About this trial
The purpose of this study is to evaluate luspatercept treatment in adults with transfusion-dependent beta-Thalassemia in the Middle East
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Male or female participants of any race aged at least 18 years at time of initiation of luspatercept treatment
Participants with documented diagnosis of transfusion-dependent β-thalassemia (TDT).
Participants who have been initiated on treatment with luspatercept as per the product's Summary of Product Characteristics (SmPC) no longer than 12 months prior to informed consent signature, and for whom therapy is ongoing.
Participants for whom the decision to prescribe luspatercept treatment is clearly separated from the physician's decision to include the participant in the current study.
Disqualifiers
Participants that meet any of the contraindications to the administration of luspatercept as outlined in the latest version of the locally approved SmPC.
Participants who are currently receiving or are planned to receive treatment with any investigational drug/device/intervention or who have received any investigational product within 1 month or 5 half-lives of the investigational agent (whichever is longer) prior to luspatercept therapy initiation.
Participants who are currently pregnant, breastfeeding, or planning a pregnancy during the study observation period.
Participants who have not provided signed informed consent for participating in the study and for collecting and analysing medical data pertinent to the objectives of this study.
Trial population
The study population will include adult patients with transfusion-dependent β-thalassemia (TDT) in the Middle East region who have initiated luspatercept treatment
Trial design
Cohort
Other
Treatments tested in this trial
Luspatercept
DrugAccording to the product label
Treatment groups
Trial outcomes
Primary outcomes
Change in transfusion burden
Change in mean pre-transfusion hemoglobin level
Secondary outcomes
Change in transfusion-related visits
Proportion of participants achieving ≥33% reduction in red blood cell (RBC) transfusion burden (number of RBC units transfused) plus a reduction of ≥2 units
Proportion of participants achieving ≥50% reduction in red blood cell transfusion burden plus a reduction of ≥2 units
Time from first luspatercept dosing date to the first erythroid response
Sponsors and contacts
Click on the lead sponsor to view all of their trials.