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| ID | Type | Description | Link |
|---|---|---|---|
| 2023-507954-34-00 | Registry Identifier | EU CT Number | |
| CPI 0610-04 | Other Identifier | Constellation Pharmaceuticals |
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A Phase 3, randomized, blinded study comparing pelabresib (CPI-0610) and ruxolitinib with placebo and ruxolitinib in myelofibrosis (MF) patients that have not been previously treated with Janus kinase inhibitors (JAKi). Pelabresib is a small molecule inhibitor of bromodomain and extra-terminal (BET) proteins.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Pelabresib + ruxolitinib | Experimental | Pelabresib monohydrate tablets + ruxolitinib phosphate tablets |
|
| Placebo + ruxolitinib | Active Comparator | Matching placebo tablets + ruxolitinib phosphate tablets |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Pelabresib | Drug | Double-blind treatment (pelabresib or matching placebo) will be administered daily for 14 consecutive days followed by a 7-day break, which is considered 1 cycle of treatment (1 cycle = 21 days). Pelabresib is a small molecule inhibitor of BET proteins with a novel mechanism of action and potential for disease-modifying effects in MF. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Splenic Response by Central Radiology Reads at Week 24 | Splenic response was characterized by a reduction of at least 35% in spleen volume from baseline (SVR35), as determined by magnetic resonance imaging (MRI) or computerized tomography (CT), and evaluated through a blinded central radiology review at Week 24. | Week 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Key Secondary: Absolute Change From Baseline in Total Symptom Score (TSS) at Week 24 | The Total Symptom Score (TSS) at Week 24, compared to baseline, was measured using the Myelofibrosis Symptom Assessment Form v4.0. This score represented the sum of seven individual symptom items, each rated on a 0-10 scale, resulting in a possible total daily score ranging from 0 to 70. A higher TSS reflected a greater disease burden and therefore a worse outcome. The baseline TSS was calculated as the average of non-missing daily total symptom scores over the seven-day period preceding the day of randomization. The TSS for each treatment week was determined as the average of non-missing daily total symptom scores for that week. However, if fewer than four daily scores were available for a given week, the weekly TSS was considered missing. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | 35294-3300 | United States | ||
| Ironwood Physicians P.C. dba Ironwood Cancer and Research Centers |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 35950489 | Result | Harrison CN, Gupta VK, Gerds AT, Rampal R, Verstovsek S, Talpaz M, Kiladjian JJ, Mesa R, Kuykendall AT, Vannucchi AM, Palandri F, Grosicki S, Devos T, Jourdan E, Wondergem MJ, Al-Ali HK, Buxhofer-Ausch V, Alvarez-Larran A, Patriarca A, Kremyanskaya M, Mead AJ, Akhani S, Sheikine Y, Colak G, Mascarenhas J. Phase III MANIFEST-2: pelabresib + ruxolitinib vs placebo + ruxolitinib in JAK inhibitor treatment-naive myelofibrosis. Future Oncol. 2022 Sep;18(27):2987-2997. doi: 10.2217/fon-2022-0484. Epub 2022 Aug 11. | |
| 40065169 |
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Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
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A total of 135 centers enrolled patients in the study in Australia, Austria, Belgium, Canada, Czech Republic, France, Germany, Greece, Hong Kong, Hungary, Israel, Italy, Malaysia, Netherlands, Poland, South Korea, Spain, Taiwan, Thailand, Turkey, United Kingdom, and United States.
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| ID | Title | Description |
|---|---|---|
| FG000 | Pelabresib + Ruxolitinib (Experimental Arm) | Pelabresib 125 mg orally (PO) once daily (QD) + ruxolitinib 10 or 15 mg orally (PO) twice a day (BID) for 14 consecutive days followed by ruxolitinib 10 or 15 mg orally (PO) twice a day (BID) for 7 days in a 21-day cycle |
| FG001 | Placebo + Ruxolitinib (Control Arm) |
| 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 | Oct 4, 2023 | Aug 14, 2024 |
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This study has a double-blind design in which patients and investigators are blinded to study drug; study drugs will be packaged identically. All patients will be randomly assigned to either treatment group in a 1:1 ratio. The blind should only be broken in the case of emergency.
|
| Ruxolitinib | Drug | Ruxolitinib is a JAK inhibitor and a current, approved treatment option for MF. |
|
| Placebo | Drug | Placebo tablets are designed to match pelabresib tablets. Each placebo tablet contains no active pharmaceutical ingredient and is visibly identical to experimental drug in size, shape, and packaging. Placebo dosing follows the same dosing conventions as pelabresib. |
|
| Baseline, Week 24 |
| Key Secondary: Number of Participants With TSS50 Response at Week 24 | The probability of a TSS response at Week 24 was estimated by calculating the TSS response rate, defined as the percentage of patients who achieved a TSS50 response at Week 24 in each of the two treatment groups. The Total Symptom Score (TSS) response was defined as a ≥50% reduction from baseline in TSS, as measured by the Myelofibrosis Symptom Assessment Form v4.0. | Week 24 |
| Percent Change From Baseline in Total Symptom Score (TSS) at Week 24 | Percent Change From Baseline in Total Symptom Score (TSS) at Week 24 measured the change in a patient's symptoms after 24 weeks of treatment, relative to baseline. A negative value indicates symptom improvement, and a reduction of ≥50% is typically considered a meaningful clinical response. | Baseline, Week 24 |
| Number of Participants With Improvement From Baseline in Bone Marrow Fibrosis of at Least 1 Grade at Week 24 | Improvement in bone marrow fibrosis by at least 1 grade, as assessed by central read compared to baseline, was analyzed by treatment group and overall. The improvement in bone marrow fibrosis grade was defined as a decrease by at least 1 grade in bone marrow fibrosis grade when compared to baseline, where a grade of MF-3 was the most severe, and MF-0 was the least severe. | Baseline, Week 24 |
| Number of Participants With Splenic Response by Central Radiology Reads at Week 48 | Splenic response is characterized by a reduction of at least 35% in spleen volume from baseline (SVR35), as determined by magnetic resonance imaging (MRI) or computerized tomography (CT), and evaluated through a blinded central radiology review at Week 48. | Week 48 |
| Number of Participants With TSS50 Response at Week 48 | The probability of a TSS response at Week 48 is estimated by calculating the TSS response rate, defined as the percentage of patients who achieve a TSS50 response at Week 48 in each of the two treatment groups. The Total Symptom Score (TSS) response is defined as a ≥50% reduction from baseline in TSS, as measured by the Myelofibrosis Symptom Assessment Form v4.0. | Week 48 |
