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
Generalized myasthenia gravis (gMG) ADAPTSC clinical research study
ADAPTSC is a clinical research study testing whether there is a difference in effect on the body between 2 administration routes of an investigational study drug in adults with generalized myasthenia gravis.
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 IPF treatment(s)?
This study aims to learn what people with idiopathic pulmonary fibrosis actually think about taking the medication(s) nintedanib (Ofev) and/or pirfenidone (Esbriet). This is not like other studies that may only focus on following doctors’ orders. 
Completed
What do you think about your myasthenia gravis treatment(s)?
This study aims to learn what people with myasthenia gravis actually think about taking the medication(s) eculizumab (Soliris), neostigmine (Bloxiverz), and/or pyridostigmine (Mestinon, Regonol). This is not like other studies that may only focus on following doctors’ orders.
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Articles and publications

Read our perspectives on news and trends in rare disease, and how patients can be powerful agents of change in health.

Blog
Solve rare disease study recruitment challenges by engaging with patients early

April 2021

Could early audience insight be the answer to the recruitment challenge in rare disease?
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Media article
Advertising Digitally for Rare Disease Clinical Trials in the 21st Century

April 2021

We look at how sponsors can best utilize digital advertising channels to help shorten recruitment timelines
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Watch and Listen
Webinar - Patient Advocacy and Access to Innovative Clinical Trials in a Changed Environment

April 2021

Our founder, Julie Walters, along with experts in bioethics and clinical trial management explored the challenges of running cell and gene therapy trials in the wake of the pandemic
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Media article
Community building empowers rare-disease patients: Xperiome

February 2021

Jeremy Edwards, CEO of Xperiome, spoke with Outsourcing-Pharma about the unique challenges rare disease patients and researchers face, and how connecting the two groups can benefit both in many ways
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Case studies

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

Case study
Patient preferences for injectable drug-delivery options

A pharmaceutical company commissioned Xperiome to survey our sickle cell community to understand how people living with the condition handle their pain crises and hospital visits, and how they would feel about different ways to take or receive a new injectable medication.

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Case study
Longitudinal, real-world data to support US FDA submission

A specialist pharmaceutical company commissioned Xperiome to recruit a group of people living with pouchitis, a rare gastrointestinal condition, and to track how it affects their lives over a six-month period.

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Case study
Phase I study to assess safety and tolerability of investigative medication in pediatric patients with Sanfilippo syndrome (MPS IIIA)

A specialty healthcare company developing treatments for rare diseases sought Xperoime’s support for an early-stage pediatric trial program testing an investigational medicine in Sanfilippo syndrome.

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Case study
Phase II study to evaluate safety and efficacy of investigative medication in adults with generalized MG

A clinical-stage biopharmaceutical company developing treatments for autoimmune diseases brought in Xperiome to boost recruitment to a Phase II study of an experimental drug for generalized myasthenia gravis (MG); a super-hot area for clinical research experiencing high competition for eligible patients.

Read more
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