Ignite Creation Date:
2025-12-25 @ 2:30 AM
Ignite Modification Date:
2025-12-26 @ 5:08 PM
Study NCT ID:
NCT06913634
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-04-06
First Post:
2025-02-25
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
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
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D017086', 'term': 'beta-Thalassemia'}], 'ancestors': [{'id': 'D013789', 'term': 'Thalassemia'}, {'id': 'D000745', 'term': 'Anemia, Hemolytic, Congenital'}, {'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D006453', 'term': 'Hemoglobinopathies'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000621232', 'term': 'luspatercept'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 140}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2023-05-30', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-02', 'completionDateStruct': {'date': '2026-06-30', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-04-03', 'studyFirstSubmitDate': '2025-02-25', 'studyFirstSubmitQcDate': '2025-04-03', 'lastUpdatePostDateStruct': {'date': '2025-04-06', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2025-04-06', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-05-30', 'type': 'ESTIMATED'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'endocrinological changes', 'timeFrame': '52-week interval period of luspatercept treatment.', 'description': 'Number of subjects who developed endocrinologicalchanges (alterations in frequency and duration of menstrual cycle, alterations in sexual function in men, thyroid dysfunction etc )'}, {'measure': 'thromboembolic events', 'timeFrame': '52-week interval period of luspatercept treatment.', 'description': 'New cases of thromboembolic events'}, {'measure': 'factors positively affect drug response', 'timeFrame': '52-week interval period of luspatercept treatment.', 'description': 'This outcome will assess the clinical, haematological, and biochemical factors that are positively associated with a favourable drug response to luspatercept, defined as a ≥33% reduction in transfusion burden sustained for at least 12 consecutive weeks during the 52-week treatment period.\n\nFactors evaluated will include:\n\n* Baseline haemoglobin level\n* Serum erythropoietin concentration\n* Reticulocyte count\n* Baseline transfusion burden'}, {'measure': 'factors negatively affect drug response', 'timeFrame': '52-week interval period of luspatercept treatment.', 'description': 'This outcome will evaluate clinical, haematological, biochemical, or genetic factors that are \\*\\*associated with a poor or absent response\\*\\* to luspatercept treatment.\n\nNon-response will be defined as failure to achieve a ≥33% reduction in transfusion burden for at least 12 consecutive weeks during the 52-week treatment period.\n\nThe following factors will be assessed as potentially associated with a negative response to treatment:\n\nHigh baseline transfusion burden Elevated baseline serum erythropoietin levels Lack of reticulocyte increase during treatment Presence of chronic comorbidities (e.g., liver disease, endocrine disorders)'}, {'measure': 'become transfusion independent while taking the drug', 'timeFrame': '52-week interval period of luspatercept treatment.', 'description': 'Number of subjects who have not had red blood cell transfusions for at least eight consecutive weeks during treatment'}], 'primaryOutcomes': [{'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.', 'description': '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.'}, {'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.', 'description': '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.'}, {'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.', 'description': '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.'}], 'secondaryOutcomes': [{'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.', 'description': 'To evaluate safety of luspatercept in subjects with comorbidities that were exclusion criteria in formal clinical trials'}, {'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.', 'description': 'To evaluate tolerability of luspatercept in subjects with comorbidities that were exclusion criteria in formal clinical trials'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Thalassemia Major']}, 'descriptionModule': {'briefSummary': '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.\n\nThe 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.', 'detailedDescription': "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.\n\nBecause 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."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': '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.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion criteria:\n\n\\- Subjects who received at least one dose of luspatercept in the compassionate phase\n\nExclusion Criteria:\n\n\\- Subjects who are not willing or able to sign the informed consent.'}, 'identificationModule': {'nctId': 'NCT06913634', 'acronym': 'LUSPA001', 'briefTitle': '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', 'organization': {'class': 'OTHER', 'fullName': 'Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia'}, 'officialTitle': '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', 'orgStudyIdInfo': {'id': 'LUSPA001'}}, 'armsInterventionsModule': {'interventions': [{'name': 'Luspatercept (ACE-536)', 'type': 'DRUG', 'description': 'Luspatercept (ACE-536)'}]}, 'contactsLocationsModule': {'locations': [{'zip': '09121', 'city': 'Cagliari', 'state': 'CA', 'country': 'Italy', 'facility': 'University of Cagliari, Ospedale Pediatrico Microcitemico, via Jenner sn, 09121 Cagliari -', 'geoPoint': {'lat': 39.23054, 'lon': 9.11917}}], 'overallOfficials': [{'name': 'RAFFAELLA ORIGA', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "UNIVERSITA' DI CAGLIARI"}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}