Beta-ThalassemiaPhase 2NCT04143724
Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
A Phase 2 study testing ACE-536 in people with Beta-Thalassemia, run by Celgene at 26 sites in 9 countries.
Trial summary
This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric participants with β-thalassemia. The study will be conducted in 2 parts for both transfusion-dependent (TD) and non-transfusion-dependent (NTD) β-thalassemia participants: TD Part A will be in adolescent participants… Read more about this study
- Who is it for?
- Ages 6 to 17 years
- Conditions the study is for
- Beta-Thalassemia
- What is being tested?
- ACE-536
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 99 participants
- Where
- 26 sitesin 9 countries
- Study dates
- 7 Nov 2019to 31 Aug 2035
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Participants must be 6 years to < 18 years of age at the time of signing the informed consent form (ICF)/informed assent form (IAF).
- Participants (and when applicable, parent/legal representative) must understand and voluntarily sign an ICF/IAF prior to conducting any study-related assessments/procedures.
- Participants (and when applicable, parent/legal representative) is willing and able to adhere to the study visit schedule and other protocol requirements.
- Participants must have documented diagnosis of β-thalassemia or Hemoglobin E/β-thalassemia.
- Transfusion dependence (TD):
- TD participant i. Participant is regularly transfused, defined as: ≥ 4 RBC transfusion events in the 24 weeks prior to enrollment with no transfusion-free period ≥ 42 days during that period.
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- Note: For the purpose of the study, transfusions administered over 2 or 3 consecutive days are considered as part of a single transfusion event. Participant must have a history of regular transfusions for at least 2 years.
- NTD participant (ex-US sites only) i. Participant must have received < 4 RBC transfusion events in the 24 weeks prior to enrollment.
- Participant must not be on a regular transfusion program and must be RBC transfusion-free for at least 8 weeks prior to enrollment.
- Participant must have mean baseline hemoglobin ≤ 10 g/dL, based on a minimum of 2 measurements ≥ 1 week apart within the 12-week screening period; at least 1 measurement should be collected within 4 weeks prior to enrollment; hemoglobin values within 21 days post-transfusion will be excluded.
- Participants have Karnofsky (age ≥16 years) or Lansky (age < 16 years) performance status score ≥ 50 at screening.
- Female children of childbearing potential (FCCBP), individuals of childbearing potential (IOCBP), and male (as assigned at birth) participants that have reached puberty (and when applicable, parent/legal representative) must agree to undergo physician-approved reproductive education and discuss the side effects of the study therapy on reproduction.
- Female children of childbearing potential, defined as females who have achieved menarche with or without breast development in Tanner Stage 2 or greater and have not undergone a hysterectomy or bilateral oophorectomy and IOCBP defined as a sexually mature woman who has achieved menarche at some point, has not undergone a hysterectomy or bilateral oophorectomy and has not been naturally postmenopausal for at least 24 consecutive months (ie, has had menses at any time in the preceding 24 consecutive months) must meet the following conditions below (Note: Secondary amenorrhea from any cause does not rule out childbearing potential. Breast development at Tanner Stage ≥2 alone does not reliably indicate reproductive potential):
- Medically supervised serum pregnancy tests with a sensitivity of at least 25 mIU/mL must be conducted in Female children of childbearing potential (FCCBP)/ individuals of childbearing potential (IOCBP), including those who commit to complete abstinence. Female children of childbearing potential/ individuals of childbearing potential (IOCBP) must have 2 negative pregnancy tests as verified by the Investigator prior to starting study therapy (one of these tests should be performed by central laboratory). Female children of childbearing potential/ individuals of childbearing potential (IOCBP) must agree to ongoing pregnancy testing during the course of the study at the End of Treatment (EOT) visit and at the 9-week Safety Follow-up visit.
- Female participants must, as appropriate to age and at the discretion of the site Investigator, either commit to true abstinence* from heterosexual contact (which must be reviewed on a monthly basis) 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 t1/2 of luspatercept based on multiple-dose PK data) after discontinuation of study therapy.
- Male (as assigned at birth) participants, as appropriate to age and the discretion of the study physician:
- Must practice true abstinence* (which must be reviewed on a monthly basis) or agree to use a synthetic or latex condom during sexual contact with a pregnant female or a Female children of childbearing potential (FCCBP)/ IOCBP while participating in the study, during dose interruptions and for at least 12 weeks (approximately 5 times the mean terminal t1/2 of luspatercept based on multiple-dose PK data) following IP discontinuation, even if he has undergone a successful vasectomy.
- True abstinence is acceptable when this is in line with the preferred and usual lifestyle of the participant. [Periodic abstinence (eg, calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception.] ** 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 progesterone/progestin containing) hormonal contraception: Oral; Intravaginal; Transdermal; Progestogen/progestin 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.
Who cannot join
- Participant has a diagnosis of Hemoglobin S/β-thalassemia or alpha (α)-thalassemia (eg, Hemoglobin H); β-thalassemia combined with α-thalassemia is allowed.
- Participant has of active hepatitis C (HCV) infection, as demonstrated by a positive HCF-ribonucleic acid (RNS) test of sufficient sensitivity, or active infectious hepatitis B (as demonstrated by the presence of hepatitis B surface antigen (HBsAG) and/or hepatitis B virus (HBV)-deoxyribonucleic acid (DNA) positive, or known positive human immunodeficiency virus (HIV).
- Note: Participants receiving antiviral therapies should have 2 negative HCV-RNA tests 3 months apart before ICF/IAF signature, ie, one test at the end of the antiviral therapy and the second test 3 months following the first test.
- Participant has deep vein thrombosis (DVT), stroke, or other thromboembolic event(s) (except clogged indwelling catheter) requiring medical intervention ≤ 24 weeks prior to enrollment.
- Participant has platelet count > 1000 x 109/L.
- Participant has treatment with another investigational drug or device ≤ 28 days prior to enrollment.
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- Participant has prior exposure to sotatercept (ACE-011) or luspatercept (ACE-536).
- Participant underwent or is scheduled for HSCT or gene therapy (candidates for HSCT or gene therapy with anticipated waiting period of ≥ 12 months are eligible).
- Participant use of iron chelation therapy (ICT), if initiated ≤ 8 weeks prior to enrollment (allowed if initiated > 8 weeks before or during treatment).
- Participant received treatment with hydroxyurea immunomodulatory drugs IMiDs (such as thalidomide), other fetal Hb (HbF) inducers or erythropoiesis-stimulating agents (ESAs) ≤ 12 weeks prior to enrollment for NTD participants and ≤ 24 weeks for TD participants.
- Participant is pregnant or breastfeeding female or plan to get pregnant during the study.
- Participant has uncontrolled hypertension. Controlled hypertension for this protocol is considered: blood pressure value corresponding to ≤ Grade 1 according to NCI CTCAE version 5.0 with or without pharmacological treatment.
- Participant has major organ damage, including:
- Symptomatic splenomegaly
- Liver disease with alanine aminotransferase (ALT)/aspartate aminotransferase (AST) > 3X the upper limit of normal (ULN) for age
- Heart disease, heart failure as classified by the New York Heart Association (NYHA) classification 3 or higher, or significant arrhythmia requiring treatment, or recent myocardial infarction within 6 months of enrollment
- Lung disease, including pulmonary fibrosis or pulmonary hypertension of Grade ≥ 3 according to NCI-CTCAE version 5.0.
- Renal insufficiency defined as:
- A serum creatinine based on age/gender based on threshold derived from Schwartz formula for estimating GFR utilizing child length and stature data published by the Centers for Disease Control.
- Participant has proteinuria ≥ Grade 3 according to NCI CTCAE version 5.0 (which is equivalent to a urine protein/creatinine ratio > 215 mg/mmol of creatinine), or a urine albumin/creatinine ratio > 129 mg/mmol of creatinine.
- Participant use high dose long-term therapy with systemic glucocorticoids ≤ 12 weeks prior to enrollment (physiologic replacement therapy for adrenal insufficiency is allowed). Low-dose long-term (defined as ≤ 0.2 mg/kg/day or ≤ 10 mg/day of prednisone equivalent), short treatment, single doses of systemic glucocorticoids (eg, for prevention or treatment of transfusion reactions), inhaled, intranasal and topical corticosteroids are allowed.
- Participant has history of severe allergic or anaphylactic reactions or hypersensitivity to recombinant proteins or excipients in the IP (refer to the IB).
- Participant use of cytotoxic agents, immunosuppressants ≤ 28 days prior to enrollment (ie, antithymocite globulin (ATG) or cyclosporine).
- Participant has history of malignancy with the exception of:
- Curatively resected nonmelanoma skin cancer.
- Curatively treated carcinoma in situ.
- Other solid tumor with no known active disease in the opinion of the Investigator.
- Participant who has extramedullary hematopoiesis (EMH) complications or requires treatment to control the growth of EMH masse(s) during the screening period.
- Any medical or psychiatric condition that in the opinion of the investigator would put the participant at unacceptable risk of participating in the study or may impact interpretation of the study results.
- Use of herbs or food supplements (eg, Chinese traditional medicine), if, per investigator's judgment, likely to impact the safety and efficacy assessment, for 24 weeks before initiating the study treatment for TD participants, and 12 weeks for NTD participants.
- Other protocol-defined Inclusion/Exclusion criteria apply.
Trial locations and contacts
26 sites in 9 countries. Contact the study team or a site near you to ask about taking part.
BMS Clinical Trials Contact Center www.BMSClinicalTrials.com contact
First line of the email MUST contain NCT # and Site #. contact
26 sites in 9 countries
China6 sites, 6 recruiting
- Recruiting
West China Hospital - Sichuan University
Chengdu, Sichuan, 610041
Ju Gao, Site 903
- Recruiting
Southern Medical University Nanfang Hospital
Guangzhou, Guangdong, 510515
Xiaoqin Feng, Site 902
- Recruiting
Sun Yat-sen Memorial Hospital, Sun Yat-Sen University
Guangzhou, 510120
Jian-Pei Fang, Site 901, +862081332199
- Recruiting
People's Liberation Army The 923rd Hospital
Nanning, GX, 530021
Xiaolin Yin, Site 904, +8613321717899
- Recruiting
The First Affiliated Hospital of Guangxi Medical University
Nanning, 530021
Yongrong Lai, Site 906, +07715356746
- Recruiting
Shenzhen Second People's Hospital
Shenzhen, Guangdong, 518028
Xin Du, Site 905, 13602523722
India6 sites, 5 recruiting
- Recruiting
Kamala Hospital and Research Center
Hyderabad, Andhra Pradesh, 500052
Suman Jain, Site 801
- Withdrawn
Local Institution - 803
Kolkata, 700094
- Recruiting
MCGM - Comprehensive Thalassemia Care, Pediatric Hematology-Oncology & BMT Centre, Borivali (E)
Mumbai, Maharashtra, 400022
Mamta V Manglani, Site 800
- Recruiting
Kingsway Hospitals
Nagpur, Maharashtra, 440001
Riya Ballikar, Site 805
- Recruiting
Post Graduate Institute of Child Health
Noida, Uttar Pradesh, 201303
Dr. Nita Radhakrishnan, Site 802, 91-9999041524
- Recruiting
Christian Medical College & Hospital
Vellore, 632004
Aby Abraham, Site 804
Italy4 sites, 4 recruiting
- Recruiting
Ente Ospedaliero Ospedali Galliera - Centro della Microcitemia e delle Anemie Congenite
Genoa, 16128
Manuela Balocco, Site 303, +390105634557
- Recruiting
AOU dell'Universita degli Studi della Campania Luigi Vanvitelli
Naples, 80131
Silverio Perrotta, Site 302, +0390815665421 0 000
- Recruiting
Azienda Ospedaliero Universitaria S. Luigi Gonzaga
Orbassano, 10043
Giovanni Battista Ferrero, Site 301
- Recruiting
Ospedale Pediatrico Bambino Gesù IRCCS
Rome, Roma, 00165
Franco Locatelli, Site 304, 390668593697
Thailand3 sites, 3 recruiting
- Recruiting
Chulalongkorn University Faculty of Medicine - King Chulalongkorn Memorial Hospital
Bangkok, 10330
Darintr Sosothikul, Site 503
- Recruiting
Siriraj Hospital Mahidol University
Bangkok, 10700
Vip Viprakasit, Site 501, 6624122113
- Recruiting
Ramathibodi Hospital, Mahidol University
Phyathai, 10400
Duantida Songdej, Site 502
United States2 sites, 1 recruiting
- Completed
Local Institution - 601
Los Angeles, California, 90027
- Recruiting
New York Presbyterian Hospital
New York, New York, 10065-4870
Sujit Sheth, Site 602, 212-746-3400
Germany2 sites, 2 recruiting
- Recruiting
Universitätsklinikum Essen
Essen, 45147
Carmen Aramayo-Singelmann, Site 102
- Recruiting
Universitatsklinikum Ulm
Ulm, 89081
Holger Cario, Site 101, +4973150027776
Greece1 site, 1 recruiting
- Recruiting
General Children's Hospital "Agia Sophia"
Athens, 115 27
Antonis Kattamis, Site 201, +302107467772 0(000)
About this study
Official title: A Phase 2a Study to Evaluate the Safety and Pharmacokinetics of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric participants with β-thalassemia.
