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| LBCTR2022125202 | DCR-PHXC-204 | A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients With Primary Hyperoxaluria Type 1 or 2 and Severe Renal Impairment, With or Without Dialysis | A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients With Primary Hyperoxaluria Type 1 or 2 and Severe Renal Impairment, With or Without Dialysis | Interventional | Pending | 21/11/2022 |
| LBCTR2019020179 | PTG300-02 | A Phase 2 Study of PTG-300 in Non-Transfusion Dependent (NTD) and Transfusion-Dependent (TD) β-Thalassemia Subjects with Chronic Anemia | A Phase 2 Study of PTG-300 in Non-Transfusion Dependent (NTD) and Transfusion-Dependent (TD) β-Thalassemia Subjects with Chronic Anemia | Interventional | Complete | 02/01/2019 |
| LBCTR2020093402 | IMR-BTL-201 | A Phase 2 Study to Evaluate the Safety and Tolerability of IMR-687 in Subjects with Beta Thalassemia | A Phase 2 Study to Evaluate the Safety and Tolerability of IMR-687 in Subjects with Beta Thalassemia | Interventional | Pending | 24/02/2020 |
| LBCTR2022105089 | GBT021601-021 | A Phase 2/3 Randomized, Multicenter Study of GBT021601 Administered Orally to Participants with Sickle Cell Disease and an Open-Label Pharmacokinetics Study in Pediatric Participants with Sickle Cell Disease | A Phase 2/3 Randomized, Multicenter Study of GBT021601 Administered Orally to Participants with Sickle Cell Disease and an Open-Label Pharmacokinetics Study in Pediatric Participants with Sickle Cell Disease | Interventional | Pending | 07/07/2022 |
| LBCTR2021064783 | VIT-2763-SCD-202 | A Phase 2a study to assess efficacy and safety of VIT-2763 in subjects with sickle cell disease | A Phase 2a, double-blind, randomised, placebo-controlled, ascending dose and maintenance dose, efficacy, and safety study of multiple doses of VIT-2763 in subjects with sickle cell disease | Interventional | Pending | 24/06/2021 |
| LBCTR2020071296 | ISIS 702843-CS2 | A Phase 2a, Randomized, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Patients with Non-Transfusion Dependent β-Thalassemia Intermedia | A Phase 2a, Randomized, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Patients with Non-Transfusion Dependent β-Thalassemia Intermedia | Interventional | Pending | 03/10/2019 |
| LBCTR2020083421 | IMR-SCD-301 | A Phase 2b Study to Evaluate the Safety and Efficacy of IMR-687 in Subjects with Sickle Cell Disease | A Phase 2b Study to Evaluate the Safety and Efficacy of IMR-687 in Subjects with Sickle Cell Disease | Interventional | Pending | 26/08/2020 |
| LBCTR2019010185 | CABL001A2301 | A Phase 3, Multi-center, Open-label, Randomized Study of Oral ABL001 Versus Bosutinib in Patients With Chronic Myelogenous Leukemia in Chronic Phase (CML-CP), Previously Treated With 2 or More Tyrosine Kinase Inhibitors | A Phase 3, Multi-center, Open-label, Randomized Study of Oral ABL001 Versus Bosutinib in Patients With Chronic Myelogenous Leukemia in Chronic Phase (CML-CP), Previously Treated With 2 or More Tyrosine Kinase Inhibitors | Interventional | Complete | 22/01/2019 |
| LBCTR2024015495 | 1517-CL-1003 | A Phase 3, Open-label, Uncontrolled Study to Evaluate the Activity, Safety, Pharmacokinetics and Pharmacodynamics of Roxadustat for the Treatment of Anemia in Pediatric Participants with Chronic Kidney Disease | A Phase 3, Open-label, Uncontrolled Study to Evaluate the Activity, Safety, Pharmacokinetics and Pharmacodynamics of Roxadustat for the Treatment of Anemia in Pediatric Participants with Chronic Kidney Disease | Interventional | Pending | 05/01/2024 |