volume was obtained by outlining the circumference of the organ and determining the volume using the validated technique of least squares. The MRI (or CT scan in applicable participants) was performed on the first or second day of the baseline period (ie, Day -7 or Day -6), and the site radiologist sent the scan to the central imaging laboratory that same day. The CT scans were processed by the same central laboratory used for MRIs. | Baseline and Week 24 |
| Percentage of Participants With ≥10% Reduction in Spleen Volume at Week 24 Compared to Baseline | MRI of the upper and lower abdomen and pelvis was performed, to assess spleen volumes. CT scan was performed if participant is not a candidate for MRI, or if MRI is not readily available. MRI was performed with a body coil. Spleen volume was obtained by outlining the circumference of the organ and determining the volume using the validated technique of least squares. The MRI (or CT scan in applicable participants) was performed on the first or second day of the baseline period (ie, Day -7 or Day -6), and the site radiologist sent the scan to the central imaging laboratory that same day. The CT scans were processed by the same central laboratory used for MRIs. | Baseline and Week 24 |
| Percentage of Participants With ≥ 50% Improvement in Total Symptom Score as Measured by the Modified MFSAF V2.0 Diary at Week 24 Compared to Baseline | Symptoms of myelofibrosis were assessed using a modified Myelofibrosis Symptom Assessment Form (MFSAF) Version 2.0 diary. Using the diary, patients rated the following symptoms on a scale from 0 (absent) to 10 (worst imaginable): night sweats, itching, abdominal discomfort, pain under ribs on left, feeling of fullness (early satiety), and muscle/bone pain. The total symptom score ranged from 0-60 and was calculated as the sum of the 6 symptom scores. A higher score indicates worse symptoms. | Baseline and Week 24 |
| Change in Spleen Length Measured by Palpation | Measurement of spleen length below the left costal margin was measured by palpation at each study visit. Investigators were provided with a soft centimeter ruler so that palpable spleen length was measured in centimeters and not in finger breadths. The edge of the spleen was determined by palpation, and measured in centimeters, using a soft ruler, from the costal margin to the point of greatest splenic protrusion. | Up to Week 156 |
| Percent Change From Baseline in Spleen Length Measured by Palpation | Measurement of spleen length below the left costal margin was measured by palpation at each study visit. Investigators were provided with a soft centimeter ruler so that palpable spleen length was measured in centimeters and not in finger breadths. The edge of the spleen was determined by palpation, and measured in centimeters, using a soft ruler, from the costal margin to the point of greatest splenic protrusion. | Up to Week 156 |
| Patient Global Impression of Change (PGIC) Score at Each Visit | Symptoms of myelofibrosis were assessed using the PGIC questionnaire. Using the questionnaire, patients rated the overall sense of treatment effect on their symptoms on a scale of 1 (very much improved)- 7(very much worse). The specific wording was: Since the start of the treatment you've received in this study, your myelofibrosis symptoms are: 1) Very much improved, 2) Much improved, 3) Minimally improved, 4) No change, 5) Minimally worse, 6) Much worse, 7) Very much worse. A higher score indicates worse symptoms. | Up to Week 156 |
| Beverly Hills |
| California |
| United States |
| Burbank | California | United States |
| La Jolla | California | United States |
| Los Angeles | California | United States |
| Pomona | California | United States |
| San Diego | California | United States |
| New Haven | Connecticut | United States |
| Fort Myers | Florida | United States |
| Jacksonville | Florida | United States |
| Orange City | Florida | United States |
| Atlanta | Georgia | United States |
| Augusta | Georgia | United States |
| Chicago | Illinois | United States |
| Iowa City | Iowa | United States |
| Louisville | Kentucky | United States |
| New Orleans | Louisiana | United States |
| Baltimore | Maryland | United States |
| Ann Arbor | Michigan | United States |
| Southfield | Michigan | United States |
| St Louis | Missouri | United States |
| Hackensack | New Jersey | United States |
| Morristown | New Jersey | United States |
| Somerville | New Jersey | United States |
| New York | New York | United States |
| Durham | North Carolina | United States |
| Hickory | North Carolina | United States |
| Canton | Ohio | United States |
| Cleveland | Ohio | United States |
| Portland | Oregon | United States |
| Danville | Pennsylvania | United States |
| Hershey | Pennsylvania | United States |
| Charleston | South Carolina | United States |
| Nashville | Tennessee | United States |
| Houston | Texas | United States |
| San Antonio | Texas | United States |
| Salt Lake City | Utah | United States |
| Burlington | Vermont | United States |
