Clinical trial NCT06875219: To Evaluate Long- Term Safety and Efficacy of Luspatercept
Official title: An Observational Study to Evaluate Long- Term Safety and Efficacy of Luspatercept in Subjects with Transfusion Dependent Who Received the First Dose of Luspatercept After Its Introduction in the Clinical Practice
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Thalassemia Major
Lead sponsor: Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia
Sponsor class: OTHER
First posted: 2025-03-13
Enrollment: 350
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Adult (male and female), living or deceased patients with transfusion-dependent beta thalassemia who received the first dose of luspatercept after its introduction in the clinical practice.

Brief summary: An observational study to evaluate long-term safety and efficacy of luspatercept in subjects with transfusion dependent who received the first dose of luspatercept after its introduction in the clinical practice

Detailed description: Luspatercept represents the first and only erythroid maturation agent (EMA) approved by the European Commission (EC) and the Food and Drug Administration (FDA) capable of enhancing advanced erythrocyte maturation. The efficacy of luspatercept was demonstrated in the phase III clinical trial called "BELIEVE." With market authorization, the drug is used in less selected patients with potentially more complex medical histories than those enrolled in the registration study. In addition, it is now also possible to prescribe the drug in patients with blood consumption at baseline between 20 and 24 blood units in the 24 weeks before the drug was prescribed, who could not be included in the "BELIEVE" study. Gathering data on the clinical characteristics of patients who are prescribed luspatercept in normal clinical practice, and of safety and efficacy in this setting, is essential to enrich the available information on this new therapy.

Eligibility criteria: Inclusion criteria:

Subjects who received at least one dose of luspatercept after its introduction in the clinical practice

Exclusion Criteria:

* Subjects who received luspatercept after participating in the "compassionate" use program
* Subjects unwilling to sign informed consent

Primary outcomes:
measure: Safety of luspatercept - Number of participants with treatment-related adverse events as assessed by CTCAE v4.0"
timeFrame: 52 WEEK
measure: Tolerability of luspatercept - Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
timeFrame: 52 WEEK
measure: Efficacy of luspatercept - Number of subjects who showed a ≥ 33% reduction from baseline in the number of transfused blood units during any 12-week interval period of luspatercept treatment
timeFrame: 12 weeks
Secondary outcomes:
measure: safety of luspatercept - Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
timeFrame: 52 weeks
measure: tolerability of luspatercept - Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
timeFrame: 52 weeks
measure: Efficacy of luspatercept - Number of subjects who showed a ≥ 33% reduction from baseline in the number of transfused blood units during any 12-week interval period of luspatercept treatment
timeFrame: 52 weeks
measure: impact of luspatercept on iron accumulation cardiac T2*
timeFrame: 52 weeks
measure: impact of luspatercept on iron accumulation hepatic T2*
timeFrame: 52 weeks
measure: incidence of endocrinological changes
timeFrame: 52 weeks
measure: incidence of thromboembolic events
timeFrame: 52 weeks
measure: positively affect drug response.
timeFrame: 52 weeks
measure: negatively affect drug response.
timeFrame: 52 weeks
Other outcomes:
Locations:
city: Cagliari
country: Italy
facility: UniversityCagliari, OspPed Microcitemico, Cagliari
state: Italia
Study officials:
References: