6 results
- 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/…
Comparison of steady-state cysteamine-trough WBC cystine levels between Cystagon® and RP103 over 3 months for each treatment period.
Please refer to protocol, section 1.2 "Rationale"
The primary objective of the study is:* To demonstrate the reduction of low-density lipoprotein cholesterol (LDL-C) by evinacumab 15 mg/kg intravenously (IV) in comparison to placebo after 24 weeks in patients with homozygous familial…
The primary objective of the study is to determine the proportion of patients with elevated TGs, without Familial ChylomicronemiaSyndrome (FCS) due to loss of function (LoF) mutations in lipoprotein lipase (LPL), and a history of…
The aim of this study is to establish pharmacokinetic data on inhaled nebulized prednisolone: Time from start nebulizing to serum peak prednisolone concentration (Tmax) and prednisolone area under. We derive this pharmacokinetic data from two…