The study will be conducted in 2 parts for both transfusion-dependent (TD) and non-transfusion-dependent (NTD) β-thalassemia participants: TD Part A will be in adolescent participants aged 12 to <18 years with two dose escalation cohorts, followed by a dose expansion cohorts. NTD Part A will be conducted in the same age group participants as TD Part A with dose confirmation and expansion cohorts. After Part A TD participants have completed at least one year of treatment, all available safety data from Part A adolescent participants will be evaluated before initiating TD and NTD Part B in the age group from 6 to <12 years old. Part B will consist of two dose escalation cohorts for TD and two dose escalation cohorts for NTD.
Read more
Upon completion of the Treatment Period, participants of any cohort who are benefiting from the study treatment, will be offered the opportunity to continue luspatercept treatment in the Long-term Treatment Period for up to 5 years from their first dose.
Participants who discontinue study treatment at any time will continue in the Posttreatment Follow-up Period for at least 5 years from their first dose of luspatercept, or 3 years from their last dose, whichever occurs later, or until they withdraw consent/assent, are lost to follow-up, or the End of Trial, whichever occurs first. If neither commercial treatment nor an LTFU (long-term follow-up) protocol is available at that time, continued treatment will be provided within this study or via an alternative mechanism, at the Sponsor's discretion.
- Conditions
- Beta-Thalassemia
- Being tested
- ACE-536
- Keywords
- ACE-536, Luspatercept, Pharmacokinetics, Beta-Thalassemia, Red Blood Cell Transfusion
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ACE-536Drug
Also called Luspatercept, BMS-986346
Specified dose on specified days
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1: TD Dose Escalation Cohort: 12 to < 18 years Luspatercept 0.75 mg/kg | Experimental | Not described Receives: Drug: ACE-536 |
| Cohort 2: TD Dose Escalation Cohort: 12 to < 18 years: Luspatercept 1.0 mg/kg | Experimental | Not described Receives: Drug: ACE-536 |
| Cohort 3: TD Dose Expansion Cohort: 12 to <18 years Luspatercept 1.0 mg/kg | Experimental | Not described Receives: Drug: ACE-536 |
| Cohort 4: TD Dose Escalation Cohort: 6 to < 12 years Luspatercept 1.0 mg/kg | Experimental | Not described Receives: Drug: ACE-536 |
| Cohort 5: TD Dose Escalation Cohort: 6 to <12 years Luspatercept 1.2 mg/kg | Experimental | Not described Receives: Drug: ACE-536 |
| Cohort 6: NTD Dose Confirmation Cohort: 12 to < 18 years Luspatercept 1.0 mg/kg | Experimental | Not described Receives: Drug: ACE-536 |
| Cohort 7: NTD Dose Expansion Cohort: NTD 12 to < 18 years | Experimental | Not described Receives: Drug: ACE-536 |
| Cohort 8: NTD Dose Escalation Cohort: 6 to < 12 years Luspatercept 1.0 mg/kg | Experimental | Not described Receives: Drug: ACE-536 |
| Cohort 9: NTD Dose Escalation Cohort: 6 to < 12 years Luspatercept 1.2 mg/kg | Experimental | Not described Receives: Drug: ACE-536 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determination of the Recommended Dose (RD
Determine the recommended dose of luspatercept that is safe and tolerable in pediatric participants with transfusion-dependent B-thalassemia or non-transfusion-dependent β-thalassemia
Measured: Cycle 1 up to the day before Cycle 2 Day 1 or Study Day 22 if not receiving the second treatment cycle
Pharmacokinetics - Cmax
Maximum serum concentration of drug
Measured: Time from Cycle 1 Day 1 of Treatment Period up to a maximum of 1 year
Pharmacokinetics - AUC
Area under the curve
Measured: Time from Cycle 1 Day 1 of Treatment Period up to a maximum of 1 year
Pharmacokinetics (PK) - t1/2
Half-life
Measured: Time from Cycle 1 Day 1 of Treatment Period up to a maximum of 1 year
Pharmacokinetics (PK) - CL/F
