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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

NCT06913634 · Status: ACTIVE_NOT_RECRUITING · Type: OBSERVATIONAL · Enrollment: 140

Last updated 2025-04-06

No results posted yet for this study

Summary

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.

Conditions

  • Thalassemia Major

Interventions

DRUG

Luspatercept (ACE-536)

Luspatercept (ACE-536)

Sponsors & Collaborators

  • Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia

    lead OTHER

Principal Investigators

  • RAFFAELLA ORIGA · UNIVERSITA' DI CAGLIARI

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-05-30
Primary Completion
2025-05-30
Completion
2026-06-30

Countries

  • Italy

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06913634 on ClinicalTrials.gov