For People with Cerebellar Ataxia

For People with Cerebellar Ataxia

Taking part in research as participants in trials is a wonderful way to get us ever closer to finding a cure. Listed below are some current research projects recruiting for participants with cerebellar ataxia.

ONLINE SURVEYS

Gene therapy survey - for people with genetic ataxias

SCA6 and SCA27B survey - a 30-minute online survey to understand the genetic and environmental factors that influence the onset, severity and progression of SCA6 and SCA27B. This survey is being conducted by the University of Chicago and is for those aged 18 years and above with a confirmed diagnosis of either SCA6 or SCA27B.

IN-PERSON STUDIES

The Cerebellum, Motor Learning and Respiration - In-person study at the University of Birmingham, Edgbaston, looking at the role of the cerebellum in breathing in adults with any form of cerebellar ataxia. If you are interested in taking part in this research, please complete the survey in the link above.

UPCOMING STUDY - Augmented Reality-Based Closed-Loop Rehabilitation for Postural Instability in Ataxia - In-person study at Aston University, Birmingham. Anyone with a progressive ataxia 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. This study will begin in November. Please click the link above to join the waiting list to take part. 

POLG-related mitochondrial disease and ataxia study - Newcastle University is recruiting individuals aged 16-75 with a genetically confirmed diagnosis of POLG-related mitochondrial disease to take part in a study looking at movement, balance and co-ordination in this condition. The study is in-person and will involve assessments at three timepoints over 12 months.

Improving diagnosis and management of gluten ataxia Study - Ataxia UK, Coeliac UK, the Sheffield Hospital Charity and the Greaves and Withey Foundation awarded funding to Professor Marios Hadjivassiliou, at the Sheffield Ataxia Centre and colleagues, to help improve the diagnosis and management of gluten ataxia.

Newcastle R-PROMS Study - a study that aims to investigate whether online assessments can be used to measure the severity of ataxia (movement, balance and co-ordination difficulties) in people with Mitochondrial Disease and any type of Spinocerebellar ataxia.

UPCOMING CLINICAL TRIALS

UPCOMING TRIAL: Arrowhead Pharmaceuticals is planning a phase I trial of their drug ARO-ATXN2 in people with SCA2. This will include sites in Newcastle and London in the UK. We will share more when the sites begin recruitment, which is expected to be later in 2025.

UPCOMING TRIAL: Intrabio is planning a phase III trial of their drug AQNEURSA (approved by the FDA to treat Niemann-Pick Disease Type C) in CACNA1A-related disorders, which includes SCA6 and Episodic Ataxia Type 2. They are currently exploring UK sites, and plan to begin recruitment in 2026.

London Ataxia Centre SCA1 and SCA3 trial (PAUSED) - this phase 1/2a trial will assess the safety and tolerability of a therapy developed by the pharmaceutical company Vico Therapeutics, known as VO659. VO659 is a small piece of genetic material that targets mutated genes. This trial is currently suspended.

NATURAL HISTORY STUDIES

Project ESMI - aims to develop disease markers, and understand the progression of SCA3.

DRPLA Natural History and Biomarker Study - Ataxia UK and CureDRPLA are coordinating the DRPLA Natural History and Biomarker Study for this very rare form of ataxia. The funding for the study is provided by CureDRPLA. This is a global study, including a UK site at the London Ataxia Centre, which is now recruiting participants. Individuals in countries with no study sites might also be able to participate in this study. See the flyer for more information.