A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

March 20, 2024 updated by: Celgene

A Phase 3b, Open-label, Single-arm, Rollover Study to Evaluate Long-term Safety in Subjects Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

A Phase 3b, open-label, single-arm, rollover study to evaluate the long-term safety of luspatercept, to the following participants:

  • 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 rollover study will be terminated, and relevant participants will discontinue from the study when all participants fulfill at least 5 years from the first dose of luspatercept in the parent protocol, or 3 years of post-treatment from last dose, whichever occurs later.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

665

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 Locations

  • Australia
      • Camperdown, Australia, 2050
        • Recruiting
        • Royal Prince Alfred Hospital
        • Contact:
          • Phoebe Joy Ho, Site 101
          • Phone Number: +61295158031 0 0000
    • Queensland
      • South Brisbane, Queensland, Australia, 4101
        • Completed
        • Local Institution - 100
    • South Australia
      • Adelaide, South Australia, Australia, 5000
        • Recruiting
        • Local Institution - 103
        • Contact:
          • Site 103
    • Victoria
      • Clayton, Victoria, Australia, 3168
        • Active, not recruiting
        • Local Institution - 102
  • Belgium
      • Brasschaat, Belgium, 2930
        • Completed
        • Local Institution - 182
      • Brugge, Belgium, 8000
        • Completed
        • Local Institution - 180
      • Ghent, Belgium, 9000
        • Completed
        • Local Institution - 183
      • Leuven, Belgium, 3000
        • Completed
        • Local Institution - 184
  • Bulgaria
      • Plovdiv, Bulgaria, 4002
        • Recruiting
        • University Multiprofile Hospital for Active Treatment Sveti Georgi EAD
        • Contact:
          • Pencho Georgiev, Site 221
          • Phone Number: +359888520139
    • Sofia
      • Boulevard, Sofia, Bulgaria, 1797
        • Recruiting
        • Local Institution - 220
        • Contact:
          • Site 220
  • Canada
    • Ontario
      • Toronto, Ontario, Canada, M4N 3M5
        • Completed
        • Local Institution - 262
      • Toronto, Ontario, Canada, M5G 2C4
        • Recruiting
        • University Health Network
        • Contact:
          • Kevin Kuo, Site 260
          • Phone Number: 4163405233
      • Toronto, Ontario, Canada, M5G 2M9
        • Completed
        • Local Institution - 263
  • China
    • Beijing
      • Beijing, Beijing, China, 100730
        • Not yet recruiting
        • Local Institution - 131
        • Contact:
          • Site 131
    • Guangdong
      • Guangzhou, Guangdong, China, 510515
        • Withdrawn
        • Local Institution - 135
    • Shanghai
      • Shanghai, Shanghai, China, 200233
        • Not yet recruiting
        • Local Institution - 132
        • Contact:
          • Site 132
    • Sichuan
      • Chengdu, Sichuan, China, 610041
        • Not yet recruiting
        • Local Institution - 134
        • Contact:
          • Site 134
    • Tianjin
      • Tianjin, Tianjin, China, 300020
        • Not yet recruiting
        • Local Institution - 130
        • Contact:
          • Site 130
    • Zhejiang
      • Hangzhou, Zhejiang, China, 310009
        • Not yet recruiting
        • Local Institution - 133
        • Contact:
          • Site 133
  • France
      • Angers, France, 49033
        • Completed
        • Local Institution - 305
      • Creteil, France, 94010
        • Recruiting
        • Local Institution - 300
        • Contact:
          • Site 300
      • La Tronche, France, 38700
        • Completed
        • Local Institution - 310
      • Lille, France, 59037
        • Recruiting
        • CHRU de Lille-Hopital Claude Huriez
        • Contact:
          • Laure Goursaud, Site 306
      • Marseille Cedex 9, France, 13385
        • Completed
        • Local Institution - 301
      • Paris, France, 75010
        • Active, not recruiting
        • Local Institution - 302
      • Pessac Cedex, France, 33604
        • Withdrawn
        • Local Institution - 307
      • Pierre Benite cedex, France, 69495
        • Completed
        • Local Institution - 304
      • Strasbourg, France, 67091
        • Completed
        • Local Institution - 308
      • Toulouse Cedex 9, France, 31059
        • Completed
        • Local Institution - 309
      • Tours, France, 37044
        • Withdrawn
        • Local Institution - 303
  • Germany
      • Berlin, Germany, 14195
        • Completed
        • Local Institution - 341
      • Dresden, Germany, 01307
        • Recruiting
        • Universitätsklinikum Carl Gustav Carus an der TU Dresden
        • Contact:
          • Katja Sockel, Site 348
          • Phone Number: 493514587666
      • Duesseldorf, Germany, 40225
        • Recruiting
        • Universitaetsklinikum Duesseldorf
        • Contact:
          • Ulrich Germing, Site 345
          • Phone Number: +492118117780
      • Dusseldorf, Germany, 40479
        • Completed
        • Local Institution - 346
      • Halle, Germany, 06120
        • Active, not recruiting
        • Local Institution - 343
      • Hamburg, Germany, 22081
        • Recruiting
        • Local Institution - 342
        • Contact:
          • Site 342
      • Hannover, Germany, 30625
        • Completed
        • Local Institution - 344
      • Leipzig, Germany, 04103
        • Withdrawn
        • Local Institution - 349
      • Mainz, Germany, 55131
        • Recruiting
        • Local Institution - 340
        • Contact:
          • Site 340
      • München, Germany, 81675
        • Recruiting
        • Klinikum Rechts der Isar der Technischen Universitaet Muenchen
        • Contact:
          • Katharina Goetze, Site 347
          • Phone Number: +498941405618 00 00
  • Greece
      • Athens, Greece, 115 27
        • Recruiting
        • Aghia Sophia' Children's General Hospital of Athens
        • Contact:
          • Antonis Kattamis, Site 382
          • Phone Number: +302107467772 0(000)
      • Athens, Greece, 115 27
        • Recruiting
        • Laiko General Hospital of Athens - Center of Thalassemia
        • Contact:
          • Maria Dimopoulou, Site 380
      • Athens, Greece, 11527
        • Recruiting
        • General Hospital Georgios Gennimatas of Athens
        • Contact:
          • Foteini Petropoulou, Site 384
          • Phone Number: +306973006723
      • Rio Patras, Greece, 26500
        • Completed
        • Local Institution - 383
      • Thessaloniki, Greece, 54642
        • Recruiting
        • General Hospital of Thessaloniki Hippokration
        • Contact:
          • Efthymia Vlachaki, Site 381
          • Phone Number: 302310892003
  • Israel
      • Afula, Israel, 18341
        • Completed
        • Local Institution - 425
      • Haifa, Israel, 3109601
        • Recruiting
        • Local Institution - 420
        • Contact:
          • Site 420
      • Jerusalem, Israel, 91031
        • Completed
        • Local Institution - 422
      • Jerusalem, Israel, 91120
        • Completed
        • Local Institution - 424
      • Nahariya, Israel, 22100
        • Recruiting
        • Galilee Medical Center
        • Contact:
          • Celia Suriu, Site 421
      • Petah Tikva, Israel, 49100
        • Completed
        • Local Institution - 423
  • Italy
      • Allessandria, Italy, 15100
        • Completed
        • Local Institution - 470
      • Bologna, Italy, 40138
        • Active, not recruiting
        • Local Institution - 464
      • Brindisi, Italy, 72100
        • Active, not recruiting
        • Local Institution - 466
      • Cagliari, Italy, 09121
        • Recruiting
        • Azienda Sanitaria Locale (ASL) Cagliari - Ospedale Regionale per le Microcitemie
        • Contact:
          • Raffaella Origa, Site 477
          • Phone Number: +390706095655 00000
      • Ferrara, Italy, 44124
        • Recruiting
        • Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant'Anna
        • Contact:
          • Maria Rita Gamberini, Site 462
          • Phone Number: +390532237349
      • Genoa, Italy, 16128
        • Recruiting
        • Ente Ospedaliero Ospedali Galliera - Centro della Microcitemia e delle Anemie Congenite
        • Contact:
          • Manuela Balocco, Site 476
          • Phone Number: +390105634557
      • Lecce, Italy, 73100
        • Completed
        • Local Institution - 473
      • Milano, Italy, 20122
        • Recruiting
        • Maggiore Polyclinic Hospital, IRCCS Ca' Granda
        • Contact:
          • Maria Domenica Cappellini, Site 467
          • Phone Number: +390250320288
      • Modena, Italy, 41124
        • Completed
        • Local Institution - 479
      • Napoli, Italy, 80131
        • Recruiting
        • AORN A Cardarelli
        • Contact:
          • Paolo Ricchi, Site 475
      • Napoli, Italy, 80131
        • Recruiting
        • AOU dell'Universita degli Studi della Campania Luigi Vanvitelli
        • Contact:
          • Silverio Perrotta, Site 461
          • Phone Number: +0390815665421 0 000
      • Orbassano, Italy, 10043
        • Recruiting
        • Azienda Ospedaliero Universitaria S. Luigi Gonzaga
        • Contact:
          • Giovanni Battista Ferrero, Site 460
      • Pavia, Italy, 27100
        • Recruiting
        • IRCCS Policlinico San Matteo
        • Contact:
          • Luca Malcovati, Site 469
          • Phone Number: +39 0382 503084
      • Reggio Di Calabria, Italy, 89124
        • Recruiting
        • Azienda Ospedaliera Bianchi Melacrino Morelli
        • Contact:
          • Esther Oliva, Site 468
          • Phone Number: +39 0965 393903
      • Roma, Italy, 133
        • Completed
        • Local Institution - 465
      • Rozzano, Italy, 20089
        • Withdrawn
        • Local Institution - 474
      • Varese, Italy, 21100
        • Recruiting
        • Ospedale di Circolo di Varese
        • Contact:
          • Marco Brociner, Site 472
      • Verona, Italy, 37134
        • Recruiting
        • Local Institution - 463
        • Contact:
          • Site 463
    • Toscana
      • Firenze, Toscana, Italy, 50134
        • Recruiting
        • Azienda Ospedaliera Universitaria Careggi
        • Contact:
          • Valeria Santini, Site 478
          • Phone Number: +390557947296 000 0
      • Firenze, Toscana, Italy, 50139
        • Active, not recruiting
        • Local Institution - 471
  • Japan
      • Chiba, Japan, 260-0852
        • Completed
        • Local Institution - 612
      • Kamakura, Japan, 247-8533
        • Recruiting
        • Local Institution - 613
        • Contact:
          • Site 613
      • Osaka, Japan, 545-8586
        • Recruiting
        • Local Institution - 609
        • Contact:
          • Site 609
      • Osaka, Japan, 589-8511
        • Recruiting
        • Local Institution - 604
        • Contact:
          • Site 604
    • Aichi
      • Nagoya, Aichi, Japan, 460-0001
        • Recruiting
        • Local Institution - 610
        • Contact:
          • Site 610
    • Chiba
      • Kamogawa, Chiba, Japan, 296-0041
        • Completed
        • Local Institution - 601
    • Ehime
      • Matsuyama, Ehime, Japan, 790-8524
        • Recruiting
        • Local Institution - 606
        • Contact:
          • Site 606
    • Gifu
      • Ogaki, Gifu, Japan, 503-8502
        • Recruiting
        • Ogaki Municipal Hospital
        • Contact:
          • Hiroshi Kosugi, Site 608
          • Phone Number: 81584813341
    • Hyogo
      • Himeji, Hyogo, Japan, 670-8540
        • Recruiting
        • Local Institution - 614
        • Contact:
          • Site 614
    • Ibaraki
      • Hitachi, Ibaraki, Japan, 317-0077
        • Recruiting
        • Hitachi General Hospital
        • Contact:
          • Atsushi Shinagawa, Site 605
          • Phone Number: +01294231111
    • Kanagawa
      • Sagamihara, Kanagawa, Japan, 252-0329
        • Recruiting
        • Local Institution - 603
        • Contact:
          • Site 603
    • Miyagi
      • Sendai, Miyagi, Japan, 980-8574
        • Recruiting
        • Tohoku University Hospital
        • Contact:
          • Koichi Onodera, Site 607
      • Sendai, Miyagi, Japan, 980-8574
        • Withdrawn
        • Local Institution - 0979
    • Nagasaki
      • Nagasaki-shi, Nagasaki, Japan, 8528511
        • Withdrawn
        • Local Institution - 611
    • Tokyo
      • Shibuya City, Tokyo, Japan, 150-8935
        • Recruiting
        • Local Institution - 602
        • Contact:
          • Site 602
      • Shinagawa City, Tokyo, Japan, 141-8625
        • Recruiting
        • Local Institution - 600
        • Contact:
          • Site 600
  • Lebanon
      • Hazmieh, Lebanon, 00961
        • Recruiting
        • Chronic Care Center
        • Contact:
          • Ali Taher, Site 500
          • Phone Number: +9613755669 000000.0
  • Malaysia
    • Johor
      • Johor Bahru, Johor, Malaysia, 80100
        • Recruiting
        • Hospital Sultanah Aminah
        • Contact:
          • Soo Min Lim, Site 545
          • Phone Number: +6072223037
    • Kedah
      • Alor Setar, Kedah, Malaysia, 05460
        • Recruiting
        • Hospital Sultanah Bahiyah
        • Contact:
          • Hong Keng Liew, Site 542
          • Phone Number: 6047406191
    • Perak
      • Ipoh, Perak, Malaysia, 30990
        • Recruiting
        • Local Institution - 546
        • Contact:
          • Site 546
    • Sabah
      • Kota Kinabalu, Sabah, Malaysia, 88586
        • Recruiting
        • Queen Elizabeth Hospital
        • Contact:
          • Lily Wong, Site 543
          • Phone Number: 6088517507
    • Sarawak
      • Kuching, Sarawak, Malaysia, 93586
        • Recruiting
        • Hospital Umum Sarawak
        • Contact:
          • Lee Ping Chew, Site 540
          • Phone Number: +60165518129
    • Wilayah Persekutuan Kuala Lumpur
      • Kuala Lumpur, Wilayah Persekutuan Kuala Lumpur, Malaysia, 59100
        • Recruiting
        • University Malaya Medical Centre
        • Contact:
          • Ping Chong Bee, Site 544
          • Phone Number: +60123048771
  • Netherlands
      • Amsterdam, Netherlands, 1081 HV
        • Recruiting
        • Vrije Universiteit Medisch Centrum (Vumc)
        • Contact:
          • Arjan van de Loosdrecht, Site 580
          • Phone Number: +31204442604
  • Spain
      • Barakaldo, Spain, 48903
        • Completed
        • Local Institution - 681
      • Barcelona, Spain, 08035
        • Completed
        • Local Institution - 685
      • Barcelona, Spain, 08908
        • Completed
        • Local Institution - 686
      • Madrid, Spain, 28007
        • Completed
        • Local Institution - 687
      • Oviedo, Spain, 33011
        • Completed
        • Local Institution - 682
      • Salamanca, Spain, 37007
        • Active, not recruiting
        • Local Institution - 684
      • Seville, Spain, 41013
        • Completed
        • Local Institution - 680
      • Valencia, Spain, 46026
        • Completed
        • Local Institution - 683
  • Sweden
      • Goteborg, Sweden, 413 45
        • Active, not recruiting
        • Local Institution - 720
      • Lund, Sweden, SE-221 85
        • Completed
        • Local Institution - 722
      • Stockholm, Sweden, 141 86
        • Completed
        • Local Institution - 721
  • Taiwan
      • Kaohsiung, San Ming Dist., Taiwan, 807
        • Recruiting
        • Local Institution - 760
        • Contact:
          • Site 760
      • Taichung, Taiwan, 40447
        • Recruiting
        • China Medical University Hospital
        • Contact:
          • Ching-Tien Peng, Site 762
          • Phone Number: 886422052121ext4636
      • Taipei, Zhongzheng Dist., Taiwan, 10002
        • Recruiting
        • National Taiwan University Hospital
        • Contact:
          • Meng-Yao Lu, Site 761
          • Phone Number: +886223123456
  • Thailand
      • Bangkok, Thailand, 10330
        • Recruiting
        • Chulalongkorn University Faculty of Medicine - King Chulalongkorn Memorial Hospital
        • Contact:
          • Pranee Sutcharitchan, Site 800
          • Phone Number: +66022564564
      • Bangkok, Thailand, 10700
        • Recruiting
        • Siriraj Hospital Mahidol University
        • Contact:
          • Vip Viprakasit, Site 802
          • Phone Number: 6624122113
      • Chiang Mai, Thailand, 50200
        • Recruiting
        • Chiang Mai University - Maharaj Nakorn Chiang Mai Hospital
        • Contact:
          • Adisak Tantiworawit, Site 801
          • Phone Number: +66815688875
  • Tunisia
      • Sousse, Tunisia, 4031
        • Recruiting
        • Local Institution - 840
        • Contact:
          • Site 840
      • Tunis, Tunisia, 1006
        • Recruiting
        • Bone Marrow Transplant Center
        • Contact:
          • Monia Ouederni, Site 841
          • Phone Number: +21693184151 0000 00
      • Tunis, Tunisia, 1008
        • Recruiting
        • Local Institution - 842
        • Contact:
          • Site 842
      • Tunis, Tunisia, 1008
        • Recruiting
        • Local Institution - 843
        • Contact:
          • Site 843
  • Turkey
      • Adana, Turkey, 01130
        • Recruiting
        • Acibadem Adana Hospital
        • Contact:
          • Ali Bulent Antmen, Site 881
          • Phone Number: +903224554444
      • Ankara, Turkey, 06590
        • Completed
        • Local Institution - 885
      • Istanbul, Turkey, 34093
        • Completed
        • Local Institution - 882
      • Istanbul, Turkey, 34098
        • Completed
        • Local Institution - 884
      • Izmir, Turkey, 35100
        • Recruiting
        • Ege Universitesi Tip Fakultesi Hastanesi
        • Contact:
          • Yesim Aydinok, Site 880
          • Phone Number: +9023244413433711
      • Mersin, Turkey, 33343
        • Completed
        • Local Institution - 883
  • United Kingdom
      • Aberdeen, United Kingdom, AB25 2ZN
        • Completed
        • Local Institution - 925
      • Leeds, United Kingdom, LS9 7TF
        • Withdrawn
        • Local Institution - 921
      • London, United Kingdom, SE1 9RT
        • Recruiting
        • Guy's and St Thomas' NHS Foundation Trust - Guy's Hospital
        • Contact:
          • Claire Harrison, Site 928
          • Phone Number: +442071882742
      • London, United Kingdom, E1 1BB
        • Completed
        • Local Institution - 923
      • London, United Kingdom, NW1 2BU
        • Recruiting
        • Local Institution - 922
        • Contact:
          • Site 922
      • London, United Kingdom, SE5 9RS
        • Recruiting
        • Kings College Hospital
        • Contact:
          • Shreyans Gandhi, Site 924
      • London, United Kingdom, N19 5NF
        • Recruiting
        • Local Institution - 920
        • Contact:
          • Site 920
      • Oxford, United Kingdom, OX3 7LE
        • Withdrawn
        • Local Institution - 929
      • Sutton in Ashfield, United Kingdom, NG17 4JL
        • Withdrawn
        • Local Institution - 926
  • United States
    • California
      • Los Angeles, California, United States, 90027-6062
        • Recruiting
        • Childrens Hospital Los Angeles RHU
        • Contact:
          • Thomas Coates, Site 968
          • Phone Number: 323-361-2352
      • Oakland, California, United States, 94609
        • Completed
        • Local Institution - 971
      • Stanford, California, United States, 94305
        • Completed
        • Local Institution - 978
    • Florida
      • Tampa, Florida, United States, 33612
        • Completed
        • Local Institution - 975
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Recruiting
        • Ann & Robert H Lurie Children's Hospital of Chicago
        • Contact:
          • Sherif Badawy, Site 970
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Recruiting
        • Boston Children's Hospital
        • Contact:
          • Erica Esrick, Site 973
          • Phone Number: 617-355-2569
    • Michigan
      • Detroit, Michigan, United States, 48201
        • Completed
        • Local Institution - 961
    • New York
      • New York, New York, United States, 10065
        • Completed
        • Local Institution - 969
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Active, not recruiting
        • Local Institution - 967
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Completed
        • Local Institution - 972
    • Tennessee
      • Nashville, Tennessee, United States, 37232-5505
        • Withdrawn
        • Vanderbilt - Ingram Cancer Center
    • Texas
      • Houston, Texas, United States, 77030
        • Withdrawn
        • The University of Texas - MD Anderson Cancer Center

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

Inclusion Criteria:

Participants must meet all the following criteria to be enrolled in this study:

  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 Long-term Post-treatment Follow-up 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) and 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. 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 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. 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 84 days following investigational product discontinuation even if he has undergone a successful vasectomy.

Exclusion Criteria:

The presence of any of the following will exclude a participant from enrollment:

  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.

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: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 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.
Drug: 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 Names:
  • ACE-536

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Adverse Events (AEs)
Time Frame: From enrollment until at least 42 Day Safety Follow-up Phase or EOS (Approximately 5 years).
Type, frequency, severity of AEs, relationship of treatment emergent adverse events to luspatercept
From enrollment until at least 42 Day Safety Follow-up Phase or EOS (Approximately 5 years).
Number of participants progressing to high/very high risk MDS or AML.
Time Frame: Enrollment to Long-term post-treatment follow-up (Approximately, 5 years)
Progression to high/very high-risk myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) (MDS and myelofibrosis [MF] only).
Enrollment to Long-term post-treatment follow-up (Approximately, 5 years)
Percentage of participants progressing to high/very high risk MDS or AML
Time Frame: Enrollment to Long-term post-treatment follow-up (Approximately, 5 years)
Progression to high/very high-risk myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) (MDS and myelofibrosis [MF] only)
Enrollment to Long-term post-treatment follow-up (Approximately, 5 years)
Number of participants developing other malignancies/pre-malignancies
Time Frame: Enrollment to Long-term post-treatment follow-up (Approximately, 5 years)
Development of other malignancies/pre-malignancies
Enrollment to Long-term post-treatment follow-up (Approximately, 5 years)
Percentage of participants developing other malignancies/pre-malignancies
Time Frame: Enrollment to Long-term post-treatment follow-up (Approximately, 5 years)
Development of other malignancies/pre-malignancies
Enrollment to Long-term post-treatment follow-up (Approximately, 5 years)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Overall Survival
Time Frame: Enrollment to Long-term post-treatment follow-up (Approximately, 5 years)
Time from date of randomization until death from any cause
Enrollment to Long-term post-treatment follow-up (Approximately, 5 years)
Number of participants developing treatment emergent extramedullary hematopoiesis (EMH) masses
Time Frame: Enrollment to Long-term post-treatment follow-up (Approximately, 5 years)
Enrollment to Long-term post-treatment follow-up (Approximately, 5 years)
Percentage of participants developing treatment emergent EMH masses
Time Frame: Enrollment to Long-term post-treatment follow-up (Approximately, 5 years)
Enrollment to Long-term post-treatment follow-up (Approximately, 5 years)

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)

August 12, 2019

Primary Completion (Estimated)

May 12, 2028

Study Completion (Estimated)

May 12, 2028

Study Registration Dates

First Submitted

August 9, 2019

First Submitted That Met QC Criteria

August 19, 2019

First Posted (Actual)

August 21, 2019

Study Record Updates

Last Update Posted (Actual)

March 21, 2024

Last Update Submitted That Met QC Criteria

March 20, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ACE-536-LTFU-001
  • U1111-1235-8123 (Registry Identifier: WHO)
  • 2018-002915-93 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

BMS will provide access to individual anonymized participant data upon request from qualified researchers, and subject to certain criteria.

Additional information regarding Bristol Myer Squibb's data sharing policy and process can be found at: https://www.bms.com/researchers-and-partners/clinical-trials-and-research/disclosurecommitment.html

IPD Sharing Time Frame

See Plan Description

IPD Sharing Access Criteria

See Plan Description

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • 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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