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Blood disorder drug luspatercept gets Long-Term safety review

NCT ID NCT04064060

Disease control Sponsor: Celgene Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jul 02, 2026 · Updated 2 times

Summary

This study follows about 665 people with myelodysplastic syndromes, beta-thalassemia, or myelofibrosis who have already taken luspatercept in earlier trials. Researchers want to see how safe the drug is over many years by tracking side effects, disease progression, and survival. Participants either continue treatment or enter long-term follow-up.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
luspatercept (a drug that helps the body make more red blood cells)
What this could lead to
If successful, this study could confirm that luspatercept is safe to use long-term for managing blood disorders like MDS and beta-thalassemia.
What could go wrong
This is a rollover study, not a new treatment test. It mainly tracks safety, so it won't prove if luspatercept works better than other options. Side effects or disease progression may still occur.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 665 people

The number the study aims to enrol. It can still change while the study runs.

Started

Aug 2019

Expected to finish

May 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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 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: 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.

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Conditions

The condition(s) this trial relates to.

beta thalassemia myelodysplastic syndrome Myelodysplastic Syndromes myelofibrosis Myeloproliferative Disorders primary myelofibrosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Beta-thalassemia Myelodysplastic syndromes (MDS) Myeloproliferative neoplasm(MPN)-associated myelofibrosis

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    27 sites in 9 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • AORN A Cardarelli

    RECRUITING

    Naples, 80131, Italy

  • AOU dell'Universita degli Studi della Campania Luigi Vanvitelli

    RECRUITING

    Naples, 80131, Italy

  • Aghia Sophia' Children's General Hospital of Athens

    RECRUITING

    Athens, 115 27, Greece

  • Azienda Ospedaliero Universitaria S. Luigi Gonzaga

    RECRUITING

    Orbassano, 10043, Italy

  • Aziza Othmana Hospital

    RECRUITING

    Tunis, 1008, Tunisia

  • Bone Marrow Transplant Center

    RECRUITING

    Tunis, 1006, Tunisia

  • Chiang Mai University - Maharaj Nakorn Chiang Mai Hospital

    RECRUITING

    Chiang Mai, 50200, Thailand

  • Chronic Care Center

    RECRUITING

    Hazmiyeh, 00961, Lebanon

  • Chulalongkorn University Faculty of Medicine - King Chulalongkorn Memorial Hospital

    RECRUITING

    Bangkok, 10330, Thailand

  • Ente Ospedaliero Ospedali Galliera - Centro della Microcitemia e delle Anemie Congenite