| Absolute Change From Baseline in Total Symptom Score (TSS) at Week 48 | The Total Symptom Score (TSS) at Week 48, compared to baseline, is measured using the Myelofibrosis Symptom Assessment Form v4.0. This score represents the sum of seven individual symptom items, each rated on a 0-10 scale, resulting in a possible total daily score ranging from 0 to 70. A higher TSS reflects a greater disease burden and therefore a worse outcome. The baseline TSS is calculated as the average of non-missing daily total symptom scores over the seven-day period preceding the day of randomization. The TSS for each treatment week is determined as the average of non-missing daily total symptom scores for that week. However, if fewer than four daily scores are available for a given week, the weekly TSS is considered missing. | Baseline, Week 48 |
| Rate of Red Blood Cell (RBC) Transfusion Over First 24 Weeks of Treatment | The rate of RBC transfusions was defined as the average number of RBC units transfused per patient month (4 weeks) during the first 24 weeks of treatment. The average number of RBC units per patient-month was calculated by dividing the total (ie, for all patients) number of RBC units in the whole exposure time by the sum of patient-months. | Week 24 |
| Number of Participants Who Transitioned From Red Blood Cell (RBC) Transfusion Dependence to Transfusion Independence | Transfusion dependence (TD) was defined as having received ≥6 units of RBCs during the 12-week baseline period prior to dosing and Transfusion independence (TI) was defined as the absence of RBC transfusions during any continuous 12-week period of the double-blind treatment phase. The conversion from TD to TI was evaluated and defined as the proportion of patients who transitioned from transfusion dependence to transfusion independence. Patients who remained in the double-blind treatment period and had not received any RBC transfusions during the most recent 12 weeks were considered responders. Patients who discontinued from the double-blind treatment before Week 12 were classified as non-responders. | From 12-week baseline period prior to dosing to any 12-week period post baseline, up to 24 weeks |
| Category Change From Baseline of Patient Global Impression of Change (PGIC) at Week 24 | The Patient Global Impression of Change (PGIC) was a single-item measure of the patient's perceived change in MF symptoms since starting treatment. Patients responded to: "Since beginning this study treatment, your myelofibrosis symptoms were: (1) Very much improved, (2) Much improved, (3) Minimally improved, (4) No change, (5) Minimally worse, (6) Much worse, (7) Very much worse." | Baseline, Week 24 |
| Progression-Free Survival (PFS) | Progression-Free Survival (PFS), defined as the time from randomization until documented progression, or until death from any cause for patients without documented progression | Through study completion, an average of 6 years |
| Overall Survival (OS) | OS, defined as the time from randomization until death from any cause | Through study completion, an average of 6 years |
| Proportion of Patients With Transformation to Blast Phase (AML) | Patients are categorized as having transformed to Acute Myelogenous Leukemia (AML) when the peripheral blood blast percentage increases to ≥20% and this elevation persists for at least two weeks, or when leukemic transformation is confirmed through disease status assessment. | Through study completion, an average of 6 years |
| Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | A treatment-emergent adverse event (TEAE) for the double-blind treatment period is defined as an AE that has a start date on or after the first dose of the pelabresib/placebo and before 30 days after the last dose of pelabresib/placebo or before the start of alternative (off-study) treatment for MF, whichever occurs first. If the AE has a start date before the date of first dose but increases in severity after first dose and before 30 days post last dose will be considered a TEAE as well. An AE that occurs after the administration of the first dose of open-label pelabresib treatment will be considered treatment-emergent for the crossover treatment period. However, a TEAE for the crossover treatment period that occurs within 30 days after the last dose of pelabresib/placebo will be considered treatment emergent for the double-blind treatment period as well. | Through study completion, an average of 6 years |
| Pharmacokinetic (PK) Parameters for Pelabresib: Area Under the Plasma Concentration-time Curve From Time Zero to Time t (AUC0-t) | Pharmacokinetic (PK) parameters will be calculated based on Pelabresib plasma concentrations and actual sampling time points. AUC0-t will be listed and summarized using descriptive statistics. | Cycle 1 Day 1, Cycle 1 Day 14 and Cycle 9 Day 1: Predose, 30 minutes to 1 hour post-dose, 3 to 4.5 hours post-dose. Cycle 3 and Cycle 7 Day 1: Predose, 5 minutes post ECG assessment. 1 Cycle = 21 days |
| Pharmacokinetic (PK) Parameters for Pelabresib: Time to Reach Maximum (Peak) Plasma Concentration Following Drug Administration (Tmax) | Pharmacokinetic (PK) parameters will be calculated based on Pelabresib plasma concentrations and actual sampling time points. AUC0-t will be listed and summarized using descriptive statistics. Tmax will be listed and summarized using descriptive statistics. | Cycle 1 Day 1, Cycle 1 Day 14 and Cycle 9 Day 1: Predose, 30 minutes to 1 hour post-dose, 3 to 4.5 hours post-dose. Cycle 3 and Cycle 7 Day 1: Predose, 5 minutes post ECG assessment. 1 Cycle = 21 days |
| Pharmacokinetic (PK) Parameters for Pelabresib: Maximum (Peak) Plasma Drug Concentration (Cmax) | Pharmacokinetic (PK) parameters will be calculated based on Pelabresib plasma concentrations and actual sampling time points. AUC0-t will be listed and summarized using descriptive statistics. Cmax will be listed and summarized using descriptive statistics. | Cycle 1 Day 1, Cycle 1 Day 14 and Cycle 9 Day 1: Predose, 30 minutes to 1 hour post-dose, 3 to 4.5 hours post-dose. Cycle 3 and Cycle 7 Day 1: Predose, 5 minutes post ECG assessment. 1 Cycle = 21 days |
| Pharmacokinetic (PK) Parameters for Pelabresib: Effective Half-Life (T1/2) | Pharmacokinetic (PK) parameters will be calculated based on Pelabresib plasma concentrations and actual sampling time points. T1/2 will be listed and summarized using descriptive statistics. | Cycle 1 Day 1, Cycle 1 Day 14 and Cycle 9 Day 1: Predose, 30 minutes to 1 hour post-dose, 3 to 4.5 hours post-dose. Cycle 3 and Cycle 7 Day 1: Predose, 5 minutes post ECG assessment. 1 Cycle = 21 days |
| Pharmacokinetic (PK) Parameters for Pelabresib: Apparent Volume of Distribution After Non-intravenous Administration (Vd/F) | Pharmacokinetic (PK) parameters will be calculated based on Pelabresib plasma concentrations and actual sampling time points. Vd/F will be listed and summarized using descriptive statistics. | Cycle 1 Day 1, Cycle 1 Day 14 and Cycle 9 Day 1: Predose, 30 minutes to 1 hour post-dose, 3 to 4.5 hours post-dose. Cycle 3 and Cycle 7 Day 1: Predose, 5 minutes post ECG assessment. 1 Cycle = 21 days |
| Pharmacokinetic (PK) Parameters for Pelabresib: Apparent Total Clearance of the Drug From Plasma After Oral Administration (CL/F) | Pharmacokinetic (PK) parameters will be calculated based on Pelabresib plasma concentrations and actual sampling time points. CL/F will be listed and summarized using descriptive statistics. | Cycle 1 Day 1, Cycle 1 Day 14 and Cycle 9 Day 1: Predose, 30 minutes to 1 hour post-dose, 3 to 4.5 hours post-dose. Cycle 3 and Cycle 7 Day 1: Predose, 5 minutes post ECG assessment. 1 Cycle = 21 days |
| Ruxolitinib Plasma Concentrations in the Presence or Absence of Pelabresib | Blood samples (approximately 4 mL each) will be collected at the time points specified in the Schedule of Assessments (SOA) to determine plasma concentrations of Ruxolitinib plasma concentrations in the presence or absence of Pelabresib | Cycle 1 Day 1, Cycle 1 Day 14 and Cycle 9 Day 1: Predose, 30 minutes to 1 hour post-dose, 3 to 4.5 hours post-dose. Cycle 3 and Cycle 7 Day 1: Predose, 5 minutes post ECG assessment. 1 Cycle = 21 days |
| Duration of the Splenic Response | Duration of splenic response, defined as the time from onset of splenic response until the time at which the patient has a <35% decrease from baseline in spleen volume and a >25% increase from nadir, as confirmed by the central review) or death, whichever comes first | Through study completion, an average of 6 years |
| Number of Participants With Modified Total Symptom Score (mTSS) Response at Week 24 | The modified TSS (mTSS) was defined as TSS without the fatigue sub-domain and with a total scale of 60 points versus 70 points for TSS. Patients were classified as responders if the percentage of change from baseline in mTSS was ≤ -50% at Week 24. | Week 24 |
| Duration of the TSS50 Response | Duration of TSS response, defined as the time from onset of TSS50 response until the time at which the patient has a <50% reduction in TSS from baseline and an increase of ≥25% from nadir | Through study completion, an average of 6 years |
| Chandler |
| Arizona |
| 85224 |
| United States |
| UC San Diego Moores Cancer Center | La Jolla | California | 92093 | United States |
| USC/Norris Comprehensive Cancer Center | Los Angeles | California | 90033 | United States |
| UCLA Medical Center | Los Angeles | California | 90095 | United States |
| Yale University School of Medicine | New Haven | Connecticut | 06510 | United States |
| Mayo Clinic | Jacksonville | Florida | 32224 | United States |
| Moffitt Cancer Center | Tampa | Florida | 33612 | United States |
| Decatur Memorial Hospital Cancer Care Center of Decatur/Cancer Care Specialists of IL | Decatur | Illinois | 62526 | United States |
| Franciscan Health/Indiana blood and Marrow Transplantation | Indianapolis | Indiana | 46237 | United States |
| Norton Cancer Institute, St. Matthews Campus | Louisville | Kentucky | 40207 | United States |
| University of Michigan Medical Center | Ann Arbor | Michigan | 48109 | United States |
| Washington University | St Louis | Missouri | 63110 | United States |
| Icahn School of Medicine at Mount Sinai | New York | New York | 10029 | United States |
| Memorial Sloan Kettering Cancer Center | New York | New York | 10065 | United States |
| Weill Cornell Medical College- New York Presbyterian Hospital | New York | New York | 10065 | United States |
| Cleveland Clinic Taussig Cancer Center | Cleveland | Ohio | 44195 | United States |
| MD Anderson Cancer Center | Houston | Texas | 77030 | United States |
| University of Texas Health Science Center - San Antonio | San Antonio | Texas | 78229 | United States |
| Center for Blood Disorders and Cellular Therapy, Swedish Cancer Institute | Seattle | Washington | 98104 | United States |
| Icon Cancer Centre | Brisbane | Queensland | 4101 | Australia |
| Royal Adelaide Hospital | Adelaide | South Australia | 5000 | Australia |
| Monash Health | Clayton | Victoria | 3168 | Australia |
| Peninsula Private Hospital Clinical Trials Unit | Frankston | Victoria | 3199 | Australia |
| USC Clinical Trials Centre Sunshine Coast Haematology and Oncology Clinic | Buderim | QLD 4556 | Australia |
| One Clinical Research PTY LTD | Nedlands | 6009 | Australia |
| LKH - Universitätsklinikum Graz; Abteilung für Hämatologie | Graz | A-8036 | Austria |
| Krankenhaus der Elisabethinen Linz | Linz | 4020 | Austria |
| Kepler University Hospital | Linz | 4021 | Austria |
| University Hospital Salzburg | Salzburg | 5020 | Austria |
| ZNA | Antwerp | Belgium |
| Cliniques universitaires Saint-Luc | Brussels | 1200 | Belgium |
| Hôpital de Jolimont | La Louvière | 7100 | Belgium |
| UZ Leuven | Leuven | Belgium |
| Domaine Universitaire du Sart Tilman | Liège | 4000 | Belgium |
| Tom Baker Cancer Centre | Calgary | Alberta | T2N 4N2 | Canada |
| University of Alberta Hospital | Edmonton | Alberta | T6G 2B7 | Canada |
| London Health Sciences Center - Victoria Hospital | London | Ontario | N6A 5W9 | Canada |
| Princess Margaret Cancer Center | Toronto | Ontario | M5G 2M9 | Canada |
| Jewish General Hospital | Montreal | Quebec | H3T 1E2 | Canada |
| Fakultni nemocnice Hradec Kralove | Hradec Králové | 500 05 | Czechia |
| University Hospital Olomouc | Olomouc | 779 00 | Czechia |
| Amiens South Hospital univerisy - Hopital Sud | Amiens | 80054 | France |
| Centre Hospitalier | Le Mans | 72000 | France |
| Hôpital l'Archet 1 | Nice | 06202 | France |
| Gard Cancer Institute | Nîmes | 30029 | France |
| Centre Hospitalier Lyon Sud Secteur 1G | Pierre-Bénite | 69310 | France |
| Chu Pontchaillou - Service Hematologie | Rennes | 35033 | France |
| Institut de Cancérologie Lucien Neuwirth | Saint-Priest-en-Jarez | 42271 | France |
| Hôpital Bretonneau | Tours | 37044 | France |
| Chru Brabois | Vandœuvre-lès-Nancy | 54500 | France |
| Universitätsklinikum Ulm | Ulm | Baden-Wurttemberg | 89081 | Germany |
| Hämatologisch-Onkologische Praxis Augsburg | Augsburg | Bavaria | 86151 | Germany |
| Universitätsklinikum Halle (Saale), Krukenberg-Krebszentrum Halle (KKH) | Halle | Saxony | 06120 | Germany |
| Universitätsklinikum Jena, Klinik für Innere Medizin II | Jena | Thuringia | 07747 | Germany |
| University Hospital Hamburg-Eppendorf | Hamburg | 20246 | Germany |
| Laiko General Hospital | Athens | Greece |
| UGH of Ioannina | Ioannina | 455 00 | Greece |
| University General Hospital of Patras | Rio | 26504 | Greece |
| Princess Margaret Hospital | Hong Kong | Kowloon | Hong Kong |
| Prince of Wales Hospital | Hong Kong | New Territories | Hong Kong |
| : Pecs University, 1st Department of Medicine | Pécs | Pecs | 7624 | Hungary |
| Szabolcs Szatmár Bereg Megyei Kórházak és Egyetemi Oktatókórház; Jósa András Oktatókórház, Hematológia | Nyíregyháza | Hungary |
| Soroka Medical Center | Beersheba | 84101 | Israel |
| Shamir Medical Center | Be’er Ya‘aqov | 60930000 | Israel |
| Rambam Health Corporation | Haifa | 3109601 | Israel |
| Lady Davis Carmel Medical Center | Haifa | 3436212 | Israel |
| Wolfson Medical Center | Holon | Israel |
| Hadassah University Hospital-Ein Kerem | Jerusalem | 9590300 | Israel |
| Shaarei Zedek | Nahariya | 2210001 | Israel |
| Rabin Medical Center - Beilinson Campus, | Petah Tikva | 49100 | Israel |
| Tel Aviv Sourasky Medical Center | Tel Aviv | 6423906 | Israel |
| Azienda Socio Sanitaria Territoriale Papa Giovanni XXIII | Bergamo | 24127 | Italy |
| A.O.U. Policlinico S. Orsola-Malpighi | Bologna | 40138 | Italy |
| Regional Hospital Spedali Civili di Brescia | Brescia | 25123 | Italy |
| Azienda Ospedaliera Universitaria Arcispedale Sant'Anna | Ferrara | Italy |
| Azienda Ospedaliera Universitaria Careggi | Florence | Italy |
| IRCCS Istituto Romagnolo per lo studio dei tumori "Dino Amadori" | Meldola | Italy |
| Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico | Milan | Italy |
| Azienda Ospedaliera San Gerardo di Monza | Monza | Italy |
| University Hospital Maggiore della Carità | Novara | 28100 | Italy |
| AOU S.Luigi Gonzaga | Orbassano | Italy |
| University Hospital of Padova | Padova | 35128 | Italy |
| University Hospital 'Paolo Giaccone' Polyclinic | Palermo | 90127 | Italy |
| Ospedale Sant'Eugenio | Rome | Italy |
| Hospital Ordine Mauriziano of Turin | Turin | 10128 | Italy |
| Ospedale di Circolo e Fondazione Macchi - ASST Sette Laghi | Varese | Italy |
| Hospital Sultanah Aminah | Johor Bahru | Johor | 80100 | Malaysia |
| Hospital Sultanah Bahiyah | Alor Star | Kedah | 05460 | Malaysia |
| Hospital Pulau Pinang | George Town | Pulau Pinang | 10450 | Malaysia |
| :Hospital Ampang | Ampang | Selangor | 68000 | Malaysia |
| Sunway Medical Centre | Petaling Jaya | Selangor | 47500 | Malaysia |
| Hospital Queen Elizabeth | Kota Kinabalu | 88586 | Malaysia |
| Amsterdam UMC | Amsterdam | 1081 | Netherlands |
| Maastricht University Medical Center | Maastricht | 6229 HX | Netherlands |
| Wojewódzki Szpital Specjalistyczny w Bialej Podlaskiej | Biała Podlaska | 21-50 | Poland |
| Uniwersyteckie Centrum Kliniczne | Gdansk | 80-293 | Poland |
| Pratia Onkologia Katowice | Katowice | Poland |
| Centrum Medyczne Pratia | Skorzewo | 60-185 | Poland |
| Nasz Lekarz Przychodnie Medyczne | Torun | 87-100 | Poland |
| Gyeongsang National University Hospital | Jinju | Gyeongsangnam-do | South Korea |
| Inje University Haeundae Paik Hospital | Busan | 48108 | South Korea |
| Pusan National University Hospital | Busan | 49241 | South Korea |
| Inje University Busan Paik Hospital | Busan | South Korea |
| Kyungpook National University Hospital | Daegu | 41944 | South Korea |
| Daegu Catholic University Medical Center | Daegu | South Korea |
| The Catholic University Of Korea St. Vincent Hospital | Gyeonggi-do | South Korea |
| Gachon University Gil Medical Center | Incheon | South Korea |
| Jeonbuk National University Hospital | Jeonju | 54907 | South Korea |
| Severance Hospital, Yonsei University Health System | Seoul | 03722 | South Korea |
| Samsung Medical Center | Seoul | 135-710 | South Korea |
| Korea University Anam Hospital | Seoul | South Korea |
| Seoul National University Hospital | Seoul | South Korea |
| The Catholic University of Korea | Seoul | South Korea |
| The Catholic University of South Korea Seoul St. Mary's Hospital | Seoul | South Korea |
| Ulsan University Hospital | Ulsan | South Korea |
| Hospital Universitario Central de Asturias | Oviedo | Principality of Asturias | 33011 | Spain |
| ICO Badalona - Hospital Universitari Germans Trias i Pujol | Badalona | 8916 | Spain |
| Hospital Clinic de Barcelona | Barcelona | Spain |
| Vall d'Hebron Institute of Oncology | Barcelona | Spain |
| Hospital San Pedro de Alcantara | Cáceres | 10003 | Spain |
| Hospital Universitario de Gran Canaria Dr. Negrin | Las Palmas de Gran Canaria | 35010 | Spain |
| Quirónsalud Madrid University Hospital | Madrid | 28223 | Spain |
| Morales Meseguer General University Hospital | Murcia | 30008 | Spain |
| Hospital Universitario de Salamanc | Salamanca | 37007 | Spain |
| Hospital Virgen Macarena | Seville | Spain |
| University Hospital of Toledo | Toledo | 45007 | Spain |
| Hospital Clínico Universitario de Valencia | Valencia | 46010 | Spain |
| Chang Gung Memorial Hospital Chiayi | Chiayi City | Taiwan |
| China Medical University Hospital | Chiayi City | Taiwan |
| Taichung Veterans General Hospital | Taichung | Taiwan |
| National Taiwan University Hospital | Taipei | Taiwan |
| Songklanagarind Hospital | Songkhla | Hat Yai District | 90110 | Thailand |
| Chulabhorn Hospital | Bangkok | Lak Si | 10210 | Thailand |
| Srinagarind hospital,Khon Kaen University | Khon Kaen | Mueang Khon Kaen District | 40002 | Thailand |
| King Chulalongkorn Memorial Hospital | Bangkok | Pathumwan | 10330 | Thailand |
| Rajavithi Hospital | Bangkok | Ratchathewi District | 10400 | Thailand |
| Ankara University Faculty of Medicine Cebeci Research and Application Hospital | Ankara | Turkey (Türkiye) |
| Dr. Abdurrahman Yurtaslan Oncology Health Application and Research Center | Ankara | Turkey (Türkiye) |
| Antalya Medstar Hospital | Antalya | Turkey (Türkiye) |
| Edirne Trakya University Faculty of Medicine Hospital | Edirne | Turkey (Türkiye) |
| Gaziantep University Sahinbey Research and Application Center Hospital | Gaziantep | Turkey (Türkiye) |
| Izmir Ege University Faculty of Medicine Hospital | Izmir | Turkey (Türkiye) |
| Erciyes University Faculty of Medicine Hospital | Kayseri | 8039 | Turkey (Türkiye) |
| Kocaeli University Application | Kocaeli | Turkey (Türkiye) |
| Mersin University | Mersin | Turkey (Türkiye) |
| Samsun 19 Mayıs University Health Application Research Hospital | Samsun | Turkey (Türkiye) |
| Western General Hospital | Edinburgh | Scotland | EH4 2XU | United Kingdom |
| University Hospital of Wales | Cardiff | Wales | CF14 4XW | United Kingdom |
| Pilgrim Hospital | Boston | PE21 9QS | United Kingdom |
| Addenbrooke's Hospital, Cambridge University Hospitals NHS Foundation Trust | Cambridge | CB2 0QQ | United Kingdom |
| The Clatterbridge Cancer Centre | Liverpool | L7 8YA | United Kingdom |
| Guy's & St Thomas NHS Foundation Trust | London | SE1 9RT | United Kingdom |
| Imperial College Healthcare NHS Trust, Hammersmith Hospital | London | W12 0HS | United Kingdom |
| University College London Hospitals | London | United Kingdom |
| Oxford University Hospitals NHS Foundation Trust, Department of Haematology, Cancer and Haematology Centre, Churchill Hospital | Oxford | United Kingdom |
| Result |
| Rampal RK, Grosicki S, Chraniuk D, Abruzzese E, Bose P, Gerds AT, Vannucchi AM, Palandri F, Lee SE, Gupta V, Lucchesi A, Oh ST, Kuykendall AT, Patriarca A, Alvarez-Larran A, Mesa R, Kiladjian JJ, Talpaz M, Scandura JM, Lavie D, Harris M, Kays SK, Li Q, Boxhammer R, Brown B, Jegg AM, Harrison CN, Mascarenhas J. Pelabresib plus ruxolitinib for JAK inhibitor-naive myelofibrosis: a randomized phase 3 trial. Nat Med. 2025 May;31(5):1531-1538. doi: 10.1038/s41591-025-03572-3. Epub 2025 Mar 10. |
Matching placebo orally (PO) once daily (QD) + ruxolitinib 10 or 15 mg orally (PO) twice a day (BID) for 14 consecutive days followed by ruxolitinib 10 or 15 mg orally (PO) twice a day (BID) for 7 days in a 21-day cycle |
|
| Modified Intent-to-Treat (mITT) Analysis Set | All randomized patients who have received at least 1 dose of any study drug(s) |
|
| Safety Analysis Set | A subset of the ITT population that includes all randomized patients who were administered at least one dose of study drug. |
|
| Per-Protocol Set (PP Set) |
|
| Pharmacokinetic (PK) Analysis Set | All patients in the Safety Set who received placebo/pelabresib + ruxolitinib treatment and had plasma samples drawn and tested for study drug concentrations. |
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| Biomarker Analysis Set | All patients in the Safety Set who received any treatment component and had evaluable biomarker data collected. |
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| Ongoing on Double-blind Treatment | Ongoing on double-blind treatment at the time of Primary Completion Date |
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| Discontinued Double-blind Treatment | Discontinued double-blind treatment at the time of Primary Completion Date |
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| On PFS/OS Follow-up | On Progression-Free Survival (PFS)/Overall Survival (OS) follow-up |
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| COMPLETED | Completed Week 24 double-blind treatment |
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| NOT COMPLETED |
|
Intent-to-Treat Population. All randomized patients. This is the primary population for all efficacy endpoints. All analyses using this population will be based on the treatment assigned by the Interactive Response Technology (IRT) system.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Pelabresib + Ruxolitinib (Experimental Arm) | Pelabresib 125 mg orally (PO) once daily (QD) + ruxolitinib 10 or 15 mg orally (PO) twice a day (BID) for 14 consecutive days followed by ruxolitinib 10 or 15 mg orally (PO) twice a day (BID) for 7 days in a 21-day cycle |
| BG001 | Placebo + Ruxolitinib (Control Arm) | Matching placebo orally (PO) once daily (QD) + ruxolitinib 10 or 15 mg orally (PO) twice a day (BID) for 14 consecutive days followed by ruxolitinib 10 or 15 mg orally (PO) twice a day (BID) for 7 days in a 21-day cycle |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
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| 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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| Dynamic International Prognostic Scoring System (DIPSS) risk category | Dynamic International Prognostic Scoring System (DIPSS) is used to predict survival in PMF patients, calculated from five variables: a. Age >65: 1 point b. Leukocyte count >25 × 10^9/L: 1 point c. Hemoglobin <10 g/dL: 2 points d. Circulating blast cells ≥1%: 1 point e. Constitutional symptoms*: 1 point The total DIPSS score categorizes patients into four risk groups, with lower scores indicating better outcomes and longer survival: • 0 points: low risk • 1-2 points: intermediate-1 risk • 3-4 points: intermediate-2 risk • 5-6 points: high risk | Count of Participants | Participants |
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| Platelet count | Count of Participants | Participants |
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| Hemoglobin | Count of Participants | Participants |
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| Diagnosis | Count of Participants | Participants |
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| Spleen volume from central reads | Mean | Standard Deviation | cm^3 |
| |||||||||||||||||
| Baseline Total Symptom Score (TSS) | Baseline TSS = Averaged daily TSS from the 7-day period prior to randomization (Day -7 to Day -1). Daily TSS is a 24-hour recall format of the MFSAF v4.0, which equals the sum of the 7 individual item responses on the 0-10 scale, with a possible total daily score ranging from 0 to 70. For each individual question, the response scale is 0 (Absent) to 10 (Worst Imaginable). | Mean | Standard Deviation | units on a scale |
|
| 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 | Number of Participants With Splenic Response by Central Radiology Reads at Week 24 | Splenic response was characterized by a reduction of at least 35% in spleen volume from baseline (SVR35), as determined by magnetic resonance imaging (MRI) or computerized tomography (CT), and evaluated through a blinded central radiology review at Week 24. | Intent-to-Treat (ITT) Analysis Set | Posted | Count of Participants | Participants | Week 24 |
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| Secondary | Key Secondary: Absolute Change From Baseline in Total Symptom Score (TSS) at Week 24 | The Total Symptom Score (TSS) at Week 24, compared to baseline, was measured using the Myelofibrosis Symptom Assessment Form v4.0. This score represented the sum of seven individual symptom items, each rated on a 0-10 scale, resulting in a possible total daily score ranging from 0 to 70. A higher TSS reflected a greater disease burden and therefore a worse outcome. The baseline TSS was calculated as the average of non-missing daily total symptom scores over the seven-day period preceding the day of randomization. The TSS for each treatment week was determined as the average of non-missing daily total symptom scores for that week. However, if fewer than four daily scores were available for a given week, the weekly TSS was considered missing. | Intent-to-Treat (ITT) Analysis Set | Posted | Least Squares Mean | Standard Error | score on a scale | Baseline, Week 24 |
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| Secondary | Key Secondary: Number of Participants With TSS50 Response at Week 24 | The probability of a TSS response at Week 24 was estimated by calculating the TSS response rate, defined as the percentage of patients who achieved a TSS50 response at Week 24 in each of the two treatment groups. The Total Symptom Score (TSS) response was defined as a ≥50% reduction from baseline in TSS, as measured by the Myelofibrosis Symptom Assessment Form v4.0. | Intent-to-Treat (ITT) Analysis Set | Posted | Count of Participants | Participants | Week 24 |