Apparent oral clearance
Measured: Time from Cycle 1 Day 1 of Treatment Period up to a maximum of 1 year
Pharmacokinetics (PK) - Vd/F
Apparent volume of distribution
Measured: Time from Cycle 1 Day 1 of Treatment Period up to a maximum of 1 year
Secondary outcomes (6)
Mean change in Red Blood Cell (RBC) Transfusion Burden for transfusion-dependent β-thalassemia participants
Change from baseline as continuous variable
Measured: 12 weeks prior to enrollment; Treatment Period and up to End of Treatment including Long-term Treatment Period - Up to 5 years
Mean change in hemoglobin levels for non-transfusion-dependent β-thalassemia participants
Change from baseline as continuous variable
Measured: 12 weeks prior to enrollment; Treatment Period and up to End of Treatment including Long-term Treatment Period - Up to 5 years
Immunogenicity
Frequency of antidrug antibodies (ADA)
Measured: Time from Cycle 1 Day 1 of Treatment Period up to a maximum of 1 year
Mean change from baseline in mean daily dose of iron chelation therapy (ICT)
Change from baseline as continuous variable
Measured: 12 weeks prior to enrollment; Treatment Period and up to End of Treatment including Long-term Treatment Period - Up to 5 years
Mean change from baseline in serum ferritin
Change from baseline as continuous variable
Measured: 12 weeks prior to enrollment; Treatment Period and up to End of Treatment including Long-term Treatment Period - Up to 5 years
Safety - Incidence of Adverse Events (AEs)
An AE is any noxious, unintended, or untoward medical occurrence that may appear or worsen in a participant during the course of a study. It may be a new intercurrent illness, a worsening concomitant illness, an injury, or any concomitant impairment of the participant's health, including laboratory test values, regardless of etiology. Any worsening (ie, any clinically significant adverse change in the frequency or intensity of a preexisting condition) should be considered an AE
Measured: From enrollment until at least 9 weeks after last dose of study treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Oct 2019
- Study start7 Nov 2019
- Primary completion31 Jan 2028 (estimated)
- Study completion31 Aug 2035 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Beta-ThalassemiaStudies of the same condition, open and under way.
- More trials run by CelgeneThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 99 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Celgene
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
- Responsible party
- Sponsor
- Investigators
- Bristol-Myers Squibb, Study director, Bristol-Myers Squibb
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04143724
- Sponsor's protocol ID
- ACE-536-B-THAL-004
- Other IDs
- 2022-502499-22-00
- Status last verified
- Jul 2026
- First submitted
- 2 Oct 2019
- Last updated
- 10 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- 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
Study at a glance
From the registry record, last updated by the sponsor on 10 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2a Study to Evaluate the Safety and Pharmacokinetics of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
- ClinicalTrials.gov ID
- NCT04143724
- Acronym / sponsor ID
- ACE-536-B-THAL-004
- Conditions
- Beta-Thalassemia
- Keywords
- ACE-536, Luspatercept, Pharmacokinetics, Beta-Thalassemia, Red Blood Cell Transfusion
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 99 participants (estimated)
- Lead sponsor
- CelgeneIndustry (pharma, biotech, devices)
- Collaborators
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
- Study start
- 7 Nov 2019 (actual)
- Primary completion
- 31 Jan 2028 (estimated)
- Study completion
- 31 Aug 2035 (estimated)
- First posted
- 29 Oct 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric participants with β-thalassemia.
