Concomitant Presence of Hb Agrinio and - -Med Deletion in a Greek Male Patient with Hemoglobinopathy H: More Severe Phenotype and Literature Review.
Michael D Diamantidis, Stefania Pitsava, Omar Zayed...
https://pubmed.ncbi.nlm.nih.gov/37606495Actively Recruiting
Phase 2
Age: 12Years +
All Genders
ID05664737
A Phase 2 Study to Evaluate the Efficacy and Safety of Luspatercept in Adults and Safety and Pharmacokinetics in Adolescents With Alpha-Thalassemia
Led by Bristol-Myers Squibb · Updated on 2026-03-24
189
Participants Needed
36
Research Sites
369 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the efficacy and safety of luspatercept combined with best supportive care (BSC) compared to placebo plus BSC for treating anemia in adults with alpha-thalassemia hemoglobin H (HbH) disease. The study also aims to assess the safety and drug levels of luspatercept in adolescent participants. This Phase 2 trial is sponsored by Bristol-Myers Squibb and focuses on improving anemia management in this specific patient group. Participants are divided into groups based on transfusion dependence and age. Adult transfusion-dependent and non-transfusion-dependent participants receive either luspatercept plus BSC or placebo plus BSC. Adolescent participants aged 12 to under 18 years are assessed for safety and pharmacokinetics of luspatercept. Treatments involve specified doses given on designated days, with monitoring across several weeks. The study includes a randomized, quadruple-masked design to compare outcomes effectively. During the study, participants undergo regular assessments including blood tests to measure hemoglobin levels, red blood cell transfusion requirements, and adverse events. Researchers also monitor pharmacokinetics and immunogenicity over extended periods up to several years. Patient-reported outcomes, quality of life, physical function tests, and biomarkers related to anemia and iron homeostasis are evaluated to understand treatment impact. The total participation may last up to 8.5 years, ensuring comprehensive safety and efficacy data collection.
CONDITIONS
Brief 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
Age: 12Years +
All Genders
Eligibility Criteria
You may qualify if you...
- Adult participants 18 years or older with alpha-thalassemia hemoglobin H disease and transfusion dependence or non-transfusion dependence as defined by RBC transfusion history
- Adolescent participants aged 12 to under 18 years with alpha-thalassemia hemoglobin H disease and specific transfusion requirements
- Adults with Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1
- Adolescents with Karnofsky (age ≥16 years) or Lansky (age <16 years) performance status score ≥50 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 homozygous HbE beta gene mutation
- Anemia caused by nutritional deficiency, chronic disease, autoimmune hemolytic anemia, or other hemolytic anemias not related to alpha-thalassemia
- Hemolysis episodes unrelated to alpha-thalassemia within 8 weeks prior to randomization
- History of deep vein thrombosis, stroke, or thromboembolic events requiring treatment within 24 weeks prior to randomization
- Uncontrolled hypertension above Grade 1 level
- Women who are pregnant, planning pregnancy, or breastfeeding
- Prior hematopoietic stem cell transplant or gene therapy (unless waiting period of 12 months met)
- Use of hydroxyurea treatment within 12 weeks for non-transfusion dependent or 24 weeks for transfusion dependent participants
- Extramedullary hematopoiesis complications requiring treatment during screening
- Any medical or psychiatric condition that may increase risk or affect study data interpretation
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - Up to 108 weeks
Participants receive Luspatercept or placebo with best supportive care according to their transfusion dependence status.
Regular visits for treatment and assessments during the study period
Follow-up
Duration - Up to approximately 7 years
Participants are monitored for safety, pharmacokinetics, and long-term outcomes after completing treatment.
Periodic visits for long-term safety and efficacy monitoring
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
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Research Team
B
BMS Clinical Trials Contact Center www.BMSClinicalTrials.com
F
First line of the email MUST contain NCT # and Site #.
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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