A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials
NCT ID: NCT04064060
Last Updated: 2025-11-12
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE3
665 participants
INTERVENTIONAL
2019-08-12
2028-05-12
Brief Summary
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* Participants receiving luspatercept on a parent protocol at the time of their transition to the rollover study, who tolerate the protocol-prescribed regimen in the parent trial and, in the opinion of the investigator, may derive clinical benefit from continuing treatment with luspatercept
* Participants in the follow-up phase previously treated with luspatercept or placebo in the parent protocol will continue into long-term post-treatment follow-up in the rollover study until the follow-up commitments are met
* The study design is divided into the Transition Phase, Treatment Phase and Follow-up Phase. Participants will enter transition phase and depending on their background will enter either the treatment phase or the Long-term Post-treatment Follow-up (LTPTFU) phase
* Transition Phase is defined as one Enrollment visit
* Treatment Phase: For participants in luspatercept treatment the dose and schedule of luspatercept in this study will be the same as the last dose and schedule in the parent luspatercept study. This does not apply to participants that are in long-term follow-up from the parent protocol
* Follow-up Phase includes:
\- 42 Day Safety Follow-up Visit
* During the Safety Follow up, the participants will be followed for 42 days after the last dose of luspatercept, for the assessment of safety-related parameters and adverse event (AE) reporting
\- Long-term Post-treatment Follow-up (LTPTFU) Phase
* Participants will be followed for overall survival every 6 months for at least 5 years from first dose of luspatercept in the parent protocol, or 3 years of post-treatment from last dose, whichever occurs later, or until death, withdrawal of consent, study termination, or until a subject is lost to follow-up. Participants will also be monitored for progression to AML or any malignancies/pre-malignancies. New anticancer or disease related therapies should be collected at the same time schedule
Participants transitioning from a parent luspatercept study in post-treatment follow-up (safety or LTPTFU) will continue from the same equivalent point in this rollover study.
The ACE-536-LTFU-001 rollover study will be terminated, and relevant participants will discontinue from the study when all participants fulfill 5 years on the study, including treatment and follow-up.
Detailed Description
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Conditions
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Keywords
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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ACE-536
Luspatercept will be administered as a subcutaneous (SC) injection to participants by the study staff at the clinical site and administration will be documented in the subject's source record.
Luspatercept
Luspatercept (ACE-536), an erythroid maturation agent, is a recombinant fusion protein consisting of a modified form of the extracellular domain (ECD) of the human activin receptor type IIB (ActRIIB) linked to the human immunoglobin G 1 (IgG1) Fc domain. ActRIIB receptor and its ligands are members of the transforming growth factor-β (TGF-β) superfamily. Members of the TGF-β superfamily ligands, through their binding to activin receptors, are involved in modulating the differentiation of late-stage erythrocyte precursors (normoblasts) in the bone marrow. Luspatercept for injection is formulated as a sterile, preservative-free, lyophilized cake/powder. Luspatercept for injection is available in 25 mg and 75 mg vials and when reconstituted with water for injection, each consists of 50 mg/mL luspatercept in a 10 mM citrate buffer-based solution
Interventions
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Luspatercept
Luspatercept (ACE-536), an erythroid maturation agent, is a recombinant fusion protein consisting of a modified form of the extracellular domain (ECD) of the human activin receptor type IIB (ActRIIB) linked to the human immunoglobin G 1 (IgG1) Fc domain. ActRIIB receptor and its ligands are members of the transforming growth factor-β (TGF-β) superfamily. Members of the TGF-β superfamily ligands, through their binding to activin receptors, are involved in modulating the differentiation of late-stage erythrocyte precursors (normoblasts) in the bone marrow. Luspatercept for injection is formulated as a sterile, preservative-free, lyophilized cake/powder. Luspatercept for injection is available in 25 mg and 75 mg vials and when reconstituted with water for injection, each consists of 50 mg/mL luspatercept in a 10 mM citrate buffer-based solution
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
1. Participant is ≥ 18 years at the time of signing the informed consent form (ICF).
2. Participant is willing and able to adhere to the study visit schedule and other protocol requirements.
3. Participant has been participating in a luspatercept trial and continues to fulfill all the requirements of the parent protocol and the participant has been either:
1. Assigned to luspatercept treatment, continues to receive clinical benefit in the opinion of the investigator and should continue to receive luspatercept treatment, OR
2. Assigned to placebo arm in the parent protocol (at the time of unblinding or in follow-up) and should cross over to luspatercept treatment, OR
3. Assigned to the Follow-up Phase of the parent protocol, previously treated with luspatercept or placebo in the parent protocol who shall continue into LTPTFU phase in the rollover study until the follow-up commitments are met (unless requirements are met as per parent protocol to crossover to luspatercept treatment).
4. Participant understands and voluntarily signs an informed consent document prior to any study-related assessments or procedures being conducted.
5. Participant demonstrates compliance, as assessed by the investigator, with the parent study protocol requirements.
6. Applies to on treatment Participants only- females of childbearing potential (FCBP) defined as a sexually mature woman 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 or amenorrhea due to other medical reasons does not rule out childbearing potential) for at least 24 consecutive months (ie, has had menses at any time in the preceding 24 consecutive months) must:
1. Have two negative pregnancy tests as verified by the investigator prior to starting study therapy. She must agree to ongoing pregnancy testing during the course of the study, and after end of study therapy. This applies even if the participant practices true abstinence from heterosexual contact.
2. Agrees to use, and be able to comply with highly effective, contraception without interruption, 35 days prior to starting investigational product (IP), during the study therapy (including dose interruptions), and for 84 days after discontinuation of study therapy.
7\. Applies to on treatment participants only- Male participants must:
a. Agrees 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 84 days following investigational product discontinuation even if he has undergone a successful vasectomy.
Exclusion Criteria
1. Applies to on treatment participants only- Concomitant use of any medications/procedures that are prohibited in the parent luspatercept protocol.
2. Participant has met one or more criteria for study discontinuation as stipulated in the parent luspatercept protocol.
3. Applies to on treatment participants only- More than 26 days between last luspatercept dose in the parent protocol and first dose into ACE-536-LTFU-001 protocol unless dose delay or dose discontinuation criteria met.
4. Applies to on treatment participants only- Pregnant or breastfeeding females.
5. Participant has 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.
6. Participant has any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study.
7. Participant has any condition that confounds the ability to interpret data from the study.
18 Years
ALL
No
Sponsors
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Celgene
INDUSTRY
Responsible Party
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Principal Investigators
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Bristol-Myers Squibb
Role: STUDY_DIRECTOR
Bristol-Myers Squibb
Locations
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Childrens Hospital Los Angeles RHU
Los Angeles, California, United States
Local Institution - 971
Oakland, California, United States
Local Institution - 978
Stanford, California, United States
Local Institution - 975
Tampa, Florida, United States
Local Institution - 970
Chicago, Illinois, United States
Local Institution - 973
Boston, Massachusetts, United States
Local Institution - 961
Detroit, Michigan, United States
Local Institution - 969
New York, New York, United States
Local Institution - 967
Cleveland, Ohio, United States
Local Institution - 972
Philadelphia, Pennsylvania, United States
Vanderbilt - Ingram Cancer Center
Nashville, Tennessee, United States
The University of Texas - MD Anderson Cancer Center
Houston, Texas, United States
Local Institution - 100
South Brisbane, Queensland, Australia
Royal Adelaide Hospital
Adelaide, South Australia, Australia
Local Institution - 102
Clayton, Victoria, Australia
Royal Prince Alfred Hospital
Camperdown, , Australia
Local Institution - 182
Brasschaat, , Belgium
Local Institution - 180
Bruges, , Belgium
Local Institution - 183
Ghent, , Belgium
Local Institution - 184
Leuven, , Belgium
Local Institution - 220
Boulevard, Sofia, Bulgaria
Local Institution - 221
Plovdiv, , Bulgaria
Local Institution - 262
Toronto, Ontario, Canada
Local Institution - 260
Toronto, Ontario, Canada
Local Institution - 263
Toronto, Ontario, Canada
Local Institution - 131
Beijing, Beijing Municipality, China
Local Institution - 135
Guangzhou, Guangdong, China
Local Institution - 132
Shanghai, Shanghai Municipality, China
Local Institution - 134
Chengdu, Sichuan, China
Local Institution - 130
Tianjin, Tianjin Municipality, China
Local Institution - 133
Hangzhou, Zhejiang, China
Local Institution - 305
Angers, , France
Local Institution - 300
Créteil, , France
Local Institution - 310
La Tronche, , France
Local Institution - 306
Lille, , France
Local Institution - 301
Marseille, , France
Local Institution - 302
Paris, , France
Local Institution - 307
Pessac, , France
Local Institution - 304
Pierre-Bénite, , France
Local Institution - 308
Strasbourg, , France
Local Institution - 309
Toulouse, , France
Local Institution - 303
Tours, , France
Local Institution - 341
Berlin, , Germany
Local Institution - 348
Dresden, , Germany
Local Institution - 345
Düsseldorf, , Germany
Local Institution - 346
Düsseldorf, , Germany
Local Institution - 343
Halle, , Germany
Local Institution - 342
Hamburg, , Germany
Local Institution - 344
Hanover, , Germany
Local Institution - 349
Leipzig, , Germany
Local Institution - 340
Mainz, , Germany
Local Institution - 347
München, , Germany
Aghia Sophia' Children's General Hospital of Athens
Athens, , Greece
Laiko General Hospital of Athens - Center of Thalassemia
Athens, , Greece
Local Institution - 384
Athens, , Greece
Local Institution - 383
Rio Patras, , Greece
Local Institution - 381
Thessaloniki, , Greece
Local Institution - 425
Afula, , Israel
Local Institution - 420
Haifa, , Israel
Local Institution - 422
Jerusalem, , Israel
Local Institution - 424
Jerusalem, , Israel
Local Institution - 421
Nahariya, , Israel
Local Institution - 423
Petah Tikva, , Israel
Local Institution - 478
Florence, Tuscany, Italy
Local Institution - 471
Florence, Tuscany, Italy
Local Institution - 470
Allessandria, , Italy
Local Institution - 464
Bologna, , Italy
Local Institution - 466
Brindisi, , Italy
Local Institution - 477
Cagliari, , Italy
Local Institution - 462
Ferrara, , Italy
Ente Ospedaliero Ospedali Galliera - Centro della Microcitemia e delle Anemie Congenite
Genoa, , Italy
Local Institution - 473
Lecce, , Italy
Maggiore Polyclinic Hospital, IRCCS Ca' Granda
Milan, , Italy
Local Institution - 479
Modena, , Italy
AORN A Cardarelli
Napoli, , Italy
AOU dell'Universita degli Studi della Campania Luigi Vanvitelli
Napoli, , Italy
Azienda Ospedaliero Universitaria S. Luigi Gonzaga
Orbassano, , Italy
Local Institution - 469
Pavia, , Italy
Local Institution - 468
Reggio Calabria, , Italy
Local Institution - 465
Roma, , Italy
Local Institution - 474
Rozzano, , Italy
Local Institution - 472
Varese, , Italy
Local Institution - 463
Verona, , Italy
Local Institution - 610
Nagoya, Aichi-ken, Japan
Local Institution - 601
Kamogawa, Chiba, Japan
Matsuyama Red Cross Hospital
Matsuyama, Ehime, Japan
Ogaki Municipal Hospital
Ōgaki, Gifu, Japan
Japanese Red Cross Society Himeji Hospital
Himeji, Hyōgo, Japan
Local Institution - 605
Hitachi, Ibaraki, Japan
Kitasato University Hospital
Sagamihara, Kanagawa, Japan
Local Institution - 0979
Sendai, Miyagi, Japan
Tohoku University Hospital
Sendai, Miyagi, Japan
Local Institution - 611
Nagasaki, Nagasaki, Japan
Japanese Red Cross Medical Center
Shibuya City, Tokyo, Japan
NTT Medical Center Tokyo
Shinagawa City, Tokyo, Japan
Local Institution - 612
Chiba, , Japan
Shonan Kamakura General Hospital
Kamakura, , Japan
Osaka Metropolitan University Hospital
Osaka, , Japan
Local Institution - 604
Osaka, , Japan
Chronic Care Center
Hazmiyeh, , Lebanon
Local Institution - 545
Johor Bahru, Johor, Malaysia
Hospital Sultanah Bahiyah
Alor Star, Kedah, Malaysia
University Malaya Medical Centre
Kuala Lumpur, Kuala Lumpur, Malaysia
Hospital Raja Permaisuri Bainun
Ipoh, Perak, Malaysia
Queen Elizabeth Hospital
Kota Kinabalu, Sabah, Malaysia
Hospital Umum Sarawak
Kuching, Sarawak, Malaysia
Local Institution - 580
Amsterdam, , Netherlands
Local Institution - 681
Barakaldo, , Spain
Local Institution - 686
Barcelona, , Spain
Local Institution - 685
Barcelona, , Spain
Local Institution - 687
Madrid, , Spain
Local Institution - 682
Oviedo, , Spain
Local Institution - 684
Salamanca, , Spain
Local Institution - 680
Seville, , Spain
Local Institution - 683
Valencia, , Spain
Local Institution - 720
Gothenburg, , Sweden
Local Institution - 722
Lund, , Sweden
Local Institution - 721
Stockholm, , Sweden
Local Institution - 760
Kaohsiung, San Ming Dist., , Taiwan
China Medical University Hospital
Taichung, , Taiwan
Local Institution - 761
Taipei, , Taiwan
Chulalongkorn University Faculty of Medicine - King Chulalongkorn Memorial Hospital
Bangkok, , Thailand
Siriraj Hospital Mahidol University
Bangkok, , Thailand
Chiang Mai University - Maharaj Nakorn Chiang Mai Hospital
Chiang Mai, , Thailand
University Hospital Farhat Hached
Sousse, , Tunisia
Bone Marrow Transplant Center
Tunis, , Tunisia
Aziza Othmana Hospital
Tunis, , Tunisia
Military Hospital of Tunis
Tunis, , Tunisia
Local Institution - 881
Adana, , Turkey (Türkiye)
Local Institution - 885
Ankara, , Turkey (Türkiye)
Local Institution - 882
Istanbul, , Turkey (Türkiye)
Local Institution - 884
Istanbul, , Turkey (Türkiye)
Local Institution - 880
Izmir, , Turkey (Türkiye)
Local Institution - 883
Mersin, , Turkey (Türkiye)
Local Institution - 925
Aberdeen, , United Kingdom
Local Institution - 921
Leeds, , United Kingdom
Local Institution - 923
London, , United Kingdom
Whittington Hospital
London, , United Kingdom
University College London Hospitals NHS Foundation Trust - University College Hospital
London, , United Kingdom
Local Institution - 928
London, , United Kingdom
Local Institution - 924
London, , United Kingdom
Local Institution - 929
Oxford, , United Kingdom
Local Institution - 926
Sutton in Ashfield, , United Kingdom
Countries
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Central Contacts
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BMS Study Connect Contact Center www.BMSStudyConnect.com
Role: CONTACT
Phone: 855-907-3286
Email: [email protected]
Facility Contacts
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Thomas Coates, Site 968
Role: primary
Devendra Hiwase, Site 103
Role: primary
Joy Ho, Site 101
Role: primary
Antonis Kattamis, Site 382
Role: primary
Maria Dimopoulou, Site 380
Role: primary
Manuela Balocco, Site 476
Role: primary
Maria Domenica Cappellini, Site 467
Role: primary
Paolo Ricchi, Site 475
Role: primary
Silverio Perrotta, Site 461
Role: primary
Giovanni Battista Ferrero, Site 460
Role: primary
Tomoaki Fujisaki, Site 606
Role: primary
Hiroshi Kosugi, Site 608
Role: primary
Yasushi Hiramatsu, Site 614
Role: primary
Takahiro Suzuki, Site 603
Role: primary
Koichi Onodera, Site 607
Role: primary
Nobuhiro Tsukada, Site 602
Role: primary
Motoshi Ichikawa, Site 600
Role: primary
Shuku sato, Site 613
Role: primary
Yasuhiro Nakashima, Site 609
Role: primary
Ali Taher, Site 500
Role: primary
Hong Keng Liew, Site 542
Role: primary
Ping Chong Bee, Site 544
Role: primary
Kamini Kirubamoorthy, Site 546
Role: primary
Lily Wong, Site 543
Role: primary
Lee Ping Chew, Site 540
Role: primary
Ching-Tien Peng, Site 762
Role: primary
Pranee Sutcharitchan, Site 800
Role: primary
Vip Viprakasit, Site 802
Role: primary
Adisak Tantiworawit, Site 801
Role: primary
Khelif Abderahim, Site 840
Role: primary
Monia Ouederni, Site 841
Role: primary
Karima Kacem, Site 842
Role: primary
Fehmi M'Sadk, Site 843
Role: primary
Emma Drasar, Site 920
Role: primary
John Porter, Site 922
Role: primary
Related Links
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BMS Clinical Trial Information
Other Identifiers
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U1111-1235-8123
Identifier Type: REGISTRY
Identifier Source: secondary_id
2022-502498-40
Identifier Type: OTHER
Identifier Source: secondary_id
ACE-536-LTFU-001
Identifier Type: -
Identifier Source: org_study_id