Clinical trial NCT06913634: 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
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
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Thalassemia Major
Lead sponsor: Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia
Sponsor class: OTHER
First posted: 2025-04-06
Enrollment: 140
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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.

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.

Eligibility criteria: Inclusion criteria:

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

Exclusion Criteria:

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

Primary outcomes:
measure: To evaluate safety of luspatercept, number of participants with treatment-related adverse events as assessed by CTCAE v4.0
timeFrame: 12-week interval period of luspatercept treatment.
measure: To evaluate tolerability of luspatercept, number of participants with treatment-related adverse events as assessed by CTCAE v4.0"
timeFrame: 12-week interval period of luspatercept treatment.
measure: 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
timeFrame: 12-week interval period of luspatercept treatment.
Secondary outcomes:
measure: subjects with comorbidities, number of participants with treatment-related adverse events as assessed by CTCAE v4.0"
timeFrame: 12-week interval period of luspatercept treatment.
measure: subjects with comorbidities, number of participants with treatment-related adverse events as assessed by CTCAE v4.0"
timeFrame: 12-week interval period of luspatercept treatment.
Other outcomes:
measure: endocrinological changes
timeFrame: 52-week interval period of luspatercept treatment.
measure: thromboembolic events
timeFrame: 52-week interval period of luspatercept treatment.
measure: factors positively affect drug response
timeFrame: 52-week interval period of luspatercept treatment.
measure: factors negatively affect drug response
timeFrame: 52-week interval period of luspatercept treatment.
measure: become transfusion independent while taking the drug
timeFrame: 52-week interval period of luspatercept treatment.
Locations:
city: Cagliari
country: Italy
facility: University of Cagliari, Ospedale Pediatrico Microcitemico, via Jenner sn, 09121 Cagliari -
state: CA
Study officials:
affiliation: UNIVERSITA' DI CAGLIARI
name: RAFFAELLA ORIGA
role: PRINCIPAL_INVESTIGATOR
References: