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 | Active Not Recruiting DMC

• 1 other identifier: LUSPA001
• Study Type: observational
• Target: 140
• Locations: 1 country
• Sites: 1

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.

Conditions

Thalassemia Major

Outcome Measures

Primary Outcomes (3)

• To evaluate 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 at least one dose of the drug in the compassionate use phase including the post- marketing authorisation phase in those who continued to receive it.

  12-week interval period of luspatercept treatment.

• To evaluate 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 at least one dose of the drug in the compassionate use phase including the post- marketing authorisation phase in those who continued to receive it.

  12-week interval period of luspatercept treatment.

• To evaluate 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 at least one dose of the drug in the compassionate use phase including the post- marketing authorisation phase in those who continued to receive it.

  12-week interval period of luspatercept treatment.

Secondary Outcomes (2)

• subjects with comorbidities, number of participants with treatment-related adverse events as assessed by CTCAE v4.0"

  12-week interval period of luspatercept treatment.

• subjects with comorbidities, number of participants with treatment-related adverse events as assessed by CTCAE v4.0"

  12-week interval period of luspatercept treatment.

Other Outcomes (5)

• endocrinological changes

  52-week interval period of luspatercept treatment.

• thromboembolic events

  52-week interval period of luspatercept treatment.

• factors positively affect drug response

  52-week interval period of luspatercept treatment.

Interventions

Luspatercept (ACE-536) DRUG

Luspatercept (ACE-536)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Adult (male and female), living or deceased patients with transfusion-dependent beta thalassemia who received at least one dose of luspatercept during the compassionate use program.

You may qualify if:

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

You may not qualify if:

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

Sponsors & Collaborators

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

Study Sites (1)

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

Cagliari, CA, 09121, Italy

Location

MeSH Terms

Conditions

beta-Thalassemia

Interventions

luspatercept

Condition Hierarchy (Ancestors)

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

Study Officials

• RAFFAELLA ORIGA

  UNIVERSITA' DI CAGLIARI

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 25, 2025
• First Posted: April 6, 2025
• Study Start: May 30, 2023
• Primary Completion: May 30, 2025
• Study Completion (Estimated): June 30, 2026
• Last Updated: April 6, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

IT (1)