The study will be conducted in 2 parts for both transfusion-dependent (TD) and non-transfusion-dependent (NTD) β-thalassemia participants: TD Part A will be in adolescent participants aged 12 to <18 years with two dose escalation cohorts, followed by a dose expansion cohorts. NTD Part A will be conducted in the same age group participants as TD Part A with dose confirmation and expansion cohorts. After Part A TD participants have completed at least one year of treatment, all available safety data from Part A adolescent participants will be evaluated before initiating TD and NTD Part B in the age group from 6 to <12 years old. Part B will consist of two dose escalation cohorts for TD and two dose escalation cohorts for NTD.
Upon completion of the Treatment Period, participants of any cohort who are benefiting from the study treatment, will be offered the opportunity to continue luspatercept treatment in the Long-term Treatment Period for up to 5 years from their first dose.
Participants who discontinue study treatment at any time will continue in the Posttreatment Follow-up Period for at least 5 years from their first dose of luspatercept, or 3 years from their last dose, whichever occurs later, or until they withdraw consent/assent, are lost to follow-up, or the End of Trial, whichever occurs first. If neither commercial treatment nor an LTFU (long-term follow-up) protocol is available at that time, continued treatment will be provided within this study or via an alternative mechanism, at the Sponsor's discretion.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 9
| Arm | Type | Receives |
|---|---|---|
| Cohort 1: TD Dose Escalation Cohort: 12 to < 18 years Luspatercept 0.75 mg/kg | Experimental | Drug: ACE-536 |
| Cohort 2: TD Dose Escalation Cohort: 12 to < 18 years: Luspatercept 1.0 mg/kg | Experimental | Drug: ACE-536 |
| Cohort 3: TD Dose Expansion Cohort: 12 to <18 years Luspatercept 1.0 mg/kg | Experimental | Drug: ACE-536 |
| Cohort 4: TD Dose Escalation Cohort: 6 to < 12 years Luspatercept 1.0 mg/kg | Experimental | Drug: ACE-536 |
| Cohort 5: TD Dose Escalation Cohort: 6 to <12 years Luspatercept 1.2 mg/kg | Experimental | Drug: ACE-536 |
| Cohort 6: NTD Dose Confirmation Cohort: 12 to < 18 years Luspatercept 1.0 mg/kg | Experimental | Drug: ACE-536 |
| Cohort 7: NTD Dose Expansion Cohort: NTD 12 to < 18 years | Experimental | Drug: ACE-536 |
| Cohort 8: NTD Dose Escalation Cohort: 6 to < 12 years Luspatercept 1.0 mg/kg | Experimental | Drug: ACE-536 |
| Cohort 9: NTD Dose Escalation Cohort: 6 to < 12 years Luspatercept 1.2 mg/kg | Experimental | Drug: ACE-536 |
Who may qualify
- Ages
- 6 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (18 listed)
- Participants must be 6 years to < 18 years of age at the time of signing the informed consent form (ICF)/informed assent form (IAF).
- Participants (and when applicable, parent/legal representative) must understand and voluntarily sign an ICF/IAF prior to conducting any study-related assessments/procedures.
- Participants (and when applicable, parent/legal representative) is willing and able to adhere to the study visit schedule and other protocol requirements.
- Participants must have documented diagnosis of β-thalassemia or Hemoglobin E/β-thalassemia.
- Transfusion dependence (TD):
Key exclusion criteria (31 listed)
- Participant has a diagnosis of Hemoglobin S/β-thalassemia or alpha (α)-thalassemia (eg, Hemoglobin H); β-thalassemia combined with α-thalassemia is allowed.
- Participant has of active hepatitis C (HCV) infection, as demonstrated by a positive HCF-ribonucleic acid (RNS) test of sufficient sensitivity, or active infectious hepatitis B (as demonstrated by the presence of hepatitis B surface antigen (HBsAG) and/or hepatitis B virus (HBV)-deoxyribonucleic acid (DNA) positive, or known positive human immunodeficiency virus (HIV).
- Note: Participants receiving antiviral therapies should have 2 negative HCV-RNA tests 3 months apart before ICF/IAF signature, ie, one test at the end of the antiviral therapy and the second test 3 months following the first test.
- Participant has deep vein thrombosis (DVT), stroke, or other thromboembolic event(s) (except clogged indwelling catheter) requiring medical intervention ≤ 24 weeks prior to enrollment.
