| LBCTR2022014845 | AG348-C-018 | A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) (ENERGIZE-T). | A Phase 3, Double-blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE-T) | Interventional | Pending | 15/07/2021 |
| LBCTR2022014844 | AG348-C-017 | A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT) | A Phase 3, Double-blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Non–Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE) | Interventional | Pending | 15/07/2021 |
| LBCTR2023075332 | ACE-536-MF-002 | An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis on Concomitant JAK2 Inhibitor Therapy and Who Require Red Blood Cell Transfusions (INDEPENDENCE) | A phase 3, double-blind, randomized study to compare the efficacy and safety of Luspatercept (ACE-536) versus placebo in subjects with Myeloproliferative Neoplasm-Associated Myelofibrosis on concomitant JAK2 inhibitor therapy and who require red blood cell transfusions | Interventional | Recruiting | 13/04/2023 |
| LBCTR2019100218 | ACE-536-LTFU-001 | A Phase 3B, open-label, single-arm, rollover study to evaluate long-term safety in subjects who have participated in other luspatercept (ACE-536) clinical trials | A Phase 3B, open-label, single-arm, rollover study to evaluate long-term safety in subjects who have participated in other luspatercept (ACE-536) clinical trials | Interventional | Recruiting | 02/04/2019 |
| LBCTR2020124661 | 56021927PCR3015 | A Study of Adding Apalutamide to Radiotherapy and LHRH Agonist in High-Risk Patients With Prostate-Specific Membrane Antigen-Positron Emission Tomography (PSMA-PET) Positive Hormone-Sensitive Prostate Cancer Participants | A Randomized, Controlled, Multicenter, Open-label Study to Investigate the Efficacy and Safety of Adding Apalutamide to Radiotherapy and LHRH Agonist in High-Risk Patients with PSMA-PET-Positive Hormone-Sensitive Prostate Cancer, with an Observational Follow-up of PSMA-PET-Negative Patients | Interventional | Pending | 06/11/2020 |
| LBCTR2021124934 | 4202-HEM-301 | An Adaptive, Randomized, Placebo-controlled, Double-blind, Multi-center Study of Oral FT-4202, a Pyruvate Kinase Activator in Patients with Sickle Cell Disease | An Adaptive, Randomized, Placebo-controlled, Double-blind, Multi-center Study of Oral FT-4202, a Pyruvate Kinase Activator in Patients with Sickle Cell Disease | Interventional | Recruiting | 10/12/2021 |
| LBCTR2022095118 | 4202-HEM-202 | Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients with Sickle Cell Disease | A Single Arm, Open Label, Phase 1/2 Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients with Sickle Cell Disease | Interventional | Pending | 08/09/2022 |
| LBCTR2022074978 | 4202-HEM-201 | A Phase 2 Open-Label Study to Evaluate Safety and Clinical Activity of FT-4202 in Patients with Thalassemia or Sickle Cell Disease | A Phase 2 Open-Label Study to Evaluate Safety and Clinical Activity of FT-4202 in Patients with Thalassemia or Sickle Cell Disease | Interventional | Pending | 18/01/2022 |
| LBCTR2022105117 | 232SM302 | Extension Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Who Previously Participated in a Study With Nusinersen | A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen | Interventional | Recruiting | 08/09/2022 |
| LBCTR2021064778 | 232SM203 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Interventional | Pending | 28/06/2021 |