To Evaluate Long- Term Safety and Efficacy of Luspatercept

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Total Enrollment

350 participants

Study Classification

OBSERVATIONAL

Study Start Date

2023-04-30

Study Completion Date

2026-02-24

Brief Summary

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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

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Detailed Description

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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

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Thalassemia Major

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Interventions

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Luspatercept (ACE-536)

Intervention Type DRUG

Eligibility Criteria

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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

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Responsible Party

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Responsibility Role SPONSOR

Locations

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UniversityCagliari, OspPed Microcitemico, Cagliari

Cagliari, Italia, Italy

Site Status

Countries

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Italy

Other Identifiers

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LUSPAREAL001

Identifier Type: -

Identifier Source: org_study_id

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