Study of Luspatercept for Pediatric Beta-Thalassemia
This study is testing a drug called luspatercept (ACE-536) in children and teenagers aged 6 to 17 years old who have beta-thalassemia. Beta-thalassemia is a blood disorder that reduces the production of hemoglobin, the protein in red blood cells that carries oxygen throughout the body. The main goals are to find a safe and effective dose of luspatercept and to understand how the body processes the drug. The study is looking at both participants who need regular blood transfusions (transfusion-dependent) and those who don't (non-transfusion-dependent). The study plans to enroll up to 99 participants and the current status is unclear.
- Study design
- This is a Phase 2a interventional study with an estimated enrollment of 99 participants. It is structured in two parts, with different age groups and dose evaluations.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- Pharmacokinetics (how the body handles the drug) will be measured for up to one year after the first dose.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
NCT04143724
Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
Recruiting
PHASE2Ages 6–17InterventionalTreatmentCelgene
~99 participants
Updated 2026-07-10 on ClinicalTrials.gov
What's tested:ACE-536
At a glance
Recruiting sites
22 of 26 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Determination of the Recommended Dose (RD
Measured over Cycle 1 up to the day before Cycle 2 Day 1 or Study Day 22 if not receiving the second treatment cycle
+5 more outcomes measured
Conditions
Where it's being run
26 sites across 17 statesItaly3
Thailand3
Guangdong2
China2
Germany2
Maharashtra2
India2
California1
Study leadership
- Bristol-Myers Squibb · STUDY_DIRECTOR · Bristol-Myers Squibb
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
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):
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.
Exclusion
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).
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.
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:
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:
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.
What this trial measures
- Determination of the Recommended Dose (RDCycle 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 - CmaxTime from Cycle 1 Day 1 of Treatment Period up to a maximum of 1 year
Maximum serum concentration of drug
- Pharmacokinetics - AUCTime from Cycle 1 Day 1 of Treatment Period up to a maximum of 1 year
Area under the curve
- Pharmacokinetics (PK) - t1/2Time from Cycle 1 Day 1 of Treatment Period up to a maximum of 1 year
Half-life
- Pharmacokinetics (PK) - CL/FTime from Cycle 1 Day 1 of Treatment Period up to a maximum of 1 year
Apparent oral clearance
- Pharmacokinetics (PK) - Vd/FTime from Cycle 1 Day 1 of Treatment Period up to a maximum of 1 year
Apparent volume of distribution