In addition to the general consortium management work package, a total of three main work packages are foreseen:
Development and testing of Orexin-A formulation approved for humans
Orexin-A has been delivered to the nose in patients with narcolepsy. In these studies, a liquid formulation for nasal use was manufactured from a lyophilized powder of Orexin-A and required repeated delivery every minute for 10 minutes of a total volume of 2 ml. A powder formulation would be a better alternative for nasal delivery of Orexin-A, and in particular for a commercial product. A formulation must be developed in a controlled environment under GMP (Good Manufacturing Practice) conditions by an approved manufacturer.
A combined single ascending dosing (SAD) and multiple ascending dosing (MAD) PK studies in healthy volunteers
This study is an open and randomised (within cohort) first-in-man study in which several dose levels of Orexin-A will be administered to healthy volunteers in a dose-escalating manner, and will be compared against placebo (P). The first phase of the study will expose the cohorts to a single, ascending dose. If there are no safety concerns, the study will advance into a Multiple Ascending Dose phase; in which the same design as in the SAD phase will be employed, except that each subject will have several, repeating doses; once daily.
Clinical trial of Intranasal Orexin A in narcolepsy patients with cataplexy ? acute administration
In this study, we will in a triple dose, placebo-controlled, double blind, cross-over experimental design, study the influence of one dose of intranasally administered Orexin-A on daytime symptoms of approx. 20 adult patients with narcolepsy and known Orexin deficiency.