NCT06875219

To Evaluate Long- Term Safety and Efficacy of Luspatercept

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

• Status: Active Not Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 350 (actual)
• Sponsor: Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Thalassemia Major

Interventions

Type	Name	Description
DRUG	Luspatercept (ACE-536)	Luspatercept (ACE-536)

Timeline

• Start date: 2023-04-30
• Primary completion: 2025-02-24
• Completion: 2026-02-24
• First posted: 2025-03-13
• Last updated: 2025-03-13

Locations

1 site across 1 country: Italy