A Phase 3 interventional study of Luspatercept in Myelodysplastic Syndromes (MDS), Beta-thalassemia and Myeloproliferative Neoplasm(MPN)-Associated Myelofibrosis, sponsored by Celgene. Recruiting at 143 sites in 22 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-07-01.
Sponsored by Celgene (part of Bristol-Myers Squibb) · Phase 3, Interventional, and Treatment
A Phase 3b, open-label, single-arm, rollover study to evaluate the long-term safety of luspatercept, to the following participants:
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 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.
1,997 studies on the registry are indexed under Myelodysplastic Syndromes; 324 are open to participants now.
This study's planned enrollment of 665 is above the median of 40 across 1,611 interventional studies indexed under Myelodysplastic Syndromes.
Browse Myelodysplastic Syndromes studies →Celgene is the lead sponsor of 428 studies on the registry; 12 are open to participants now.
Of its 104 completed or terminated interventional studies of FDA-regulated products, 30 (29%) have results posted.
First submitted before Celgene became part of Bristol-Myers Squibb.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Participants must meet all the following criteria to be enrolled in this study:
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) has achieved menarche at some point, 2) has not undergone a hysterectomy or bilateral oophorectomy, or 3) has not been naturally (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:
Agrees to use, and be able to comply with highly effective, without interruption, 35 days prior to starting 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:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Postmenopausal
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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
Also known as: ACE-536
Adverse Events (AEs)
Type, frequency, severity of AEs, relationship of treatment emergent adverse events to luspatercept
Time frame: From enrollment until at least 42 Day Safety Follow-up Phase
Number of participants progressing to high/very high risk MDS or AML.
Progression to high/very high-risk myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) (MDS and myelofibrosis \[MF\] only).
Time frame: Enrollment to Long-term post-treatment follow-up (Approximately, 5 years)
Percentage of participants progressing to high/very high risk MDS or AML
Progression to high/very high-risk myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) (MDS and myelofibrosis \[MF\] only)
Time frame: Enrollment to Long-term post-treatment follow-up (Approximately, 5 years)
Number of participants developing other malignancies/pre-malignancies
Development of other malignancies/pre-malignancies
Time frame: Enrollment to Long-term post-treatment follow-up (Approximately, 5 years)
Percentage of participants developing other malignancies/pre-malignancies
Development of other malignancies/pre-malignancies
Time frame: Enrollment to Long-term post-treatment follow-up (Approximately, 5 years)
Overall Survival
Time from date of randomization until death from any cause
Time frame: 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)
Percentage of participants developing treatment emergent EMH masses
Time frame: Enrollment to Long-term post-treatment follow-up (Approximately, 5 years)
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Showing the first 100 of 143 sites across 22 countries. Choose a country and city above to see the rest.
Plan to share: Yes — 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.html
Supporting information: Study protocol, Statistical analysis plan, Clinical study report
Time frame: See Plan Description
Access criteria: See Plan Description
Data sharing details ↗ · www.bms.com
Started Aug 2019 (actual). Primary completion is estimated for May 2028, 105 months after the start.
Of 26 industry-sponsored phase 3 interventional studies under Myelodysplastic Syndromes, half reached primary completion within 42 months of starting (the median), counting those still running; a quarter within 33 and three quarters within 52. This study's planned 105 months is above that.
Counting every sponsor, half of 45 studies reached it within 50 months; a quarter within 38 and three quarters within 95.
How these figures are worked out, and which studies are left out of them.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
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Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drug tested here: Luspatercept