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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT04717414
Other study ID # ACE-536-MF-002
Secondary ID 2020-000607-36U1
Status Recruiting
Phase Phase 3
First received
Last updated
Start date February 25, 2021
Est. completion date August 23, 2025

Study information

Verified date June 2024
Source Celgene
Contact BMS Clinical Trials Contact Center www.BMSClinicalTrials.com
Phone 855-907-3286
Email Clinical.Trials@bms.com
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

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.


Description:

Permitted Concomitant Medications and Procedures - Subjects are receiving a JAK2 inhibitor for the treatment of MPN-associated MF that is approved in the country where the study is being conducted. JAK2 inhibitors are to be used according to their respective label and as prescribed as part of the subject's standard-of-care therapy as prescribed by their physician prior to study entry. - Best supportive care (BSC) includes, but is not limited to, treatment with transfusions (eg, RBC, platelet, whole blood), ICTs, antibiotic, antiviral and/or antifungal therapy, and nutritional support as needed. - Granulocyte colony-stimulating factors (ie, G-CSF, granulocyte macrophage colony-stimulating factor [GM-CSF]) are allowed only in cases of neutropenic fever or as clinically indicated per product label. - Prophylactic antithrombotic therapy is permitted. - Thrombopoietin and platelet transfusions are permitted. - Treatment with systemic corticosteroids is permitted for nonhematological conditions providing the subject is receiving a constant dose equivalent to ≤ 10 mg prednisone during the study. - Administration of attenuated vaccines (eg, influenza vaccine) is allowed if clinically indicated per Investigator discretion. - Iron chelation therapy (ICT) is to be used according to the product label. If the label permits, the ICT dose should be stable during at least the first 24 weeks of IP. Initiation of ICT while within the first 24 weeks of IP should be clinically indicated to treat an AE. Prohibited Concomitant Medications The following concomitant medications are specifically excluded during the course of study treatment: - Cytotoxic, chemotherapeutic, targeted, or investigational agents/therapies (excluding JAK2 inhibitor therapy) - Azacitidine, decitabine, or other hypomethylating agents - Lenalidomide, thalidomide, and pomalidomide - Erythropoietin stimulating agents (ESAs) and other RBC hematopoietic growth factors (eg, IL-3) - Hydroxyurea or other alkylating agents - Androgens (unless given to treat hypogonadism) - Oral retinoids (topical retinoids are permitted) - Arsenic trioxide - Interferon - Anagrelide - Systemic corticosteroids at a dose equivalent to > 10 mg prednisone - Investigational products for the treatment of MPN-associated MF


Recruitment information / eligibility

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.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
ACE-536
Subcutaneous Injection
Other:
Placebo
Subcutaneous Injection

Locations

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

Sponsors (1)

Lead Sponsor Collaborator
Celgene

Countries where clinical trial is conducted

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, 

Outcome

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
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