    RECRUITING

    Genoa, 16128, Italy

  • Hospital Raja Permaisuri Bainun

    RECRUITING

    Ipoh, Perak, 30990, Malaysia

  • Hospital Sultanah Bahiyah

    RECRUITING

    Alor Star, Kedah, 05460, Malaysia

  • Hospital Umum Sarawak

    RECRUITING

    Kuching, Sarawak, 93586, Malaysia

  • Japanese Red Cross Society Himeji Hospital

    RECRUITING

    Himeji, Hyōgo, 6708540, Japan

  • Local Institution - 0979

    WITHDRAWN

    Sendai, Miyagi, 980-8574, Japan

  • Local Institution - 100

    COMPLETED

    South Brisbane, Queensland, 4101, Australia

  • Local Institution - 102

    COMPLETED

    Clayton, Victoria, 3168, Australia

  • Local Institution - 130

    WITHDRAWN

    Tianjin, Tianjin Municipality, 300020, China

  • Local Institution - 131

    WITHDRAWN

    Beijing, Beijing Municipality, 100730, China

  • Local Institution - 132

    WITHDRAWN

    Shanghai, Shanghai Municipality, 200233, China

  • Local Institution - 133

    WITHDRAWN

    Hangzhou, Zhejiang, 310009, China

  • Local Institution - 134

    WITHDRAWN

    Chengdu, Sichuan, 610041, China

  • Local Institution - 135

    WITHDRAWN

    Guangzhou, Guangdong, 510515, China

  • Local Institution - 180

    COMPLETED

    Bruges, 8000, Belgium

  • Local Institution - 182

    COMPLETED

    Brasschaat, 2930, Belgium

  • Local Institution - 183

    COMPLETED

    Ghent, 9000, Belgium

  • Local Institution - 184

    COMPLETED

    Leuven, 3000, Belgium

  • Local Institution - 220

    COMPLETED

    Boulevard, Sofia, 1797, Bulgaria

  • Local Institution - 221

    COMPLETED

    Plovdiv, 4002, Bulgaria

  • Local Institution - 260

    COMPLETED

    Toronto, Ontario, M5G 2C4, Canada

  • Local Institution - 262

    COMPLETED

    Toronto, Ontario, M4N 3M5, Canada

  • Local Institution - 263

    COMPLETED

    Toronto, Ontario, M5G 2M9, Canada

  • Local Institution - 300

    COMPLETED

    Créteil, 94010, France

  • Local Institution - 301

    COMPLETED

    Marseille, 13385, France

  • Local Institution - 302

    COMPLETED

    Paris, 75010, France

  • Local Institution - 303

    WITHDRAWN

    Tours, 37044, France

  • Local Institution - 304

    COMPLETED

    Pierre-Bénite, 69495, France

  • Local Institution - 305

    COMPLETED

    Angers, 49033, France

  • Local Institution - 306

    COMPLETED

    Lille, 59037, France

  • Local Institution - 307

    WITHDRAWN

    Pessac, 33604, France

  • Local Institution - 308

    COMPLETED

    Strasbourg, 67091, France

  • Local Institution - 309

    COMPLETED

    Toulouse, 31059, France

  • Local Institution - 310

    COMPLETED

    La Tronche, 38700, France

  • Local Institution - 340

    COMPLETED

    Mainz, 55131, Germany

  • Local Institution - 341

    COMPLETED

    Berlin, 14195, Germany

  • Local Institution - 342

    COMPLETED

    Hamburg, 22081, Germany

  • Local Institution - 343

    COMPLETED

    Halle, 06120, Germany

  • Local Institution - 344

    COMPLETED

    Hanover, 30625, Germany

  • Local Institution - 345

    COMPLETED

    Düsseldorf, 40225, Germany

  • Local Institution - 346

    COMPLETED

    Düsseldorf, 40479, Germany

  • Local Institution - 347

    COMPLETED

    München, 81675, Germany

  • Local Institution - 348

    COMPLETED

    Dresden, 01307, Germany

  • Local Institution - 349

    WITHDRAWN

    Leipzig, 04103, Germany

  • Local Institution - 380

    COMPLETED

    Athens, 115 27, Greece

  • Local Institution - 381

    COMPLETED

    Thessaloniki, 54642, Greece

  • Local Institution - 383

    COMPLETED

    Rio Patras, 26500, Greece

  • Local Institution - 384

    COMPLETED

    Athens, 11527, Greece

  • Local Institution - 420

    COMPLETED

    Haifa, 3109601, Israel

  • Local Institution - 421

    COMPLETED

    Nahariya, 22100, Israel

  • Local Institution - 422

    COMPLETED

    Jerusalem, 91031, Israel

  • Local Institution - 423

    COMPLETED

    Petah Tikva, 49100, Israel

  • Local Institution - 424

    COMPLETED

    Jerusalem, 91120, Israel

  • Local Institution - 425

    COMPLETED

    Afula, 1834111, Israel

  • Local Institution - 462

    COMPLETED

    Ferrara, 44124, Italy

  • Local Institution - 463

    COMPLETED

    Verona, 37134, Italy

  • Local Institution - 464

    COMPLETED

    Bologna, 40138, Italy

  • Local Institution - 465

    COMPLETED

    Roma, 133, Italy

  • Local Institution - 466

    COMPLETED

    Brindisi, 72100, Italy

  • Local Institution - 468

    COMPLETED

    Reggio Calabria, 89124, Italy

  • Local Institution - 469

    COMPLETED

    Pavia, 27100, Italy

  • Local Institution - 470

    COMPLETED

    Allessandria, 15100, Italy

  • Local Institution - 471

    ACTIVE_NOT_RECRUITING

    Florence, Tuscany, 50139, Italy

  • Local Institution - 472

    COMPLETED

    Varese, 21100, Italy

  • Local Institution - 473

    COMPLETED

    Lecce, 73100, Italy

  • Local Institution - 474

    WITHDRAWN

    Rozzano, 20089, Italy

  • Local Institution - 477

    COMPLETED

    Cagliari, 09121, Italy

  • Local Institution - 478

    COMPLETED

    Florence, Tuscany, 50134, Italy

  • Local Institution - 479

    COMPLETED

    Modena, 41124, Italy

  • Local Institution - 544

    COMPLETED

    Kuala Lumpur, Kuala Lumpur, 59100, Malaysia

  • Local Institution - 545

    COMPLETED

