Clinical trial NCT06596642: A Real-World Study of β-Thalassemia Major Treatment With Luspatercept in Taiwan
Official title: Taiwan β-Thalassemia Major Real-World Study for Luspatercept
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: β-Thalassemia Major
Keywords: β-Thalassemia major
Lead sponsor: Bristol-Myers Squibb
Sponsor class: INDUSTRY
First posted: 2024-09-19
Enrollment: 61
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Adults participants in Taiwan diagnosed with β-thalassemia major and treated with luspatercept.

Brief 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.


Eligibility criteria: 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.

Primary outcomes:
measure: Number of Red Blood Cell (RBC) transfusion units received by participants
timeFrame: Baseline, every 3 weeks thereafter until week 96
measure: Participant hemoglobin (Hb) level results
timeFrame: Baseline, every 3 weeks thereafter until week 96
measure: Participant serum ferritin results
timeFrame: Baseline, week 12, 24, 36, 48, 60, 72, 84 and 96
Secondary outcomes:
measure: Participant platelet level results
timeFrame: Baseline, and every 3 weeks thereafter up to week 96
measure: Participant white blood cell count results
timeFrame: Baseline, and every 3 weeks thereafter up to week 96
measure: Participant reticulocytes/normoblast count results
timeFrame: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
measure: Participant Hemoglobin F (HbF) levels
timeFrame: Baseline and weeks 6 and 12
measure: Participant uric acid level results
timeFrame: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
measure: Participant lactate dehydrogenase (LDH) level results
timeFrame: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
measure: Participant aspartate aminotransferase (AST)/alanine aminotransferase (ALT) ratio
timeFrame: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
measure: Participant creatinine level results
timeFrame: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
measure: Participant bilirubin (total and direct) laboratory values
timeFrame: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
measure: Type of iron chelating therapy prescribed to participants
timeFrame: Baseline, every 3 weeks thereafter until week 96
measure: Iron chelating therapy treatment dosage prescribed to participants
timeFrame: Baseline, every 3 weeks thereafter until week 96
measure: Participant Adverse Events (AEs)
timeFrame: Baseline and every 3 weeks until week 12, then weeks 24, 36, 48, 60, 72, 84 and 96
measure: Participant health-related quality of life as assessed by Transfusion-dependent Quality of Life (TranQoL) questionnaire
timeFrame: Baseline, week 12, 24, 48, 72 and 96
measure: Participant health-related quality of life as assessed by EQ-5D-5L questionnaire
timeFrame: Baseline, week 12, 24, 48, 72 and 96
measure: Luspatercept treatment dosage prescribed to participants
timeFrame: Baseline, every 3 weeks thereafter until week 96
measure: Duration of luspatercept treatment
timeFrame: Baseline, every 3 weeks thereafter until week 96
measure: Participant reason(s) for luspatercept treatment discontinuation as assessed by the treating clinician
timeFrame: Baseline, every 3 weeks thereafter until week 96
measure: Participant demographics
timeFrame: Baseline
measure: Participant weight
timeFrame: Baseline
measure: Participant β0/β0 genotype status
timeFrame: Baseline
measure: Participant disease status
timeFrame: Baseline
measure: Participant comorbidities
timeFrame: Baseline
measure: Participant concomitant medication
timeFrame: Baseline
measure: Participant β-thalassemia treatment history
timeFrame: Baseline
measure: Participant surgical history (splenectomy and/or cholecystectomy)
timeFrame: Baseline
Other outcomes:
Locations:
city: Kaohsiung
country: Taiwan
facility: Local Institution - 02
city: Tainan
country: Taiwan
facility: Local Institution - 04
city: Taipei
country: Taiwan
facility: Local Institution - 01
city: Taipei
country: Taiwan
facility: Local Institution - 03
city: Taoyuan
country: Taiwan
facility: Local Institution - 05
Study officials:
affiliation: Bristol-Myers Squibb
name: Bristol-Myers Squibb
role: STUDY_DIRECTOR
References: