4 results
The FAST Therapy Trial is a prospective trial of patients with a new diagnosis of fetal SVA aimed to compare the impact of different perinatal treatment strategies from the time of SVA diagnosis to birth or death1) The primary outcome will be the…
- 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/…
The main objective of this trial is to investigate safety of long term use of inhaled molgramostim by collecting information on the side effects.
Comparison of steady-state cysteamine-trough WBC cystine levels between Cystagon® and RP103 over 3 months for each treatment period.