| LBCTR2021064778 | 232SM203 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Interventional | Pending | 28/06/2021 |
| LBCTR2021044780 | CUER342021 | Evaluation of the efficacy of oral swabs and oropharyngeal saliva as diagnostic tools for COVID-19 | Evaluation of the efficacy of oral swabs and oropharyngeal saliva as diagnostic tools for COVID-19 | Interventional | Pending | 27/03/2021 |
| 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 |
| LBCTR2021074838 | GBT2104-133 | An Open-label Extension Study to Evaluate the Long-term Safety of Inclacumab Administered to Participants with Sickle Cell Disease Who Have Participated in an Inclacumab Clinical Trial | An Open-label Extension Study to Evaluate the Long-term Safety of Inclacumab Administered to Participants with Sickle Cell Disease Who Have Participated in an Inclacumab Clinical Trial | Interventional | Pending | 05/07/2021 |
| LBCTR2022024840 | ISRCTN52076264 | Supporting Mothers’ Mental Health with Interpersonal Therapy | Evaluating the impact of group interpersonal psychotherapy compared to high-quality standard care for mothers with postnatal depression in Lebanon and Kenya on child developmental outcomes, maternal depression and the mother-child relationship | Interventional | Pending | 13/07/2021 |
| LBCTR2022014844 | AG348-C-017 | A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT) | A Phase 3, Double-blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Non–Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE) | Interventional | Pending | 15/07/2021 |
| LBCTR2022014845 | AG348-C-018 | A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) (ENERGIZE-T). | A Phase 3, Double-blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE-T) | Interventional | Pending | 15/07/2021 |
| 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 |
| LBCTR2021114915 | WA40404 | A Study to Evaluate the Efficacy and Safety of Ocrelizumab in Adults With Primary Progressive Multiple Sclerosis | A PHASE IIIb MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF OCRELIZUMAB IN ADULTS WITH PRIMARY PROGRESSIVE MULTIPLE SCLEROSIS | Interventional | Pending | 05/11/2021 |
| 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 |