A Real-World Study of β-Thalassemia Major Treatment With Luspatercept in Taiwan

June 3, 2025 updated by: Bristol-Myers Squibb

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
61

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.
  • Name: BMS Study Connect Contact Center www.BMSStudyConnect.com
  • Phone Number: 855-907-3286
  • Email: Clinical.Trials@bms.com

Study Contact Backup

  • Name: First line of the email MUST contain NCT # and Site #.

Study Locations

  • Taiwan
      • Kaohsiung, Taiwan, 807
        • Local Institution - 02
      • Tainan, Taiwan, 704
        • Local Institution - 04
      • Taipei, Taiwan, 100229
        • Local Institution - 01
      • Taipei, Taiwan, 10449
        • Local Institution - 03
      • Taoyuan, Taiwan, 333
        • Local Institution - 05

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Adults participants in Taiwan diagnosed with β-thalassemia major and treated with luspatercept.

Description

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • Hypersensitivity to the active substance or to any of the excipients.
  • Pregnancy.
  • Participants requiring treatment to control the growth of extramedullary hematopoiesis (EMH) masses.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Adults participants diagnosed with β-thalassemia major
Drug: Luspatercept
As per product label

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Number of Red Blood Cell (RBC) transfusion units received by participants
Time Frame: Baseline, every 3 weeks thereafter until week 96
Baseline, every 3 weeks thereafter until week 96
Participant hemoglobin (Hb) level results
Time Frame: Baseline, every 3 weeks thereafter until week 96
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
Baseline, week 12, 24, 36, 48, 60, 72, 84 and 96

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Participant weight
Time Frame: Baseline
Baseline
Participant comorbidities
Time Frame: Baseline
Baseline
Participant demographics
Time Frame: Baseline
Baseline
Participant platelet level results
Time Frame: Baseline, and every 3 weeks thereafter up to week 96
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
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
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
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
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
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
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
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
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
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
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
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
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
Baseline, week 12, 24, 48, 72 and 96
Luspatercept treatment dosage prescribed to participants
Time Frame: Baseline, every 3 weeks thereafter until week 96
Baseline, every 3 weeks thereafter until week 96
Duration of luspatercept treatment
Time Frame: Baseline, every 3 weeks thereafter until week 96
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
Baseline, every 3 weeks thereafter until week 96
Participant β0/β0 genotype status
Time Frame: Baseline
Baseline
Participant disease status
Time Frame: Baseline
Baseline
Participant concomitant medication
Time Frame: Baseline
Baseline
Participant β-thalassemia treatment history
Time Frame: Baseline
Baseline
Participant surgical history (splenectomy and/or cholecystectomy)
Time Frame: Baseline
Baseline

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Bristol-Myers Squibb

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Bristol-Myers Squibb, Bristol-Myers Squibb

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
August 21, 2024

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
October 1, 2026

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 17, 2026

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
July 29, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 17, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
September 19, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
June 4, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 3, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
June 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CA056-1132

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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