Anemia Clinical Trial
— INDEPENDENCEOfficial title:
A Phase 3, Double-blind, Randomized Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Placebo in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis on Concomitant JAK Inhibitor Therapy and Who Require Red Blood Cell Transfusions
The purpose of this Phase 3 study is to evaluate the efficacy and safety of Luspatercept compared with placebo in subjects with myeloproliferative neoplasm (MPN)-associated Myelofibrosis (MF) and anemia on concomitant Janus kinase 2 (JAK2) inhibitor therapy and who require red blood cell count (RBC) transfusions. The study is divided into Screening Period, a Treatment Phase (consisting of a Blinded Core Treatment Period, a Day 169 Response Assessment, a Blinded Extension Treatment Period, and an Open-label Extension Treatment Period), and a Posttreatment Follow-up Period. Following the Day 169 Response Assessment, subjects who did not show clinical benefit will have the option to unblind. Subjects who were on placebo during the Blinded Core Treatment Period will have the opportunity to crossover into the Open-Label Extension Treatment Period and receive Luspatercept.
Status | Recruiting |
Enrollment | 309 |
Est. completion date | August 23, 2025 |
Est. primary completion date | March 27, 2025 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility | Subjects must satisfy the following criteria to be randomized in the study: Inclusion Criteria - Subject is =18 years of age at the time of signing the ICF. - Subject has a diagnosis of PMF according to the 2016 World Health Organization (WHO) criteria or diagnosis of post-ET or post-PV MF according to the IWG-MRT 2007 criteria, confirmed by the most recent local pathology report. - Subject is requiring RBC transfusions as defined as:. i) Average RBC-transfusion frequency: 4 to 12 RBC units/12 weeks immediately up to randomization. There must be no interval > 6 weeks (42 days) without = 1 RBC transfusion. ii) RBC transfusions are scored in determining eligibility when given for treatment of:. A. Symptomatic (ie, fatigue or shortness of breath) anemia with a pretransfusion Hgb = 9.5 g/dL or. B. Asymptomatic anemia with a pretransfusion Hgb = 7 g/dL. iii) RBC transfusions given for worsening of anemia due to bleeding or infections are not scored in determining eligibility. - Subjects on continuous (eg, absent of dose interruptions lasting = 2 consecutive weeks) JAK2 inhibitor therapy as approved in the country of the study site for the treatment for MPN-associated MF as part of their standard-of-care therapy for at least 32 weeks, on stable daily dose for at least 16 weeks immediately up to the date of randomization and anticipated to be on a stable daily dose of that JAK2 inhibitor for at least 24 weeks after randomization. - Subject has an Eastern Cooperative Oncology Group (ECOG) performance score of = 2. - A female of childbearing potential (FCBP) for this study is defined as a female who: 1) has achieved menarche at some point, 2) has not undergone a hysterectomy or bilateral oophorectomy or 3) has not been naturally postmenopausal (amenorrhea following cancer therapy does not rule out childbearing potential) for at least 24 consecutive months (eg, has had menses at any time in the preceding 24 consecutive months). Females of childbearing potential (FCBP)participating in the study must:. i) Have 2 negative pregnancy tests as verified by the Investigator prior to starting study therapy. She must agree to ongoing pregnancy testing during the study, and after end of IP. This applies even if the subject practices true abstinence* from heterosexual contact. ii) Either commit to true abstinence* from heterosexual contact (which must be reviewed on a monthly basis and source documented) or agree to use, and be able to comply with, effective contraception** without interruption, 28 days prior to starting IP, during the study therapy (including dose interruptions), and for 12 weeks (approximately 5 times the mean terminal half-life of IP based on multiple-dose PK data) after discontinuation of study therapy. - Male subjects must: Practice true abstinence* (which must be reviewed on a monthly basis) or agree to use a condom during sexual contact with a pregnant female or a female of childbearing potential** while participating in the study, during dose interruptions and for at least 12 weeks (approximately 5 times the mean terminal half-life of IP based on multiple-dose PK data) following IP discontinuation, even if he has undergone a successful vasectomy. i) True abstinence is acceptable when it is in line with the preferred and usual lifestyle of the subject. [Periodic abstinence (eg, calendar, ovulation, symptothermal, postovulation methods) and withdrawal are not acceptable methods of contraception.]. ii) Agreement to use highly effective methods of contraception that alone or in combination result in a failure rate of a Pearl index of less than 1% per year when used consistently and correctly throughout the course of the study. Such methods include: Combined (estrogen and progestogen containing) hormonal contraception: Oral, Intravaginal, Transdermal; Progestogen-only hormonal contraception associated with inhibition of ovulation: Oral, Injectable hormonal contraception, Implantable hormonal contraception; Placement of an intrauterine device (IUD); Placement of an intrauterine hormone-releasing system (IUS); Bilateral tubal occlusion; Vasectomized partner; Sexual Abstinence. - Subject must understand and voluntarily sign an ICF prior to any study-related assessments/procedures being conducted. - Subject is willing and able to adhere to the study visit schedule and other protocol requirements including the use of the electronic patient reported outcomes device. Exclusion Criteria - The presence of any of the following will exclude a subject from randomization:. - Subject with anemia from cause other than MPN-associated MForJAK2 inhibitor therapy (eg, iron deficiency, vitamin B12 and/or folate deficiencies, autoimmune or hemolytic anemia, infection, or any type of known clinically significant bleeding or sequestration). - Subject use of hydroxyurea, immunomodulatory compounds such as pomalidomide, thalidomide, ESAs, androgenic steroids or other drugs with potential effects on hematopoiesis = 8 weeks immediately up to the date of randomization. i) Systemic corticosteroids are permitted for nonhematological conditions providing the subject is receiving a constant dose equivalent to = 10 mg prednisone for the 4 weeks immediately up to randomization. ii) Iron chelation therapy (ICT) is permitted providing the subject is receiving a stable dose for the 8 weeks immediately up to randomization. - Subject with any of the following laboratory abnormalities at screening:. i) Neutrophils: < 1 x 10^9/L. ii) White blood count (WBC): > 100 x 10^9/L. iii) Platelets: the lowest allowable level as approved for the concomitant JAK2 inhibitor but not < 25 x 10^9/L or > 1000 x 10^9/L. iv) Peripheral blood myeloblasts:> 5%. v) Estimated glomerular filtration rate:< 30 mL/min/1.73 m2 (via the 4-variable modification of diet in renal disease [MDRD] formula) or nephrotic subjects (eg, urine albumin-to-creatinine ratio > 3500 mg/g). vi) Aspartate aminotransferase (AST) or alanine aminotransferase (ALT):> 3.0 x upper limit of normal (ULN). vii) Direct bilirubin: = 2 x ULN. A. Higher levels are acceptable if these can be attributed to active red blood cell precursor destruction within the bone marrow (eg, ineffective erythropoiesis). - Subject with uncontrolled hypertension, defined as repeated elevations of systolic blood pressure = 140 mmHg or diastolic blood pressure = 90 mmHg, that is not resolved at the time of randomization. - Subject with prior history of malignancies, other than disease under study, unless the subject has been free of the disease for = 3 years. However, subject with the following history/concurrent conditions is allowed:. i) Basal or squamous cell carcinoma of the skin. ii) Carcinoma in situ of the cervix. iii) Carcinoma in situ of the breast. iv) Incidental histologic finding of prostate cancer (T1a or T1b using the tumor, nodes, metastasis [TNM] clinical staging system). - Subject with prior hematopoietic cell transplant or subject anticipated to receive a hematopoietic cell transplant during the 24 weeks from the date of randomization. 7. Subject with stroke, myocardial infarction, deep venous thrombosis, pulmonary or arterial embolism within 6 months immediately up to the date of randomization. - Subject with major surgery within 2 months up to the date of randomization. Subject must have completely recovered from any previous surgery immediately up to the date of randomization. - Subject with a major bleeding event (defined as symptomatic bleeding in a critical area or organ and/or bleeding causing a decrease in Hgb of = 2 g/dL or leading to transfusion of = 2 units of packed red cells) in the last 6 months prior to the date of randomization. - Subject with inadequately controlled heart disease and/or have a known left ventricular ejection fraction < 35%. - Subject with uncontrolled systemic fungal, bacterial, or viral infection (defined as ongoing signs/symptoms related to the infection without improvement despite appropriate antibiotics, antiviral therapy, and/or other treatment). - Subject with known human immunodeficiency virus (HIV), evidence of active Hepatitis B (HepB) as demonstrated by the presence of Hepatitis B surface antigen (HBsAg) and/or positive for Hepatitis B virus DNA (HBVDNA-positive), and/or evidence of active Hepatitis C (HepC) as demonstrated by a positive Hepatitis C virus RNA (HCV-RNA) test of sufficient sensitivity. - Subject with prior therapy of luspatercept or sotatercept. - Subject with history of severe allergic or anaphylactic reactions or hypersensitivity to recombinant proteins or excipients in the investigational product. - Pregnant or breastfeeding females. - Subject participation in any other clinical protocol or investigational trial that involves use of experimental therapy (including investigational agents) and/or therapeutic devices within 30 days or for investigational agents within five half-lives, whichever comes later, immediately up to the date of randomization. - Subject with any significant medical condition, laboratory abnormality, psychiatric illness, or is considered vulnerable by local regulations (eg, imprisoned or institutionalized) that would prevent the subject from participating in the study or places the subject at unacceptable risk if he/she were to participate in the study. 18.Subject with any condition or concomitant medication that confounds the ability to interpret data from the study. - Other protocol-defined Inclusion/Exclusion criteria apply. |
Country | Name | City | State |
---|---|---|---|
Argentina | Hospital Italiano de Buenos Aires | Ciudad Autonoma de Buenos Aires | |
Argentina | Hospital Britanico de Buenos Aires | Ciudad Autónoma de BuenosAires | Buenos Aires |
Argentina | Hospital Italiano de La Plata | La Plata | Buenos Aires |
Australia | Monash Medical Centre | Clayton | Victoria |
Australia | Gosford Hospital | Gosford | |
Australia | Royal Hobart Hospital | Hobart | |
Australia | The Alfred Hospital | Melbourne | Victoria |
Australia | Sir Charles Gairdner Hospital | Nedlands | Western Australia |
Austria | Local Institution - 272 | Graz | |
Austria | Krankenhaus der Elisabethinen Linz, I Interne Abteilung | Linz | |
Austria | Local Institution - 271 | Vienna | |
Austria | Local Institution - 274 | Vienna | |
Belgium | AZ Sint-Jan AV Brugge | Brugge | |
Belgium | Cliniques Universitaires Saint-Luc | Brussels | |
Belgium | Local Institution - 313 | Hasselt | |
Belgium | Uz Leuven | Leuven | |
Belgium | Centre Hospitalier Universitaire de Liège - Domaine Universitaire du Sart Tilman | Liège | |
Belgium | AZ Delta vzw | Roeselare | |
Belgium | Centre Hospitalier Peltzer - La Tourelle | Verviers | |
Belgium | Cliniques Universitaires UCL de Mont-Godine | Yvoir | |
Canada | Local Institution - 181 | Calgary | Alberta |
Canada | University Of Alberta Hospital | Edmonton | Alberta |
Canada | University Hospital - London Health Sciences Centre | London | Ontario |
Canada | Hopital Maisonneuve-Rosemont | Montreal | Quebec |
Canada | Sir Mortimer B. Davis - Jewish Genl | Montreal | Quebec |
Canada | Local Institution - 176 | Sherbrooke | Quebec |
Canada | Princess Margaret Cancer Centre | Toronto | Ontario |
Canada | St. Paul'S Hospital | Vancouver | British Columbia |
Chile | IC La Serena Research | La Serena | Coquimbo |
Chile | Centro de Oncología de Precisión | Las Condes | Metropolitana De Santiago |
Chile | Enroll SpA - PPDS | Santiago | |
China | Beijing Peking Union Medical College Hospital | Beijing | |
China | First Hospital of Jilin University | Changchun | |
China | Xiangya Hospital Central-South University | Changsha | Hunan |
China | Guangdong General Hospital | Guangzhou | |
China | Nanfang Hospital of Southern Medical University | Guangzhou | Guangdong |
China | The First Affiliated Hospital Of Harbin Medical University | Harbin | |
China | The Second Affiliated Hospital Of Kunming Medical University | Kunming | Yunnan |
China | Nanchang University - The Second Affiliated Hospital | Nanchang | Jiangxi |
China | The First Affiliated Hospital of NanChang University | Nanchang | Jiangxi |
China | Jiangsu Province Hospital, The First Affiliated Hospital of Nanjing Medical University | Nanjing | Jiangsu |
China | Affiliated Hospital of Nantong University | Nantong | Jiangsu |
China | The First Affiliated Hospital of Nanyang Medical College | Nanyang | Henan |
China | The Affiliated Hospital Of Qingdao University | Qingdao | Shandong |
China | Ruijin Hospital Shanghai Jiaotong University | Shanghai | |
China | Shanghai 6th Hospital | Shanghai | |
China | The First Affiliated Hospital Of Soochow University | Suzhou | |
China | Second Hospital of Shanxi Medical University | Taiyuan | Shanxi |
China | Chinese Academy of Medical Sciences & Peking Union Medical College | Tianjin | |
China | Tianjin Medical University General Hospital | Tianjin | |
China | Henan Cancer Hospital | Zhengzhou | |
Colombia | Instituto Nacional De Cancerologia | Bogota | Distrito Capital De Bogotai |
Colombia | Fundacion Oftalmologica De Santander - Foscal | Floridablanca | Soto |
Colombia | Hospital Pablo Tobon Uribe | Medellin | Antioquia |
Czechia | Vseobecna Fakultni Nemocnice V Praze | Prague 2 | |
France | CHRU Hopital du bocage | Angers | |
France | Chu Estaing | Clermont Ferrand | |
France | Local Institution - 324 | Creteil | |
France | Chu De Grenoble | Grenoble | |
France | CHRU de Lille-Hopital Claude Huriez | Lille | |
France | Centre Leon Berard | Lyon | |
France | CHU de Nice Archet I | Nice | |
France | Centre Hospitalier Universitaire de Nimes (CHU) - Hopital Universitaire Caremeau | Nimes Cedex 9 | |
France | Hopital Saint Louis | Paris Cedex 10 | |
France | Groupe Hospitalier Sud Hopital Haut Leveque USN | Pessac | |
France | CHU La Miletrie | Poitiers Cedex | |
France | ICANS Institut de cancerologie Strasbourg Europe | Strasbourg | |
France | Local Institution - 330 | Toulouse Cedex 9 | |
Germany | Unviversitatsklinikum Aachen | Aachen | |
Germany | Stauferklinikum Schwab. Gmund | Baden-Warttemberg | |
Germany | Universitaetsklinikum Duesseldorf | Dusseldorf | |
Germany | Universitatsklinikum Halle Saale | Halle | |
Germany | Local Institution - 300 | Hamburg | |
Germany | Universitaetsklinikum Jena | Jena | |
Germany | Universitatsklinikum Leipzig | Leipzig | |
Germany | Local Institution - 301 | Mannheim | |
Germany | Johannes Wiesling Klinikum Minden | Minden | |
Greece | University Hospital of Alexandroupolis | Alexandroupolis | |
Greece | Attikon University General Hospital | Athens | |
Greece | Evangelismos General Hospital of Athens | Athens | |
Greece | Local Institution - 386 | Athens | |
Greece | Local Institution - 387 | Patra | Achaia |
Greece | University General Hospital of Patras | Rio Patras | |
Greece | Georgios Papanikolaou General Hospital of Thessaloniki | Thessaloniki | |
Hong Kong | Queen Mary Hospital | Hong Kong | |
Hong Kong | Prince of Wales Hospital the Chinese University of Hong Kong | Sha Tin | |
Hungary | Del-pesti Centrumkorhaz- Orszagos Hematologiai és Infektologiai Intezet | Budapest | |
Hungary | Petz Aladár Egyetemi Oktató Kórház | Gyor | |
Ireland | Cork University Hospital | Cork | |
Ireland | St James Hospital | Dublin | |
Ireland | Mater Misercordiae Hospital | Dublin 7 | |
Israel | Rambam Medical Center | Haifa | |
Israel | Hadassah Medical Organization | Jerusalem | |
Israel | Meir Medical Center | Kfar-Saba | |
Israel | Tel-Aviv Sourasky Medical Center | Tel-Aviv | Tel Aviv |
Israel | Shamir Medical Center - Assaf Harofeh | Zerifin | |
Italy | Azienda Ospedaliero Universitaria Ospedali Riuniti Umberto I, G.M. Lancisi, G. Salesi | Ancona | |
Italy | Azienda Ospedaliero-Universitaria di Bologna - Policlinico S.Orsola-Malpighi | Bologna | |
Italy | Asst Spedali Civili Di Brescia | Brescia | |
Italy | Azienda Ospedaliero - Universitaria Policlinico - Vittorio Emanuele - Ospedale Gaspare Rodolico | Catania | |
Italy | Azienda Ospedaliera Universitaria Careggi | Firenze | |
Italy | IRCCS - Istituto Romagnolo per lo Studio Dei Tumori "Dino Amadori" (IRST) | Meldola (fc) | Fc |
Italy | Fondazione Irccs Ca' Granda Ospedale Maggiore Policlinico | Milano | |
Italy | Azienda Ospedaliera Universitaria Federico Ii | Napoli Campania | |
Italy | A.O.U. Maggiore della Carit | Novara | |
Italy | Azienda Ospedaliera Di Padova | Padova | |
Italy | Azienda Ospedaliero Universitaria Pisana | Pisa | |
Italy | Grande Ospedale Metropolitano Bianchi-Melacrino-Morelli | Reggio Di Calabria | |
Italy | Azienda Ospedaliera Sant Andrea | Roma | |
Italy | Azienda Policlinico Universitario Umberto I | Roma | |
Italy | Ospedale S Eugenio | Roma | |
Italy | Local Institution - 245 | Terni | |
Italy | A.O.U. Citta Della Salute E Della Scienza , Presidio Molinette,S.C. Ematologia Universitaria | Torino | |
Italy | Universita degli Studi dell'Insubria - Ospedale di Circolo e Fondazione Macchi - Varese | Varese | |
Italy | Centro Ricerche Cliniche di Verona S.r.l. | Verona | |
Japan | Aomori Prefectural Central Hospital | Aomori | |
Japan | Juntendo University Hospital | Bunkyo-ku | Tokyo |
Japan | Tokyo Metropolitan Cancer and Infectious Diseases Center Komagome Hospital | Bunkyo-ku | |
Japan | University of Yamanashi Hospital | Chuo | |
Japan | Tokai University Hospital | Isehara City, Kanagawa | |
Japan | Shonan Kamakura General Hospital | Kamakura | |
Japan | Kameda General Hospital | Kamogawa | |
Japan | Local Institution - 706 | Maebashi | |
Japan | University of Miyazaki Hospital | Miyazaki | |
Japan | The Japanese Red Cross Nagasaki Genbaku Hospital | Nagasaki-shi | Nagasaki |
Japan | Ogaki Municipal Hospital | Ogaki | |
Japan | Osaka Metropolitan university Hospital | Osaka | |
Japan | Kindai University Hospital- Osakasayama Campus | Osakasayama | Osaka |
Japan | Local Institution - 708 | Sapporo | |
Japan | NTT Medical Center Tokyo | Shinagawa-ku, Tokyo | |
Japan | Tokyo Women's Medical University Hospital | Shinjuku City | |
Japan | Local Institution - 710 | Shinjyuku-ku | |
Japan | Toyohashi Municipal Hospital | Toyohashi | |
Korea, Republic of | Kyungpook National University Hospital | Daegu | |
Korea, Republic of | Chonnam National University Hwasun Hospital | Hwasun-Gun | |
Korea, Republic of | Seoul National University Bundang Hospital | Seongnam-si | |
Korea, Republic of | Asan Medical Center | Seoul | |
Korea, Republic of | Samsung Medical Center | Seoul | |
Korea, Republic of | Seoul National University Hospital | Seoul | |
Korea, Republic of | The Catholic University of Korea Seoul - Saint Mary's Hospital | Seoul | |
Lebanon | American Univ of Beirut Med Center | Badaro Beirut | |
Lebanon | LAU Medical Center Rizk Hospital | Beirut | |
Lebanon | Hammoud Hospital University Medical Center | Saida | South |
Poland | Uniwersyteckie Centrum Kliniczne | Gdansk | |
Poland | Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki w Krakowie | Krakow | |
Poland | Wojewódzki Szpital Specjalistyczny im. M. Kopernika w Lodzi | Lodz | |
Poland | ALVAMED | Poznan | |
Poland | Specjalistyczny Szpital im. dra Alfreda Sokolowskiego | Walbrzych | |
Poland | Samodzielny Publiczny Szpital Kliniczny Nr 1 we Wroclawiu | Wroclaw | |
Romania | Onco Card SRL | Brasov | |
Romania | Fundeni Clinical Institute | Bucharest | |
Romania | Prof. Dr. I. Chiricuta Institute of Oncology | Cluj-Napoca | |
Romania | Spitalul Clinic Municipal Filantropia Craiova | Craiova | Dolj |
Russian Federation | Local Institution - 500 | Moscow | |
Russian Federation | Local Institution - 502 | St Petersburg | |
Russian Federation | Local Institution - 503 | St. Petersburg | |
Spain | Hospital Clinic de Barcelona | Barcelona | |
Spain | Hospital Universitari Germans Trias i Pujol ICO Badalona | Barcelona | |
Spain | Hospital Virgenes de las Nieves | Granada | |
Spain | Hospital Universitario De Gran Canaria Dr. Negrin | Las Palmas de Gran Canaria | |
Spain | Hospital Universitario 12 De Octubre | Madrid | |
Spain | Hospital Universitario Ramón y Cajal | Madrid | |
Spain | Local Institution - 202 | Palma de Mallorca | |
Spain | Universitario de Salamanca - Hospital Clinico | Salamanca | |
Spain | Complejo Hospitalario Universitario De Santiago | Santiago de Compostela | |
Spain | Hospital Universitario Virgen del Rocio | Seville | |
Spain | Hospital Clinico Universitario De Valencia | Valencia | |
United Kingdom | Heart of England NHS Foundation Trust | Birmingham | |
United Kingdom | United Lincolnshire Hospitals NHS Trust | Boston | |
United Kingdom | Nottingham City Hospital | Nottingham | Nottinghamshire |
United Kingdom | Churchhill Hospital | Oxford | |
United States | Local Institution - 114 | Ann Arbor | Michigan |
United States | Local Institution - 112 | Chicago | Illinois |
United States | John Theurer Cancer Center | Hackensack | New Jersey |
United States | The University of Texas - MD Anderson Cancer Center | Houston | Texas |
United States | University of Tennessee Medical Center | Knoxville | Tennessee |
United States | University Of Kentucky Markey Cancer Center | Lexington | Kentucky |
United States | University Of California Los Angeles | Los Angeles | California |
United States | Mount Sinai Medical Center | New York | New York |
United States | Local Institution - 135 | Orlando | Florida |
United States | Allegheny Health Network | Pittsburgh | Pennsylvania |
United States | University of Pittsburg Medical Center | Pittsburgh | Pennsylvania |
United States | BRCR Medical Center Inc. | Plantation | Florida |
United States | Local Institution - 108 | Saint Louis | Missouri |
United States | University of Utah - Huntsman Cancer Institute | Salt Lake City | Utah |
Lead Sponsor | Collaborator |
---|---|
Celgene |
United States, Argentina, Australia, Austria, Belgium, Canada, Chile, China, Colombia, Czechia, France, Germany, Greece, Hong Kong, Hungary, Ireland, Israel, Italy, Japan, Korea, Republic of, Lebanon, Poland, Romania, Russian Federation, Spain, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Red blood cell-transfusion independence (RBC-TI) = 12 weeks (RBC-TI 12) | Proportion of subjects who become RBC-transfusion free over any consecutive 12-week period starting within the first 24 weeks. | Up to 24 weeks | |
Secondary | Red blood cell-transfusion independence = 16 weeks (RBC-TI 16) | Proportion of subjects who become RBC-transfusion free over any consecutive 16-week period | Up to 24 weeks | |
Secondary | Duration of Red blood cell-transfusion independence (RBC-TI 12) | Maximum duration of RBC-TI response | Up to end of treatment, approximately 3 years | |
Secondary | Reduction of transfusion burden by = 50% and by = 4 units/12 weeks from baseline over any consecutive 12-week period | Proportion of subjects who reduce their transfusion burden by = 50% and by = 4 units/12 weeks from baseline over any consecutive 12-week period | Up to 24 weeks | |
Secondary | Duration of reduction in transfusion burden | Maximum duration of when RBC-transfusion dependent subjects who reduce their transfusion burden by = 50% and by = 4 units/12 weeks from baseline over any consecutive 12 week period | Up to end of treatment, approximately 3 years | |
Secondary | Red blood cell-transfusion independence = 12 weeks in the treatment period (RBC-TI 12/TP) | Proportion of subjects who become RBC-transfusion free over any consecutive 12-week period | Up to end of treatment, approximately 3 years | |
Secondary | Red blood cell-transfusion independence = 16 weeks in the treatment period (RBC-TI 16/TP) | Proportion of subjects who become RBC-transfusion free over any consecutive 16-week period | Up to end of treatment, approximately 3 years | |
Secondary | Change in RBC transfusion burden | Mean change in transfusion burden (RBC units) from baseline | Up to 24 weeks | |
Secondary | Cumulative duration of RBC-transfusion independence | Cumulative response duration for subjects achieving multiple episodes of RBC-TI 12 | Up to end of treatment, approximately 3 years | |
Secondary | Mean Hgb increase = 1 g/dL from baseline over any consecutive 12-week period in absence of RBC transfusions | Proportion of subjects achieving a mean Hgb increase = 1 g/dL from baseline over any consecutive 12-week period in absence of RBC transfusions | Up to end of treatment, approximately 3 years | |
Secondary | Change in serum ferritin from baseline | Change in serum ferritin | Up to end of treatment, approximately 3 years | |
Secondary | Incidence of Adverse Events (AEs) | Number of participants with adverse events | From screening up to 42 days post last dose | |
Secondary | Transformation to blast phase: Number of subjects who transform into AML | AML = acute myeloid leukemia | Up to approximately 5 years | |
Secondary | Frequency of Antidrug antibodies (ADA) | Will be collected for assessment of anti-drug antibodies (ADA) against Luspatercept in serum in all subjects | From randomization and up to including 48 weeks post first dose | |
Secondary | Pharmacokinetics - Area Under the Concentration-Time Curve (AUC) | Measures of luspatercept exposure area under the curve | From randomization and up to including 48 weeks post first dose | |
Secondary | Pharmacokinetics - Maximum plasma concentration of drug (Cmax) | Maximum plasma concentration of drug | From randomization and up to including 48 weeks post first dose |
Status | Clinical Trial | Phase | |
---|---|---|---|
Terminated |
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