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.

CLINICAL TRIALS

London Ataxia Centre SCA1 and SCA3 trial (Added February 2026) - 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 now recruiting participants through the London Ataxia Centre.

Trial to test the Exopulse Mollii electrostimulation suit in ataxias (Added March 2026) - This trial will be run by Dr Lisa Bunn at the University of Plymouth and is for adults and children over the age of 4 with any confirmed genetic or inherited cerebellar ataxia. The researchers will look at whether wearing the Exopulse Mollii suit in the home environment for 4 weeks can improve motor function, balance, coordination, and quality of life in ataxias. Participants will be randomly assigned to either wear a working version of the suit or a 'sham' non-working version of the suit.

Intrabio phase III trial of AQNEURSA (approved by the FDA to treat Niemann-Pick Disease Type C) in CACNA1A-related disorders, which includes SCA6 and Episodic Ataxia Type 2 (Added September 2025) - Newcastle and Oxford are UK sites in this trial.

Arrowhead Pharmaceuticals is planning a phase I trial of their drug ARO-ATXN2 in people with SCA2 (Added September 2025) - They are currently exploring UK sites, and plan to begin recruitment in 2026.

ONLINE STUDIES/ SURVEYS

Exploring identity in people with hereditary ataxias (Added October 2026) - This study, led by a researcher at the University of Southampton, involves an an online interview to explore how a diagnosis of a hereditary ataxia impacts sense of identity in young adults with any hereditary ataxia, aged, 18-35. If you find answering these questions upsetting in any way then please discuss this with a healthcare professional, someone in your support network, or contact the Ataxia UK helpline (Mon-Fri 10:30-14:30 on 0800 995 6037 or email help@ataxia.org.uk).

SCA2 and SCA3 survey (Added Feb 2026) - This survey, being conducted on behalf of the pharmaceutical company Servier, aims to gather opinions about antisense oligonucleotide (ASO) therapy research. The information gathered will be used to help develop future clinical studies for ataxia. This survey is open to individuals who have a confirmed diagnosis of SCA2 or SCA3, or who are at risk of developing SCA2 or SCA3 (meaning they have a family history and may carry the gene but have not had genetic testing). Individuals who are presymptomatic can also take part.  To take part in the survey, individuals must be at least 18 years old, and must be able to walk without permanent use of walking aids. Family members or caregivers may answer on behalf of the person with SCA2/SCA3. For more information click here.

SCA6 and SCA27B survey (Added August 2024) - 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

A project testing brain stimulation in people with ataxia (Added Oct 2026) - Dr Tim West, neuroscientist, is leading a study based at the Institute of Neurology, University College London. His team is developing a non-invasive form of brain stimulation that targets the part of the brain that coordinates movement, the cerebellum. The long-term aim is a personalised treatment for ataxia using neurostimulation. The study is now under way, and Dr West is looking for adults with ataxia whose symptoms mainly affect coordination, balance and speech to take part and Ataxia UK is supporting Dr West with recruitment.

POLG-related mitochondrial disease and ataxia study (Added Nov 2024) - 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.

Newcastle R-PROMS Study (Added Nov 2023) - 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.

NATURAL HISTORY STUDIES

Project ESMI (Added March 2024) - Aims to develop disease markers, and understand the progression of SCA3.

DRPLA Natural History and Biomarker Study (Added Nov 2022) - 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.