| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| LBCTR2021104866 | DCR-PHXC-203 | A Phase 2 Open-Label Multicenter Study to Evaluate the Safety, Pharmacokinetics, and Efficacy of Nedosiran in Pediatric Patients from Birth to 5 Years of Age with Primary Hyperoxaluria and Relatively Intact Renal Function | A Phase 2 Open-Label Multicenter Study to Evaluate the Safety, Pharmacokinetics, and Efficacy of Nedosiran in Pediatric Patients from Birth to 5 Years of Age with Primary Hyperoxaluria and Relatively Intact Renal Function | Interventional | Pending | 08/10/2021 |
| LBCTR2021054742 | DCR-PHXC-502 | A Natural History Study of Patients with Genetically Confirmed Primary Hyperoxaluria Type 3 with a History of Stone Events | A Natural History Study of Patients with Genetically Confirmed Primary Hyperoxaluria Type 3 with a History of Stone Events | Observational | Pending | 07/07/2021 |
| LBCTR2019010183 | CLBH589D2222 | A multicenter, randomized, open-label Phase 2 study evaluating the safety and efficacy of three different regimens of oral panobinostat in combination with subcutaneous bortezomib and oral dexamethasone in patients with relapsed or relapsed/refractory multiple myeloma who have been previously exposed to immunomodulatory agents | “A multicenter, randomized, open-label Phase 2 study evaluating the safety and efficacy of three different regimens of oral panobinostat in combination with subcutaneous bortezomib and oral dexamethasone in patients with relapsed or relapsed/refractory multiple myeloma who have been previously exposed to immunomodulatory agents” | Interventional | Complete | 12/11/2018 |
| LBCTR2019020192 | CQGE031C2303 | A multi-center, randomized, double-blind, active and placebo-controlled study to investigate the efficacy and safety of ligelizumab (QGE031) in the treatment of Chronic Spontaneous Urticaria (CSU) in adolescents and adults inadequately controlled with H1-antihistamines | A multi-center, randomized, double-blind, active and placebo-controlled study to investigate the efficacy and safety of ligelizumab (QGE031) in the treatment of Chronic Spontaneous Urticaria (CSU) in adolescents and adults inadequately controlled with H1-antihistamines | Interventional | Complete | 08/02/2019 |