| 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 |
| 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 |
| 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 |
| LBCTR2021124932 | MO42921 | Vanessa | A MULTI-COUNTRY OBSERVATIONAL RETROSPECTIVE STUDY TO EVALUATE THE PREVALENCE OF PD-L1 AND ITS ROLE IN PATIENTS WITH TNBC TREATED WITH SYSTEMIC THERAPY | Observational | Pending | 05/12/2021 |
| LBCTR2024015480 | NN7533-4470 | ACENT 1 | A multicentre trial evaluating the efficacy and safety of oral decitabine-tetrahydrouridine (NDec) in patients with sickle cell disease | Interventional | Pending | 01/02/2024 |
| LBCTR2023115324 | P3-IMU-838-RMS-01 (ENSURE-1) | A Study to Evaluate the Efficacy, Safety, and Tolerability of IMU-838 (vidofludimus calcium) versus Placebo in Adults with Relapsing Multiple Sclerosis (RMS) | A Multi-Center, Randomized, Double-Blinded Phase 3 Study to Evaluate the Efficacy, Safety, and Tolerability of IMU-838 versus Placebo in Adults with Relapsing Multiple Sclerosis (ENSURE-1) | Interventional | Pending | 30/03/2023 |
| LBCTR2022014919 | CQGE031E12301 | Study of Efficacy and Safety of Ligelizumab in Adolescents and Adults With Chronic Inducible Urticaria Who Remain Symptomatic Despite Treatment With H1- Antihistamines | A Multi-center, Randomized, Double-blind, Placebo Controlled Study to Investigate the Efficacy and Safety of Ligelizumab (QGE031) in the Treatment of Chronic Inducible Urticaria (CINDU) in Adolescents and Adults Inadequately Controlled With H1-antihistamines | Interventional | Suspended | 09/11/2021 |