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| Secondary | Percent Change From Baseline in Total Symptom Score (TSS) at Week 24 | Percent Change From Baseline in Total Symptom Score (TSS) at Week 24 measured the change in a patient's symptoms after 24 weeks of treatment, relative to baseline. A negative value indicates symptom improvement, and a reduction of ≥50% is typically considered a meaningful clinical response. | Intent-to-Treat (ITT) Analysis Set | Posted | Mean | 95% Confidence Interval | Percent change from baseline to Week 24 | Baseline, Week 24 |
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| Secondary | Number of Participants With Improvement From Baseline in Bone Marrow Fibrosis of at Least 1 Grade at Week 24 | Improvement in bone marrow fibrosis by at least 1 grade, as assessed by central read compared to baseline, was analyzed by treatment group and overall. The improvement in bone marrow fibrosis grade was defined as a decrease by at least 1 grade in bone marrow fibrosis grade when compared to baseline, where a grade of MF-3 was the most severe, and MF-0 was the least severe. | Intent-to-Treat (ITT) Analysis Set: Only participants with available data at the specified time point were included in the analysis. | Posted | Count of Participants | Participants | Baseline, Week 24 |
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| Secondary | Number of Participants With Splenic Response by Central Radiology Reads at Week 48 | Splenic response is characterized by a reduction of at least 35% in spleen volume from baseline (SVR35), as determined by magnetic resonance imaging (MRI) or computerized tomography (CT), and evaluated through a blinded central radiology review at Week 48. | Not Posted | Dec 2027 | Week 48 | Participants | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With TSS50 Response at Week 48 | The probability of a TSS response at Week 48 is estimated by calculating the TSS response rate, defined as the percentage of patients who achieve a TSS50 response at Week 48 in each of the two treatment groups. The Total Symptom Score (TSS) response is defined as a ≥50% reduction from baseline in TSS, as measured by the Myelofibrosis Symptom Assessment Form v4.0. | Not Posted | Dec 2027 | Week 48 | Participants | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change From Baseline in Total Symptom Score (TSS) at Week 48 | The Total Symptom Score (TSS) at Week 48, compared to baseline, is measured using the Myelofibrosis Symptom Assessment Form v4.0. This score represents the sum of seven individual symptom items, each rated on a 0-10 scale, resulting in a possible total daily score ranging from 0 to 70. A higher TSS reflects a greater disease burden and therefore a worse outcome. The baseline TSS is calculated as the average of non-missing daily total symptom scores over the seven-day period preceding the day of randomization. The TSS for each treatment week is determined as the average of non-missing daily total symptom scores for that week. However, if fewer than four daily scores are available for a given week, the weekly TSS is considered missing. | Not Posted | Dec 2027 | Baseline, Week 48 | Participants | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Rate of Red Blood Cell (RBC) Transfusion Over First 24 Weeks of Treatment | The rate of RBC transfusions was defined as the average number of RBC units transfused per patient month (4 weeks) during the first 24 weeks of treatment. The average number of RBC units per patient-month was calculated by dividing the total (ie, for all patients) number of RBC units in the whole exposure time by the sum of patient-months. | Intent-to-Treat (ITT) Analysis Set | Posted | Number | 95% Confidence Interval | RBC units transfused/patient-months | Week 24 |
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| Secondary | Number of Participants Who Transitioned From Red Blood Cell (RBC) Transfusion Dependence to Transfusion Independence | Transfusion dependence (TD) was defined as having received ≥6 units of RBCs during the 12-week baseline period prior to dosing and Transfusion independence (TI) was defined as the absence of RBC transfusions during any continuous 12-week period of the double-blind treatment phase. The conversion from TD to TI was evaluated and defined as the proportion of patients who transitioned from transfusion dependence to transfusion independence. Patients who remained in the double-blind treatment period and had not received any RBC transfusions during the most recent 12 weeks were considered responders. Patients who discontinued from the double-blind treatment before Week 12 were classified as non-responders. | Intent-to-Treat (ITT) Analysis Set | Posted | Count of Participants | Participants | From 12-week baseline period prior to dosing to any 12-week period post baseline, up to 24 weeks |
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| Secondary | Category Change From Baseline of Patient Global Impression of Change (PGIC) at Week 24 | The Patient Global Impression of Change (PGIC) was a single-item measure of the patient's perceived change in MF symptoms since starting treatment. Patients responded to: "Since beginning this study treatment, your myelofibrosis symptoms were: (1) Very much improved, (2) Much improved, (3) Minimally improved, (4) No change, (5) Minimally worse, (6) Much worse, (7) Very much worse." | Intent-to-Treat (ITT) Analysis Set - Only participants with evaluable data at the pre-specified time points | Posted | Count of Participants | Participants | Baseline, Week 24 |
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| Secondary | Progression-Free Survival (PFS) | Progression-Free Survival (PFS), defined as the time from randomization until documented progression, or until death from any cause for patients without documented progression | Not Posted | Dec 2027 | Through study completion, an average of 6 years | Participants | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Overall Survival (OS) | OS, defined as the time from randomization until death from any cause | Not Posted | Dec 2027 | Through study completion, an average of 6 years | Participants | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Proportion of Patients With Transformation to Blast Phase (AML) | Patients are categorized as having transformed to Acute Myelogenous Leukemia (AML) when the peripheral blood blast percentage increases to ≥20% and this elevation persists for at least two weeks, or when leukemic transformation is confirmed through disease status assessment. | Not Posted | Dec 2027 | Through study completion, an average of 6 years | Participants | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | A treatment-emergent adverse event (TEAE) for the double-blind treatment period is defined as an AE that has a start date on or after the first dose of the pelabresib/placebo and before 30 days after the last dose of pelabresib/placebo or before the start of alternative (off-study) treatment for MF, whichever occurs first. If the AE has a start date before the date of first dose but increases in severity after first dose and before 30 days post last dose will be considered a TEAE as well. An AE that occurs after the administration of the first dose of open-label pelabresib treatment will be considered treatment-emergent for the crossover treatment period. However, a TEAE for the crossover treatment period that occurs within 30 days after the last dose of pelabresib/placebo will be considered treatment emergent for the double-blind treatment period as well. | Not Posted | Dec 2027 | Through study completion, an average of 6 years | Participants | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Pharmacokinetic (PK) Parameters for Pelabresib: Area Under the Plasma Concentration-time Curve From Time Zero to Time t (AUC0-t) | Pharmacokinetic (PK) parameters will be calculated based on Pelabresib plasma concentrations and actual sampling time points. AUC0-t will be listed and summarized using descriptive statistics. | Not Posted | Dec 2027 | Cycle 1 Day 1, Cycle 1 Day 14 and Cycle 9 Day 1: Predose, 30 minutes to 1 hour post-dose, 3 to 4.5 hours post-dose. Cycle 3 and Cycle 7 Day 1: Predose, 5 minutes post ECG assessment. 1 Cycle = 21 days | Participants | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Pharmacokinetic (PK) Parameters for Pelabresib: Time to Reach Maximum (Peak) Plasma Concentration Following Drug Administration (Tmax) | Pharmacokinetic (PK) parameters will be calculated based on Pelabresib plasma concentrations and actual sampling time points. AUC0-t will be listed and summarized using descriptive statistics. Tmax will be listed and summarized using descriptive statistics. | Not Posted | Dec 2027 | Cycle 1 Day 1, Cycle 1 Day 14 and Cycle 9 Day 1: Predose, 30 minutes to 1 hour post-dose, 3 to 4.5 hours post-dose. Cycle 3 and Cycle 7 Day 1: Predose, 5 minutes post ECG assessment. 1 Cycle = 21 days | Participants | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Pharmacokinetic (PK) Parameters for Pelabresib: Maximum (Peak) Plasma Drug Concentration (Cmax) | Pharmacokinetic (PK) parameters will be calculated based on Pelabresib plasma concentrations and actual sampling time points. AUC0-t will be listed and summarized using descriptive statistics. Cmax will be listed and summarized using descriptive statistics. | Not Posted | Dec 2027 | Cycle 1 Day 1, Cycle 1 Day 14 and Cycle 9 Day 1: Predose, 30 minutes to 1 hour post-dose, 3 to 4.5 hours post-dose. Cycle 3 and Cycle 7 Day 1: Predose, 5 minutes post ECG assessment. 1 Cycle = 21 days | Participants | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Pharmacokinetic (PK) Parameters for Pelabresib: Effective Half-Life (T1/2) | Pharmacokinetic (PK) parameters will be calculated based on Pelabresib plasma concentrations and actual sampling time points. T1/2 will be listed and summarized using descriptive statistics. | Not Posted | Dec 2027 | Cycle 1 Day 1, Cycle 1 Day 14 and Cycle 9 Day 1: Predose, 30 minutes to 1 hour post-dose, 3 to 4.5 hours post-dose. Cycle 3 and Cycle 7 Day 1: Predose, 5 minutes post ECG assessment. 1 Cycle = 21 days | Participants | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Pharmacokinetic (PK) Parameters for Pelabresib: Apparent Volume of Distribution After Non-intravenous Administration (Vd/F) | Pharmacokinetic (PK) parameters will be calculated based on Pelabresib plasma concentrations and actual sampling time points. Vd/F will be listed and summarized using descriptive statistics. | Not Posted | Dec 2027 | Cycle 1 Day 1, Cycle 1 Day 14 and Cycle 9 Day 1: Predose, 30 minutes to 1 hour post-dose, 3 to 4.5 hours post-dose. Cycle 3 and Cycle 7 Day 1: Predose, 5 minutes post ECG assessment. 1 Cycle = 21 days | Participants | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Pharmacokinetic (PK) Parameters for Pelabresib: Apparent Total Clearance of the Drug From Plasma After Oral Administration (CL/F) | Pharmacokinetic (PK) parameters will be calculated based on Pelabresib plasma concentrations and actual sampling time points. CL/F will be listed and summarized using descriptive statistics. | Not Posted | Dec 2027 | Cycle 1 Day 1, Cycle 1 Day 14 and Cycle 9 Day 1: Predose, 30 minutes to 1 hour post-dose, 3 to 4.5 hours post-dose. Cycle 3 and Cycle 7 Day 1: Predose, 5 minutes post ECG assessment. 1 Cycle = 21 days | Participants | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Ruxolitinib Plasma Concentrations in the Presence or Absence of Pelabresib | Blood samples (approximately 4 mL each) will be collected at the time points specified in the Schedule of Assessments (SOA) to determine plasma concentrations of Ruxolitinib plasma concentrations in the presence or absence of Pelabresib | Not Posted | Dec 2027 | Cycle 1 Day 1, Cycle 1 Day 14 and Cycle 9 Day 1: Predose, 30 minutes to 1 hour post-dose, 3 to 4.5 hours post-dose. Cycle 3 and Cycle 7 Day 1: Predose, 5 minutes post ECG assessment. 1 Cycle = 21 days | Participants | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Duration of the Splenic Response | Duration of splenic response, defined as the time from onset of splenic response until the time at which the patient has a <35% decrease from baseline in spleen volume and a >25% increase from nadir, as confirmed by the central review) or death, whichever comes first | Not Posted | Dec 2027 | Through study completion, an average of 6 years | Participants | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Modified Total Symptom Score (mTSS) Response at Week 24 | The modified TSS (mTSS) was defined as TSS without the fatigue sub-domain and with a total scale of 60 points versus 70 points for TSS. Patients were classified as responders if the percentage of change from baseline in mTSS was ≤ -50% at Week 24. | Intent-to-Treat (ITT) Analysis Set | Posted | Count of Participants | Participants | Week 24 |
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| Secondary | Duration of the TSS50 Response | Duration of TSS response, defined as the time from onset of TSS50 response until the time at which the patient has a <50% reduction in TSS from baseline and an increase of ≥25% from nadir | Not Posted | Dec 2027 | Through study completion, an average of 6 years | Participants |
From first dose of study treatment up to the cut-off date for the interim analysis (31-Aug-2023), approximately 28 months
Adverse Events were reported that had a start date on or after the first dose of the pelabresib/placebo and before 30 days after the last dose of pelabresib/placebo or before the start of alternative (off study) treatment for MF, whichever occured first. In addition, Serious Adverse Events, including deaths, that were reported at any time after 30 days after the last dose of pelabresib/placebo are presented.