- Participant has platelet count > 1000 x 109/L.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ACE-536Also: Luspatercept, BMS-986346Specified dose on specified days | Drug | Cohort 1: TD Dose Escalation Cohort: 12 to < 18 years Luspatercept 0.75 mg/kg, Cohort 2: TD Dose Escalation Cohort: 12 to < 18 years: Luspatercept 1.0 mg/kg, Cohort 3: TD Dose Expansion Cohort: 12 to <18 years Luspatercept 1.0 mg/kg, Cohort 4: TD Dose Escalation Cohort: 6 to < 12 years Luspatercept 1.0 mg/kg, Cohort 5: TD Dose Escalation Cohort: 6 to <12 years Luspatercept 1.2 mg/kg, Cohort 6: NTD Dose Confirmation Cohort: 12 to < 18 years Luspatercept 1.0 mg/kg, Cohort 7: NTD Dose Expansion Cohort: NTD 12 to < 18 years, Cohort 8: NTD Dose Escalation Cohort: 6 to < 12 years Luspatercept 1.0 mg/kg, Cohort 9: NTD Dose Escalation Cohort: 6 to < 12 years Luspatercept 1.2 mg/kg |
Primary outcome
Determination of the Recommended Dose (RD
Time frame: Cycle 1 up to the day before Cycle 2 Day 1 or Study Day 22 if not receiving the second treatment cycle
Determine the recommended dose of luspatercept that is safe and tolerable in pediatric participants with transfusion-dependent B-thalassemia or non-transfusion-dependent β-thalassemia
Pharmacokinetics - Cmax
Time frame: Time from Cycle 1 Day 1 of Treatment Period up to a maximum of 1 year
Maximum serum concentration of drug
Pharmacokinetics - AUC
Time frame: Time from Cycle 1 Day 1 of Treatment Period up to a maximum of 1 year
Area under the curve
Pharmacokinetics (PK) - t1/2
Time frame: Time from Cycle 1 Day 1 of Treatment Period up to a maximum of 1 year
Half-life
Pharmacokinetics (PK) - CL/F
Time frame: Time from Cycle 1 Day 1 of Treatment Period up to a maximum of 1 year
Apparent oral clearance
Pharmacokinetics (PK) - Vd/F
Time frame: Time from Cycle 1 Day 1 of Treatment Period up to a maximum of 1 year
Apparent volume of distribution
6 secondary outcomes
Mean change in Red Blood Cell (RBC) Transfusion Burden for transfusion-dependent β-thalassemia participants
Time frame: 12 weeks prior to enrollment; Treatment Period and up to End of Treatment including Long-term Treatment Period - Up to 5 years
Mean change in hemoglobin levels for non-transfusion-dependent β-thalassemia participants
Time frame: 12 weeks prior to enrollment; Treatment Period and up to End of Treatment including Long-term Treatment Period - Up to 5 years
Immunogenicity
Time frame: Time from Cycle 1 Day 1 of Treatment Period up to a maximum of 1 year
Mean change from baseline in mean daily dose of iron chelation therapy (ICT)
Time frame: 12 weeks prior to enrollment; Treatment Period and up to End of Treatment including Long-term Treatment Period - Up to 5 years
Mean change from baseline in serum ferritin
Time frame: 12 weeks prior to enrollment; Treatment Period and up to End of Treatment including Long-term Treatment Period - Up to 5 years
Safety - Incidence of Adverse Events (AEs)
Time frame: From enrollment until at least 9 weeks after last dose of study treatment
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jul 2026.
- BMS Clinical Trial InformationLink provided by the sponsor.
- BMS Clinical Trial Patient RecruitingLink provided by the sponsor.
- Individual participant dataSharing planned: 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
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
BMS Clinical Trials Contact Center www.BMSClinicalTrials.com contact
First line of the email MUST contain NCT # and Site #. contact
Bristol-Myers Squibb study director
Bristol-Myers Squibb
26 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ACE-536 in people with Beta-Thalassemia, run by Celgene at 26 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 18 inclusion and 31 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 10 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 26 sites in China, India, Italy, Thailand, United States and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact BMS Clinical Trials Contact Center www.BMSClinicalTrials.com (855-907-3286, Clinical.Trials@bms.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Determination of the Recommended Dose (RD (time frame: Cycle 1 up to the day before Cycle 2 Day 1 or Study Day 22 if not receiving the second treatment cycle). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Jul 2026.