To Evaluate Long- Term Safety and Efficacy of Luspatercept (LUSPAREAL001)

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

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

Study Overview

• Status: Active, not recruiting
• Conditions: Thalassemia Major
• Intervention / Treatment: Drug: Luspatercept (ACE-536)

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.

• Study Type: Observational
• Enrollment (Actual): 350

Contacts and Locations

Study Locations

• Italy
  • Italia
    • Cagliari, Italia, Italy, 09121
      • UniversityCagliari, OspPed Microcitemico, Cagliari

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Adult (male and female), living or deceased patients with transfusion-dependent beta thalassemia who received the first dose of luspatercept after its introduction in the clinical practice.

Description

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

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety of luspatercept - Number of participants with treatment-related adverse events as assessed by CTCAE v4.0"	- To evaluate safety of luspatercept in subjects with transfusion-dependent beta thalassemia who received the first dose of luspatercept after its introduction in the clinical practice	52 WEEK
Tolerability of luspatercept - Number of participants with treatment-related adverse events as assessed by CTCAE v4.0	- To evaluate tolerability of luspatercept in subjects with transfusion-dependent beta thalassemia who received the first dose of luspatercept after its introduction in the clinical practice	52 WEEK
Efficacy of luspatercept - Number of subjects who showed a ≥ 33% reduction from baseline in the number of transfused blood units during any 12-week interval period of luspatercept treatment	- To evaluate efficacy of luspatercept in subjects with transfusion-dependent beta thalassemia who received the first dose of luspatercept after its introduction in the clinical practice	12 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
safety of luspatercept - Number of participants with treatment-related adverse events as assessed by CTCAE v4.0	- To evaluate safety of luspatercept in subjects with transfusion-dependent beta thalassemia with baseline transfusion needs and comorbidities that were exclusion criteria in formal clinical trials	52 weeks
tolerability of luspatercept - Number of participants with treatment-related adverse events as assessed by CTCAE v4.0	- To evaluate tolerability of luspatercept in subjects with transfusion-dependent beta thalassemia with baseline transfusion needs and comorbidities that were exclusion criteria in formal clinical trials	52 weeks
Efficacy of luspatercept - Number of subjects who showed a ≥ 33% reduction from baseline in the number of transfused blood units during any 12-week interval period of luspatercept treatment	- To evaluate efficacy of luspatercept in subjects with transfusion-dependent beta thalassemia with baseline transfusion needs and comorbidities that were exclusion criteria in formal clinical trials	52 weeks
impact of luspatercept on iron accumulation cardiac T2*	- To increase available data on the impact of luspatercept on iron accumulation by assessing the change in serum ferritin and cardiac T2* value on nuclear magnetic resonance imaging	52 weeks
impact of luspatercept on iron accumulation hepatic T2*	- To increase available data on the impact of luspatercept on iron accumulation by assessing the change in serum ferritin and hepatic T2* value on nuclear magnetic resonance imaging	52 weeks
incidence of endocrinological changes	- To evaluate the incidence of endocrinological changes in patients treated with luspatercept	52 weeks
incidence of thromboembolic events	- To assess the incidence of thromboembolic events in this category of patients	52 weeks
positively affect drug response.	- To identify the factors that positively affect drug response.	52 weeks
negatively affect drug response.	- To identify the factors that negatively affect drug response.	52 weeks

Collaborators and Investigators

• Sponsor: Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia

Study record dates

Study Major Dates

• Study Start (Actual): April 30, 2023
• Primary Completion (Actual): February 24, 2025
• Study Completion (Estimated): February 24, 2026

Study Registration Dates

• First Submitted: February 25, 2025
• First Submitted That Met QC Criteria: March 10, 2025
• First Posted (Actual): March 25, 2025

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: March 10, 2025
• Last Verified: February 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hemoglobinopathies; Thalassemia; beta-Thalassemia; Hematinics; Luspatercept
• Other Study ID Numbers: LUSPAREAL001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No