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MOMENTUM is a randomized, double-blind, active control Phase 3 trial intended to confirm the differentiated clinical benefits of the investigational drug momelotinib (MMB) versus danazol (DAN) in symptomatic and anemic participants who have previously received an approved Janus kinase inhibitor (JAKi) therapy for myelofibrosis (MF). The purpose of this clinical study is to compare the effectiveness and safety of MMB to DAN in treating and reducing: 1) disease related symptoms, 2) the need for blood transfusions and 3) splenomegaly, in adults with primary MF, post-polycythemia vera MF or post-essential thrombocythemia MF. The study is planned in countries including, but not limited to: Australia, Austria, Belgium, Bulgaria, Canada, Czech Republic, Denmark, France, Germany, Hungary, Israel, Italy, New Zealand, Poland, Romania, Singapore, South Korea, Spain, Sweden, Taiwan, United Kingdom (UK) and United States (US).
Participants must be symptomatic with a Myelofibrosis Symptom Assessment Form (MFSAF) version (v) 4.0 Total Symptom Score of >= 10 at screening, and be anemic with hemoglobin (Hgb) < 10 gram/deciliter (g/dL). For participants with ongoing JAKi therapy at screening, JAKi therapy must be tapered over a period of at least 1 week, followed by a 2-week non-treatment washout interval prior to randomization.
Participants will be randomized 2:1 to orally self-administer blinded treatment: MMB plus placebo or DAN plus placebo. Participants randomized to receive MMB who complete the randomized treatment period to the end of Week 24 may continue to receive MMB in the open-label extended treatment period to the end of Week 204 (a total period of treatment of approximately 4 years) if the participants tolerates and continues to benefit from MMB.
Participants randomized to receive DAN may cross-over to MMB open-label treatment in the following circumstances: at the end of Week 24 if they complete the randomized treatment period; or at the end of Week 24 if they discontinue treatment with DAN but continue study assessments and do not receive prohibited medications including alternative active anti-MF therapy; or at any time during the randomized treatment period if they meet the protocol-defined criteria for radiographically confirmed symptomatic splenic progression. Participants randomized to receive DAN who are receiving clinical benefit at the end of Week 24 may choose to continue DAN therapy up to Week 48. The comparator treatment, DAN, is an approved medication in the US and in some other countries and is recommended by national guidelines as a treatment for anemia in MF.
MOMENTUM Contact Email: GSKClinicalSupportHD@gsk.com
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Momelotinib | Experimental | Participants will receive momelotinib plus placebo to match danazol |
|
| Danazol | Active Comparator | Participants will receive danazol plus placebo to match momelotinib |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Momelotinib | Drug | Momelotinib tablets will be self-administered orally once daily |
|
| Measure | Description | Time Frame |
|---|---|---|
| Total Symptom Score (TSS) Response Rate at Week 24 | Myelofibrosis Symptom Assessment Form (MFSAF) TSS version (v) 4.0 response rate was defined as percentage of participants with a >= 50 percent (%) reduction from Baseline in mean MFSAF TSS over consecutive 28-day period immediately before end of Week 24. TSS response rate was measured using MFSAF v4.0. MFSAF v4.0 comprises 7 domains representing 7 most relevant symptoms of myelofibrosis (MF) identified through existing participant and clinician-based evidence: fatigue,night sweats,pruritus,abdominal discomfort,pain under left ribs,early satietyand bone pain. Participants scored each symptom domain using an 11-point numeric rating scale ranging from 0(absent) to 10(worst imaginable). The MFSAF TSS was calculated as sum of scores of 7 domains for a possible range of scores of 0 to 70, with a higher TSS corresponding to more severe symptoms. A reduction from Baseline corresponded to a lessening of MF symptoms. Baseline was the last assessment done before or on the day of first dose date. | Baseline and Week 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Transfusion Independence (TI) at Week 24 | TI status was defined as not receiving red blood cell (RBC) or whole blood transfusion for >=12 weeks, with no hemoglobin (Hgb) level < 8 grams per deciliter (g/dL) during the same interval. Percentage of participants with TI have been presented. | Week 24 |
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Inclusion Criteria:
Age >= 18 years.
Confirmed diagnosis of PMF in accordance with the World Health Organization (WHO) 2016 criteria, or Post- polycythemia vera/essential thrombocythemia (PV/ET) MF in accordance with the International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT criteria).
Symptomatic, defined as a TSS of >= 10 units assessed by a single MFSAF v4.0 assessment during Screening prior to Baseline period (Day BL1).
Anemic, defined as a Hgb < 10 g/dL in Screening/Baseline period.
Previously treated with an approved JAK inhibitor for PMF or Post-PV/ET MF for >= 90 days, or >= 28 days if JAK inhibitor therapy is complicated by RBC transfusion requirement of >= 4 units in 8 weeks, or Grade 3/4 AEs of thrombocytopenia, anemia, or hematoma.
Baseline splenomegaly, defined as having a palpable spleen at >= 5 centimeter (cm), below the left costal margin, or with volume >= 450 cubic centimeter (cm^3) on imaging (ultrasound, magnetic resonance imaging [MRI] or computed tomography [CT] are acceptable), assessed during Screening at any point prior to Randomization.
High risk, intermediate-2, or intermediate-1 risk MF as defined by Dynamic International Prognostic Scoring System (DIPSS), or DIPSS-plus.
No allogeneic stem cell transplant planned.
Acceptable laboratory assessments:
Exclusion Criteria:
Use of the following treatments within the time periods noted:
History of prostate cancer, with the exception of localized prostate cancer that has been treated surgically or by radiotherapy with curative intent and presumed cured.
Prostate specific antigen (PSA) > 4 nanograms per milliliter (ng/mL).
Unsuitable for spleen volume measurements due to prior splenectomy or unwilling or unable to undergo an MRI scan or CT scan for spleen volume measurement per protocol requirements.
Any of the following (criteria a - k):
Participants with a prior or concurrent malignancy, whose natural history or treatment has a significant potential to interfere with the safety or efficacy assessment of the investigational regimen.
Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding or thalassemia.
Known positive status for human immunodeficiency viruses (HIV).
Chronic active or acute viral hepatitis A, B, or C infection, or hepatitis B or C carrier (testing required for hepatitis B and C).
Unresolved non-hematologic toxicities from prior therapies that are > Grade 1 per CTCAE v5.0.
Presence of peripheral neuropathy >= Grade 2 per CTCAE v5.0.
Women who are already pregnant or lactating. Additional inclusion/exclusion criteria may apply.
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| Name | Affiliation | Role |
|---|---|---|
| Srdan Verstovsek, M.D., Ph.D. | Department of Leukemia, The University of Texas MD Anderson Cancer Center | Principal Investigator |
| Ruben Mesa | UT Health San Antonio Cancer Center, San Antonio, TX, USA | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Mayo Clinic Hospital - Phoenix | Phoenix | Arizona | 85054 | United States | ||
| Irvine Center for Clinical Research |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41958363 | Derived | Mesa R, Palmer JM, Palandri F, Masarova L, Harrison CN, Mazerolle F, M'hari M, Liu T, Zhang S, Cardellino A, Wang Z, Ellis CE, Patnaik D, Regnault A, LeBlanc TW. Association Between Transfusion Status, Hemoglobin Levels, and Patient-Reported Outcomes in Myelofibrosis: A Post Hoc Clinical Trial Analysis. Cancer Med. 2026 Apr;15(4):e71729. doi: 10.1002/cam4.71729. | |
| 40691344 |
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A total of 195 participants were enrolled in the study.
This study evaluated the activity of Momelotinib (MMB) versus Danazol (DAN) in Symptomatic, Anemic participants. This study consists of Randomized Double-blind (DB) Treatment Period (TP) and Open-label extended Treatment Period.
