Thalassemia MajorLUSPA001NCT06913634
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
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
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. 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
- 140 participants
- Where
- 1 siteItaly
- Study dates
- 30 May 2023to 30 Jun 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 at least one dose of luspatercept during the compassionate use program.
Who may be able to join
- Subjects who received at least one dose of luspatercept in the compassionate phase
Who cannot join
- Subjects who are not willing or able to sign the informed consent.
Trial locations and contacts
1 site in 1 country.
Italy1 site
University of Cagliari, Ospedale Pediatrico Microcitemico, via Jenner sn, 09121 Cagliari -
Cagliari, CA, 09121
About this study
Official title: Observational 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
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.
Read the detailed description
One month after the publication in the Official Gazette of the Italian Republic of the AIFA determination of price and reimbursement for the treatment indication covered by the program, those who were still on treatment and were, in clinical judgment, eligible to continue it, continued to receive the drug through dispensation by the NHS.
Because access to the compassionate phase was less restrictive than access to that in formal clinical trials and the patients included particularly representative of real life, collecting data on clinical characteristics at baseline, safety, and efficacy is critical to enriching the information available on this new therapy. Continuing to collect efficacy, safety, and tolerability information beyond the compassionate phase is also essential for a more complete and accurate assessment, given the short duration of 'compassionate' treatment for a portion of the patients under study.
- 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.
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.
Measured: 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.
Measured: 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.
Measured: 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"
To evaluate safety of luspatercept in subjects with comorbidities that were exclusion criteria in formal clinical trials
Measured: 12-week interval period of luspatercept treatment.
subjects with comorbidities, number of participants with treatment-related adverse events as assessed by CTCAE v4.0"
To evaluate tolerability of luspatercept in subjects with comorbidities that were exclusion criteria in formal clinical trials
Measured: 12-week interval period of luspatercept treatment.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Apr 2025
- Study start30 May 2023
- Primary completion30 May 2025 (estimated)
- Study completion30 Jun 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
- 140 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- RAFFAELLA ORIGA, Principal investigator, UNIVERSITA' DI CAGLIARI
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06913634
- Sponsor's protocol ID
- LUSPA001
- Acronym
- LUSPA001
- Status last verified
- Feb 2025
- First submitted
- 25 Feb 2025
- Last updated
- 6 Apr 2025
- Results posted
- No
- Data monitoring committee
- Yes
- 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 6 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Observational 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
- ClinicalTrials.gov ID
- NCT06913634
- Acronym / sponsor ID
- LUSPA001 / LUSPA001
- Conditions
- Thalassemia Major
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 140 participants (estimated)
- Lead sponsor
- Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in ItaliaUniversity, hospital or other
- Study start
- 30 May 2023 (actual)
- Primary completion
- 30 May 2025 (estimated)
- Study completion
- 30 Jun 2026 (estimated)
- First posted
- 6 Apr 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.
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.
Detailed description
One month after the publication in the Official Gazette of the Italian Republic of the AIFA determination of price and reimbursement for the treatment indication covered by the program, those who were still on treatment and were, in clinical judgment, eligible to continue it, continued to receive the drug through dispensation by the NHS.
Because access to the compassionate phase was less restrictive than access to that in formal clinical trials and the patients included particularly representative of real life, collecting data on clinical characteristics at baseline, safety, and efficacy is critical to enriching the information available on this new therapy. Continuing to collect efficacy, safety, and tolerability information beyond the compassionate phase is also essential for a more complete and accurate assessment, given the short duration of 'compassionate' treatment for a portion of the patients under study.
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 at least one dose of luspatercept during the compassionate use program.
Key inclusion criteria (1 listed)
- Subjects who received at least one dose of luspatercept in the compassionate phase
Key exclusion criteria (1 listed)
- Subjects who are not willing or able to sign the 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
To evaluate safety of luspatercept, number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Time frame: 12-week interval period of luspatercept treatment.
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.
To evaluate tolerability of luspatercept, number of participants with treatment-related adverse events as assessed by CTCAE v4.0"
Time frame: 12-week interval period of luspatercept treatment.
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.
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
Time frame: 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.
2 secondary outcomes
subjects with comorbidities, number of participants with treatment-related adverse events as assessed by CTCAE v4.0"
Time frame: 12-week interval period of luspatercept treatment.
subjects with comorbidities, number of participants with treatment-related adverse events as assessed by CTCAE v4.0"
Time frame: 12-week interval period of luspatercept treatment.
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 6 Apr 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.
RAFFAELLA ORIGA principal investigator
UNIVERSITA' DI CAGLIARI
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 1 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 6 Apr 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?
To evaluate safety of luspatercept, number of participants with treatment-related adverse events as assessed by CTCAE v4.0 (time frame: 12-week interval period of luspatercept treatment.). 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 6 Apr 2025.