5 results
The objective of this pivotal study is to assess the PK and PD as well as safety and tolerability of RP103 compared to Cystagon® in patients with nephropathic cystinosis. Results of this Phase 3 study will be used to support the registration…
The primary objective of this study is to compare the efficacy of pitavastatin 1 mg once daily (QD), 2 mg QD, and 4 mg QD (after up-titration) to placebo in terms of the percentage reduction in LDL-C in children or adolescent patients with high-risk…
The primary objective of this study is to assess the safety of pitavastatin 1 mg QD, 2 mg QD, and 4 mg QD in children or adolescent patients with high-risk hyperlipidaemia over a period of 52 weeks.The secondary objective of this study is to assess…
To investigate if antiplatelet therapy consisting of ticagrelor plus ASA is superior to clopidogrel plus ASA in preventing ischaemic brain lesions occurring as a result of CAS assessed on magnetic resonance imaging (MRI).
- To assess the pharmacokinetic profile of cysteamine base after single dose administration of the new sustained-release cysteamine bitartrate (PO-001) to healthy volunteers, in comparison with Cystagon® and Procysbi®.- To collect safety/…