Study to Evaluate the Long- Term Safety and Efficacy of Luspatercept in Subjects Who Received at Least One Dose of Luspatercept in the Compassionate Use Phase

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." More than 200 Italian patients with transfusion- dependent beta thalassemia aged ≥18 years who had no approved therapeutic alternatives to improve their clinical course were considered eligible for the 'compassionate' use program related to luspatercept and most of them received at least one dose of the drug before it was dispensed by the National Health System (NHS), after approval by the pharmaceutical company and the Ethics Committee of the Clinical Center in which they were being followed.

NCT Number	NCT06913634
Lead Sponsor	Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia
Conditions	Thalassemia Major
Enrollment	140 participants
Start Date	2023-05-30
Primary Completion	2025-05-30 (estimated)
Study Completion	2026-06-30 (estimated)
Updated on ClinicalTrials.gov	2025-04-06