    Johor Bahru, Johor, 80100, Malaysia

  • Local Institution - 580

    COMPLETED

    Amsterdam, 1081 HV, Netherlands

  • Local Institution - 601

    COMPLETED

    Kamogawa, Chiba, 296-0041, Japan

  • Local Institution - 602

    COMPLETED

    Shibuya City, Tokyo, 150-8935, Japan

  • Local Institution - 603

    COMPLETED

    Sagamihara, Kanagawa, 252-0329, Japan

  • Local Institution - 604

    COMPLETED

    Sakai, Osaka, 5900197, Japan

  • Local Institution - 605

    COMPLETED

    Hitachi, Ibaraki, 317-0077, Japan

  • Local Institution - 607

    COMPLETED

    Sendai, Miyagi, 980-8574, Japan

  • Local Institution - 609

    COMPLETED

    Osaka, 545-8586, Japan

  • Local Institution - 610

    COMPLETED

    Nagoya, Aichi-ken, 460-0001, Japan

  • Local Institution - 611

    WITHDRAWN

    Nagasaki, Nagasaki, 8528511, Japan

  • Local Institution - 612

    COMPLETED

    Chiba, 260-0852, Japan

  • Local Institution - 680

    COMPLETED

    Seville, 41013, Spain

  • Local Institution - 681

    COMPLETED

    Barakaldo, 48903, Spain

  • Local Institution - 682

    COMPLETED

    Oviedo, 33011, Spain

  • Local Institution - 683

    COMPLETED

    Valencia, 46026, Spain

  • Local Institution - 684

    COMPLETED

    Salamanca, 37007, Spain

  • Local Institution - 685

    COMPLETED

    Barcelona, 8035, Spain

  • Local Institution - 686

    COMPLETED

    Barcelona, 08908, Spain

  • Local Institution - 687

    COMPLETED

    Madrid, 28028, Spain

  • Local Institution - 720

    COMPLETED

    Gothenburg, 413 45, Sweden

  • Local Institution - 721

    COMPLETED

    Stockholm, 141 86, Sweden

  • Local Institution - 722

    COMPLETED

    Lund, SE-221 85, Sweden

  • Local Institution - 760

    COMPLETED

    Kaohsiung, San Ming Dist., 807, Taiwan

  • Local Institution - 761

    COMPLETED

    Taipei, 100225, Taiwan

  • Local Institution - 762

    COMPLETED

    Taichung, 40447, Taiwan

  • Local Institution - 880

    COMPLETED

    Izmir, 35100, Turkey (Türkiye)

  • Local Institution - 881

    COMPLETED

    Adana, 01130, Turkey (Türkiye)

  • Local Institution - 882

    COMPLETED

    Istanbul, 34093, Turkey (Türkiye)

  • Local Institution - 883

    COMPLETED

    Mersin, 33343, Turkey (Türkiye)

  • Local Institution - 884

    COMPLETED

    Istanbul, 34098, Turkey (Türkiye)

  • Local Institution - 885

    COMPLETED

    Ankara, 06590, Turkey (Türkiye)

  • Local Institution - 921

    WITHDRAWN

    Leeds, LS9 7TF, United Kingdom

  • Local Institution - 923

    COMPLETED

    London, E1 1BB, United Kingdom

  • Local Institution - 924

    COMPLETED

    London, SE5 9RS, United Kingdom

  • Local Institution - 925

    COMPLETED

    Aberdeen, AB25 2ZN, United Kingdom

  • Local Institution - 926

    WITHDRAWN

    Sutton in Ashfield, NG17 4JL, United Kingdom

  • Local Institution - 928

    COMPLETED

    London, SE1 9RT, United Kingdom

  • Local Institution - 929

    WITHDRAWN

    Oxford, OX3 7LE, United Kingdom

  • Local Institution - 961

    COMPLETED

    Detroit, Michigan, 48201, United States

  • Local Institution - 967

    COMPLETED

    Cleveland, Ohio, 44195, United States

  • Local Institution - 968

    COMPLETED

    Los Angeles, California, 90027-6062, United States

  • Local Institution - 969

    COMPLETED

    New York, New York, 10065, United States

  • Local Institution - 970

    COMPLETED

    Chicago, Illinois, 60611, United States

  • Local Institution - 971

    COMPLETED

    Oakland, California, 94609, United States

  • Local Institution - 972

    COMPLETED

    Philadelphia, Pennsylvania, 19104, United States

  • Local Institution - 973

    COMPLETED

    Boston, Massachusetts, 02115, United States

  • Local Institution - 975

    COMPLETED

    Tampa, Florida, 33612, United States

  • Local Institution - 978

    COMPLETED

    Stanford, California, 94305, United States

  • Maggiore Polyclinic Hospital, IRCCS Ca' Granda

    RECRUITING

    Milan, 20122, Italy

  • Matsuyama Red Cross Hospital

    RECRUITING

    Matsuyama, Ehime, 790-8524, Japan

  • Military Hospital of Tunis

    RECRUITING

    Tunis, 1008, Tunisia

  • NTT Medical Center Tokyo

    RECRUITING

    Shinagawa City, Tokyo, 141-8625, Japan

  • Ogaki Municipal Hospital

    RECRUITING

    Ōgaki, Gifu, 503-8502, Japan

  • Queen Elizabeth Hospital

    RECRUITING

    Kota Kinabalu, Sabah, 88586, Malaysia

  • Royal Adelaide Hospital

    RECRUITING

    Adelaide, South Australia, 5000, Australia

  • Royal Prince Alfred Hospital

    RECRUITING

    Camperdown, 2050, Australia

  • Shonan Kamakura General Hospital

    RECRUITING

    Kamakura, 247-8533, Japan

  • Siriraj Hospital Mahidol University

    RECRUITING

    Bangkok, 10700, Thailand

  • The University of Texas - MD Anderson Cancer Center

    WITHDRAWN

    Houston, Texas, 77030, United States

  • University College London Hospitals NHS Foundation Trust - University College Hospital

    RECRUITING

    London, NW1 2BU, United Kingdom

  • University Hospital Farhat Hached

    RECRUITING

    Sousse, 4031, Tunisia

  • Vanderbilt - Ingram Cancer Center

    WITHDRAWN

    Nashville, Tennessee, 37232-5505, United States

  • Whittington Hospital

    RECRUITING

    London, N19 5NF, United Kingdom

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