β-Thalassemia MajorNCT06596642
A Real-World Study of β-Thalassemia Major Treatment With Luspatercept in Taiwan
An observational study of β-Thalassemia Major, run by Bristol-Myers Squibb at 5 sites in Taiwan.
Trial summary
This real-world study will assess the efficacy and safety of luspatercept treatment for β-thalassemia major in Taiwan as well as the impact on quality of life. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- β-Thalassemia Major
- What is being tested?
- Luspatercept
- How many people
- 61 participants
- Where
- 5 sitesTaiwan
- Study dates
- 21 Aug 2024to 23 Sep 2026
- Phase
- Observational
- Are there results?
- None posted in the registry
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Adults participants in Taiwan diagnosed with β-thalassemia major and treated with luspatercept.
Who may be able to join
- Participants ≥18 years of age.
- Participants with the diagnosis of transfusion-dependent β-thalassemia major who are eligible to the treatment of luspatercept.
- Before luspatercept treatment, participant's transfusion burden ≥ 24 Red Blood Cell units in 24 weeks, without ≥35-day transfusion free.
Who cannot join
- Hypersensitivity to the active substance or to any of the excipients.
- Pregnancy.
- Participants requiring treatment to control the growth of extramedullary hematopoiesis (EMH) masses.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
Taiwan5 sites
Local Institution - 02
Kaohsiung City, 807
Local Institution - 04
Tainan, 704
Local Institution - 01
Taipei, 100229
Local Institution - 03
Taipei, 10449
Local Institution - 05
Taoyuan, 333
About this study
Official title: Taiwan β-Thalassemia Major Real-World Study for Luspatercept
This real-world study will assess the efficacy and safety of luspatercept treatment for β-thalassemia major in Taiwan as well as the impact on quality of life.
- Conditions
- β-Thalassemia Major
- Being tested
- Luspatercept
- Keywords
- β-Thalassemia major
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LuspaterceptDrug
As per product label
| Study group | Role | What they receive |
|---|---|---|
| Adults participants diagnosed with β-thalassemia major | Not described Receives: Drug: Luspatercept |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Red Blood Cell (RBC) transfusion units received by participants
Measured: Baseline, every 3 weeks thereafter until week 96
Participant hemoglobin (Hb) level results
Measured: Baseline, every 3 weeks thereafter until week 96
Participant serum ferritin results
Measured: Baseline, week 12, 24, 36, 48, 60, 72, 84 and 96
Secondary outcomes (25)
Participant platelet level results
Measured: Baseline, and every 3 weeks thereafter up to week 96
Participant white blood cell count results
Measured: Baseline, and every 3 weeks thereafter up to week 96
Participant reticulocytes/normoblast count results
Measured: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
Participant Hemoglobin F (HbF) levels
Measured: Baseline and weeks 6 and 12
Participant uric acid level results
Measured: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
Participant lactate dehydrogenase (LDH) level results
Measured: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
Participant aspartate aminotransferase (AST)/alanine aminotransferase (ALT) ratio
Measured: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
Participant creatinine level results
Measured: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
Participant bilirubin (total and direct) laboratory values
Measured: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
Type of iron chelating therapy prescribed to participants
Measured: Baseline, every 3 weeks thereafter until week 96
Iron chelating therapy treatment dosage prescribed to participants
Measured: Baseline, every 3 weeks thereafter until week 96
Participant Adverse Events (AEs)
Measured: Baseline and every 3 weeks until week 12, then weeks 24, 36, 48, 60, 72, 84 and 96
Participant health-related quality of life as assessed by Transfusion-dependent Quality of Life (TranQoL) questionnaire
Measured: Baseline, week 12, 24, 48, 72 and 96
Participant health-related quality of life as assessed by EQ-5D-5L questionnaire
Measured: Baseline, week 12, 24, 48, 72 and 96
Luspatercept treatment dosage prescribed to participants
Measured: Baseline, every 3 weeks thereafter until week 96
Duration of luspatercept treatment
Measured: Baseline, every 3 weeks thereafter until week 96
Participant reason(s) for luspatercept treatment discontinuation as assessed by the treating clinician
Measured: Baseline, every 3 weeks thereafter until week 96
Participant demographics
Measured: Baseline
Participant weight
Measured: Baseline
Participant β0/β0 genotype status
Measured: Baseline
Participant disease status
Measured: Baseline
Participant comorbidities
Measured: Baseline
Participant concomitant medication
Measured: Baseline
Participant β-thalassemia treatment history
Measured: Baseline
Participant surgical history (splenectomy and/or cholecystectomy)
Measured: Baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2024
- Study start21 Aug 2024
- Primary completion23 Sep 2026
- Study completion23 Sep 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for β-Thalassemia MajorStudies of the same condition, open and under way.
- More trials run by Bristol-Myers SquibbThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 61 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bristol-Myers Squibb
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Bristol-Myers Squibb, Study director, Bristol-Myers Squibb
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06596642
- Sponsor's protocol ID
- CA056-1132
- Status last verified
- Nov 2026
- First submitted
- 29 Jul 2024
- Last updated
- 5 Nov 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Taiwan β-Thalassemia Major Real-World Study for Luspatercept
- ClinicalTrials.gov ID
- NCT06596642
- Acronym / sponsor ID
- CA056-1132
- Conditions
- β-Thalassemia Major
- Keywords
- β-Thalassemia major
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 61 participants (actual)
- Lead sponsor
- Bristol-Myers SquibbIndustry (pharma, biotech, devices)
- Study start
- 21 Aug 2024 (actual)
- Primary completion
- 23 Sep 2026 (actual)
- Study completion
- 23 Sep 2026 (actual)
- First posted
- 19 Sep 2024 (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 real-world study will assess the efficacy and safety of luspatercept treatment for β-thalassemia major in Taiwan as well as the impact on quality of life.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Adults participants diagnosed with β-thalassemia major | – | Drug: Luspatercept |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Adults participants in Taiwan diagnosed with β-thalassemia major and treated with luspatercept.
Key inclusion criteria (3 listed)
- Participants ≥18 years of age.
- Participants with the diagnosis of transfusion-dependent β-thalassemia major who are eligible to the treatment of luspatercept.
- Before luspatercept treatment, participant's transfusion burden ≥ 24 Red Blood Cell units in 24 weeks, without ≥35-day transfusion free.
Key exclusion criteria (3 listed)
- Hypersensitivity to the active substance or to any of the excipients.
- Pregnancy.
- Participants requiring treatment to control the growth of extramedullary hematopoiesis (EMH) masses.
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 |
|---|---|---|
| LuspaterceptAs per product label | Drug | Adults participants diagnosed with β-thalassemia major |
Primary outcome
Number of Red Blood Cell (RBC) transfusion units received by participants
Time frame: Baseline, every 3 weeks thereafter until week 96
Participant hemoglobin (Hb) level results
Time frame: Baseline, every 3 weeks thereafter until week 96
Participant serum ferritin results
Time frame: Baseline, week 12, 24, 36, 48, 60, 72, 84 and 96
25 secondary outcomes
Participant platelet level results
Time frame: Baseline, and every 3 weeks thereafter up to week 96
Participant white blood cell count results
Time frame: Baseline, and every 3 weeks thereafter up to week 96
Participant reticulocytes/normoblast count results
Time frame: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
Participant Hemoglobin F (HbF) levels
Time frame: Baseline and weeks 6 and 12
Participant uric acid level results
Time frame: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
Participant lactate dehydrogenase (LDH) level results
Time frame: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
Participant aspartate aminotransferase (AST)/alanine aminotransferase (ALT) ratio
Time frame: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
Participant creatinine level results
Time frame: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
Participant bilirubin (total and direct) laboratory values
Time frame: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
Type of iron chelating therapy prescribed to participants
Time frame: Baseline, every 3 weeks thereafter until week 96
Iron chelating therapy treatment dosage prescribed to participants
Time frame: Baseline, every 3 weeks thereafter until week 96
Participant Adverse Events (AEs)
Time frame: Baseline and every 3 weeks until week 12, then weeks 24, 36, 48, 60, 72, 84 and 96
Participant health-related quality of life as assessed by Transfusion-dependent Quality of Life (TranQoL) questionnaire
Time frame: Baseline, week 12, 24, 48, 72 and 96
Participant health-related quality of life as assessed by EQ-5D-5L questionnaire
Time frame: Baseline, week 12, 24, 48, 72 and 96
Luspatercept treatment dosage prescribed to participants
Time frame: Baseline, every 3 weeks thereafter until week 96
Duration of luspatercept treatment
Time frame: Baseline, every 3 weeks thereafter until week 96
Participant reason(s) for luspatercept treatment discontinuation as assessed by the treating clinician
Time frame: Baseline, every 3 weeks thereafter until week 96
Participant demographics
Time frame: Baseline
Participant weight
Time frame: Baseline
Participant β0/β0 genotype status
Time frame: Baseline
Participant disease status
Time frame: Baseline
Participant comorbidities
Time frame: Baseline
Participant concomitant medication
Time frame: Baseline
Participant β-thalassemia treatment history
Time frame: Baseline
Participant surgical history (splenectomy and/or cholecystectomy)
Time frame: Baseline
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Nov 2026.
- BMS Clinical Trial InformationLink provided by the sponsor.
- FDA Safety Alerts and RecallsLink provided by the sponsor.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
Bristol-Myers Squibb study director
Bristol-Myers Squibb
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of β-Thalassemia Major, run by Bristol-Myers Squibb at 5 sites in Taiwan.
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 3 inclusion and 3 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: finished, as of the sponsor's last update on 5 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Taiwan. 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Red Blood Cell (RBC) transfusion units received by participants (time frame: Baseline, every 3 weeks thereafter until week 96). The study lists 3 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 5 Nov 2026.