Xperiome™ Labs

Learn how we turn patient experience into insights through our knowledge bank for the lived experience of rare disease.

Research

Learn about the research studies we conduct with commercial and academic partners, and the clinical trials we support for pharmaceutical sponsors.


Active
Ornithine transcarbamylase (OTC) deficiency clinical research study
This study is a randomized, placebo-controlled study, testing an investigational medicine called ARCT-810 on adults with ornithine transcarbamylase (OTC) deficiency.

Completed
Anti-AChR antibody-positive generalized myasthenia gravis clinical research study
This placebo-controlled Phase 3 study is testing an investigational medicine called ravulizumab-cwvz, also known in this study as ALXN1210, on adults with anti-AChR antibody-positive, generalized myasthenia gravis (gMG).

Completed
What do you think about your cystic fibrosis treatment(s)?
This study aims to learn what people with cystic fibrosis actually think about taking the CFTR modulator therapies Kalydeco, Orkambi, Symdeko/Symkevi, and Trikafta/Kaftrio. This is not like other studies that may only focus on following doctors’ orders.

Completed
What do you think about your sickle cell treatment(s)?
This study aims to learn what people with sickle cell actually think about taking the newer sickle cell medications – Adakveo (crizanlizumab), Endari (L-glutamine), and Oxbryta (voxelotor) – and see how they compare with the older drug, hydroxyurea. This is not like other studies that may only focus on following doctors’ orders.

Case studies

Discover how we’re making our mark in research and accelerating the development and reach of new treatments.

Visit our Virtual Venue to find out how Xperiome can help you unlock deep insights into rare patient experience.