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| ID | Title | Description |
|---|---|---|
| FG000 | MMB 200 mg Once Daily (QD) + Placebo | Participants were randomized to receive 200 milligrams (mg) of MMB orally QD and a DAN-placebo orally twice daily (BID) during the randomized 24-week double-blind treatment period. Participants who completed the randomized treatment period or discontinued treatment early for other reasons but completed scheduled assessments through Week 24 were enrolled in an open-label extended treatment period. Participants continued to receive 200 mg of MMB orally QD during the open-label extended treatment period. Participants who discontinued treatment prior to Week 24 due to splenic progression or other reasons, but completed assessments, were unblinded. Participants who discontinued MMB during the randomized treatment period were not eligible to receive MMB in the open-label extended treatment period. Participants who were randomized to MMB during the randomized treatment period with confirmed splenic progression could not proceed to the open-label extended treatment period. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Randomized DB TP (Up to Week 24) |
|
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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 | Dec 18, 2020 | Dec 1, 2022 |
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During the 24 week randomized treatment phase of the study, participants, investigators and sponsor and relevant vendor personnel (with the exception of specified unblinded personnel, for example clinical supply) will remain blinded to the participant's treatment assignment and to aggregate data that may lead to inadvertent unblinding. Participants who continue treatment with momelotinib or danazol after Week 24 in the extended treatment phase will receive unblinded treatment.
| Placebo to match danazol | Drug | Danazol placebo capsules will be self-administered orally twice daily |
|
| Danazol | Drug | Danazol capsules will be self-administered orally twice daily |
|
|
| Placebo to match momelotinib | Drug | Momelotinib placebo tablets will be self-administered orally once daily |
|
| Splenic Response Rate (SRR) of >=25% at Week 24 |
Splenic response rate (SRR) is defined as the percentage of participants who have reduction in spleen volume of >=25% from Baseline at the end of Week 24. Baseline was the last assessment done before or on the day of first dose date. |
| Baseline and Week 24 |
| Change From Baseline in MFSAF TSS at Week 24 | TSS was measured using the MFSAF v4.0. The MFSAF v4.0 comprises 7 domains representing the 7 most relevant symptoms of MF identified through existing participant and clinician-based evidence: fatigue, night sweats, pruritus, abdominal discomfort, pain under the left ribs, early satiety, and bone pain. Participants scored each symptom domain using an 11-point numeric rating scale ranging from 0 (absent) to 10 (worst imaginable). The MFSAF TSS was calculated as the sum of scores of the 7 domains for a possible range of scores of 0 to 70, with a higher TSS corresponding to more severe symptoms. A reduction from Baseline corresponded to a lessening of MF symptoms. Baseline was the last assessment done before or on the day of first dose date. Change from Baseline was defined as the post-Baseline value minus Baseline value. | Baseline and Week 24 |
| Splenic Response Rate (SRR) of >= 35% at Week 24 | Splenic response rate (SRR) is defined as the percentage of participants who have reduction in spleen volume of >=35 % from Baseline at the end of Week 24. Baseline was the last assessment done before or on the day of first dose date. | Baseline and Week 24 |
| Percentage of Participants With Zero RBC Units Transfused Over 24-Weeks | Percentage of participants with zero RBC units transfused over 24-weeks were reported. | Up to 24 weeks |
| Percentage of Participants With <=4 RBC Units Transfused Over 24-weeks | Percentage of participants with <=4 RBC units transfused over 24-weeks were reported. | Up to 24 weeks |
| Duration of MFSAF TSS Response | Duration of MFSAF TSS response is defined as the number of days from the start of the initial 28-day period in which a participant had a >= 50% reduction from Baseline TSS to the first day of the 7-day assessment that determines the mean TSS for the 28-day period during which the participants TSS equals or exceeds their Baseline value. | Up to a maximum of 151 weeks |
| Duration of TI Response | Duration of TI is defined as the number of days from (a) the first day of a 12-week period that satisfies the 12-week TI status definition, to (b) the first RBC transfusion or Hgb level < 8 g/dL (except in the case of clinically overt bleeding). | Up to a maximum of 151 weeks |
| Mean Cumulative Number of Whole Blood Units Transfused Over 24 Weeks | Cumulative transfusion risk was calculated as the estimated mean cumulative number of whole blood units transfused during the study. | Up to Week 24 |
| Percentage of Participants With Transfusion Dependence (TD) Status at Week 24 | TD status at Week 24 is defined as requirement of >=4 RBC units in an 8-week period immediately prior to the end of Week 24. | Week 24 |
| Percentage of Participants With a Hemoglobin Response | Hemoglobin responses are defined as increases of >= 1, >= 1.5, or >= 2 g/dL from Baseline in Hgb, as measured over a (rolling) period of at least 12 consecutive weeks falling entirely before the end of Week 24. Baseline was the last assessment done before or on the day of first dose date. Data has been reported for percentage of participants who had >= 1, >= 1.5, or >= 2 g/dL increase from Baseline in hemoglobin. | Baseline and Week 24 |
| Number of Baseline TD Participants With TI Status at Week 24 | Participants were defined as having TD if they met both of the following requirements in the 8 weeks immediately before the end of Week 24: >= 4 red blood cell or whole blood units were transfused (except in the case of clinically overt bleeding), each in response to a hemoglobin assessment of <= 9.5 g/dL; and there were >= 2 hemoglobin assessments with >= 28 days between the earliest and latest hemoglobin assessments. TI status was defined as not requiring red blood cell transfusion (except in the case of clinically overt bleeding) for >= 12 weeks immediately prior to the end of Week 24, with hemoglobin levels >= 8 g/dL. | Week 24 |
| Duration of TI in Baseline TD Participants | Duration of TI is defined as the number of days from (a) the first day of a 12-week period that satisfies the 12-week TI status definition, to (b) the first RBC transfusion or Hgb level < 8 g/dL (except in the case of clinically overt bleeding). | Up to a maximum of 151 weeks |
| Number of Participants With Serious Adverse Events (SAEs) and Non-serious Adverse Events (Non-SAEs)- up to Week 24 | An adverse event (AE) is any untoward medical occurrence in a trial participant administered an investigational product(s), a comparator product, or an approved drug regardless of the causal relationship with treatment. An SAE is an AE that Results in death, life threatening, requires inpatient hospitalization or prolongation of an existing hospitalization, results in persistent or significant disability or incapacity, results in a congenital anomaly/birth defect or any important medical events as per medical or scientific judgment. Adverse events which were not Serious were considered as Non-Serious adverse events. | Up to Week 24 |
| Number of Participants With Serious Adverse Events (SAEs) and Non-serious Adverse Events (Non-SAEs)- From Week 24 to a Maximum of 151 Weeks | An AE is any untoward medical occurrence in a trial participant administered an investigational product(s), a comparator product, or an approved drug regardless of the causal relationship with treatment. An SAE is an AE that Results in death, life threatening, requires inpatient hospitalization or prolongation of an existing hospitalization, results in persistent or significant disability or incapacity, results in a congenital anomaly/birth defect or any important medical events as per medical or scientific judgment. Adverse events which were not Serious were considered as Non-Serious adverse events. | From Week 24 to a maximum of 151 weeks |
| Overall Survival (OS) | Overall survival is defined as the interval from the first study drug dosing date (or randomization date for participants who did not receive treatment) to death from any cause. | Up to a maximum of 151 weeks |
| Leukemia-free Survival (LFS) | LFS is defined as the interval from first study drug dosing date (or randomization date for participants who did not receive treatment) to any evidence of leukemic transformation and/or death (from any cause). | Up to a maximum of 151 weeks |
| Change From Baseline in Disease-related Fatigue as Assessed by MFSAF TSS v4.0 at Week 24 | The MFSAF v4.0 comprises 7 domains representing the 7 most relevant symptoms of MF identified through existing participant- and clinician-based evidence: fatigue, night sweats, pruritus, abdominal discomfort, pain under the left ribs, early satiety, and bone pain. Participants scored each symptom domain using an 11-point numeric rating scale ranging from 0 (absent) to 10 (worst imaginable). Data has been reported for Disease-related Fatigue domain measured using an 11-point numeric rating scale ranging from 0 (absent) to 10 (worst imaginable), higher score indicates worst outcome. An increase in score from Baseline indicated a worsening of fatigue and a decrease in score from Baseline indicated an improvement in fatigue. Baseline was the last assessment done before or on the day of first dose date. Change from Baseline was defined as the post-Baseline value minus Baseline value. | Baseline and Week 24 |
| Change From Baseline in Cancer-related Fatigue as Assessed by European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) at Week 24 | The EORTC QLQ-C30 is comprised of 5 functional scales (physical, role, emotional, social, cognitive), eight single item symptom scales (fatigue, pain, nausea/vomiting, appetite loss, constipation, diarrhea, insomnia, dyspnea), as well as sub-scales assessing global health/quality of life and financial impact. Most items use a 4-point Likert scale from "not at all" to "very much" and a one-week recall period with the exception of the final two items which use a 7 point scale response from "very poor" to "excellent". Scores were averaged and transformed to a 0-100 scale, with higher scores representing better functioning/quality of life. An increase in scores from Baseline indicated an improved functioning/quality of life, and a decrease in scores from Baseline indicated a worsened functioning/quality of life. Baseline was the last assessment done before or on the day of first dose date. Change from Baseline was defined as the post-Baseline value minus Baseline value. | Baseline and Week 24 |
| Change From Baseline in Physical Function Score as Assessed by Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function Short Form 10b at Week 24 | PROMIS Physical Function Short Form 10b consists of 14 questions; each with a 5-point response. PROMIS short form assesses self-reported capability of a participant rather than actual performance of physical activities. This includes functioning of one's upper extremities (dexterity), lower extremities (walking or mobility), and central regions (neck, back) as well as instrumental activities of daily living, such as running errands. Participants scored each response on a scale from 1 (unable to do) to 5 (without any difficulty, or not at all). Total possible range of scores was 14 to 70, with higher scores corresponding to a greater physical function ability. An increase in score from Baseline indicated an improvement in physical function ability and a decrease in score from Baseline indicated a reduction in physical function ability. Baseline was last assessment done before or on the day of first dose date. Change from Baseline was defined as post-Baseline value minus Baseline value. | Baseline and Week 24 |
| Irvine |
| California |
| 92614 |
| United States |
| Norris Comprehensive Cancer Center | Los Angeles | California | 91011 | United States |
| American Institute of Research - Whittier | Whittier | California | 90603 | United States |
| University of Colorado Hospital Anschutz Cancer Pavilion | Aurora | Colorado | 80045 | United States |
| Georgetown University Medical Center | Washington D.C. | District of Columbia | 20057 | United States |
| Moffitt Cancer Center | Tampa | Florida | 33612 | United States |
| Northwest Oncology & Hematology - Rolling Meadows | Rolling Meadows | Illinois | 60008 | United States |
| Washington University School of Medicine in Saint Louis | St Louis | Missouri | 63110 | United States |
| Hackensack University Medical Center | Hackensack | New Jersey | 07601 | United States |
| Columbia University Irving Medical Center - Presbyterian Hospital | New York | New York | 10032 | United States |
| Cleveland Clinic - Richard E. Jacobs Health Center | Avon | Ohio | 44011 | United States |
| Gabrail Cancer Center | Canton | Ohio | 44718 | United States |
| University Hospitals Cleveland Medical Center | Cleveland | Ohio | 44106 | United States |
| Allegheny Health Network | Pittsburgh | Pennsylvania | 15224 | United States |
| The University of Texas MD Anderson Cancer Center | Houston | Texas | 77030 | United States |
| The University of Texas Health Science Center at San Antonio | San Antonio | Texas | 78229 | United States |
| Canberra Region Cancer Centre | Garran | Australian Capital Territory | 2605 | Australia |
| Calvary Mater Newcastle Hospital | Waratah | New South Wales | 2298 | Australia |
| Flinders Medical Centre | Bedford Park | South Australia | 5042 | Australia |
| The Alfred Hospital | Melbourne | Victoria | 3004 | Australia |
| Perth Radiological Clinic - Magnetic Resonance Centre | Perth | Western Australia | 6000 | Australia |
| Medizinische Universität Wien | Vienna | State of Vienna | 1090 | Austria |
| Medizinische Universität Innsbruck | Innsbruck | Tyrol | 6020 | Austria |
| Ordensklinikum Linz Elisabethinen | Linz | Upper Austria | 4020 | Austria |
| Oberösterreichische Gesundheitsholding GmbH | Steyr | 4400 | Austria |
| Hôpital Erasme | Brussels | Brussels Capital | 1070 | Belgium |
| Grand Hôpital de Charleroi - Notre Dame | Charleroi | Hainaut | 6000 | Belgium |
| Algemeen Ziekenhuis Sint-Jan Brugge-Oostende - Campus Sint-Jan | Bruges | West-Vlaanderen | 8000 | Belgium |
| Ziekenhuis Netwerk Antwerpen Stuivenberg | Antwerp | 2060 | Belgium |
| Centre Hospitalier Universitaire de Liège | Liège | B-4000 | Belgium |
| University Multiprofile Hospital For Active Treatment Dr. Georgi Stranski EAD | Pleven | 5800 | Bulgaria |
| University Hospital St. Ivan Rilski | Sofia | 1431 | Bulgaria |
| University Multiprofile Hospital For Active Treatment Aleksandrovska | Sofia | 1431 | Bulgaria |
| National Specialized Hospital for Active Treatment of Haematologic Diseases | Sofia | 1756 | Bulgaria |
| Saint Paul's Hospital | Vancouver | British Columbia | V6Z 1Y6 | Canada |
| Queen Elizabeth II Health Sciences Centre - Halifax Infirmary | Halifax | Nova Scotia | B3H 1V7 | Canada |
| McMaster University Medical Center | Hamilton | Ontario | L8V 5C2 | Canada |
| Princess Margaret Cancer Centre | Toronto | Ontario | M5G 2M9 | Canada |
| Research Institute of the McGill University Health Centre | Montreal | Quebec | H3H 2R9 | Canada |
| Hôpital de l'Enfant-Jésus | Québec | G1J 1Z4 | Canada |
| Fakultni Nemocnice Brno | Brno | Jihormoravsky Kraj | 625 00 | Czechia |
| Herlev Hospital | Herlev | Capital Region | 2730 | Denmark |
| Aalborg Universitetshospital - Syd | Aalborg | North Denmark | 9000 | Denmark |
| Sjællands Universitetshospital - Roskilde | Roskilde | Region Sjælland | 4000 | Denmark |
| Odense Universitetshospital | Odense | Region Syddanmark | 5000 | Denmark |
| Hôpital Haut-Lévêque | Pessac | Aquitaine | 33604 | France |
| Hospital Center University Of Caen Normandie | Caen | Basse-Normandie | 14033 | France |
| Hôpital Claude Huriez | Lille | Hauts-de-France | 59037 | France |
| Centre Hospitalier Universitaire Limoges | Limoges | Limousin | 87042 | France |
| Centre Hospitalier De Lens | Lens | Nord Pas-Des-Calais | 62307 | France |
| Centre Hospitalier Le Mans | Le Mans | Pays de la Loire Region | 72037 | France |
| Centre Hospitalier Universitaire Amiens-Picardie - Site Sud | Amiens | Picardie | 80054 | France |
| Hôpital l'Archet | Nice | Provence-Alpes-Côte d'Azur Region | 06200 | France |
| Centre Hospitalier Lyon-Sud | Pierre-Bénite | Rhone-Alps | 69495 | France |
| Centre Hosptitalier Universitaire Angers | Angers | 49 933 | France |
| Hôpital Saint-Vincent De Paul - Lille | Lille | 59000 | France |
| Hôpital De La Conception | Marseille | 13385 | France |
| Hôpital Emile Muller | Mulhouse | 68100 | France |
| Centre Hospitalier Universitaire Nantes - Hôtel Dieu | Nantes | 44000 | France |
| Centre Hospitalier Universitaire de Poitiers | Poitiers | 86021 | France |
| Hôpital Saint-Louis | Paris | Île-de-France Region | 75010 | France |
| Hôpital Saint-Antoine | Paris | Île-de-France Region | 75571 | France |
| Universitätsklinikum Aachen | Aachen | North Rhine-Westphalia | 52074 | Germany |
| Universitätsklinikum Essen | Essen | North Rhine-Westphalia | 45147 | Germany |
| Johannes Wesling Klinikum Minden | Minden | North Rhine-Westphalia | 32429 | Germany |
| Universitätsklinikum Carl Gustav Carus Dresden | Dresden | Saxony | 01307 | Germany |
| Universitätsklinikum Leipzig | Leipzig | Saxony | 04103 | Germany |
| Universitätsklinikum Halle | Halle | Saxony-Anhalt | 06120 | Germany |
| Universitätsklinikum Jena | Jena | Thuringia | 07747 | Germany |
| Uniklinik Köln | Cologne | 50937 | Germany |
| Universitätsklinikum Schleswig-Holstein - Campus Lübeck | Lübeck | 23538 | Germany |
| Kliniken Ostalb - Stauferklinikum Schwäbisch Gmünd | Mutlangen | 73557 | Germany |
| Pécsi Tudományegyetem Klinikai Központ | Pécs | Baranya | 7624 | Hungary |
| Debreceni Egyetem Klinikai Központ | Debrecen | Hajdú-Bihar | 4032 | Hungary |
| Szent Borbála Kórház | Tatabánya | Komárom-Esztergom | 2800 | Hungary |
| Dél-pesti Centrumkórház - Országos Hematológiai és Infektológiai Intézet - Szent László Telephely | Budapest | Pest County | 1097 | Hungary |
| Somogy Megyei Kaposi Mór Oktató Kórház | Kaposvár | Somogy County | 7400 | Hungary |
| Szabolcs-Szatmár-Bereg Megyei Kórházak És Egyetemi Oktatókórház | Nyíregyháza | Szabolcs-Szatmár-Bereg | 4400 | Hungary |
| Markusovszky Egyetemi Oktatókórház Szombathely | Szombathely | Vas County | 9700 | Hungary |
| Semmelweis Egyetem | Budapest | 1083 | Hungary |
| Petz Aladár Megyei Oktató Kórház | Győr | 9023 | Hungary |
| Yitzhak Shamir Medical Center | Be’er Ya‘aqov | Central District | 7030000 | Israel |
| Bnai Zion Medical Center | Haifa | Haifa District | 31048 | Israel |
| Hadassah University Hospital Ein Kerem | Jerusalem | Jerusalem | Israel |
| Rambam Health Care Campus | Haifa | 3109601 | Israel |
| Carmel Medical Center | Haifa | 3436212 | Israel |
| Shaare Zedek Medical Center | Jerusalem | 9103102 | Israel |
| Meir Medical Center | Kfar Saba | 4428164 | Israel |
| Western Galilee Hospital-Nahariya | Nahariya | Israel |
| Rabin Medical Center - Beilinson Hospital | Petah Tikva | 4941492 | Israel |
| Tel Aviv Sourasky Medical Center | Tel Aviv | Israel |
| Azienda Ospedaliero - Universitaria Careggi | Florence | Florence | 50134 | Italy |
| Ospedale Casa Sollievo della Sofferenza | San Giovanni Rotondo | Foggia | 71013 | Italy |
| Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori | Meldola | Forli-Cesena | 47014 | Italy |
| Azienda Socio Sanitaria Territoriale Monza - Ospedale San Gerardo | Monza | Monza E Brianza | 20900 | Italy |
| Azienda Ospedaliera Ospedali Riuniti Marche Nord | Pesaro | Pesaro E Urbino | 61121 | Italy |
| IRCCS Centro di Riferimento Oncologico di Basilicata | Rionero in Vulture | Potenza | 85028 | Italy |
| Azienda Ospedaliero-Universitaria Citta della Salute e della Scienza di Torino | Torino | Turin | 10126 | Italy |
| Azienda Ospedaliera Ordine Mauriziano di Torino | Torino | Turin | 10128 | Italy |
| Azienda Ospedaliera Nazionale SS. Antonio e Biagio e C. Arrigo - Alessandria | Alessandria | 15121 | Italy |
| Azienda Ospedaliero-Universitaria di Bologna Policlinico Sant Orsola-Malpighi | Bologna | 40138 | Italy |
| Ospedale Policlinico San Martino | Genova | 16132 | Italy |
| Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico | Milan | 20122 | Italy |
| Azienda Socio Sanitaria Territoriale Grande Ospedale Metropolitano Niguarda | Milan | 20162 | Italy |
| Azienda Ospedaliera Universitaria Federico II | Naples | 80131 | Italy |
| Azienda Ospedaliero-Universitaria Maggiore della Carità di Novara | Novara | 28100 | Italy |
| Umberto I - Policlinico di Roma | Roma | 00161 | Italy |
| Fondazione Policlinico Universitario Agostino Gemelli | Roma | 20123 | Italy |
| Presidio Ospedaliero Universitario Santa Maria della Misericordia | Udine | 33100 | Italy |
| Ospedale di Circolo e Fondazione Macchi | Varese | 21100 | Italy |
| Ospedale Policlinico Giambattista Rossi Borgo Roma | Verona | 37134 | Italy |
| North Shore Hospital | Auckland | 0622 | New Zealand |
| Middlemore Clinical Trials | Auckland | 2025 | New Zealand |
| Szpital Uniwersytecki w Krakowie | Krakow | Lesser Poland Voivodeship | 31-501 | Poland |
| Szpital Specjalistyczny im. Ludwika Rydygiera w Krakowie | Krakow | Lesser Poland Voivodeship | 31-826 | Poland |
| Klinika Hematologii Nowotworów Krwi i Transplantacji Szpiku | Wroclaw | Lower Silesian Voivodeship | 50-367 | Poland |
| Samodzielny Publiczny Szpital Kliniczny nr 1 w Lublinie | Lublin | Lublin Voivodeship | 20-081 | Poland |
| Instytut Hematologii I Transfuzjologii | Warsaw | Masovian Voivodeship | 02-776 | Poland |
| Alvamed Zakład Specjalistycznej Opieki Zdrowotnej | Warsaw | Masovian Voivodeship | 03-401 | Poland |
| Szpital Wojewódzki w Opolu | Opole | Opole Voivodeship | 45-064 | Poland |
| Uniwersyteckie Centrum Kliniczne w Gdańsku | Gdansk | Pomeranian Voivodeship | 80-214 | Poland |
| Silesian Healthy Blood Clinic | Chorzów | Salskie | 41-503 | Poland |
| Wojewódzkie Wielospecjalistyczne Centrum Onkologii i Traumatologii im. M. Kopernika w Łodzi | Lodz | 93-513 | Poland |
| Spitalul Filantropia - Craiova | Craiova | Dolj | 200143 | Romania |
| Institutul Regional De Oncologie Iasi | Iași | Iaşi | 700483 | Romania |
| Spitalul Clinic Judetean De Urgenta Târgu Mureș | Târgu Mureş | Mureș County | 540136 | Romania |
| Laboratul clinic MedLife-Policlinica de Diagnostic Rapid Brasov | Brasov | 500366 | Romania |
| Coltea - Spital Clinic | Bucharest | 030 171 | Romania |
| Singapore General Hospital | Singapore | 169608 | Singapore |
| Tan Tock Seng Hospital | Singapore | 308433 | Singapore |
| Kyungpook National University Hospital | Daegu | Daegu Gwang'yeogsi | 41944 | South Korea |
| Inje University Busan Paik Hospital | Busan | 47392 | South Korea |
| Inje University Haeundae Paik Hospital | Busan | 48108 | South Korea |
| Seoul National University Bundang Hospital | Seongnam-si | 13620 | South Korea |
| Severance Hospital | Seoul | 03722 | South Korea |
| The Catholic University of Korea Seoul Saint Mary's Hospital | Seoul | 06591 | South Korea |
| Samsung Medical Center | Seoul | South Korea |
| Seoul National University Hospital | Seoul | South Korea |
| Ulsan University Hospital | Ulsan | 44033 | South Korea |
| Hospital Universitario Central de Asturias | Oviedo | Principality of Asturias | 33011 | Spain |
| Institut Hospital del Mar d'Investigacions Mèdiques | Barcelona | 08003 | Spain |
| Hospital Universitari Vall d'Hebrón | Barcelona | 08035 | Spain |
| Hospital Germans Trias i Pujol | Barcelona | 08916 | Spain |
| Hospital San Pedro de Alcantara | Cáceres | 10003 | Spain |
| Institut Català d'Oncologia Girona | Girona | 17007 | Spain |
| Hospital Universitario Ramón y Cajal | Madrid | 28034 | Spain |
| Hospital Universitario 12 de Octubre | Madrid | 28041 | Spain |
| Hospital Universitario Virgen de la Victoria | Málaga | 29010 | Spain |
| Complejo Asistencial Universitario de Salamanca - Hospital Clínico | Salamanca | 37007 | Spain |
| Hospital Universitario Virgen del Rocío | Seville | 41013 | Spain |
| Hospital Universitario La Fe | Valencia | 46009 | Spain |
| Hospital de Día Quirónsalud Zaragoza | Zaragoza | 50012 | Spain |
| Karolinska Universitetssjukhuset Solna | Solna | Stockholm County | 171 76 | Sweden |
| Sahlgrenska Universitetssjukhuset | Gothenburg | Västra Götaland County | 413 45 | Sweden |
| Uddevalla Sjukhus | Uddevalla | 45153 | Sweden |
| Chiayi Chang Gung Memorial Hospital | Buzi | Chaiyi | 613 | Taiwan |
| China Medical University Hospital | Taichung | Taichung | 40447 | Taiwan |
| National Taiwan University Hospital | Taipei | 10002 | Taiwan |
| Chang Gung Memorial Hospital - Linkou Branch | Taoyuan | 333 | Taiwan |
| United Lincolnshire Hospitals NHS Trust | Boston | England | PE21 9QS | United Kingdom |
| University Hospitals Bristol NHS Foundation Trust | Bristol | England | BS2 8ED | United Kingdom |
| University College London Hospitals NHS Foundation Trust | London | England | NW1 2PG | United Kingdom |
| Guy's and Saint Thomas' NHS Foundation Trust | London | England | SE1 9RT | United Kingdom |
| Imperial College Healthcare NHS Trust | London | England | W12 0HS | United Kingdom |
| University Hospital Southampton NHS Foundation Trust | Southampton | England | SO16 6YD | United Kingdom |
| NHS Lanarkshire | Airdrie | Scotland | ML6 0JS | United Kingdom |
| Yoon SS, Chen CC, Lee SE, Chang H, Cheong JW, Hou HA, Lee WS, Lim SN, Moon JH, Ong KH, Dai Y, Liu C, Kawashima J, Goh YT. Efficacy and safety of momelotinib in Janus kinase inhibitor-experienced Asian patients with myelofibrosis and anemia. Int J Hematol. 2025 Nov;122(5):660-670. doi: 10.1007/s12185-025-04037-6. Epub 2025 Jul 21. |
| 40535755 | Derived | Mesa RA, Talpaz M, Mazerolle F, Gorsh B, M'Hari M, Regnault A, Ellis C, Wang Z, Purser M, Liu T, Strouse B, Patnaik D. Time Without Transfusion Reliance (TWiTR): Integrating Survival Quality Into Myelofibrosis Treatment Strategies Based on the Phase 3 SIMPLIFY-1, SIMPLIFY-2, and MOMENTUM Trials. EJHaem. 2025 Jun 18;6(3):e70075. doi: 10.1002/jha2.70075. eCollection 2025 Jun. |
| 39516087 | Derived | Harrison CN, Mesa R, Talpaz M, Gupta V, Gerds AT, Perkins A, Goh YT, Fox ML, McLornan D, Palmer J, Foltz L, Vannucchi A, Koschmieder S, Passamonti F, Lee SE, Ellis C, Strouse B, Gonzalez Carreras FJ, Oh ST. Longitudinal Assessment of Transfusion Intensity in Patients With JAK Inhibitor-Naive or -Experienced Myelofibrosis Treated With Momelotinib. Clin Lymphoma Myeloma Leuk. 2025 Mar;25(3):199-211. doi: 10.1016/j.clml.2024.10.001. Epub 2024 Oct 16. |
| 37517413 | Derived | Gerds AT, Verstovsek S, Vannucchi AM, Al-Ali HK, Lavie D, Kuykendall AT, Grosicki S, Iurlo A, Goh YT, Lazaroiu MC, Egyed M, Fox ML, McLornan D, Perkins A, Yoon SS, Gupta V, Kiladjian JJ, Granacher N, Lee SE, Ocroteala L, Passamonti F, Harrison CN, Oh S, Klencke BJ, Yu J, Donahue R, Kawashima J, Mesa R. Momelotinib versus danazol in symptomatic patients with anaemia and myelofibrosis previously treated with a JAK inhibitor (MOMENTUM): an updated analysis of an international, double-blind, randomised phase 3 study. Lancet Haematol. 2023 Sep;10(9):e735-e746. doi: 10.1016/S2352-3026(23)00174-6. Epub 2023 Jul 27. |
| 37042865 | Derived | Verstovsek S, Mesa R, Gupta V, Lavie D, Dubruille V, Cambier N, Platzbecker U, Hus M, Xicoy B, Oh ST, Kiladjian JJ, Vannucchi AM, Gerds A, Egyed M, Mayer J, Sacha T, Kawashima J, Morris M, Huang M, Harrison C. Momelotinib long-term safety and survival in myelofibrosis: integrated analysis of phase 3 randomized controlled trials. Blood Adv. 2023 Jul 25;7(14):3582-3591. doi: 10.1182/bloodadvances.2022009311. |
| 36709073 | Derived | Verstovsek S, Gerds AT, Vannucchi AM, Al-Ali HK, Lavie D, Kuykendall AT, Grosicki S, Iurlo A, Goh YT, Lazaroiu MC, Egyed M, Fox ML, McLornan D, Perkins A, Yoon SS, Gupta V, Kiladjian JJ, Granacher N, Lee SE, Ocroteala L, Passamonti F, Harrison CN, Klencke BJ, Ro S, Donahue R, Kawashima J, Mesa R; MOMENTUM Study Investigators. Momelotinib versus danazol in symptomatic patients with anaemia and myelofibrosis (MOMENTUM): results from an international, double-blind, randomised, controlled, phase 3 study. Lancet. 2023 Jan 28;401(10373):269-280. doi: 10.1016/S0140-6736(22)02036-0. |
