| 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 |
| LBCTR2020033423 | MO40653 | IMReal | A NON-INTERVENTIONAL, MULTICENTER, MULTIPLE COHORT STUDY INVESTIGATING THE OUTCOMES AND SAFETY OF ATEZOLIZUMAB UNDER REAL-WORLD CONDITIONS IN PATIENTS TREATED IN ROUTINE CLINICAL PRACTICE | Observational | Recruiting | 04/03/2020 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |