NCT06875219

Brief Summary

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

Trial Health

55
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
350

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Apr 2023

Typical duration for all trials

Geographic Reach
1 country

1 active site

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

April 30, 2023

Completed
1.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 24, 2025

Completed
1 day until next milestone

First Submitted

Initial submission to the registry

February 25, 2025

Completed
16 days until next milestone

First Posted

Study publicly available on registry

March 13, 2025

Completed
12 months until next milestone

Study Completion

Last participant's last visit for all outcomes

February 24, 2026

Completed
Last Updated

March 13, 2025

Status Verified

February 1, 2025

Enrollment Period

1.8 years

First QC Date

February 25, 2025

Last Update Submit

March 10, 2025

Conditions

Thalassemia Major

Outcome Measures

Primary Outcomes (3)

  • 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 Outcomes (9)

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

    52 weeks

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

    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

    52 weeks

  • impact of luspatercept on iron accumulation cardiac T2*

    52 weeks

  • impact of luspatercept on iron accumulation hepatic T2*

    52 weeks

  • +4 more secondary outcomes

Interventions

Luspatercept (ACE-536)DRUG

Luspatercept (ACE-536)

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-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.

You may qualify if:

  • Subjects who received at least one dose of luspatercept after its introduction in the clinical practice

You may not qualify if:

  • Subjects who received luspatercept after participating in the "compassionate" use program
  • Subjects unwilling to sign informed consent

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

UniversityCagliari, OspPed Microcitemico, Cagliari

Cagliari, Italia, 09121, Italy

Location

MeSH Terms

Conditions

beta-Thalassemia

Interventions

luspatercept

Condition Hierarchy (Ancestors)

ThalassemiaAnemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

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

March 13, 2025

Study Start

April 30, 2023

Primary Completion

February 24, 2025

Study Completion

February 24, 2026

Last Updated

March 13, 2025

Record last verified: 2025-02

Data Sharing

IPD Sharing
Will not share

Locations

IT(1)