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

NCT ID: NCT06913634

Last Updated: 2025-04-06

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Total Enrollment: 140 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2023-05-30
• Study Completion Date: 2026-06-30

Brief 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.

Detailed Description

One month after the publication in the Official Gazette of the Italian Republic of the AIFA determination of price and reimbursement for the treatment indication covered by the program, those who were still on treatment and were, in clinical judgment, eligible to continue it, continued to receive the drug through dispensation by the NHS.

Because access to the compassionate phase was less restrictive than access to that in formal clinical trials and the patients included particularly representative of real life, collecting data on clinical characteristics at baseline, safety, and efficacy is critical to enriching the information available on this new therapy. Continuing to collect efficacy, safety, and tolerability information beyond the compassionate phase is also essential for a more complete and accurate assessment, given the short duration of 'compassionate' treatment for a portion of the patients under study.

Conditions

Thalassemia Major

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Interventions

Luspatercept (ACE-536)

Luspatercept (ACE-536)

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Subjects who received at least one dose of luspatercept in the compassionate phase

Exclusion Criteria

• Subjects who are not willing or able to sign the informed consent.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

RAFFAELLA ORIGA

Role: PRINCIPAL_INVESTIGATOR

UNIVERSITA' DI CAGLIARI

Locations

University of Cagliari, Ospedale Pediatrico Microcitemico, via Jenner sn, 09121 Cagliari -

Cagliari, CA, Italy

Countries

Italy

Other Identifiers

LUSPA001

Identifier Type: -

Identifier Source: org_study_id