ProjektportalDevelopment of meaningful COA-based endpoint for clinical trials in cerebellar ataxias
Terrmin
HT24
Beskrivning
Autosomal Recessive Cerebellar Ataxia (ARCA) is heterogenous group of > 200 different rare and ultra rare neurodegenrative diseases that affect the cerebellum and associated tracts. The Scale for Assessment and Rating of Ataxia (SARA) is the most commonly used measure for assessing the disease severity of cerebellar ataxias in clinics and natural history studies. The usefullness of SARA assessment, and its modifications, as a clinical outcome assessment (COA) in clinical studies is now being discussed in the ataxia field. An important aspect to be addressed is the clinical and patient meaningfullness of SARA-based endpoints.
In this project, data from the ARCA registry will be used in the analysis. The dataset comprises of item-level data from SARA as well as other assessments that measured the disease severity including functional satging (FARS), activities of daily living (ADL), self-rated assessment of health status (EQ-5D), and others. Anchor-based approaches along with Item Response Theory (IRT) methodology will be used to develop and support the interpretability of SARA-based endpoints. The research will be conducted in light with the recent FDA guidance series on patient-focused drug development.
This project requires programming skills in R and NONMEM.
The work will be carried out in Uppsala, however in a research group, that offers an international environment. The working language, including supervision, will be English.
Does this project sounds like something for you? Apply here or contact the supervisor (see below) or Maria Kjellsson (maria.kjellsson@farmaci.uu.se) if you have additional questions. We look forward to your application!
Huvudområde
Farmaceutisk vetenskap
Ämne
Farmakometri
Typ
Beräkningsstudie
Företag
Uppsala University
Ort/Plats
Uppsala
Handledarens namn
Alzahra Hamdan, Mats Karlsson
Handledarens e-post
alzahra.hamdan@farmaci.uu.se; mats.karlsson@farmaci.uu.se
Institution
Institutionen för farmaci
Program
Masterprogram i läkemedelsmodellering
Kurs
Degree Project in Pharmaceutical Modeling within Pharmacometrics 45 c - 3FB029
Omfattning/hp
45hp
Hur många studenter kan antagas för detta projekt?
1