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Active, Not Recruiting

NCT Number: NCT06875219

To Evaluate Long- Term Safety and Efficacy of Luspatercept

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

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Thalassemia Major Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Thalassemia beta-Thalassemia

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

UniversityCagliari, OspPed Microcitemico, Cagliari

Cagliari, Italia, 09121, Italy

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Luspatercept (ACE-536)

Drug

Luspatercept (ACE-536)

Primary outcomes

  1. Safety of luspatercept - Number of participants with treatment-related adverse events as assessed by CTCAE v4.0"

    Time frame: 52 WEEK

    • 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

  2. Tolerability of luspatercept - Number of participants with treatment-related adverse events as assessed by CTCAE v4.0

    Time frame: 52 WEEK

    • 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

  3. 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

    Time frame: 12 weeks

    • 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

Secondary outcomes

  1. safety of luspatercept - Number of participants with treatment-related adverse events as assessed by CTCAE v4.0

    Time frame: 52 weeks

    • 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

  2. tolerability of luspatercept - Number of participants with treatment-related adverse events as assessed by CTCAE v4.0

    Time frame: 52 weeks

    • 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

  3. 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

    Time frame: 52 weeks

    • 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

  4. impact of luspatercept on iron accumulation cardiac T2*

    Time frame: 52 weeks

    • 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

  5. impact of luspatercept on iron accumulation hepatic T2*

    Time frame: 52 weeks

    • 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

  6. incidence of endocrinological changes

    Time frame: 52 weeks

    • To evaluate the incidence of endocrinological changes in patients treated with luspatercept

  7. incidence of thromboembolic events

    Time frame: 52 weeks

    • To assess the incidence of thromboembolic events in this category of patients

  8. positively affect drug response.

    Time frame: 52 weeks

    • To identify the factors that positively affect drug response.

  9. negatively affect drug response.

    Time frame: 52 weeks

    • To identify the factors that negatively affect drug response.

Sponsors and collaborators

Lead sponsor

Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia

Other

Registry information

Official study title

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

Acronym: LUSPAREAL001

Important dates

Study start
2023
Primary completion
2025
Study completion
2026
First posted
Mar 13, 2025
Registry last updated
Mar 13, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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