The purpose of this study is to evaluate luspatercept treatment in adults with transfusion-dependent beta-Thalassemia in the Middle East
Study Type
OBSERVATIONAL
Enrollment
200
According to the product label
Sultan Qaboos University Hospital
Seeb, Muḩāfaz̧at Masqaţ, Oman
RECRUITINGPrince Muhammad bin Nasser Hospital
Jizan, Saudi Arabia
NOT_YET_RECRUITINGChange in transfusion burden
Time frame: Baseline and up to week 144
Change in mean pre-transfusion hemoglobin level
Time frame: Baseline and up to week 144
Change in transfusion-related visits
Time frame: Baseline and up to week 144
Proportion of participants achieving ≥33% reduction in red blood cell (RBC) transfusion burden (number of RBC units transfused) plus a reduction of ≥2 units
Time frame: Baseline and up to week 144
Proportion of participants achieving ≥50% reduction in red blood cell transfusion burden plus a reduction of ≥2 units
Time frame: Baseline and up to week 144
Time from first luspatercept dosing date to the first erythroid response
Time frame: Baseline and up to week 144
Time from the date the erythroid response is first observed until the last day of response
Time frame: Baseline and up to week 144
Participant pretransfusion hemoglobin level
Time frame: Baseline and up to week 144
Participant baseline transfusion burden
Time frame: Baseline
Proportion of participants without red blood cell transfusion during any consecutive 12-week or 24-week treatment period
Time frame: Baseline and up to week 144
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Time from first luspatercept dosing date to first onset of red blood cell-transfusion independence ≥12 weeks and ≥24 weeks
Time frame: Baseline and up to week 144
Time from the date a 12-week and 24-week red blood cell-transfusion independence is first observed until the date the participant has a subsequently documented red blood cell transfusion
Time frame: Baseline and up to week 144
Change in mean serum ferritin (SF) level
Time frame: Baseline and up to week 144
Change in proportion of participants with mean serum ferritin <1,000, 1000-2500, and >2,500 μg/L
Time frame: Baseline and up to week 144
Types (drug formulation - mono and combination therapy) of iron chelation therapy received
Time frame: Baseline and up to week 144
Change in mean daily dose of iron chelation therapy from baseline
Time frame: Baseline and up to week 144
Number of medical encounters (inpatient hospitalizations, emergency department attendances, hospital outpatient visits, visits at office-based physicians)
Time frame: Baseline and up to week 144
Inpatient length of stay
Time frame: Baseline and up to week 144
Proportion of participants remaining on luspatercept treatment
Time frame: Up to week 144
Length of time from initiation to discontinuation of luspatercept treatment
Time frame: Up to week 144
Frequencies of reasons for discontinuation of luspatercept treatment
Time frame: Up to week 144
Participant sociodemographics
Sociodemographics of participants such as age, sex, ethnicity, country of treatment, height, weight, and body mass index
Time frame: Baseline
Participant disease characteristics
Disease characteristics of participants, describing age of diagnosis of β-thalassemia, genotype (β0/β0, non-β0/β0), splenectomy status (yes/no), pretransfusion hemoglobin level (mean of all documented), transfusion burden (total number of red blood cell units transfused).
Time frame: Baseline
Participant comorbidities
Disease- and non-disease-related comorbid conditions
Time frame: Baseline
Concomitant treatment(s) received
Disease-related best supportive care (BSC) and treatments for other comorbidities.
Time frame: Baseline
Number of luspatercept doses administered
Time frame: Up to week 144
Number of luspatercept dose modifications
Time frame: Up to week 144
Frequencies of reasons for luspatercept dose modifications
Time frame: Up to week 144