| Talpaz M, Paquette R, Afrin L, Hamburg SI, Prchal JT, Jamieson K, Terebelo HR, Ortega GL, Lyons RM, Tiu RV, Winton EF, Natrajan K, Odenike O, Claxton D, Peng W, O'Neill P, Erickson-Viitanen S, Leopold L, Sandor V, Levy RS, Kantarjian HM, Verstovsek S. Interim analysis of safety and efficacy of ruxolitinib in patients with myelofibrosis and low platelet counts. J Hematol Oncol. 2013 Oct 29;6(1):81. doi: 10.1186/1756-8722-6-81. |
| COMPLETED |
|
| NOT COMPLETED |
|
|
Safety evaluable population included participants who received at least 1 dose of study drug.
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| ID | Title | Description |
|---|---|---|
| BG000 | Ruxolitinib 5 mg | Doses may not exceed 10 mg bid except in subjects who continue to meet the above dose escalation criteria, and who have, in addition, a PGIC score of minimally worse, much worse or very much worse while receiving 10 mg bid. Such subjects may continue dose escalation to a maximum dose of 15 mg bid. During the extended treatment phase, doses of ruxolitinib may be increased in 5 mg qd increments up to a dose of 25 mg bid if the subject meets the above dose escalation criteria, or per the investigator's discretion. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| ||||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percent Change From Baseline in Spleen Volume at Week 24 by Final Titrated Dose | Magnetic resonance imaging (MRI) of the upper and lower abdomen and pelvis was performed to assess spleen volumes. Computed tomography (CT) scan was performed if participant was not a candidate for MRI or if MRI was not readily available. MRI was performed with a body coil. Spleen volume was obtained by outlining the circumference of the organ and determining the volume using the validated technique of least squares. The MRI (or CT scan in applicable participants) was performed on the first or second day of the baseline period (ie, Day -7 or Day -6), and the site radiologist sent the scan to the central imaging laboratory that same day. The CT scans were processed by the same central laboratory used for MRIs. | Intent-to-treat (ITT) population included all participants enrolled and treated in the study. | Posted | Mean | Standard Deviation | Percentage change from baseline | Baseline and Week 24 |
|
|
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| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Primary | Percent Change From Baseline in Total Symptom Score (TSS) as Measured by the Modified Myelofibrosis Symptom Assessment Form (MFSAF) V2.0 Diary at Week 24 by Final Titrated Dose | Symptoms of myelofibrosis were assessed using a modified Myelofibrosis Symptom Assessment Form (MFSAF) Version 2.0 diary. Using the diary, patients rated the following symptoms on a scale from 0 (absent) to 10 (worst imaginable): night sweats, itching, abdominal discomfort, pain under ribs on left, feeling of fullness (early satiety), and muscle/bone pain. The total symptom score ranged from 0-60 and was calculated as the sum of the 6 symptom scores. A higher score indicates worse symptoms. | ITT population included all participants enrolled and treated in the study. For the 5 mg AM/ 10 mg PM arm, the t-test was not calculated due to only having a single participant. | Posted | Mean | Standard Deviation | Percentage change from baseline | Baseline and Week 24 |
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| Primary | Percentage of Participants With Treatment-emergent Adverse Events (TEAE) | TEAE was defined as adverse events that began or worsened from baseline after the first administration of the study drug. Participants were analyzed based on the number of subjects who received a dose within the dose group. The percentages for each column are calculated using this N. Participants who had more than 1 event in an AE category (eg, treatment-related TEAE) are counted once at each dose level the event occurred. | Safety evaluable population included participants who received at least 1 dose of study drug. | Posted | Number | percentage of participants | Up to Week 156 |
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| Primary | Percentage of Participants With New Onset Grade 4 Thrombocytopenia Events as Assessed by Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE V4.03) | Participants with platelet count between 50 and 100 × 10^9/L at the screening and/or baseline visit were enrolled in the study. Thrombocytopenia is defined as a condition with low blood platelet count. Grade 4 thrombocytopenia was platelet count < 25 × 10^9/L. Participants were analyzed based on the number of subjects who received a dose within the dose group. The percentages for each column are calculated using this N. Participants who had more than 1 event in an AE category (eg, treatment-related TEAE) are counted once at each dose level the event occurred. | Safety evaluable population included participants who received at least 1 dose of study drug. | Posted | Number | percentage of participants | Up to Week 156 |
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| Primary | Percentage of Participants With New Onset Grade 2 or Higher Hemorrhage as Assessed by CTCAE V4.03 | Hemorrhages were defined as any lower level terms by MedDRA included in the Standardized MedDRA Query (SMQ) for hemorrhage terms. Participants were analyzed based on the number of subjects who received a dose within the dose group. The percentages for each column are calculated using this N. Participants who had more than 1 event in an AE category (eg, treatment-related TEAE) are counted once at each dose level the event occurred. | Safety evaluable population included participants who received at least 1 dose of study drug. | Posted | Number | percentage of participants | Up to Week 156 |
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| Secondary | Percent Change in Spleen Volume at Week 24 Compared to Baseline | MRI of the upper and lower abdomen and pelvis was performed, to assess spleen volumes. CT scan was performed if participant was not a candidate for MRI, or if MRI was not readily available. MRI was performed with a body coil. Spleen volume was obtained by outlining the circumference of the organ and determining the volume using the validated technique of least squares. The MRI (or CT scan in applicable participants) was performed on the first or second day of the baseline period (ie, Day -7 or Day -6), and the site radiologist sent the scan to the central imaging laboratory that same day. The CT scans were processed by the same central laboratory used for MRIs. | ITT population included all participants enrolled and treated in the study. | Posted | Mean | Standard Deviation | Percentage | Baseline and Week 24 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percent Change in Total Symptom Score as Measured by the Modified MFSAF V2.0 Diary at Week 24 Compared to Baseline | Symptoms of myelofibrosis were assessed using a modified Myelofibrosis Symptom Assessment Form (MFSAF) Version 2.0 diary. Using the diary, patients rated the following symptoms on a scale from 0 (absent) to 10 (worst imaginable): night sweats, itching, abdominal discomfort, pain under ribs on left, feeling of fullness (early satiety), and muscle/bone pain. The total symptom score ranged from 0-60 and was calculated as the sum of the 6 symptom scores. A higher score indicates worse symptoms.. | ITT population included all participants enrolled and treated in the study. | Posted | Mean | Standard Deviation | Percentage change from baseline | Baseline and Week 24 |
|
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| Secondary | Percentage of Participants With ≥ 35% Reduction in Spleen Volume at Week 24 Compared to Baseline | MRI of the upper and lower abdomen and pelvis was performed, to assess spleen volumes. CT scan was performed if participant is not a candidate for MRI, or if MRI is not readily available. MRI was performed with a body coil. Spleen volume was obtained by outlining the circumference of the organ and determining the volume using the validated technique of least squares. The MRI (or CT scan in applicable participants) was performed on the first or second day of the baseline period (ie, Day -7 or Day -6), and the site radiologist sent the scan to the central imaging laboratory that same day. The CT scans were processed by the same central laboratory used for MRIs. | Efficacy evaluable participants included all participants enrolled and treated in the study. | Posted | Number | 95% Confidence Interval | percentage of participants | Baseline and Week 24 |
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| Secondary | Percentage of Participants With ≥10% Reduction in Spleen Volume at Week 24 Compared to Baseline | MRI of the upper and lower abdomen and pelvis was performed, to assess spleen volumes. CT scan was performed if participant is not a candidate for MRI, or if MRI is not readily available. MRI was performed with a body coil. Spleen volume was obtained by outlining the circumference of the organ and determining the volume using the validated technique of least squares. The MRI (or CT scan in applicable participants) was performed on the first or second day of the baseline period (ie, Day -7 or Day -6), and the site radiologist sent the scan to the central imaging laboratory that same day. The CT scans were processed by the same central laboratory used for MRIs. | Efficacy evaluable participants included all participants enrolled and treated in the study. | Posted | Number | 95% Confidence Interval | percentage of participants | Baseline and Week 24 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With ≥ 50% Improvement in Total Symptom Score as Measured by the Modified MFSAF V2.0 Diary at Week 24 Compared to Baseline | Symptoms of myelofibrosis were assessed using a modified Myelofibrosis Symptom Assessment Form (MFSAF) Version 2.0 diary. Using the diary, patients rated the following symptoms on a scale from 0 (absent) to 10 (worst imaginable): night sweats, itching, abdominal discomfort, pain under ribs on left, feeling of fullness (early satiety), and muscle/bone pain. The total symptom score ranged from 0-60 and was calculated as the sum of the 6 symptom scores. A higher score indicates worse symptoms. | Efficacy evaluable participants included all participants enrolled and treated in the study. | Posted | Number | 95% Confidence Interval | percentage of participants | Baseline and Week 24 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change in Spleen Length Measured by Palpation | Measurement of spleen length below the left costal margin was measured by palpation at each study visit. Investigators were provided with a soft centimeter ruler so that palpable spleen length was measured in centimeters and not in finger breadths. The edge of the spleen was determined by palpation, and measured in centimeters, using a soft ruler, from the costal margin to the point of greatest splenic protrusion. | Efficacy evaluable participants included all participants enrolled and treated in the study. 'Number Analyzed' is number of participants with data available at a particular time point. | Posted | Mean | Standard Deviation | cm | Up to Week 156 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percent Change From Baseline in Spleen Length Measured by Palpation | Measurement of spleen length below the left costal margin was measured by palpation at each study visit. Investigators were provided with a soft centimeter ruler so that palpable spleen length was measured in centimeters and not in finger breadths. The edge of the spleen was determined by palpation, and measured in centimeters, using a soft ruler, from the costal margin to the point of greatest splenic protrusion. | Efficacy evaluable participants included all participants enrolled and treated in the study. 'Number Analyzed' is number of participants with data available at a particular time point. | Posted | Mean | Standard Deviation | Percentage | Up to Week 156 |
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| Secondary | Patient Global Impression of Change (PGIC) Score at Each Visit | Symptoms of myelofibrosis were assessed using the PGIC questionnaire. Using the questionnaire, patients rated the overall sense of treatment effect on their symptoms on a scale of 1 (very much improved)- 7(very much worse). The specific wording was: Since the start of the treatment you've received in this study, your myelofibrosis symptoms are: 1) Very much improved, 2) Much improved, 3) Minimally improved, 4) No change, 5) Minimally worse, 6) Much worse, 7) Very much worse. A higher score indicates worse symptoms. | Efficacy evaluable participants included all participants enrolled and treated in the study. 'Number Analyzed' is number of participants with data available at the particular time point. | Posted | Mean | Standard Deviation | score on a scale | Up to Week 156 |
|
Up to approximately 161 weeks
Safety evaluable population included participants who received at least 1 dose of study drug. Data for adverse events was reported as per the total daily dose the participant was receiving at the time of the event.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | >0 - 5 mg | Participants received ruxolitinib up to 5 mg. | 0 | 15 | 5 | 15 | 13 | 15 |
| EG001 | >5 - 10 mg | Participants received ruxolitinib in the range of 5 to 10 mg. | 0 | 66 | 8 | 66 | 43 | 66 |
| EG002 | >10 - 15 mg | Participants received ruxolitinib in the range of 10 to 15 mg. | 0 | 54 | 4 | 54 | 26 | 54 |
| EG003 | >15 - 20 mg | Participants received ruxolitinib in the range of 15 to 20 mg. | 2 | 44 | 9 | 44 | 31 | 44 |
| EG004 | >20 mg | Participants received ruxolitinib, more than 20 mg. | 0 | 9 | 1 | 9 | 5 | 9 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Leukocytosis | Blood and lymphatic system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Bradycardia | Cardiac disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Cardiac failure congestive | Cardiac disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Colitis | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Rectal haemorrhage | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Retroperitoneal haemorrhage | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Death | General disorders | MedDRA (12.0) | Systematic Assessment | General disorders and administration site conditions |
|
| Cholelithiasis | Hepatobiliary disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Subdural haematoma | Injury, poisoning and procedural complications | MedDRA (12.0) | Systematic Assessment |
| |
| Tumour lysis syndrome | Metabolism and nutrition disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Lung adenocarcinoma metastatic | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (12.0) | Systematic Assessment |
| |
| Squamous cell carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (12.0) | Systematic Assessment |
| |
| Cerebrovascular accident | Nervous system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Hypnagogic hallucination | Psychiatric disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Epididymitis | Reproductive system and breast disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Pneumonitis | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Haematoma | Vascular disorders | MedDRA (12.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Reticulocytosis | Blood and lymphatic system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Splenomegaly | Blood and lymphatic system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Cardiomegaly | Cardiac disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Cyanosis | Cardiac disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Scleral haemorrhage | Eye disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Visual impairment | Eye disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA (12.0) | Systematic Assessment | General disorders and administration site conditions |
|
| Chest discomfort | General disorders | MedDRA (12.0) | Systematic Assessment | General disorders and administration site conditions |
|
| Early satiety | General disorders | MedDRA (12.0) | Systematic Assessment | General disorders and administration site conditions |
|
| Fatigue | General disorders | MedDRA (12.0) | Systematic Assessment | General disorders and administration site conditions |
|
| Oedema | General disorders | MedDRA (12.0) | Systematic Assessment | General disorders and administration site conditions |
|
| Oedema peripheral | General disorders | MedDRA (12.0) | Systematic Assessment | General disorders and administration site conditions |
|
| Pain | General disorders | MedDRA (12.0) | Systematic Assessment | General disorders and administration site conditions |
|
| Pyrexia | General disorders | MedDRA (12.0) | Systematic Assessment | General disorders and administration site conditions |
|
| Cholelithiasis | Hepatobiliary disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Gallbladder disorder | Hepatobiliary disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Seasonal allergy | Immune system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Herpes simplex | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Oral herpes | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Vaginal infection | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (12.0) | Systematic Assessment |
| |
| Transfusion reaction | Injury, poisoning and procedural complications | MedDRA (12.0) | Systematic Assessment |
| |
| Blast cell count increased | Investigations | MedDRA (12.0) | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA (12.0) | Systematic Assessment |
| |
| Blood creatinine decreased | Investigations | MedDRA (12.0) | Systematic Assessment |
| |
| Cardioactive drug level increased | Investigations | MedDRA (12.0) | Systematic Assessment |
| |
| Electrocardiogram QT prolonged | Investigations | MedDRA (12.0) | Systematic Assessment |
| |
| Haemoglobin decreased | Investigations | MedDRA (12.0) | Systematic Assessment |
| |
| Neutrophil count decreased | Investigations | MedDRA (12.0) | Systematic Assessment |
| |
| Occult blood positive | Investigations | MedDRA (12.0) | Systematic Assessment |
| |
| Platelet count decreased | Investigations | MedDRA (12.0) | Systematic Assessment |
| |
| Serum ferritin increased | Investigations | MedDRA (12.0) | Systematic Assessment |
| |
| White blood cell count decreased | Investigations | MedDRA (12.0) | Systematic Assessment |
| |
| White blood cell count increased | Investigations | MedDRA (12.0) | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Hypermagnesaemia | Metabolism and nutrition disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Hyperuricaemia | Metabolism and nutrition disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Hypocalcaemia | Metabolism and nutrition disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Iron overload | Metabolism and nutrition disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Bone pain | Musculoskeletal and connective tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Presyncope | Nervous system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Mental status changes | Psychiatric disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Urine flow decreased | Renal and urinary disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Dyspnoea exertional | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Pulmonary hypertension | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Upper-airway cough syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Ecchymosis | Skin and subcutaneous tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Night sweats | Skin and subcutaneous tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (12.0) | Systematic Assessment |
|
Participants with platelet counts of 50 to 100 x 10^9/L were enrolled to begin ruxolitinib at 5 mg BID. Subjects could then titrate their dose to an dose that offers benefit. Only the overall data was calculated for the Participant and Baseline data.
Clinical Study Agreement
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Incyte Corporation | 1-855-463-3463 | medinfo@incyte.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jan 17, 2012 | Dec 13, 2019 | SAP_001.pdf |
| ID | Term |
|---|---|
| D009196 | Myeloproliferative Disorders |
| ID | Term |
|---|---|
| D001855 | Bone Marrow Diseases |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C540383 | ruxolitinib |
Not provided
Not provided
Not provided
| Asian |
|
| Native Hawaiian or Pacific Islander |
|
| Other |
|
| 0.0163 |
1-sample t-test was used. |
| Other |
1-sample t-test with null hypothesis mean >= 0 |
| t-test, 1 sided | <0.0001 | 1-sample t-test was used. | Other | 1-sample t-test with null hypothesis mean >= 0 |
| t-test, 1 sided | 0.2019 | 1-sample t-test was used. | Other | 1-sample t-test with null hypothesis mean >= 0 |
| OG003 | 10 mg AM/ 15 mg PM or 15 mg BID | Participants received the final titrated dose of ruxolitinib 10 mg AM plus 15 mg PM or 15 mg BID. |
|
|
|
| OG004 | >20 mg | Participants received ruxolitinib, more than 20 mg. |
|
|
| OG003 |
| >15 - 20 mg |
Participants received ruxolitinib in the range of 15 to 20 mg. |
| OG004 | >20 mg | Participants received ruxolitinib, more than 20 mg. |
|
|
Participants received ruxolitinib in the range of 15 to 20 mg.
| OG004 | >20 mg | Participants received ruxolitinib, more than 20 mg. |
|
|
|
|
|
|
Participants received the final titrated dose of ruxolitinib 10 mg BID.
| OG003 | 10 mg AM/ 15 mg PM or 15 mg BID | Participants received the final titrated dose of ruxolitinib 10 mg AM plus 15 mg PM or 15 mg BID. |
|
|
Participants received the final titrated dose of ruxolitinib 10 mg BID.
| OG003 | 10 mg AM/ 15 mg PM or 15 mg BID | Participants received the final titrated dose of ruxolitinib 10 mg AM plus 15 mg PM or 15 mg BID. |
|
|
| OG003 | 10 mg AM/ 15 mg PM or 15 mg BID | Participants received the final titrated dose of ruxolitinib 10 mg AM plus 15 mg PM or 15 mg BID. |
|
|
Participants received the final titrated dose of ruxolitinib 10 mg AM plus 15 mg PM or 15 mg BID. |
|
|
| 10 mg AM/ 15 mg PM or 15 mg BID |
Participants received the final titrated dose of ruxolitinib 10 mg AM plus 15 mg PM or 15 mg BID. |
|
|
| OG003 | 10 mg AM/ 15 mg PM or 15 mg BID | Participants received the final titrated dose of ruxolitinib 10 mg AM plus 15 mg PM or 15 mg BID. |
|
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