| FG001 | DAN 300 mg BID + Placebo | Participants were randomized to receive 300 mg of DAN orally BID and MMB-placebo orally QD during the randomized 24-week double-blind treatment period. Participants who completed the randomized treatment period, discontinued treatment early due to splenic progression, or discontinued treatment early for other reasons but completed scheduled assessments through Week 24 enrolled in an open-label extended treatment period. Participants switched to receive 200 mg of MMB orally QD during open-label extended treatment period. All participants elected to receive MMB as open-label treatment during the open-label extended treatment period. |
| COMPLETED |
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| NOT COMPLETED |
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| Open-Label Extended TP(Weeks 24 to 151) |
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Baseline characteristics were measured in the Intention-To-Treat (ITT) Analysis Set, which included all randomized participants.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | MMB 200 mg QD + Placebo | Participants were randomized to receive 200 milligrams (mg) of MMB orally QD and a DAN-placebo orally twice daily (BID) during the randomized 24-week double-blind treatment period. Participants who completed the randomized treatment period or discontinued treatment early for other reasons but completed scheduled assessments through Week 24 were enrolled in an open-label extended treatment period. Participants continued to receive 200 mg of MMB orally QD during the open-label extended treatment period. Participants who discontinued treatment prior to Week 24 due to splenic progression or other reasons, but completed assessments, were unblinded. Participants who discontinued MMB during the randomized treatment period were not eligible to receive MMB in the open-label extended treatment period. Participants who were randomized to MMB during the randomized treatment period with confirmed splenic progression could not proceed to the open-label extended treatment period. |
| BG001 | DAN 300 mg BID + Placebo | Participants were randomized to receive 300 mg of DAN orally BID and MMB-placebo orally QD during the randomized 24-week double-blind treatment period. Participants who completed the randomized treatment period, discontinued treatment early due to splenic progression, or discontinued treatment early for other reasons but completed scheduled assessments through Week 24 were enrolled in an open-label extended treatment period. Participants who received DAN 300 mg BID during randomized period switched to receive 200 mg of MMB orally QD during open-label extended treatment period. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
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| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| 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 | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | Total Symptom Score (TSS) Response Rate at Week 24 | Myelofibrosis Symptom Assessment Form (MFSAF) TSS version (v) 4.0 response rate was defined as percentage of participants with a >= 50 percent (%) reduction from Baseline in mean MFSAF TSS over consecutive 28-day period immediately before end of Week 24. TSS response rate was measured using MFSAF v4.0. MFSAF v4.0 comprises 7 domains representing 7 most relevant symptoms of myelofibrosis (MF) identified through existing participant and clinician-based evidence: fatigue,night sweats,pruritus,abdominal discomfort,pain under left ribs,early satietyand bone pain. Participants scored each symptom domain using an 11-point numeric rating scale ranging from 0(absent) to 10(worst imaginable). The MFSAF TSS was calculated as sum of scores of 7 domains for a possible range of scores of 0 to 70, with a higher TSS corresponding to more severe symptoms. A reduction from Baseline corresponded to a lessening of MF symptoms. Baseline was the last assessment done before or on the day of first dose date. | Measured in the Intent-To-Treat (ITT) Analysis Set, which included all randomized participants. | Posted | Number | 95% Confidence Interval | Percentage of participants | Baseline and Week 24 |
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| Secondary | Percentage of Participants With Transfusion Independence (TI) at Week 24 | TI status was defined as not receiving red blood cell (RBC) or whole blood transfusion for >=12 weeks, with no hemoglobin (Hgb) level < 8 grams per deciliter (g/dL) during the same interval. Percentage of participants with TI have been presented. | ITT Analysis Set | Posted | Number | 95% Confidence Interval | Percentage of Participants | Week 24 |
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| Secondary | Splenic Response Rate (SRR) of >=25% at Week 24 | Splenic response rate (SRR) is defined as the percentage of participants who have reduction in spleen volume of >=25% from Baseline at the end of Week 24. Baseline was the last assessment done before or on the day of first dose date. | ITT Analysis Set | Posted | Number | 95% Confidence Interval | Percentage of Participants | Baseline and Week 24 |
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| Secondary | Change From Baseline in MFSAF TSS at Week 24 | TSS was measured using the MFSAF v4.0. The MFSAF v4.0 comprises 7 domains representing the 7 most relevant symptoms of MF identified through existing participant and clinician-based evidence: fatigue, night sweats, pruritus, abdominal discomfort, pain under the left ribs, early satiety, and bone pain. Participants scored each symptom domain using an 11-point numeric rating scale ranging from 0 (absent) to 10 (worst imaginable). The MFSAF TSS was calculated as the sum of scores of the 7 domains for a possible range of scores of 0 to 70, with a higher TSS corresponding to more severe symptoms. A reduction from Baseline corresponded to a lessening of MF symptoms. Baseline was the last assessment done before or on the day of first dose date. Change from Baseline was defined as the post-Baseline value minus Baseline value. | ITT Analysis Set. Only those participants with data available at specified time points were analyzed. | Posted | Least Squares Mean | Standard Error | Scores on a scale | Baseline and Week 24 |
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| Secondary | Splenic Response Rate (SRR) of >= 35% at Week 24 | Splenic response rate (SRR) is defined as the percentage of participants who have reduction in spleen volume of >=35 % from Baseline at the end of Week 24. Baseline was the last assessment done before or on the day of first dose date. | ITT Analysis Set | Posted | Number | 95% Confidence Interval | Percentage of Participants | Baseline and Week 24 |
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| Secondary | Percentage of Participants With Zero RBC Units Transfused Over 24-Weeks | Percentage of participants with zero RBC units transfused over 24-weeks were reported. | ITT Analysis Set | Posted | Number | 95% Confidence Interval | Percentage of participants | Up to 24 weeks |
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| Secondary | Percentage of Participants With <=4 RBC Units Transfused Over 24-weeks | Percentage of participants with <=4 RBC units transfused over 24-weeks were reported. | ITT Analysis Set | Posted | Number | 95% Confidence Interval | Percentage of participants | Up to 24 weeks |
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| Secondary | Duration of MFSAF TSS Response | Duration of MFSAF TSS response is defined as the number of days from the start of the initial 28-day period in which a participant had a >= 50% reduction from Baseline TSS to the first day of the 7-day assessment that determines the mean TSS for the 28-day period during which the participants TSS equals or exceeds their Baseline value. | ITT Analysis Set. Only those participants with data available at specified time point were analyzed. | Posted | Median | Inter-Quartile Range | Days | Up to a maximum of 151 weeks |
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| Secondary | Duration of TI Response | Duration of TI is defined as the number of days from (a) the first day of a 12-week period that satisfies the 12-week TI status definition, to (b) the first RBC transfusion or Hgb level < 8 g/dL (except in the case of clinically overt bleeding). | ITT Analysis Set. Only those participants with data available at specified time point were analyzed. | Posted | Median | Inter-Quartile Range | Days | Up to a maximum of 151 weeks |
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| Secondary | Mean Cumulative Number of Whole Blood Units Transfused Over 24 Weeks | Cumulative transfusion risk was calculated as the estimated mean cumulative number of whole blood units transfused during the study. | ITT Analysis Set. | Posted | Mean | Standard Deviation | Whole blood units | Up to Week 24 |
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| Secondary | Percentage of Participants With Transfusion Dependence (TD) Status at Week 24 | TD status at Week 24 is defined as requirement of >=4 RBC units in an 8-week period immediately prior to the end of Week 24. | ITT Analysis Set | Posted | Number | 95% Confidence Interval | Percentage of participants | Week 24 |
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| Secondary | Percentage of Participants With a Hemoglobin Response | Hemoglobin responses are defined as increases of >= 1, >= 1.5, or >= 2 g/dL from Baseline in Hgb, as measured over a (rolling) period of at least 12 consecutive weeks falling entirely before the end of Week 24. Baseline was the last assessment done before or on the day of first dose date. Data has been reported for percentage of participants who had >= 1, >= 1.5, or >= 2 g/dL increase from Baseline in hemoglobin. | ITT Analysis Set | Posted | Number | 95% Confidence Interval | Percentage of participants | Baseline and Week 24 |
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| Secondary | Number of Baseline TD Participants With TI Status at Week 24 | Participants were defined as having TD if they met both of the following requirements in the 8 weeks immediately before the end of Week 24: >= 4 red blood cell or whole blood units were transfused (except in the case of clinically overt bleeding), each in response to a hemoglobin assessment of <= 9.5 g/dL; and there were >= 2 hemoglobin assessments with >= 28 days between the earliest and latest hemoglobin assessments. TI status was defined as not requiring red blood cell transfusion (except in the case of clinically overt bleeding) for >= 12 weeks immediately prior to the end of Week 24, with hemoglobin levels >= 8 g/dL. | ITT Analysis Set. Only those participants with data available at specified time points were analyzed. | Posted | Count of Participants | Participants | Week 24 |
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| Secondary | Duration of TI in Baseline TD Participants | Duration of TI is defined as the number of days from (a) the first day of a 12-week period that satisfies the 12-week TI status definition, to (b) the first RBC transfusion or Hgb level < 8 g/dL (except in the case of clinically overt bleeding). | ITT Analysis Set. Only those participants with data available at specified time points were analyzed. | Posted | Median | Inter-Quartile Range | Days | Up to a maximum of 151 weeks |
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| Secondary | Number of Participants With Serious Adverse Events (SAEs) and Non-serious Adverse Events (Non-SAEs)- up to Week 24 | An adverse event (AE) is any untoward medical occurrence in a trial participant administered an investigational product(s), a comparator product, or an approved drug regardless of the causal relationship with treatment. An SAE is an AE that Results in death, life threatening, requires inpatient hospitalization or prolongation of an existing hospitalization, results in persistent or significant disability or incapacity, results in a congenital anomaly/birth defect or any important medical events as per medical or scientific judgment. Adverse events which were not Serious were considered as Non-Serious adverse events. | Safety analysis set included all participants in the ITT Analysis set who received at least one dose of study drug. | Posted | Count of Participants | Participants | Up to Week 24 |
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| Secondary | Number of Participants With Serious Adverse Events (SAEs) and Non-serious Adverse Events (Non-SAEs)- From Week 24 to a Maximum of 151 Weeks | An AE is any untoward medical occurrence in a trial participant administered an investigational product(s), a comparator product, or an approved drug regardless of the causal relationship with treatment. An SAE is an AE that Results in death, life threatening, requires inpatient hospitalization or prolongation of an existing hospitalization, results in persistent or significant disability or incapacity, results in a congenital anomaly/birth defect or any important medical events as per medical or scientific judgment. Adverse events which were not Serious were considered as Non-Serious adverse events. | Safety analysis set | Posted | Count of Participants | Participants | From Week 24 to a maximum of 151 weeks |
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| Secondary | Overall Survival (OS) | Overall survival is defined as the interval from the first study drug dosing date (or randomization date for participants who did not receive treatment) to death from any cause. | ITT Analysis Set. Values are presented based on the Kaplan-Meier analysis. All participants (overall population) were included in analysis. | Posted | Median | Inter-Quartile Range | Days | Up to a maximum of 151 weeks |
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| Secondary | Leukemia-free Survival (LFS) | LFS is defined as the interval from first study drug dosing date (or randomization date for participants who did not receive treatment) to any evidence of leukemic transformation and/or death (from any cause). | ITT Analysis Set. Values are presented based on the Kaplan-Meier analysis. All participants (overall population) were included in analysis. | Posted | Median | Inter-Quartile Range | Days | Up to a maximum of 151 weeks |
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| Secondary | Change From Baseline in Disease-related Fatigue as Assessed by MFSAF TSS v4.0 at Week 24 | The MFSAF v4.0 comprises 7 domains representing the 7 most relevant symptoms of MF identified through existing participant- and clinician-based evidence: fatigue, night sweats, pruritus, abdominal discomfort, pain under the left ribs, early satiety, and bone pain. Participants scored each symptom domain using an 11-point numeric rating scale ranging from 0 (absent) to 10 (worst imaginable). Data has been reported for Disease-related Fatigue domain measured using an 11-point numeric rating scale ranging from 0 (absent) to 10 (worst imaginable), higher score indicates worst outcome. An increase in score from Baseline indicated a worsening of fatigue and a decrease in score from Baseline indicated an improvement in fatigue. Baseline was the last assessment done before or on the day of first dose date. Change from Baseline was defined as the post-Baseline value minus Baseline value. | ITT Analysis Set. Only those participants with data available at specified time points were analyzed. | Posted | Least Squares Mean | Standard Error | Scores on a scale | Baseline and Week 24 |
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| Secondary | Change From Baseline in Cancer-related Fatigue as Assessed by European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) at Week 24 | The EORTC QLQ-C30 is comprised of 5 functional scales (physical, role, emotional, social, cognitive), eight single item symptom scales (fatigue, pain, nausea/vomiting, appetite loss, constipation, diarrhea, insomnia, dyspnea), as well as sub-scales assessing global health/quality of life and financial impact. Most items use a 4-point Likert scale from "not at all" to "very much" and a one-week recall period with the exception of the final two items which use a 7 point scale response from "very poor" to "excellent". Scores were averaged and transformed to a 0-100 scale, with higher scores representing better functioning/quality of life. An increase in scores from Baseline indicated an improved functioning/quality of life, and a decrease in scores from Baseline indicated a worsened functioning/quality of life. Baseline was the last assessment done before or on the day of first dose date. Change from Baseline was defined as the post-Baseline value minus Baseline value. | ITT Analysis Set. Only those participants with data available at specified time points were analyzed. | Posted | Least Squares Mean | Standard Error | Scores on a scale | Baseline and Week 24 |
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| Secondary | Change From Baseline in Physical Function Score as Assessed by Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function Short Form 10b at Week 24 | PROMIS Physical Function Short Form 10b consists of 14 questions; each with a 5-point response. PROMIS short form assesses self-reported capability of a participant rather than actual performance of physical activities. This includes functioning of one's upper extremities (dexterity), lower extremities (walking or mobility), and central regions (neck, back) as well as instrumental activities of daily living, such as running errands. Participants scored each response on a scale from 1 (unable to do) to 5 (without any difficulty, or not at all). Total possible range of scores was 14 to 70, with higher scores corresponding to a greater physical function ability. An increase in score from Baseline indicated an improvement in physical function ability and a decrease in score from Baseline indicated a reduction in physical function ability. Baseline was last assessment done before or on the day of first dose date. Change from Baseline was defined as post-Baseline value minus Baseline value. | ITT Analysis Set. Only those participants with data available at specified time points were analyzed. | Posted | Least Squares Mean | Standard Error | Scores on a scale | Baseline and Week 24 |
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All-cause mortality, Serious adverse events (SAEs) and non-SAEs were collected up to Week 24 for Randomized Double-Blind Treatment Period and From Week 24 to a maximum of 151 weeks for the Open-Label Extended Treatment Period
Serious adverse events (SAEs) and non-SAEs were measured in the Safety analysis set, which included all participants in the ITT analysis set who received at least one dose of study drug. All-cause mortality was measured in the ITT analysis set, which included all randomized participants. Data are presented per treatment received.
