Actively Recruiting
A Study to Determine the Efficacy and Safety of Luspatercept in Adult Participants and to Evaluate the Safety and Pharmacokinetics in and Adolescent Participants With Alpha (α)-Thalassemia
Led by Bristol-Myers Squibb · Updated on 2026-03-24
189
Participants Needed
36
Research Sites
609 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The purpose of the study is to evaluate the efficacy and safety of luspatercept plus best supportive care (BSC) vs placebo plus BSC on anemia in adult participants with α-thalassemia hemoglobin H (HbH) disease and determine the safety and drug levels in adolescent participants.
CONDITIONS
Official Title
A Study to Determine the Efficacy and Safety of Luspatercept in Adult Participants and to Evaluate the Safety and Pharmacokinetics in and Adolescent Participants With Alpha (α)-Thalassemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Adult participants aged 18 years or older with documented alpha-thalassemia hemoglobin H disease
- Transfusion-dependent adults: at least 6 red blood cell units transfused in the 24 weeks before randomization
- Non-transfusion-dependent adults: fewer than 6 red blood cell units transfused in the 24 weeks before randomization, no transfusions for at least 8 weeks prior unless for acute conditions, and mean baseline hemoglobin of 10 g/dL or less based on at least 2 measurements
- Eastern Cooperative Oncology Group (ECOG) score of 0 or 1 for adult participants
- Adolescent participants aged 12 to less than 18 years with documented alpha-thalassemia hemoglobin H disease
- Transfusion-dependent adolescents: at least 4 red blood cell events in the 24 weeks before enrollment with no transfusion-free period longer than 56 days during that time and history of regular transfusions for at least 2 years
- Non-transfusion-dependent adolescents: fewer than 4 red blood cell events in the 24 weeks before enrollment, transfusion-free for at least 8 weeks prior, and mean baseline hemoglobin of 10 g/dL or less based on at least 2 measurements
- Karnofsky (for age 16 years and older) or Lansky (for under 16 years) performance status score of 50 or higher at screening
You will not qualify if you...
- Diagnosis of alpha-thalassemia trait, Hb Bart hydrops, ATRx alpha-thalassemia, hemoglobin S/beta-thalassemia, myelodysplasia subtype anemia, or HbE homozygous beta gene mutation
- Anemia caused by nutritional deficiency, chronic disease, autoimmune hemolytic anemia, or other hemolytic anemias
- Hemolysis episodes unrelated to alpha-thalassemia within 8 weeks before randomization
- Deep vein thrombosis, stroke, or other thromboembolic events requiring treatment within 24 weeks before randomization (except clogged catheters)
- Uncontrolled hypertension exceeding Grade 1 blood pressure levels
- Women who are pregnant, planning pregnancy during the study, or breastfeeding
- Previous hematopoietic stem cell transplant or gene therapy (unless waiting period of 12 months is met)
- Use of hydroxyurea treatment within 12 weeks for non-transfusion-dependent or 24 weeks for transfusion-dependent participants prior to enrollment
- Extramedullary hematopoiesis complications requiring treatment during screening
- Any medical or psychiatric condition that poses unacceptable risk or affects data interpretation according to the investigator
- Other protocol-specified inclusion or exclusion criteria apply
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 36 locations
1
Local Institution - 0008
Halifax, Nova Scotia, Canada, B3K 6R8
Withdrawn
2
Sun Yat-sen Memorial Hospital, Sun Yat-Sen University
Guangzhou, GD, China, 510120
Actively Recruiting
3
Nanfang Hospital of Southern Medical University
Guangzhou, GD, China, 510515
Actively Recruiting
4
The First People's Hospital of Foshan
Foshan, Guangdong, China, 528000
Actively Recruiting
5
Maoming People's Hospital
Maoming Shi, Guangdong, China, 525000
Actively Recruiting
6
Shenzhen Second People's Hospital
Shenzhen Shi, Guangdong, China, 518025
Actively Recruiting
7
Liuzhou People's Hospital
Liuzhou, Guangxi, China, 545006
Actively Recruiting
8
People's Liberation Army The 923rd Hospital
Nanning, GX, China, 530021
Actively Recruiting
9
Local Institution - 0011
Haikou, Hainan, China, 570203
Active, Not Recruiting
10
Local Institution - 0012
Kunming, Yunnan, China, 650032
Completed
11
Hainan General Hospital
Haikou, China, 570311
Actively Recruiting
12
The First Affiliated Hospital of Guangxi Medical University
Nanning, China, 530021
Actively Recruiting
13
Local Institution - 0005
Thessaloniki, B, Greece, 546 42
Withdrawn
14
Local Institution - 0007
Larissa, E, Greece, 412 21
Active, Not Recruiting
15
Local Institution - 0018
Rio, G, Greece, 265 04
Active, Not Recruiting
16
Local Institution - 0006
Athens, Greece, 115 27
Active, Not Recruiting
17
Local Institution - 0009
Goudi, Greece, 11527
Active, Not Recruiting
18
Local Institution - 0025
Hong Kong, HK, Hong Kong
Completed
19
Local Institution - 0024
Hong Kong Island, Hong Kong
Withdrawn
20
Local Institution - 0022
Cagliari, CA, Italy, 09121
Withdrawn
21
Local Institution - 0026
Genova, GE, Italy, 16128
Active, Not Recruiting
22
Local Institution - 0020
Orbassano, TO, Italy, 10043
Active, Not Recruiting
23
Local Institution - 0028
Naples, Italy, 80131
Active, Not Recruiting
24
"Universita degli Studi della Campania ""Luigi Vanvitelli"" - AOU - Clinica Pediatrica"
Naples, Italy, 80138
Actively Recruiting
25
Hospital Tunku Azizah
Kuala Lumpur, WP, Malaysia, 50586
Actively Recruiting
26
Hospital Sultanah Aminah
Johor Bahru, Malaysia, 80100
Actively Recruiting
27
Local Institution - 0035
Al-Ahsa, Saudi Arabia, 31982
Withdrawn
28
King Saud University (KSU) - College of Medicine
Riyadh, Saudi Arabia, 11411
Actively Recruiting
29
KK Women's and Children's Hospital
Singapore, Singapore, 229899
Actively Recruiting
30
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, KHH, Taiwan, 807
Actively Recruiting
31
National Taiwan University Hospital
Nan Gang Qu, TPE, Taiwan, 10002
Actively Recruiting
32
China Medical University Hospital
Taichung, TXG, Taiwan, 40447
Actively Recruiting
33
Siriraj Hospital
Bangkok Noi, Bangkok, Thailand, 10700
Actively Recruiting
34
Naresuan University Hospital
Mueang Phitsanulok, Thailand, 65000
Actively Recruiting
35
Hacettepe Üniversitesi Tıp Fakültesi
Altındağ, Turkey (Türkiye), 06230
Actively Recruiting
36
Istanbul Universitesi - Istanbul Tip Fakultesi (ITF) Hastanesi
Topkapı, Turkey (Türkiye), 34093
Actively Recruiting
Research Team
B
BMS Clinical Trials Contact Center www.BMSClinicalTrials.com
CONTACT
F
First line of the email MUST contain NCT # and Site #.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
4
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