For People with Friedreich's Ataxia

For People with Friedreich's Ataxia

Taking part in research as a patient is a wonderful way to bring us ever closer to finding a cure for ataxia. Below is a list of current research trials and projects that are recruiting for patients with FA.

Yanita was diagnosed with FA in her 20s and took part in the Omaveloxolone clinical trial in the UK.   

“I’m so glad I could take part in the trial and be part of the research for a drug that is now approved by the FDA,” Yanita shared.   

CLINICAL TRIALS

BRAVE paediatric study of Omaveloxolone for FA - this phase 3 clinical trial looks at the effects and long-term safety of Omaveloxolone (SKYCLARYS) in children with FA aged 2 - 15 years. This trial is being run by Biogen. The London Ataxia Centre, Oxford Ataxia Centre and Sheffield Ataxia Centre are UK trial sites. The trial is now open for recruitment in the UK.

Sheffield Ataxia Centre contact: ​Dr Santosh Mordekar, santosh.mordekar@nhs.net ​

​London Ataxia Centre contact:​ Dr Shpresa Pula, shpresa.pula1@nhs.net 

Oxford Ataxia Centre contact: Prof Andrea Nemeth, andrea.nemeth@ndcn.ox.ac.uk. 

ONLINE SURVEYS

FA care pathway survey - The purpose of this study is to gather the experiences of care of people with FA in the UK, in order to understand ataxia patients’ care pathways for the management of specific symptoms associated with FA. More specifically, this questionnaire aims to identify which aspects of FA management are most important to patients, including their perceptions of the quality of the medical care they receive for both FA progression and associated conditions. Anyone age 16 and above with a confirmed diagnosis of FA can take part. For people younger than 16, their parent or guardian can take part on their behalf.

IN-PERSON STUDIES

Augmented Reality-Based Closed-Loop Rehabilitation for Postural Instability in Ataxia - In-person study at Aston University, Birmingham. Anyone with a progressive ataxia including FA who is ambulant and aged 18+ can take part. Participants in the AR-based balance training study will visit Aston University twice weekly for 8–12 weeks, depending on their progression, with additional pre- and post-intervention assessment visits. Each session will last approximately 30 minutes.

FA balance and gait study - a study using wearable technology to investigate the impacts of Friedreich's ataxia on balance and gait, both in the laboratory setting and in real-world settings such as the home

NATURAL HISTORY STUDIES

Natural history study in Friedreich's ataxia (EFACTS)

APPS AND WEBSITES

The FA app - The FA app, developed by the charity End FA, is an app which aims to connect with and empower all people with Friedreich’s ataxia (FA) worldwide. The objectives of the app are to connect people with FA with researchers running clinical and virtual research trials, to empower people with FA with useful tools for managing their life and treatment, and to educate people by sharing the latest FA news.