| LBCTR2020021295 | VIT-2763-THAL-201 | A study to evaluate if VIT-2763 may be beneficial in the treatment of Nontransfusion Dependent Beta-thalassaemia. | A Phase 2a, Double-blind, Randomised, Placebo-controlled, Parallel Group, Multicentre Study on Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of Multiple Doses of VIT-2763 in Subjects with Non-transfusion Dependent Beta-thalassaemia | Interventional | Complete | 02/10/2019 |
| 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 |
| LBCTR2022095104 | RBSC2161 | Pharmacokinetics and Pharmacodynamics of Rifaximin Novel Formulations in Patients With Sickle Cell Disease | A Phase 2a Randomized, Double-Blind, Placebo-Controlled Study to Characterize the Pharmacokinetics and Pharmacodynamics of Rifaximin Novel Formulations in Patients With Sickle Cell Disease | Interventional | Complete | 27/07/2022 |
| LBCTR2019010167 | CNTO1959CRD3001 | A Study of the Efficacy and Safety of Guselkumab in Participants with Moderately to Severely Active Crohn's Disease | A Phase 2/3, Randomized, Double-blind, Placebo- and Active-controlled, Parallel group, Multicenter Protocol to Evaluate the Efficacy and Safety of Guselkumab in Participants with Moderately to Severely Active Crohn's Disease | Interventional | Complete | 24/10/2018 |
| LBCTR2022034916 | AG348-C-020 | A Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Sickle Cell Disease (SCD) | A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Mitapivat in Subjects With Sickle Cell Disease | Interventional | Pending | 08/11/2021 |
| 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 |
| LBCTR2019020198 | SEG101B2201 | Study of Dose Confirmation and Safety of Crizanlizumab in Pediatric Sickle Cell Disease Patients | A Phase 2,Multicenter,Open-Label Study to Assess Appropriate Dosing and to Evaluate Safety of Crizanlizumab,With or Without Hydroxyurea/Hydroxycarbamide,in Sequential,Descending Age Groups of Pediatric Sickle Cell Disease Patients With Vaso-Occlusive Crisis | Interventional | Recruiting | 20/02/2019 |
| 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 |
| 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 |