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 | MMB 200 mg QD + Placebo- Randomized Double-Blind Treatment Period | Participants were randomized to receive 200 mg of MMB orally QD and a DAN-placebo orally BID during the randomized 24-week double-blind treatment period. | 25 | 130 | 45 | 130 | 108 | 130 |
| EG001 | DAN 300 mg BID + Placebo- Randomized Double-Blind Treatment Period | Participants were randomized to receive 300 mg of DAN orally BID and MMB-placebo orally QD during the randomized 24-week double-blind treatment period. | 16 | 65 | 26 | 65 | 55 | 65 |
| EG002 | MMB 200 mg QD- Open-Label Extended Treatment Period | Participants who completed the randomized treatment period or discontinued treatment early for other reasons but completed scheduled assessments through Week 24 were enrolled in an open-label extended treatment period. Participants continued to receive 200 mg of MMB orally QD during the open-label extended treatment period. | 23 | 93 | 30 | 93 | 57 | 93 |
| EG003 | DAN 300 mg BID to MMB 200 mg QD- Open-Label Extended Treatment Period | Participants who completed the randomized treatment period, discontinued treatment early due to splenic progression, or discontinued treatment early for other reasons but completed scheduled assessments through Week 24 were enrolled in an open-label extended treatment period. Participants who received DAN 300 mg BID during randomized period switched to receive 200 mg of MMB orally QD during open-label extended treatment period. | 8 | 41 | 12 | 41 | 28 | 41 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA v24.0 | Systematic Assessment |
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| Blood loss anaemia | Blood and lymphatic system disorders | MedDRA v24.0 | Systematic Assessment |
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| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA v24.0 | Systematic Assessment |
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| Splenic haematoma | Blood and lymphatic system disorders | MedDRA v24.0 | Systematic Assessment |
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| Splenic infarction | Blood and lymphatic system disorders | MedDRA v24.0 | Systematic Assessment |
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| Splenic vein thrombosis | Blood and lymphatic system disorders | MedDRA v24.0 | Systematic Assessment |
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| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA v24.0 | Systematic Assessment |
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| Acute myocardial infarction | Cardiac disorders | MedDRA v24.0 | Systematic Assessment |
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| Atrial fibrillation | Cardiac disorders | MedDRA v24.0 | Systematic Assessment |
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| Cardiogenic shock | Cardiac disorders | MedDRA v24.0 | Systematic Assessment |
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| Coronary artery disease | Cardiac disorders | MedDRA v24.0 | Systematic Assessment |
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| Coronary artery stenosis | Cardiac disorders | MedDRA v24.0 | Systematic Assessment |
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| Myocardial ischaemia | Cardiac disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Inappropriate antidiuretic hormone secretion | Endocrine disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Gastric ulcer perforation | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Haematochezia | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Haemoperitoneum | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Melaena | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Oesophageal varices haemorrhage | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Small intestinal obstruction | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Upper gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Death | General disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Disease progression | General disorders | MedDRA v24.0 | Systematic Assessment |
| |
| General physical health deterioration | General disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Sudden death | General disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Cholangitis acute | Hepatobiliary disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Cholecystitis acute | Hepatobiliary disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Hepatitis | Hepatobiliary disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Hepatotoxicity | Hepatobiliary disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Hypersensitivity | Immune system disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Biliary sepsis | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| COVID-19 pneumonia | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Clostridium difficile colitis | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Cystitis | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Enterococcal sepsis | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Escherichia sepsis | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Joint abscess | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Listeria sepsis | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Pyelonephritis acute | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Septic shock | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Staphylococcal sepsis | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Streptococcal bacteraemia | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Tooth abscess | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Cervical vertebral fracture | Injury, poisoning and procedural complications | MedDRA v24.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA v24.0 | Systematic Assessment |
| |
| Femur fracture | Injury, poisoning and procedural complications | MedDRA v24.0 | Systematic Assessment |
| |
| Subdural haematoma | Injury, poisoning and procedural complications | MedDRA v24.0 | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA v24.0 | Systematic Assessment |
| |
| General physical condition abnormal | Investigations | MedDRA v24.0 | Systematic Assessment |
| |
| Liver function test increased | Investigations | MedDRA v24.0 | Systematic Assessment |
| |
| Fluid overload | Metabolism and nutrition disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Tenosynovitis | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Acute myeloid leukaemia | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA v24.0 | Systematic Assessment |
| |
| Leukaemia cutis | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA v24.0 | Systematic Assessment |
| |
| Metastatic malignant melanoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA v24.0 | Systematic Assessment |
| |
| Tongue neoplasm malignant stage unspecified | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA v24.0 | Systematic Assessment |
| |
| Transformation to acute myeloid leukaemia | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA v24.0 | Systematic Assessment |
| |
| Cerebrovascular accident | Nervous system disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Subarachnoid haemorrhage | Nervous system disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Delirium | Psychiatric disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Chronic kidney disease | Renal and urinary disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Renal failure | Renal and urinary disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Prostatitis | Reproductive system and breast disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Pneumonia aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Aortic stenosis | Vascular disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Circulatory collapse | Vascular disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Shock haemorrhagic | Vascular disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Arrhythmia | Cardiac disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Atrial thrombosis | Cardiac disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Cardiac arrest | Cardiac disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Cardiac failure | Cardiac disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Cardiac failure chronic | Cardiac disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Cardiac failure congestive | Cardiac disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Myocardial infarction | Cardiac disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Enteritis | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Haemorrhagic erosive gastritis | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Complication associated with device | General disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Multiple organ dysfunction syndrome | General disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Abscess limb | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Endocarditis | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Enterobacter sepsis | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Escherichia infection | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Gastroenteritis rotavirus | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Infective exacerbation of chronic obstructive airways disease | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Periorbital cellulitis | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Pneumonia bacterial | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Pneumonia influenzal | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Pneumonia staphylococcal | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Pulmonary sepsis | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Splenic abscess | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Urosepsis | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Lower limb fracture | Injury, poisoning and procedural complications | MedDRA v24.0 | Systematic Assessment |
| |
| Periprosthetic fracture | Injury, poisoning and procedural complications | MedDRA v24.0 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Sarcopenia | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Adenocarcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA v24.0 | Systematic Assessment |
| |
| Basal cell carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA v24.0 | Systematic Assessment |
| |
| Squamous cell carcinoma of skin | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA v24.0 | Systematic Assessment |
| |
| Cerebral haemorrhage | Nervous system disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Personality change | Psychiatric disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Pulmonary arterial hypertension | Respiratory, thoracic and mediastinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Pulmonary oedema | Respiratory, thoracic and mediastinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Restrictive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Haematoma | Vascular disorders | MedDRA v24.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA v24.0 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA v24.0 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA v24.0 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA v24.0 | Systematic Assessment |
| |
| Blood alkaline phosphatase increased | Investigations | MedDRA v24.0 | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA v24.0 | Systematic Assessment |
| |
| Platelet count decreased | Investigations | MedDRA v24.0 | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA v24.0 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Hyperuricaemia | Metabolism and nutrition disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Night sweats | Skin and subcutaneous tissue disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Systematic Assessment |
|
GlaxoSmithKline (GSK) agreements may vary with individual investigators, but will not prohibit any investigator from publishing. GSK supports the publication of results from all centers of a multi-center trial but requests that reports based on single site data not precede the primary publication of the entire clinical trial.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| GSK Response Center | Sierra Oncology LLC - a GSK company | 866-435-7343 | GSKClinicalSupportHD@gsk.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Dec 30, 2021 | Dec 1, 2022 | SAP_001.pdf |
| ID | Term |
|---|---|
| D055728 | Primary Myelofibrosis |
| D000740 | Anemia |
| D009196 | Myeloproliferative Disorders |
| D001855 | Bone Marrow Diseases |
| D006402 | Hematologic Diseases |
| ID | Term |
|---|---|
| D006425 | Hemic and Lymphatic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C546012 | N-(cyanomethyl)-4-(2-((4-(4-morpholinyl)phenyl)amino)-4-pyrimidinyl)benzamide |
| D003613 | Danazol |
| ID | Term |
|---|---|
| D011245 | Pregnadienes |
| D011278 | Pregnanes |
| D013256 | Steroids |
| D000072473 | Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
Not provided
Not provided
| Lack of Efficacy |
|
| Physician Decision |
|
| Withdrawal by Subject |
|
| Leukemic transformation |
|
| Disease progression |
|
| Continuing in MMB extension study |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
Participants were randomized to receive 300 mg of DAN orally BID and MMB-placebo orally QD during the randomized 24-week double-blind treatment period. Participants who completed the randomized treatment period, discontinued treatment early due to splenic progression, or discontinued treatment early for other reasons but completed scheduled assessments through Week 24 were enrolled in an open-label extended treatment period. Participants who received DAN 300 mg BID during randomized period switched to receive 200 mg of MMB orally QD during open-label extended treatment period.
