An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis on Concomitant JAK2 Inhibitor Therapy and Who Require Red Blood Cell Transfusions (INDEPENDENCE)

April 10, 2024 updated by: Celgene

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed 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

Study Type

Interventional

Enrollment (Estimated)

309

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: BMS Study Connect Contact Center www.BMSStudyConnect.com
  • Phone Number: 855-907-3286
  • Email: Clinical.Trials@bms.com

Study Contact Backup

  • Name: First line of email MUST contain NCT # and Site #.

Study Locations

  • Argentina
      • Ciudad Autonoma de Buenos Aires, Argentina, C1199ABB
        • Recruiting
        • Hospital Italiano de Buenos Aires
        • Contact:
          • Georgina Bendek, Site 171
          • Phone Number: +5491169370731
    • Buenos Aires
      • Ciudad Autónoma de BuenosAires, Buenos Aires, Argentina, C1280AEB
        • Recruiting
        • Hospital Britanico de Buenos Aires
        • Contact:
          • Silvina Palmer, Site 172
          • Phone Number: +5491162101221
      • La Plata, Buenos Aires, Argentina, B1900AX
        • Recruiting
        • Hospital Italiano de La Plata
        • Contact:
          • Jorge Milone, Site 173
          • Phone Number: 2214173869
  • Australia
      • Gosford, Australia, 2250
        • Recruiting
        • Gosford Hospital
        • Contact:
          • Cecily Forsyth, Site 601
          • Phone Number: 0404484360
      • Hobart, Australia, 7000
        • Recruiting
        • Royal Hobart Hospital
        • Contact:
          • Rosemary Harrup, Site 603
          • Phone Number: +0362228308 000 0000
    • Victoria
      • Clayton, Victoria, Australia, 3168
        • Recruiting
        • Monash Medical Centre
        • Contact:
          • Paul Yeh, Site 600
          • Phone Number: 0000000000
      • Melbourne, Victoria, Australia, 3004
        • Recruiting
        • The Alfred Hospital
        • Contact:
          • Andrew Perkins, Site 602
    • Western Australia
      • Nedlands, Western Australia, Australia, 6009
        • Recruiting
        • Sir Charles Gairdner Hospital
        • Contact:
          • Carolyn Grove, Site 604
          • Phone Number: 000-000-0000
  • Austria
      • Graz, Austria, 8036
        • Recruiting
        • Medizinische Universität Graz
        • Contact:
          • Albert Woelfler, Site 272
          • Phone Number: +4331638514087
      • Linz, Austria, 4020
        • Recruiting
        • Krankenhaus der Elisabethinen Linz, I Interne Abteilung
        • Contact:
          • Veronika Buxhofer-Ausch, Site 273
          • Phone Number: 0732 7676 4409
      • Vienna, Austria, 1090
        • Recruiting
        • Medizinische Universitat Wien, Universitatsklinik fur Dermatologie. Abteilung fur Immundermatologie
        • Contact:
          • Maria Krauth, Site 271
      • Vienna, Austria, 1140
        • Completed
        • Local Institution - 274
  • Belgium
      • Brugge, Belgium, 8000
        • Recruiting
        • AZ Sint-Jan AV Brugge
        • Contact:
          • Jan Van Droogenbroeck, Site 318
          • Phone Number: +3250452627
      • Brussels, Belgium, 1200
        • Recruiting
        • Cliniques Universitaires Saint-Luc
        • Contact:
          • Violaine Havelange, Site 312
      • Hasselt, Belgium, 3500
        • Completed
        • Local Institution - 313
      • Leuven, Belgium, 3000
        • Recruiting
        • UZ Leuven
        • Contact:
          • Timothy Devos, Site 311
          • Phone Number: 3216346880
      • Liège, Belgium, 4000
        • Recruiting
        • Centre Hospitalier Universitaire de Liège - Domaine Universitaire du Sart Tilman
        • Contact:
          • Gaelle Vertenoeil, Site 319
      • Roeselare, Belgium, 8800
        • Recruiting
        • AZ Delta vzw
        • Contact:
          • Lien Deleu, Site 316
          • Phone Number: 3251237560
      • Verviers, Belgium, 4800
        • Recruiting
        • Centre Hospitalier Peltzer - La Tourelle
        • Contact:
          • Gaetan Vanstraelen, Site 315
          • Phone Number: +3287212171
      • Yvoir, Belgium, 5530
        • Recruiting
        • Cliniques Universitaires UCL de Mont-Godine
        • Contact:
          • Julien Depaus, Site 314
  • Canada
    • Alberta
      • Calgary, Alberta, Canada, T2N 4N2
        • Completed
        • Local Institution - 181
      • Edmonton, Alberta, Canada, T6G 2S2
        • Recruiting
        • University of Alberta Hospital
        • Contact:
          • Elena Liew, Site 179
    • British Columbia
      • Vancouver, British Columbia, Canada, V6Z 2A5
        • Recruiting
        • St. Paul's Hospital
        • Contact:
          • Lynda Foltz, Site 183
    • Ontario
      • London, Ontario, Canada, N6C 6B5
        • Recruiting
        • University Hospital - London Health Sciences Centre
        • Contact:
          • Cyrus Hsia, Site 178
          • Phone Number: 5198525308
      • Toronto, Ontario, Canada, M5G 2M9
        • Recruiting
        • Princess Margaret Cancer Centre
        • Contact:
          • Dawn Maze, Site 180
          • Phone Number: 4169464501
    • Quebec
      • Montreal, Quebec, Canada, H1T 2M4
        • Recruiting
        • Hopital Maisonneuve-Rosemont
        • Contact:
          • Lambert Busque, Site 177
          • Phone Number: 5142523404
      • Montreal, Quebec, Canada, H3T 1E2
        • Recruiting
        • Sir Mortimer B. Davis - Jewish Genl
        • Contact:
          • Shireen Sirhan, Site 182
      • Sherbrooke, Quebec, Canada, J1H5N4
        • Completed
        • Local Institution - 176
  • Chile
      • Santiago, Chile, 7500587
        • Recruiting
        • Enroll SpA - PPDS
        • Contact:
          • Ximena Valladares, Site 193
          • Phone Number: 569000000000
    • Coquimbo
      • La Serena, Coquimbo, Chile, 1720430
        • Recruiting
        • IC La Serena Research
        • Contact:
          • Luisa Morales, Site 191
          • Phone Number: 56993718661
    • Metropolitana De Santiago
      • Las Condes, Metropolitana De Santiago, Chile, 7560742
        • Recruiting
        • Centro de Oncología de Precisión
        • Contact:
          • Marcelo Garrido Salvo, Site 192
          • Phone Number: 56223592703
  • China
      • Beijing, China, 100730
        • Recruiting
        • Beijing Peking Union Medical College Hospital
        • Contact:
          • Minghui Duan, Site 806
      • Changchun, China, 130021
        • Recruiting
        • First Hospital of Jilin University
        • Contact:
          • Sujun Gao, Site 802
          • Phone Number: +8615843073208
      • Guangzhou, China, 510030
        • Recruiting
        • Guangdong General Hospital
        • Contact:
          • Jianyu Weng, Site 805
          • Phone Number: 8602083889772
      • Harbin, China, 150081
        • Recruiting
        • The First Affiliated Hospital of Harbin Medical University
        • Contact:
          • Tiejun Gong, Site 808
      • Shanghai, China, 200025
        • Recruiting
        • Ruijin Hospital Shanghai Jiaotong University
        • Contact:
          • Junmin Li, Site 809
          • Phone Number: +8613817712211 0000
      • Shanghai, China, 200233
        • Recruiting
        • Shanghai 6th Hospital
        • Contact:
          • Chunkang Chang, Site 801
          • Phone Number: 13764643870
      • Suzhou, China, 215006
        • Recruiting
        • The First Affiliated Hospital of Soochow University
        • Contact:
          • Suning Chen, Site 811
      • Tianjin, China, 300041
        • Recruiting
        • Chinese Academy of Medical Sciences & Peking Union Medical College
        • Contact:
          • zhijian xiao, Site 800
          • Phone Number: +86 13821085716
      • Tianjin, China, 300052
        • Recruiting
        • Tianjin Medical University General Hospital
        • Contact:
          • RONG FU, Site 813
          • Phone Number: 13920350233
      • Zhengzhou, China
        • Recruiting
        • Henan Cancer Hospital
        • Contact:
          • Xudong Wei, Site 810
          • Phone Number: +8613837169301
    • Guangdong
      • Guangzhou, Guangdong, China, 510515
        • Recruiting
        • Nanfang Hospital of Southern Medical University
        • Contact:
          • Na Xu, Site 807
    • Henan
      • Nanyang, Henan, China
        • Recruiting
        • The First affiliated Hospital of Nanyang Medical College
        • Contact:
          • Huibing Dang, Site 814
    • Hunan
      • Changsha, Hunan, China, 410008
        • Recruiting
        • Xiangya Hospital Central-South University
        • Contact:
          • yanjuan He, Site 812
          • Phone Number: 15973130688
    • Jiangsu
      • Nanjing, Jiangsu, China, 210029
        • Recruiting
        • Jiangsu Province Hospital, The First Affiliated Hospital of Nanjing Medical University
        • Contact:
          • Wenyi Shen, Site 804
      • Nantong, Jiangsu, China, 226001
        • Recruiting
        • Affiliated Hospital Of Nantong University
        • Contact:
          • Hong Liu, Site 818
          • Phone Number: +8613951300660
    • Jiangxi
      • Nanchang, Jiangxi, China, 330006
        • Recruiting
        • The First Affiliated Hospital of NanChang University
        • Contact:
          • Fei Li, Site 820
      • Nanchang, Jiangxi, China, 330008
        • Recruiting
        • Nanchang University - The Second Affiliated Hospital
        • Contact:
          • Aiping Tang, Site 822
    • Shandong
      • Qingdao, Shandong, China
        • Recruiting
        • The Affiliated Hospital of Qingdao University
        • Contact:
          • Chunting Zhao, Site 821
    • Shanxi
      • Taiyuan, Shanxi, China, 030001
        • Recruiting
        • Second Hospital of Shanxi Medical University
        • Contact:
          • Yanping Ma, Site 816
    • Yunnan
      • Kunming, Yunnan, China, 650101
        • Recruiting
        • The Second Affiliated hospital of Kunming Medical University
        • Contact:
          • Zeping Zhou, Site 819
          • Phone Number: 18788571605
  • Colombia
    • Antioquia
      • Medellin, Antioquia, Colombia, 50034
        • Recruiting
        • Hospital Pablo Tobon Uribe
        • Contact:
          • Kenny Galvez, Site 161
          • Phone Number: 5731489056030000
    • Distrito Capital De Bogotai
      • Bogota, Distrito Capital De Bogotai, Colombia, 111511
        • Not yet recruiting
        • Local Institution - 163
        • Contact:
          • Site 163
    • Soto
      • Floridablanca, Soto, Colombia, 681002
        • Recruiting
        • Fundacion Oftalmologica de Santander - FOSCAL
        • Contact:
          • Claudia Lucia Sossa Melo, Site 162
          • Phone Number: 5776382828
  • Czechia
      • Prague 2, Czechia, 128 08
        • Recruiting
        • Vseobecna Fakultni Nemocnice V Praze
        • Contact:
          • Anna JONASOVA, Site 341
          • Phone Number: +420224966209
  • France
      • Angers, France, 49033
        • Recruiting
        • CHRU Hopital du bocage
        • Contact:
          • Francoise Boyer, Site 331
          • Phone Number: +33241354472 000000
      • Clermont Ferrand, France, 63000
        • Recruiting
        • CHU Estaing
        • Contact:
          • Gaspar Aspas Requena, Site 333
      • Creteil, France, 94010
        • Completed
        • Local Institution - 324
      • Grenoble, France, 38043
        • Recruiting
        • CHU de Grenoble
        • Contact:
          • Frederic Garban, Site 329
          • Phone Number: +33476765096
      • Lille, France, 59037
        • Recruiting
        • CHRU de Lille-Hopital Claude Huriez
        • Contact:
          • Mathieu Wemeau, Site 327
      • Lyon, France, 69008
        • Recruiting
        • Centre LEON BERARD
        • Contact:
          • Franck Nicolini, Site 332
      • Nice, France, 06202
        • Recruiting
        • CHU de Nice Archet I
        • Contact:
          • Michael Loschi, Site 325
          • Phone Number: 33492035841
      • Nimes Cedex 9, France, 30029
        • Recruiting
        • Centre Hospitalier Universitaire de Nimes (CHU) - Hopital Universitaire Caremeau
        • Contact:
          • stefan Wickenhauser, Site 322
          • Phone Number: 0466684673
      • Paris Cedex 10, France, 75475
        • Recruiting
        • Hôpital Saint Louis
        • Contact:
          • Jean-Jacques Kiladjian, Site 321
          • Phone Number: +33142499494
      • Pessac, France, 33604
        • Recruiting
        • Groupe Hospitalier Sud Hopital Haut Leveque USN
        • Contact:
          • Clémence Mediavilla, Site 323
          • Phone Number: +33557656514
      • Poitiers Cedex, France, 86021
        • Recruiting
        • CHU La Milétrie
        • Contact:
          • Jose Torregrosa Diaz, Site 326
      • Strasbourg, France, 67200
        • Recruiting
        • Icans Institut de Cancerologie Strasbourg Europe
        • Contact:
          • Shanti Natarajan-Ame, Site 328
          • Phone Number: +33388127676
      • Toulouse Cedex 9, France, 31059
        • Completed
        • Local Institution - 330
  • Germany
      • Aachen, Germany, 52074
        • Recruiting
        • Unviversitatsklinikum Aachen
        • Contact:
          • Steffen Koschmieder, Site 291
          • Phone Number: +492518352671
      • Baden-Warttemberg, Germany, 73557
        • Recruiting
        • Stauferklinikum Schwab. Gmund
        • Contact:
          • Holger Hebart, Site 294
          • Phone Number: 4971717011302
      • Dusseldorf, Germany, 40225
        • Recruiting
        • Universitaetsklinikum Duesseldorf
        • Contact:
          • Norbert Gattermann, Site 299
          • Phone Number: 0211-81 16500
      • Halle, Germany, 06120
        • Recruiting
        • Universitatsklinikum Halle Saale
        • Contact:
          • Haifa-Kathrin Al-Ali, Site 293
          • Phone Number: +493455574959 000 0
      • Hamburg, Germany, 22081
        • Recruiting
        • OncoResearch Lerchenfeld GmbH
        • Contact:
          • Thomas Wolff, Site 300
          • Phone Number: +494022604650
      • Jena, Germany, 07740
        • Recruiting
        • Universitaetsklinikum Jena
        • Contact:
          • Carl Crodel, Site 295
          • Phone Number: 4936419324236
      • Leipzig, Germany, 04103
        • Recruiting
        • Universitatsklinikum Leipzig
        • Contact:
          • Barbara Madlen Jentzsch, Site 297
      • Mannheim, Germany, 68167
        • Recruiting
        • Universitaetsklinikum Mannheim
        • Contact:
          • Andreas Reiter, Site 301
      • Minden, Germany, 32429
        • Recruiting
        • Johannes Wiesling Klinikum Minden
        • Contact:
          • Martin Griesshammer, Site 292
          • Phone Number: +4905718014812
  • Greece
      • Alexandroupolis, Greece, 08100
        • Recruiting
        • University Hospital of Alexandroupolis
        • Contact:
          • Ioannis Kotsianidis, Site 383
          • Phone Number: +00302551030320 0000
      • Athens, Greece, 10676
        • Recruiting
        • Evangelismos General Hospital of Athens
        • Contact:
          • Maria Pagoni, Site 384
          • Phone Number: +306977343651
      • Athens, Greece, 12464
        • Recruiting
        • Attikon University General Hospital
        • Contact:
          • Vassiliki Pappa, Site 385
          • Phone Number: +302105831000 000 00
      • Athens, Greece, 11 527
        • Completed
        • Local Institution - 386
      • Rio Patras, Greece, 26500
        • Recruiting
        • University General Hospital of Patras
        • Contact:
          • Alexandros Spyridonidis, Site 381
          • Phone Number: 306977448977
      • Thessaloniki, Greece, 57010
        • Recruiting
        • Georgios Papanikolaou General Hospital of Thessaloniki
        • Contact:
          • Damianos Sotiropoulos, Site 382
          • Phone Number: +306945971300
    • Achaia
      • Patra, Achaia, Greece, 264 43
        • Not yet recruiting
        • Local Institution - 387
        • Contact:
          • Site 387
  • Hong Kong
      • Hong Kong, Hong Kong
        • Recruiting
        • Queen Mary Hospital
        • Contact:
          • Harry Gill-Harinder Singh, Site 661
      • Sha Tin, Hong Kong
        • Recruiting
        • Prince of Wales Hospital The Chinese University of Hong Kong
        • Contact:
          • Raymond Wong, Site 662
  • Hungary
      • Budapest, Hungary, 1096
        • Recruiting
        • Del-pesti Centrumkorhaz- Orszagos Hematologiai és Infektologiai Intezet
        • Contact:
          • Gabor Mikala, Site 462
      • Gyor, Hungary, 9023
        • Recruiting
        • Petz Aladár Egyetemi Oktató Kórház
        • Contact:
          • Eszter Sári, Site 463
  • Ireland
      • Cork, Ireland, T12 DFK4
        • Recruiting
        • Cork University Hospital
        • Contact:
          • Clodagh Keohane, Site 423
      • Dublin, Ireland, Dublin 8
        • Recruiting
        • St James Hospital
        • Contact:
          • Eibhlin Conneally, Site 421
          • Phone Number: +35314103545
      • Dublin 7, Ireland, 7
        • Recruiting
        • Mater Misercordiae Hospital
        • Contact:
          • Anne Fortune, Site 422
          • Phone Number: +353018545075
  • Israel
      • Haifa, Israel, 31096
        • Recruiting
        • Rambam Medical Center
        • Contact:
          • Noa Lavi, Site 523
          • Phone Number: 97248542541
      • Jerusalem, Israel, 91120
        • Recruiting
        • Hadassah Medical Organization
        • Contact:
          • David Lavie, Site 522
          • Phone Number: 97226778180
      • Kfar-Saba, Israel, 44281
        • Recruiting
        • Meir Medical Center
        • Contact:
          • Martin Ellis, Site 521
          • Phone Number: +97297471504
      • Zerifin, Israel, 70300
        • Recruiting
        • Shamir Medical Center - Assaf Harofeh
        • Contact:
          • Maya Koren-Michowitz, Site 525
          • Phone Number: +00972505191131
    • Tel Aviv
      • Tel-Aviv, Tel Aviv, Israel, 64239
        • Recruiting
        • Tel-Aviv Sourasky Medical Center
        • Contact:
          • Ilya Kirgner, Site 524
          • Phone Number: 972524266648
  • Italy
      • Ancona, Italy, 60126
        • Recruiting
        • Azienda Ospedaliero Universitaria Ospedali Riuniti Umberto I, G.M. Lancisi, G. Salesi
        • Contact:
          • Attilio Olivieri, Site 250
          • Phone Number: +00390971613660 0000
      • Bologna, Italy, 40138
        • Recruiting
        • Azienda Ospedaliero-Universitaria di Bologna - Policlinico S.Orsola-Malpighi
        • Contact:
          • FRANCESCA PALANDRI, Site 247
          • Phone Number: +39 0512143044
      • Brescia, Italy, 25123
        • Recruiting
        • ASST Spedali Civili di Brescia
        • Contact:
          • Mariella Dadda, Site 256
      • Catania, Italy, 95123
        • Recruiting
        • Azienda Ospedaliero - Universitaria Policlinico - Vittorio Emanuele - Ospedale Gaspare Rodolico
        • Contact:
          • Giuseppe Palumbo, Site 243
          • Phone Number: +390957436250
      • Firenze, Italy, 50134
        • Recruiting
        • Azienda Ospedaliera Universitaria Careggi
        • Contact:
          • Paola Guglielmelli, Site 241
          • Phone Number: 3318518346
      • Milano, Italy, 20122
        • Recruiting
        • Fondazione Irccs Ca' Granda Ospedale Maggiore Policlinico
        • Contact:
          • Francesco Passamonti, Site 255
          • Phone Number: +39 02 5503 3422
      • Napoli Campania, Italy, 80131
        • Recruiting
        • Azienda Ospedaliera Universitaria Federico II
        • Contact:
          • Fabrizio Pane, Site 246
      • Novara, Italy, 28100
        • Recruiting
        • A.O.U. Maggiore della Carit
        • Contact:
          • Andrea Patriarca, Site 252
      • Padova, Italy, 35128
        • Recruiting
        • Azienda Ospedaliera di Padova
        • Contact:
          • Gianni Binotto, Site 253
          • Phone Number: +39498212298 00 000
      • Pisa, Italy, 56100
        • Recruiting
        • Azienda Ospedaliero Universitaria Pisana
        • Contact:
          • Sara Galimberti, Site 248
          • Phone Number: +393470038656
      • Reggio Di Calabria, Italy, 89124
        • Recruiting
        • Grande Ospedale Metropolitano Bianchi-Melacrino-Morelli
        • Contact:
          • Caterina Alati, Site 244
      • Roma, Italy, 00189
        • Recruiting
        • Azienda Ospedaliera Sant Andrea
        • Contact:
          • Agostino Tafuri, Site 251
          • Phone Number: 06337715113
      • Roma, Italy, 144
        • Recruiting
        • Ospedale S Eugenio
        • Contact:
          • Elisabetta Abruzzese, Site 249
          • Phone Number: +390651008984
      • Roma, Italy, 00100
        • Recruiting
        • Azienda Policlinico Universitario Umberto I
        • Contact:
          • Massimo Breccia, Site 254
      • Terni, Italy, 05100
        • Completed
        • Local Institution - 245
      • Torino, Italy, 10126
        • Recruiting
        • A.O.U. Citta Della Salute E Della Scienza , Presidio Molinette,S.C. Ematologia Universitaria
        • Contact:
          • Giulia Benevolo, Site 259
          • Phone Number: +00390116335937 0000
      • Varese, Italy, 21100
        • Recruiting
        • Universita degli Studi dell'Insubria - Ospedale di Circolo e Fondazione Macchi - Varese
        • Contact:
          • Marco Brociner, Site 242
      • Verona, Italy, 37134
        • Recruiting
        • Centro Ricerche Cliniche Di Verona S.R.L.
        • Contact:
          • Massimiliano Bonifacio, Site 257
          • Phone Number: +390458124420
    • Fc
      • Meldola (fc), Fc, Italy, 47014
        • Recruiting
        • IRCCS - Istituto Romagnolo per lo Studio Dei Tumori "Dino Amadori" (IRST)
        • Contact:
          • Alessandro Lucchesi, Site 258
          • Phone Number: +390543739100
  • Japan
      • Aomori, Japan, 030-8553
        • Recruiting
        • Aomori Prefectural Central Hospital
        • Contact:
          • Kohmei Kubo, Site 700
          • Phone Number: 09075200987
      • Bunkyo-ku, Japan, 113-8677
        • Recruiting
        • Tokyo Metropolitan Cancer and Infectious Diseases Center Komagome Hospital
        • Contact:
          • Toya Takashi, Site 713
      • Chuo, Japan, 409-3898
        • Recruiting
        • University of Yamanashi Hospital
        • Contact:
          • KEITA KIRITO, Site 709
          • Phone Number: +81-55-273-9432
      • Isehara City, Kanagawa, Japan, 259-1193
        • Recruiting
        • Tokai University Hospital
        • Contact:
          • Yoshiaki Ogawa, Site 707
          • Phone Number: 8181463931121
      • Kamakura, Japan, 247-8533
        • Recruiting
        • Shonan Kamakura General Hospital
        • Contact:
          • Yotaro Tamai, Site 717
          • Phone Number: 0467-46-1717
      • Kamogawa, Japan, 296-8602
        • Recruiting
        • Kameda General Hospital
        • Contact:
          • Kosei Matsue, Site 703
          • Phone Number: 0470922211
      • Maebashi, Japan, 371-8511
        • Completed
        • Local Institution - 706
      • Miyazaki, Japan, 889-1692
        • Recruiting
        • University of Miyazaki Hospital
        • Contact:
          • Kazuya Shimoda, Site 712
          • Phone Number: 81985859121
      • Ogaki, Japan, 503-8502
        • Recruiting
        • Ogaki Municipal Hospital
        • Contact:
          • Hiroshi Kosugi, Site 716
          • Phone Number: 81584813341
      • Osaka, Japan, 545-8586
        • Recruiting
        • Osaka Metropolitan University Hospital
        • Contact:
          • Teruhito Takakuwa, Site 705
      • Sapporo, Japan, 003-0006
        • Completed
        • Local Institution - 708
      • Shinagawa-ku, Tokyo, Japan, 141-8625
        • Recruiting
        • Ntt Medical Center Tokyo
        • Contact:
          • Motoshi Ichikawa, Site 704
          • Phone Number: 03-3448-6111
      • Shinjuku City, Japan, 162-8666
        • Recruiting
        • Tokyo Women's Medical University Hospital
        • Contact:
          • Kentaro Yoshinaga, Site 714
      • Shinjyuku-ku, Japan, 160-0023
        • Completed
        • Local Institution - 710
      • Toyohashi, Japan, 441-8570
        • Recruiting
        • Toyohashi Municipal Hospital
        • Contact:
          • Shingo Kurahashi, Site 718
          • Phone Number: 81-532-33-6111
    • Nagasaki
      • Nagasaki-shi, Nagasaki, Japan, 8528511
        • Recruiting
        • The Japanese Red Cross Nagasaki Genbaku Hospital
        • Contact:
          • Tatsuro Jo, Site 702
          • Phone Number: +81-95-847-1511
    • Osaka
      • Osakasayama, Osaka, Japan, 5898511
        • Recruiting
        • Kindai University Hospital- Osakasayama Campus
        • Contact:
          • Hirokazu Tanaka, Site 711
          • Phone Number: 81723660221
    • Tokyo
      • Bunkyo-ku, Tokyo, Japan, 113-0033
        • Recruiting
        • Juntendo University Hospital
        • Contact:
          • Tadaaki Inano, Site 701
  • Korea, Republic of
      • Daegu, Korea, Republic of, 700-721
        • Recruiting
        • Kyungpook National University Hospital
        • Contact:
          • Joon-Ho Moon, Site 646
          • Phone Number: +82534205587-00-000
      • Hwasun-Gun, Korea, Republic of, 58128
        • Recruiting
        • Chonnam National University Hwasun Hospital
        • Contact:
          • Deok-Hwan Yang, Site 645
      • Seongnam-si, Korea, Republic of, 13620
        • Recruiting
        • Seoul National University Bundang Hospital
        • Contact:
          • Soo-Mee Bang, Site 643
          • Phone Number: +82-10-2760-9350
      • Seoul, Korea, Republic of, 06351
        • Recruiting
        • Samsung Medical Center
        • Contact:
          • Chul Won Jung, Site 647
      • Seoul, Korea, Republic of, 3080
        • Recruiting
        • Seoul National University Hospital
        • Contact:
          • Ja Min Byun, Site 641
      • Seoul, Korea, Republic of, 5505
        • Recruiting
        • Asan Medical Center
        • Contact:
          • Je-Hwan Lee, Site 642
          • Phone Number: 0230103218
      • Seoul, Korea, Republic of, 06591
        • Recruiting
        • The Catholic University of Korea Seoul - Saint Mary's Hospital
        • Contact:
          • Sung- Eun Lee, Site 644
  • Lebanon
      • Badaro Beirut, Lebanon, 11072280
        • Recruiting
        • American Univ of Beirut Med Center
        • Contact:
          • Ali Taher, Site 550
          • Phone Number: +9613755669 000000.0
      • Beirut, Lebanon, 11-3288
        • Recruiting
        • LAU Medical Center Rizk Hospital
        • Contact:
          • Colette Hanna, Site 552
    • South
      • Saida, South, Lebanon, 652
        • Recruiting
        • Hammoud Hospital University Medical Center
        • Contact:
          • Fadi Farhat, Site 551
  • Poland
      • Gdansk, Poland, 80-952
        • Recruiting
        • Uniwersyteckie Centrum Kliniczne
        • Contact:
          • Maria Bieniaszewska, Site 436
          • Phone Number: +48585844611
      • Krakow, Poland, 31-501
        • Recruiting
        • Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki w Krakowie
        • Contact:
          • Tomasz Sacha, Site 432
          • Phone Number: +48 602516924
      • Lodz, Poland, 93-510
        • Recruiting
        • Wojewodzki Szpital Specjalistyczny im. M. Kopernika w Lodzi
        • Contact:
          • Joanna Gora-Tybor, Site 431
      • Poznan, Poland, 61-696
        • Recruiting
        • ALVAMED
        • Contact:
          • Tomasz Wozny, Site 433
          • Phone Number: +48618464550
      • Walbrzych, Poland, 58-309
        • Recruiting
        • Specjalistyczny Szpital im. dra Alfreda Sokolowskiego
        • Contact:
          • Aleksandra Butrym, Site 434
          • Phone Number: +48502657840
      • Wroclaw, Poland, 50367
        • Recruiting
        • Samodzielny Publiczny Szpital Kliniczny Nr 1 we Wroclawiu
        • Contact:
          • Tomasz Wrobel, Site 435
          • Phone Number: +48 71 784 25 76
  • Romania
      • Brasov, Romania, 500052
        • Recruiting
        • Local Institution - 393
        • Contact:
          • Site 393
      • Bucharest, Romania, 022328
        • Recruiting
        • Fundeni Clinical Institute
        • Contact:
          • IULIA URSULEAC, Site 391
          • Phone Number: +40724710323
      • Cluj-Napoca, Romania, 400015
        • Recruiting
        • Prof. Dr. I. Chiricuta Institute of Oncology
        • Contact:
          • Ciprian Tomuleasa, Site 394
          • Phone Number: 0741337480
    • Dolj
      • Craiova, Dolj, Romania, 200143
        • Recruiting
        • Spitalul Clinic Municipal Filantropia Craiova
        • Contact:
          • Luminita Ocroteala, Site 395
          • Phone Number: 0040735439439
  • Russian Federation
      • Moscow, Russian Federation, 125284
        • Completed
        • Local Institution - 500
      • St Petersburg, Russian Federation, 197341
        • Completed
        • Local Institution - 502
      • St. Petersburg, Russian Federation, 197022
        • Completed
        • Local Institution - 503
  • Spain
      • Barcelona, Spain, 08036
        • Recruiting
        • Hospital Clinic de Barcelona
        • Contact:
          • Alberto Alvarez, Site 204
      • Barcelona, Spain, 08916
        • Recruiting
        • Hospital Universitari Germans Trias i Pujol ICO Badalona
        • Contact:
          • Blanca Xicoy, Site 207
          • Phone Number: +34934978987
      • Granada, Spain, 18014
        • Recruiting
        • Hospital Virgenes de las Nieves
        • Contact:
          • Francisca Hernandez Mohedo, Site 208
          • Phone Number: +958038301 0000 000
      • Las Palmas de Gran Canaria, Spain, 35012
        • Recruiting
        • Hospital Universitario de Gran Canaria Dr. Negrin
        • Contact:
          • Maria Gomez, Site 200
      • Madrid, Spain, 28034
        • Recruiting
        • Hospital Universitario Ramon y Cajal
        • Contact:
          • Jose Valentin Garcia-Gutierrez, Site 205
      • Madrid, Spain, 28041
        • Recruiting
        • Hospital Universitario 12 de Octubre
        • Contact:
          • Rosa Maria Ayala Diaz, Site 206
      • Palma de Mallorca, Spain, 7120
        • Recruiting
        • Hospital Son Espases
        • Contact:
          • Maria Antonia Duran Pastor, Site 202
      • Salamanca, Spain, 37007
        • Recruiting
        • Universitario de Salamanca - Hospital Clinico
        • Contact:
          • Jesus Hernandez Rivas, Site 209
      • Santiago de Compostela, Spain, 15706
        • Recruiting
        • Complejo Hospitalario Universitario de Santiago
        • Contact:
          • Manuel Perez Encinas, Site 201
      • Seville, Spain, 41013
        • Recruiting
        • Hospital Universitario Virgen del Rocio
        • Contact:
          • Maria Isabel Montero Cuadrado, Site 210
      • Valencia, Spain, 46010
        • Recruiting
        • Hospital Clínico Universitario de Valencia
        • Contact:
          • Juan-Carlos Hernandez Boluda, Site 203
          • Phone Number: +34963987832
  • United Kingdom
      • Birmingham, United Kingdom, B9 5SS
        • Recruiting
        • Heart of England NHS Foundation Trust
        • Contact:
          • Hayder Hussein, Site 366
      • Boston, United Kingdom, PE21 9QS
        • Recruiting
        • United Lincolnshire Hospitals NHS Trust
        • Contact:
          • Ciro Rinaldi, Site 365
          • Phone Number: +01205446311 0 00000
      • Oxford, United Kingdom, OX3 7LI
        • Recruiting
        • Churchhill Hospital
        • Contact:
          • Bethan Psaila, Site 362
    • Nottinghamshire
      • Nottingham, Nottinghamshire, United Kingdom, NG5 1PB
        • Recruiting
        • Nottingham City Hospital
        • Contact:
          • Frances Wadelin, Site 363
          • Phone Number: +441159691169
  • United States
    • California
      • Los Angeles, California, United States, 90095
        • Recruiting
        • University of California Los Angeles
        • Contact:
          • Gary Schiller, Site 110
          • Phone Number: 310-993-5779
    • Florida
      • Jacksonville, Florida, United States, 32207
        • Not yet recruiting
        • Local Institution - 127
        • Contact:
          • Site 127
      • Orlando, Florida, United States, 32804
        • Not yet recruiting
        • Local Institution - 135
        • Contact:
          • Site 135
      • Plantation, Florida, United States, 33322
        • Recruiting
        • BRCR Medical Center Inc.
        • Contact:
          • Jason Tache, Site 133
          • Phone Number: 516-447-0614
    • Illinois
      • Chicago, Illinois, United States, 60612
        • Completed
        • Local Institution - 112
    • Kentucky
      • Lexington, Kentucky, United States, 40536-0293
        • Recruiting
        • University of Kentucky Markey Cancer Center
        • Contact:
          • Fevzi Yalniz, Site 124
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Completed
        • Local Institution - 114
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Completed
        • Local Institution - 108
    • New Jersey
      • Hackensack, New Jersey, United States, 07601-2191
        • Recruiting
        • John Theurer Cancer Center
        • Contact:
          • James McCloskey, Site 104
          • Phone Number: 551-996-3925
    • New York
      • New York, New York, United States, 10029
        • Recruiting
        • Mount Sinai Medical Center
        • Contact:
          • Marina Kremyanskaya, Site 105
          • Phone Number: 212-241-4106
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15224
        • Recruiting
        • Allegheny Health Network
        • Contact:
          • Salman Fazal, Site 122
          • Phone Number: 412-578-4355
      • Pittsburgh, Pennsylvania, United States, 15213
        • Recruiting
        • University of Pittsburg Medical Center
        • Contact:
          • James Rossetti, Site 126
          • Phone Number: 412-578-4355
    • Tennessee
      • Knoxville, Tennessee, United States, 37920
        • Recruiting
        • University of Tennessee Medical Center
        • Contact:
          • Raleigh Cutrer, Site 130
    • Texas
      • Houston, Texas, United States, 77030
        • Recruiting
        • The University of Texas - MD Anderson Cancer Center
        • Contact:
          • Prithviraj Bose, Site 116
          • Phone Number: 713-792-2063
    • Utah
      • Salt Lake City, Utah, United States, 84112
        • Recruiting
        • University of Utah - Huntsman Cancer Institute
        • Contact:
          • Srinivas Tantravahi, Site 119

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Experimental Arm: Luspatercept (ACE-536)
Luspatercept will be given to participants via subcutaneous injection (administered on Day 1 of each 21-day treatment cycle)
Drug: ACE-536
Subcutaneous Injection
Other Names:
  • Luspatercept
  • BMS-986346
Placebo Comparator: Control Arm: Placebo
Placebo starting dose with volume equivalent to experimental arm subcutaneous injection every 3 weeks (administered on Day 1 of each 21-day treatment cycle)
Other: Placebo
Subcutaneous Injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Red blood cell-transfusion independence (RBC-TI) ≥ 12 weeks (RBC-TI 12)
Time Frame: Up to 24 weeks
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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
Red blood cell-transfusion independence ≥ 16 weeks (RBC-TI 16)
Time Frame: Up to 24 weeks
Proportion of subjects who become RBC-transfusion free over any consecutive 16-week period
Up to 24 weeks
Duration of Red blood cell-transfusion independence (RBC-TI 12)
Time Frame: Up to end of treatment, approximately 3 years
Maximum duration of RBC-TI response
Up to end of treatment, approximately 3 years
Reduction of transfusion burden by ≥ 50% and by ≥ 4 units/12 weeks from baseline over any consecutive 12-week period
Time Frame: Up to 24 weeks
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
Duration of reduction in transfusion burden
Time Frame: Up to end of treatment, approximately 3 years
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
Red blood cell-transfusion independence ≥ 12 weeks in the treatment period (RBC-TI 12/TP)
Time Frame: Up to end of treatment, approximately 3 years
Proportion of subjects who become RBC-transfusion free over any consecutive 12-week period
Up to end of treatment, approximately 3 years
Red blood cell-transfusion independence ≥ 16 weeks in the treatment period (RBC-TI 16/TP)
Time Frame: Up to end of treatment, approximately 3 years
Proportion of subjects who become RBC-transfusion free over any consecutive 16-week period
Up to end of treatment, approximately 3 years
Change in RBC transfusion burden
Time Frame: Up to 24 weeks
Mean change in transfusion burden (RBC units) from baseline
Up to 24 weeks
Cumulative duration of RBC-transfusion independence
Time Frame: Up to end of treatment, approximately 3 years
Cumulative response duration for subjects achieving multiple episodes of RBC-TI 12
Up to end of treatment, approximately 3 years
Mean Hgb increase ≥ 1 g/dL from baseline over any consecutive 12-week period in absence of RBC transfusions
Time Frame: Up to end of treatment, approximately 3 years
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
Change in serum ferritin from baseline
Time Frame: Up to end of treatment, approximately 3 years
Change in serum ferritin
Up to end of treatment, approximately 3 years
Incidence of Adverse Events (AEs)
Time Frame: From screening up to 42 days post last dose
Number of participants with adverse events
From screening up to 42 days post last dose
Transformation to blast phase: Number of subjects who transform into AML
Time Frame: Up to approximately 5 years
AML = acute myeloid leukemia
Up to approximately 5 years
Frequency of Antidrug antibodies (ADA)
Time Frame: From randomization and up to including 48 weeks post first dose
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
Pharmacokinetics - Area Under the Concentration-Time Curve (AUC)
Time Frame: From randomization and up to including 48 weeks post first dose
Measures of luspatercept exposure area under the curve
From randomization and up to including 48 weeks post first dose
Pharmacokinetics - Maximum plasma concentration of drug (Cmax)
Time Frame: From randomization and up to including 48 weeks post first dose
Maximum plasma concentration of drug
From randomization and up to including 48 weeks post first dose

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Celgene

Investigators

  • Study Director: Bristol-Myers Squibb, Bristol-Myers Squibb

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 25, 2021

Primary Completion (Estimated)

March 27, 2025

Study Completion (Estimated)

August 23, 2025

Study Registration Dates

First Submitted

December 29, 2020

First Submitted That Met QC Criteria

January 15, 2021

First Posted (Actual)

January 22, 2021

Study Record Updates

Last Update Posted (Actual)

April 12, 2024

Last Update Submitted That Met QC Criteria

April 10, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ACE-536-MF-002
  • 2020-000607-36 (EudraCT Number)
  • U1111-1260-9595 (Registry Identifier: WHO)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Information relating to our policy on data sharing and the process for requesting data can be found at the following link:

https://www.celgene.com/research-development/clinical-trials/clinical-trials-data-sharing/

IPD Sharing Time Frame

See Plan Description

IPD Sharing Access Criteria

See Plan Description

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • ICF
  • ANALYTIC_CODE
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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