Usability assessment of an OptiNose exhalation delivery system in children

Data suggest that oxytocin delivered with an OptiNose system may offer nose-to-brain (N2B) activity in adults. It is desirable to make future treatment regimens also available, for example for autism spectrum disorder (ASD), to children and adolescents, but it is not known if the delivery system is suitable for pediatric use. Unfortunately, ASD is a complex disorder, with a heterogeneous etiology, and the research program required is commensurately challenging. It has been determined that Prader Willy syndrome (PWS) will be the first planned indication for an OptiNose exhalation delivery form of oxytocin. Nasal oxytocin has shown promising effects in PWS, a complex genetic neurodevelopmental disorder with cognitive/behavioral problems and obesity starting in early childhood where there is a high level of unsatisfied medical need for new treatment options. PWS is also classified as an orphan indication, offering important advantages in development time and exclusivity. This extension project will test fit and usability in subjects under 18 of an exhalation delivery system known to be usable in an adult population and will collect anthropometric data. The study will begin with the oldest age sub-group (16-17 year old) and progress down towards the youngest (6-7 year old) until failure occurs. Anthropometric data will help establish appropriate dimensions of an exhalation delivery system. If the fit or usability among age groups of subjects do not meet pre-defined levels, it may be determined that changes to the product design (for example, length of the flexible mouthpiece) may help subjects obtain an appropriate fit. OptiNose has developed relevant prototypes and test fit and functionality in vitro. We have obtained answers to what age groups can use the delivery system designed for adults and in what age groups it is necessary to adapt the system. Based on these results, Optinose has developed prototypes that are specially adapted for use in young children and we have tested fit and functionality of these prototypes. In addition, we have created new nasal and sinuses silicone models that allow us to study drug deposition in patients who have undergone sinus surgery. We have produced sophisticated silicone models that we can split so that we can coat the surfaces with a gel that simulates the normal mucosal surfaces. We can visualize the deposition by using a gel that changes the color when it comes into contact with liquids or by delivering different colored dyes. Deposition studies in these advanced silicone models have provided very exciting and partly unexpected results that could lead to new patents. We have presented results from these studies at several international scientific conferences and the results may also have commercial significance. In the extension of the work described above, we have also developed, in collaboration with Norwegian companies, 3D-computer models of the complex nasal and sinus geometry and visualization of how the OptiNose exhalation delivery system works ("augmented reality"). These new visualization tools are very useful instruction and training of professionals, patients of all ages and their caregivers and the can also be utilized for promotional purposes.

Vi har utviklet prototyper som er fullt funksjonelle og har dimensjoner som er tilpasset små barn og den har funksjoner som gjør det mye lettere å motiviere små barn og særlig barn med Prader Willi Syndrom (PWS) og deres omsorgspersoner til å bruke «devicen». Vi har produsert avanserte og unike slikonmodeller og ny metode- Deponeringstudier som vi har gjennomført med denne metoden i silikonmodellen har gitt svært spennende og til dels uventede resultater som trolig kan føre til nye patenter. Utvikling av nye metoder for visualisering av deponering i nesemodeller. 3D-animasjoner og «kunstig virkelighet»-Disse nye visualiseringsverktøyene er svært nyttige for instruksjon og opplæring av fagpersoner, patienter i alle aldre og deres omsorgspersoner og vi vil også bruke dem kommersielt.

The primary objective of this project extension is to assess if an OptiNose exhalation delivery system used in adults is suitable for use in adolescents and children. Data suggest that oxytocin delivered with an OptiNose system may offer nose-to-brain (N2B) activity in adults. It is desirable to make future treatment regimens also available, for example for autism spectrum disorder (ASD), to children and adolescents, but it is not known if the delivery system is suitable for pediatric use. Unfortunately, ASD is a complex disorder, with a heterogeneous etiology, and the research program required is commensurately challenging. It has been determined that Prader Willy syndrome (PWS) will be the first planned indication for an OptiNose exhalation delivery form of oxytocin. Nasal oxytocin has shown promising effects in PWS, a complex genetic neurodevelopmental disorder with cognitive/behavioral problems and obesity starting in early childhood where there is a high level of unsatisfied medical need for new treatment options. PWS is also classified as an orphan indication, offering important advantages in development time and exclusivity. This extension project will test fit and usability in subjects under 18 of an exhalation delivery system known to be usable in an adult population and will collect anthropometric data. The study will begin with the oldest age sub-group (16-17 year old) and progress down towards the youngest (6-7 year old) until failure occurs. Anthropometric data will help establish appropriate dimensions of an exhalation delivery system. If the fit or usability among age groups of subjects do not meet pre-defined levels, it may be determined that changes to the product design (for example, length of the flexible mouthpiece) may help subjects obtain an appropriate fit. In that case, OptiNose may develop prototypes and test fit and usability in relevant age groups.