|
|
|
Participants were randomized to receive 300 mg of DAN orally BID and MMB-placebo orally QD during the randomized 24-week double-blind treatment period. Participants who completed the randomized treatment period, discontinued treatment early due to splenic progression, or discontinued treatment early for other reasons but completed scheduled assessments through Week 24 were enrolled in an open-label extended treatment period. Participants who received DAN 300 mg BID during randomized period switched to receive 200 mg of MMB orally QD during open-label extended treatment period.
|
|
|
| OG001 | DAN 300 mg BID + Placebo | Participants were randomized to receive 300 mg of DAN orally BID and MMB-placebo orally QD during the randomized 24-week double-blind treatment period. Participants who completed the randomized treatment period, discontinued treatment early due to splenic progression, or discontinued treatment early for other reasons but completed scheduled assessments through Week 24 were enrolled in an open-label extended treatment period. Participants who received DAN 300 mg BID during randomized period switched to receive 200 mg of MMB orally QD during open-label extended treatment period. |
|
|
|
Participants were randomized to receive 300 mg of DAN orally BID and MMB-placebo orally QD during the randomized 24-week double-blind treatment period. Participants who completed the randomized treatment period, discontinued treatment early due to splenic progression, or discontinued treatment early for other reasons but completed scheduled assessments through Week 24 were enrolled in an open-label extended treatment period. Participants who received DAN 300 mg BID during randomized period switched to receive 200 mg of MMB orally QD during open-label extended treatment period.
|
|
|
|
|
|
|
|
|
| DAN 300 mg BID + Placebo |
Participants were randomized to receive 300 mg of DAN orally BID and MMB-placebo orally QD during the randomized 24-week double-blind treatment period. Participants who completed the randomized treatment period, discontinued treatment early due to splenic progression, or discontinued treatment early for other reasons but completed scheduled assessments through Week 24 were enrolled in an open-label extended treatment period. Participants who received DAN 300 mg BID during randomized period switched to receive 200 mg of MMB orally QD during open-label extended treatment period. |
|
|
Participants were randomized to receive 300 mg of DAN orally BID and MMB-placebo orally QD during the randomized 24-week double-blind treatment period. Participants who completed the randomized treatment period, discontinued treatment early due to splenic progression, or discontinued treatment early for other reasons but completed scheduled assessments through Week 24 were enrolled in an open-label extended treatment period. Participants who received DAN 300 mg BID during randomized period switched to receive 200 mg of MMB orally QD during open-label extended treatment period.
|
|
|
|
|
|
|
|
| OG001 |
| DAN 300 mg BID + Placebo |
Participants were randomized to receive 300 mg of DAN orally BID and MMB-placebo orally QD during the randomized 24-week double-blind treatment period. Participants who completed the randomized treatment period, discontinued treatment early due to splenic progression, or discontinued treatment early for other reasons but completed scheduled assessments through Week 24 were enrolled in an open-label extended treatment period. Participants who received DAN 300 mg BID during randomized period switched to receive 200 mg of MMB orally QD during open-label extended treatment period. |
|
|
|
| OG001 | DAN 300 mg BID + Placebo | Participants were randomized to receive 300 mg of DAN orally BID and MMB-placebo orally QD during the randomized 24-week double-blind treatment period. Participants who completed the randomized treatment period, discontinued treatment early due to splenic progression, or discontinued treatment early for other reasons but completed scheduled assessments through Week 24 were enrolled in an open-label extended treatment period. Participants who received DAN 300 mg BID during randomized period switched to receive 200 mg of MMB orally QD during open-label extended treatment period. |
|
|
Participants were randomized to receive 300 mg of DAN orally BID and MMB-placebo orally QD during the randomized 24-week double-blind treatment period. Participants who completed the randomized treatment period, discontinued treatment early due to splenic progression, or discontinued treatment early for other reasons but completed scheduled assessments through Week 24 were enrolled in an open-label extended treatment period. Participants who received DAN 300 mg BID during randomized period switched to receive 200 mg of MMB orally QD during open-label extended treatment period. |
|
|
|
|
|
|
Participants were randomized to receive 300 mg of DAN orally BID and MMB-placebo orally QD during the randomized 24-week double-blind treatment period. Participants who completed the randomized treatment period, discontinued treatment early due to splenic progression, or discontinued treatment early for other reasons but completed scheduled assessments through Week 24 were enrolled in an open-label extended treatment period. Participants who received DAN 300 mg BID during randomized period switched to receive 200 mg of MMB orally QD during open-label extended treatment period.
|
|
|
Participants were randomized to receive 300 mg of DAN orally BID and MMB-placebo orally QD during the randomized 24-week double-blind treatment period. Participants who completed the randomized treatment period, discontinued treatment early due to splenic progression, or discontinued treatment early for other reasons but completed scheduled assessments through Week 24 were enrolled in an open-label extended treatment period. Participants who received DAN 300 mg BID during randomized period switched to receive 200 mg of MMB orally QD during open-label extended treatment period. |
|
|
|
| OG001 | DAN 300 mg BID + Placebo | Participants were randomized to receive 300 mg of DAN orally BID and MMB-placebo orally QD during the randomized 24-week double-blind treatment period. Participants who completed the randomized treatment period, discontinued treatment early due to splenic progression, or discontinued treatment early for other reasons but completed scheduled assessments through Week 24 were enrolled in an open-label extended treatment period. Participants who received DAN 300 mg BID during randomized period switched to receive 200 mg of MMB orally QD during open-label extended treatment period. |
|
|
|
| OG001 | DAN 300 mg BID + Placebo | Participants were randomized to receive 300 mg of DAN orally BID and MMB-placebo orally QD during the randomized 24-week double-blind treatment period. Participants who completed the randomized treatment period, discontinued treatment early due to splenic progression, or discontinued treatment early for other reasons but completed scheduled assessments through Week 24 were enrolled in an open-label extended treatment period. Participants who received DAN 300 mg BID during randomized period switched to receive 200 mg of MMB orally QD during open-label extended treatment period. |
|
|
|
| OG001 | DAN 300 mg BID + Placebo | Participants were randomized to receive 300 mg of DAN orally BID and MMB-placebo orally QD during the randomized 24-week double-blind treatment period. Participants who completed the randomized treatment period, discontinued treatment early due to splenic progression, or discontinued treatment early for other reasons but completed scheduled assessments through Week 24 were enrolled in an open-label extended treatment period. Participants who received DAN 300 mg BID during randomized period switched to receive 200 mg of MMB orally QD during open-label extended treatment period. |
|
|
|