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 | Pelabresib + Ruxolitinib (Experimental Arm) | Pelabresib 125 mg orally (PO) once daily (QD) + ruxolitinib 10 or 15 mg orally (PO) twice a day (BID) for 14 consecutive days followed by ruxolitinib 10 or 15 mg orally (PO) twice a day (BID) for 7 days in a 21-day cycle | 11 | 212 | 63 | 212 | 201 | 212 |
| EG001 | Placebo + Ruxolitinib (Control Arm) | Matching placebo orally (PO) once daily (QD) + ruxolitinib 10 or 15 mg orally (PO) twice a day (BID) for 14 consecutive days followed by ruxolitinib 10 or 15 mg orally (PO) twice a day (BID) for 7 days in a 21-day cycle | 10 | 214 | 63 | 214 | 204 | 214 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Febrile Neutropenia | Blood and lymphatic system disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Splenomegaly | Blood and lymphatic system disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Cardiac Failure | Cardiac disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Arrhythmia | Cardiac disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Atrial Fibrillation | Cardiac disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Cardiac Arrest | Cardiac disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Cardiac Failure Congestive | Cardiac disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Myocardial Infarction | Cardiac disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Acute Myocardial Infarction | Cardiac disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Left Ventricular Failure | Cardiac disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Myocardial Ischaemia | Cardiac disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Sinus Node Dysfunction | Cardiac disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Vertigo | Ear and labyrinth disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Thyroid Mass | Endocrine disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Small Intestinal Obstruction | Gastrointestinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Abdominal Pain | Gastrointestinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Abdominal Pain Upper | Gastrointestinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Chronic Gastritis | Gastrointestinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Colitis | Gastrointestinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Gastric Ulcer | Gastrointestinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Gastric Varices Haemorrhage | Gastrointestinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Gastrointestinal Haemorrhage | Gastrointestinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Oesophageal Varices Haemorrhage | Gastrointestinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Varices Oesophageal | Gastrointestinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Chest Pain | General disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Drug Withdrawal Syndrome | General disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Sudden Cardiac Death | General disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Bile Duct Stone | Hepatobiliary disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Cholecystitis | Hepatobiliary disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Cholecystitis Acute | Hepatobiliary disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Herpes Zoster | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Covid-19 | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Covid-19 Pneumonia | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Diverticulitis | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Urinary Tract Infection | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Abscess Neck | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Appendicitis | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Bacteraemia | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Campylobacter Infection | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Enterococcal Infection | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Fungal Infection | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Gastroenteritis Viral | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Gastrointestinal Viral Infection | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Herpes Zoster Meningoencephalitis | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Herpes Zoster Oticus | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Liver Abscess | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Norovirus Infection | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Peritonitis | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Pneumocystis Jirovecii Pneumonia | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Pneumonia Bacterial | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Pyelonephritis | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Pyelonephritis Acute | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Septic Shock | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Urosepsis | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Accidental Overdose | Injury, poisoning and procedural complications | MedDRA 26.0 | Non-systematic Assessment |
| |
| Femur Fracture | Injury, poisoning and procedural complications | MedDRA 26.0 | Non-systematic Assessment |
| |
| Road Traffic Accident | Injury, poisoning and procedural complications | MedDRA 26.0 | Non-systematic Assessment |
| |
| Skin Graft Necrosis | Injury, poisoning and procedural complications | MedDRA 26.0 | Non-systematic Assessment |
| |
| Splenic Rupture | Injury, poisoning and procedural complications | MedDRA 26.0 | Non-systematic Assessment |
| |
| Thoracic Vertebral Fracture | Injury, poisoning and procedural complications | MedDRA 26.0 | Non-systematic Assessment |
| |
| Wound | Injury, poisoning and procedural complications | MedDRA 26.0 | Non-systematic Assessment |
| |
| Platelet Count Decreased | Investigations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Arthritis | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Bone Pain | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Haematoma Muscle | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Intervertebral Disc Protrusion | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Basal Cell Carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Non-systematic Assessment |
| |
| Squamous Cell Carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Non-systematic Assessment |
| |
| Acute Myeloid Leukaemia | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Non-systematic Assessment |
| |
| Squamous Cell Carcinoma Of Skin | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Non-systematic Assessment |
| |
| Adenocarcinoma Gastric | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Non-systematic Assessment |
| |
| Atypical Fibroxanthoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Non-systematic Assessment |
| |
| B-Cell Lymphoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Non-systematic Assessment |
| |
| Bladder Transitional Cell Carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Non-systematic Assessment |
| |
| Bowen's Disease | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Non-systematic Assessment |
| |
| Chloroma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Non-systematic Assessment |
| |
| Malignant Fibrous Histiocytoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Non-systematic Assessment |
| |
| Malignant Melanoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Non-systematic Assessment |
| |
| Metastatic Malignant Melanoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Non-systematic Assessment |
| |
| Neoplasm | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Non-systematic Assessment |
| |
| Papillary Renal Cell Carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Non-systematic Assessment |
| |
| Precursor T-Lymphoblastic Lymphoma/Leukaemia | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Non-systematic Assessment |
| |
| Prostate Cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Non-systematic Assessment |
| |
| Renal Cell Carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Non-systematic Assessment |
| |
| Squamous Cell Carcinoma Of The Tongue | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Non-systematic Assessment |
| |
| Uterine Leiomyoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Non-systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Cerebrovascular Accident | Nervous system disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Haemorrhage Intracranial | Nervous system disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Post Herpetic Neuralgia | Nervous system disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Presyncope | Nervous system disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Transient Ischaemic Attack | Nervous system disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Acute Kidney Injury | Renal and urinary disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Hydronephrosis | Renal and urinary disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Ureterolithiasis | Renal and urinary disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Pulmonary Embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Pulmonary Hypertension | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Acute Pulmonary Oedema | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Chronic Obstructive Pulmonary Disease | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Pleural Effusion | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Embolism Arterial | Vascular disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Shock Haemorrhagic | Vascular disorders | MedDRA 26.0 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Abdominal Pain Upper | Gastrointestinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Abdominal Pain | Gastrointestinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Oedema Peripheral | General disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Covid-19 | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Upper Respiratory Tract Infection | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Herpes Zoster | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Urinary Tract Infection | Infections and infestations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 26.0 | Non-systematic Assessment |
| |
| Platelet Count Decreased | Investigations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Alanine Aminotransferase Increased | Investigations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Weight Increased | Investigations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Aspartate Aminotransferase Increased | Investigations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Blood Creatinine Increased | Investigations | MedDRA 26.0 | Non-systematic Assessment |
| |
| Decreased Appetite | Metabolism and nutrition disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Hyperuricaemia | Metabolism and nutrition disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Back Pain | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Pain In Extremity | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Dysgeusia | Nervous system disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Muscle Spasms | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Non-systematic Assessment |
| |
| Bone Pain | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Non-systematic Assessment |
|
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Novartis Pharmaceuticals | +1 862 778 8300 | Novartis.email@Novartis.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 26, 2023 | Aug 14, 2024 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D055728 | Primary Myelofibrosis |
| ID | Term |
|---|---|
| D009196 | Myeloproliferative Disorders |
| 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
| >=65 years |
|
| 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 |
|
| Intermediate-2 |
|
| High |
|
| 100-200 × 10^9 cells/L |
|
| <100 × 10^9 cells/L |
|
| ≤10 g/dL |
|
| Post-Polycythemia Vera Myelofibrosis (PPV-MF) |
|
| Post-Essential Thrombocythemia Myelofibrosis (PET-MF) |
|
|
|
|
|
|
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