Actively Recruiting
Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
Led by Celgene · Updated on 2026-01-06
99
Participants Needed
26
Research Sites
813 weeks
Total Duration
On this page
Sponsors
C
Celgene
Lead Sponsor
A
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants must be 6 years to less than 18 years old at the time of consent.
- Participants and their guardians must understand and voluntarily sign informed consent/assent forms.
- Participants must be willing and able to follow the study visit schedule and protocol requirements.
- Participants must have a confirmed diagnosis of beta-thalassemia or Hemoglobin E/beta-thalassemia.
- Transfusion-dependent participants must have had 4 or more red blood cell transfusions in the past 24 weeks with no transfusion-free period of 42 days or more and a history of regular transfusions for at least 2 years.
- Non-transfusion-dependent participants (ex-US sites only) must have had fewer than 4 transfusions in the past 24 weeks, not be on a regular transfusion program, and be transfusion-free for at least 8 weeks before enrollment.
- Non-transfusion-dependent participants must have a mean baseline hemoglobin of 10 g/dL or less based on at least 2 measurements taken at least 1 week apart within 4 weeks before enrollment.
- Participants must have a Karnofsky (age 16 years and older) or Lansky (under 16 years) performance status score of 50 or greater at screening.
- Female children of childbearing potential, individuals of childbearing potential, and male participants who have reached puberty must agree to physician-approved reproductive education and discuss reproductive side effects.
- Female participants of childbearing potential must have 2 negative pregnancy tests before starting therapy and agree to ongoing pregnancy testing.
- Females must agree to true abstinence or use effective contraception from 28 days before starting study drug until 12 weeks after stopping.
- Male participants must agree to true abstinence or use condoms during sexual contact with pregnant females or females of childbearing potential during the study and for 12 weeks after stopping.
You will not qualify if you...
- Participants with Hemoglobin S/beta-thalassemia or alpha-thalassemia (except beta-thalassemia combined with alpha-thalassemia) are excluded.
- Participants with active hepatitis C or B infection or known HIV infection are excluded.
- Participants with deep vein thrombosis, stroke, or other thromboembolic events requiring medical intervention within 24 weeks prior to enrollment are excluded.
- Participants with platelet counts greater than 1000 x 10^9/L are excluded.
- Participants who used other investigational drugs or devices within 28 days prior to enrollment are excluded.
- Participants with prior exposure to sotatercept or luspatercept are excluded.
- Participants who underwent or are scheduled for hematopoietic stem cell transplant or gene therapy are excluded.
- Participants who started iron chelation therapy less than or equal to 8 weeks prior to enrollment are excluded.
- Participants who received hydroxyurea, immunomodulatory drugs, fetal hemoglobin inducers, or erythropoiesis-stimulating agents less than 12 weeks (non-transfusion-dependent) or 24 weeks (transfusion-dependent) prior to enrollment are excluded.
- Pregnant or breastfeeding females or those planning pregnancy during the study are excluded.
- Participants with uncontrolled hypertension or major organ damage including symptomatic splenomegaly, significant liver, heart, lung, or kidney disease are excluded.
- Participants with proteinuria grade 3 or higher or high urine protein/creatinine or albumin/creatinine ratios are excluded.
- Participants using high dose long-term systemic glucocorticoids within 12 weeks prior to enrollment are excluded.
- Participants with severe allergic reactions or hypersensitivity to study drug components are excluded.
- Participants using cytotoxic agents or immunosuppressants within 28 days prior to enrollment are excluded.
- Participants with a history of malignancy except certain treated cancers are excluded.
- Participants with extramedullary hematopoiesis complications requiring treatment during screening are excluded.
- Participants with medical or psychiatric conditions that pose unacceptable risk or affect study results are excluded.
- Participants using herbs or food supplements likely to impact safety or efficacy within 24 weeks (transfusion-dependent) or 12 weeks (non-transfusion-dependent) before treatment are excluded.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 26 locations
1
Local Institution - 601
Los Angeles, California, United States, 90027
Completed
2
New York Presbyterian Hospital
New York, New York, United States, 10065-4870
Actively Recruiting
3
Southern Medical University Nanfang Hospital
Guangzhou, Guangdong, China, 510515
Actively Recruiting
4
Shenzhen Second People's Hospital
Shenzhen, Guangdong, China, 518028
Actively Recruiting
5
People's Liberation Army The 923rd Hospital
Nanning, GX, China, 530021
Actively Recruiting
6
West China Hospital - Sichuan University
Chengdu, Sichuan, China, 610041
Actively Recruiting
7
Sun Yat-sen Memorial Hospital, Sun Yat-Sen University
Guangzhou, China, 510120
Actively Recruiting
8
The First Affiliated Hospital of Guangxi Medical University
Nanning, China, 530021
Actively Recruiting
9
Universitätsklinikum Essen
Essen, Germany, 45147
Actively Recruiting
10
Universitatsklinikum Ulm
Ulm, Germany, 89081
Actively Recruiting
11
General Children's Hospital "Agia Sophia"
Athens, Greece, 115 27
Actively Recruiting
12
Kamala Hospital and Research Center
Hyderabad, Andhra Pradesh, India, 500052
Actively Recruiting
13
MCGM - Comprehensive Thalassemia Care, Pediatric Hematology-Oncology & BMT Centre, Borivali (E)
Mumbai, Maharashtra, India, 400022
Actively Recruiting
14
Kingsway Hospitals
Nagpur, Maharashtra, India, 440001
Actively Recruiting
15
Post Graduate Institute of Child Health
Noida, Uttar Pradesh, India, 201303
Actively Recruiting
16
Local Institution - 803
Kolkata, India, 700094
Withdrawn
17
Christian Medical College & Hospital
Vellore, India, 632004
Actively Recruiting
18
Ospedale Pediatrico Bambino Gesù IRCCS
Rome, Roma, Italy, 00165
Actively Recruiting
19
Ente Ospedaliero Ospedali Galliera - Centro della Microcitemia e delle Anemie Congenite
Genoa, Italy, 16128
Actively Recruiting
20
AOU dell'Universita degli Studi della Campania Luigi Vanvitelli
Naples, Italy, 80131
Actively Recruiting
21
Azienda Ospedaliero Universitaria S. Luigi Gonzaga
Orbassano, Italy, 10043
Actively Recruiting
22
Local Institution - 700
Beirut, Lebanon, 0
Withdrawn
23
Chulalongkorn University Faculty of Medicine - King Chulalongkorn Memorial Hospital
Bangkok, Thailand, 10330
Actively Recruiting
24
Siriraj Hospital Mahidol University
Bangkok, Thailand, 10700
Actively Recruiting
25
Ramathibodi Hospital, Mahidol University
Phyathai, Thailand, 10400
Actively Recruiting
26
Local Institution - 401
Izmir, Turkey (Türkiye), 35100
Completed
Research Team
B
BMS Study Connect Contact Center www.BMSStudyConnect.com
CONTACT
F
First line of email MUST contain NCT # and Site #.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
9
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