Thalassaemia trials in Italy
11 trials · From ClinicalTrials.gov, checked 11 November 2026. Ask the study team whether a trial fits you.
- Phase 3Taking peopleA Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
- Where
- Cagliari, Padova
- Age
- 1 year to 17 years
- People
- 54 worldwide
- Run by
- Agios Pharmaceuticals, Inc.
See details and how to joinNCT07506863 · EU 2025-524707-71-00 - Phase 3Taking peopleA Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia
- Where
- Cagliari
- Age
- 1 year to 17 years
- People
- 45 worldwide
- Run by
- Agios Pharmaceuticals, Inc.
See details and how to joinNCT07517133 · EU 2025-524706-15-00 - Phase 3Taking peopleA Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
- Where
- Orbassano, Padova, Pavia
- Age
- 2 years and over
- People
- 480 worldwide
- Run by
- Novo Nordisk A/S
See details and how to joinNCT06609226 · EU 2024-510805-27-00 - Phase 3Enrolling by invitationA Long-term Follow-up Study in Participants Who Received CTX001
- Where
- Rome
- Age
- 2 years to 64 years
- People
- 160 worldwide
- Run by
- Vertex Pharmaceuticals Incorporated
See details and how to joinNCT04208529 · EU 2024-512654-19-00 - Phase 2Taking peopleGene-Modified Stem Cell Therapy for Subjects With Transfusion-dependent Beta-thalassemia
- Where
- Milan, Rome
- Age
- 3 years to 35 years
- People
- 9
- Run by
- Fondazione Telethon
See details and how to joinNCT07680803 · EU 2025-522160-32-00 - Phase 2Taking peopleStudy of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
- Where
- Genoa, Naples, Orbassano, Rome
- Age
- 6 years to 17 years
- People
- 99 worldwide
- Run by
- Celgene
See details and how to joinNCT04143724 · EU 2022-502499-22-00 - Phase 3Taking peopleA Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials
- Where
- Allessandria, Bologna, Brindisi, Cagliari, Ferrara, Florence, Genoa, Lecce, Milan, Modena, Naples, Orbassano, Pavia, Reggio Calabria, Roma, Rozzano, Varese, Verona
- Age
- 18 years and over
- People
- 665 worldwide
- Run by
- Celgene
See details and how to joinNCT04064060 - Phase 3Taking peopleEvaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
- Where
- Rome
- Age
- 12 years to 35 years
- People
- 26 worldwide
- Run by
- Vertex Pharmaceuticals Incorporated
See details and how to joinNCT05477563 · EU 2024-514641-12-00 - No phaseTaking peopleAtrial Fibrillation in Beta-Thalassemia
- Where
- Ferrara
- Age
- 18 years and over
- People
- 350
- Run by
- University Hospital of Ferrara
See details and how to joinNCT05508932 - Phase 2Taking peopleA Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic Syndromes
- Where
- Bologna, Cagliari, Florence, Genoa, Milan, Modena, Naples, Pavia
- Age
- 18 years to 64 years
- People
- 90 worldwide
- Run by
- Pharmacosmos A/S
See details and how to joinNCT05693909 · EU 2023-507396-21-00 - No phaseTaking peopleStudy of the Role of Genetic Modifiers in Hemoglobinopathies
- Where
- Turin
- Age
- 2 years and over
- People
- 30,000 worldwide
- Run by
- Cyprus Institute of Neurology and Genetics
See details and how to joinNCT05799118