Thalassemia MajorLUSPAREAL001NCT06875219
To Evaluate Long- Term Safety and Efficacy of Luspatercept
An observational study of Thalassemia Major, run by Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia at 1 site in Italy.
Trial 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 Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Thalassemia Major
- What is being tested?
- Luspatercept (ACE-536)
- How many people
- 350 participants
- Where
- 1 siteItaly
- Study dates
- 30 Apr 2023to 24 Feb 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
Who may be able to join
- Subjects who received at least one dose of luspatercept after its introduction in the clinical practice
Who cannot join
- Subjects who received luspatercept after participating in the "compassionate" use program
- Subjects unwilling to sign informed consent
Trial locations and contacts
1 site in 1 country.
Italy1 site
UniversityCagliari, OspPed Microcitemico, Cagliari
Cagliari, Italia, 09121
About this study
Official 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
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
Read the 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.
- Conditions
- Thalassemia Major
- Being tested
- Luspatercept (ACE-536)
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Luspatercept (ACE-536)Drug
Luspatercept (ACE-536)
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Measured: 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
Measured: 12 weeks
Secondary outcomes (9)
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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 52 weeks
incidence of endocrinological changes
- To evaluate the incidence of endocrinological changes in patients treated with luspatercept
Measured: 52 weeks
incidence of thromboembolic events
- To assess the incidence of thromboembolic events in this category of patients
Measured: 52 weeks
positively affect drug response.
- To identify the factors that positively affect drug response.
Measured: 52 weeks
negatively affect drug response.
- To identify the factors that negatively affect drug response.
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Mar 2025
- Study start30 Apr 2023
- Primary completion24 Feb 2025
- Study completion24 Feb 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thalassemia MajorStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 350 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06875219
- Sponsor's protocol ID
- LUSPAREAL001
- Acronym
- LUSPAREAL001
- Status last verified
- Feb 2025
- First submitted
- 25 Feb 2025
- Last updated
- 13 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT06875219
- Acronym / sponsor ID
- LUSPAREAL001 / LUSPAREAL001
- Conditions
- Thalassemia Major
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 350 participants (actual)
- Lead sponsor
- Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in ItaliaUniversity, hospital or other
- Study start
- 30 Apr 2023 (actual)
- Primary completion
- 24 Feb 2025 (actual)
- Study completion
- 24 Feb 2026 (estimated)
- First posted
- 13 Mar 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (1 listed)
- Subjects who received at least one dose of luspatercept after its introduction in the clinical practice
Key exclusion criteria (2 listed)
- Subjects who received luspatercept after participating in the "compassionate" use program
- Subjects unwilling to sign informed consent
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Luspatercept (ACE-536)Luspatercept (ACE-536) | Drug | – |
Primary outcome
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
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
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
9 secondary outcomes
safety of luspatercept - Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Time frame: 52 weeks
tolerability of luspatercept - Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Time frame: 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
Time frame: 52 weeks
impact of luspatercept on iron accumulation cardiac T2*
Time frame: 52 weeks
impact of luspatercept on iron accumulation hepatic T2*
Time frame: 52 weeks
incidence of endocrinological changes
Time frame: 52 weeks
incidence of thromboembolic events
Time frame: 52 weeks
positively affect drug response.
Time frame: 52 weeks
negatively affect drug response.
Time frame: 52 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Mar 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Thalassemia Major, run by Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia at 1 site in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 13 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety of luspatercept - Number of participants with treatment-related adverse events as assessed by CTCAE v4.0" (time frame: 52 WEEK). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Mar 2025.