Condition

Ataxia clinical trials and research

• 10Clinical trials
• 64NIH research projects
• 8Countries

Newest Ataxia clinical trials

All recruiting trials (10)

• How foot braces might help people with ataxia walk better

  This study tests whether wearing foot orthoses at home can improve gait and balance for adults with cerebellar ataxia.

  Recruiting University of Texas Southwestern Medical Center · Dallas, Texas · NCT07720700

• Registry and natural history of epilepsy-dyskinesia syndromes

  This registry will collect health data and biological samples from people of all ages who have a genetic diagnosis of epilepsy-dyskinesia syndromes to see how their genes relate to their movement and seizure symptoms over time.

  Recruiting Boston Children's Hospital · Boston, Massachusetts · NCT06967727

• Registry and biobank for neuroimmunological diseases

  This study is trying to see if certain antibodies in the brain are linked to neurological disorders like encephalopathy and psychosis, and how they might affect patients' health.

  Recruiting at 2 sites Charite University, Berlin, Germany · Berlin, State of Berlin and 1 other locations · NCT06958341

• Home-based training for improving gait and balance in movement disorders

  This study is testing whether a three-week home training program can help people with movement disorders like ataxia and Parkinson's improve their balance and walking.

  Recruiting Forschungszentrum Juelich · Düsseldorf, North Rhine-Westphalia · NCT06617884

• Understanding epilepsy-dyskinesia syndromes in children

  This study is trying to learn more about epilepsy-dyskinesia syndromes in children by gathering information from different countries to see how these movement and seizure disorders are related.

  Recruiting Boston Children's Hospital · Boston, Massachusetts · NCT06585605

• Cerebellar tACS plus iTBS combined with exercise for ataxia balance

  This test will see if combining cerebellar tACS and iTBS with guided exergaming improves balance in people with ataxia.

  Recruiting LIFE - Research and Care Institute - Santa Lucia IRCCS · Rome, Lazio · NCT06420271

• Home exercise program for people with neurodegenerative diseases

  This study is testing a home exercise program to see if it can help people with neurodegenerative diseases improve their walking and balance.

  Recruiting Hugo W. Moser Research Institute at Kennedy Krieger, Inc. · Baltimore, Maryland · NCT05443906

• Observing the natural progression of RFC1 repeat expansion disease

  This study is trying to understand how RFC1 repeat expansion disease affects people over time and to find helpful markers for diagnosis and future treatments.

  Recruiting at 10 sites University Hospital Tuebingen · Melbourne, Victoria and 9 other locations · NCT05177809

• Rehabilitation for balance issues in patients with neuropathy

  This study tests whether different types of balance rehabilitation can help people with neuropathy improve their balance and walking stability.

  Recruiting Assistance Publique - Hôpitaux de Paris · Paris · NCT03881930

• Registry and clinical study for Wolfram syndrome and related disorders

  This study is trying to gather information from people with Wolfram syndrome to better understand the condition and its effects, while also looking for specific gene mutations.

  Recruiting Washington University School of Medicine · St Louis, Missouri · NCT02841553

NIH-funded research (newest 40 of 64)

Early-stage science funded by the US National Institutes of Health. Most of these projects don't enroll patients, but they show what is being worked on.

• Modeling Ataxia Telangiectasia in a human stem cell model of Purkinje cells

  Researchers are creating a patient-derived, human cell model of Purkinje cells to understand why this brain disease causes cerebellar degeneration in people with Ataxia Telangiectasia.

  NIH research Rockefeller University · New York, United States · NIH-11605226

• VEGF-mimetic nanoparticles to help spinocerebellar ataxia type 1

  A new, patient-friendly approach is being developed to deliver a VEGF-like therapy using tiny particles that could protect brain cells in spinocerebellar ataxia type 1.

  NIH research Northwestern University · Chicago, United States · NIH-11565549

• Understanding early Friedreich ataxia in children to improve how we measure outcomes

  This study looks at how Friedreich ataxia starts and progresses in kids to create better, child-friendly measures for tracking the disease and testing treatments.

  NIH research Children's Hosp of Philadelphia · Philadelphia, United States · NIH-11494983

• Targeting FICD to Help Spinocerebellar Ataxia Type 3 (SCA3)

  A brain-focused project tests whether blocking FICD, a protein in cells’ ER quality system, can slow or prevent damage in people (via animal and cell studies) who have spinocerebellar ataxia type 3.

  NIH research University of Michigan at Ann Arbor · Ann Arbor, United States · NIH-11437540

• Understanding how unstable GAA repeats affect Friedreich’s ataxia progression

  A study in adults and children with Friedreich’s ataxia will look at how changes in GAA repeat length inside the FXN gene relate to the disease’s start and progression.

  NIH research Ut Southwestern Medical Center · Dallas, United States · NIH-11432740

• Biomarkers to measure how well QTE therapy works for Friedreich's ataxia

  Aiming to find blood and muscle signals that show whether a new FRDA treatment (QTE therapy) is working.

  NIH research Anekabio LLC · Gainesville, United States · NIH-11420527

• Investigating how motor neurons contribute to premature death in spinocerebellar ataxia type 1

  A study at Baylor College of Medicine is exploring why people with spinocerebellar ataxia type 1 also develop muscle weakness and breathing problems, and how motor neurons are involved.

  NIH research Baylor College of Medicine · Houston, United States · NIH-11387891

• Understanding GEMIN5 mutations and cerebellar ataxia

  Researchers are studying how changes in the GEMIN5 gene cause motor problems and cerebellar atrophy in people with Aran-Duchenne-like symptoms.

  NIH research University of Pittsburgh at Pittsburgh · Pittsburgh, United States · NIH-11382302

• Understanding nerve damage in Spinocerebellar Ataxia Type 3

  A study in people with SCA3 explores how the disease affects the peripheral nerves and what that means for symptoms like numbness or weakness.

  NIH research University of Michigan at Ann Arbor · Ann Arbor, United States · NIH-11381061

• Understanding how frataxin is turned back on in Friedreich’s ataxia

  A patient-friendly look at a Promising approach that aims to turn on the frataxin gene in people with Friedreich’s ataxia.

  NIH research St. Jude Children's Research Hospital · Memphis, United States · NIH-11376716

• Biomarkers in blood for detecting and tracking ataxia progression

  A small, patient-friendly study from UCLA will look for blood signals that reflect ataxia progression to help test new treatments.

  NIH research University of California Los Angeles · Los Angeles, United States · NIH-11371286

• CRISPR therapy targeting CAG repeats in SCA12

  A new gene-editing approach using CRISPR to remove a disease-causing repeat in a gene linked to spinocerebellar ataxia type 12, tested in models and human cell lines.

  NIH research Johns Hopkins University · Baltimore, United States · NIH-11364275

• Role of TOX3 in cerebellar development and movement disorders

  A new study will explore how TOX3 controls the formation of cerebellar cells and how its loss leads to ataxia and related conditions.

  NIH research Cedars-Sinai Medical Center · Los Angeles, United States · NIH-11362481

• m6A RNA modifications and FXTAS: uncovering new paths to treatment

  A small, patient-focused study will explore how a brain RNA modification called m6A may influence the progression of Fragile X-associated tremor/ataxia syndrome (FXTAS) and point toward new treatment ideas.

  NIH research Emory University · Atlanta, United States · NIH-11356174

• Understanding and slowing the brain changes in Spinocerebellar Ataxia Type 1

  Researchers are studying how a gene mistake causes brain cells to accumulate harmful proteins in Spinocerebellar Ataxia type 1 and what that means for people who might develop this condition.

  NIH research Baylor College of Medicine · Houston, United States · NIH-11352582

• Astrocyte problems in Spinocerebellar Ataxia Type 1

  Researchers are studying how supporting brain cells called astrocytes contribute to Spinocerebellar Ataxia Type 1 and how fixing them might help people with the disease.

  NIH research University of Minnesota · Minneapolis, United States · NIH-11346022

• RSSH donors to help Fe-S cluster formation in Friedreich's ataxia cells

  A scientist is testing whether hydropersulfide donors can restore iron-sulfur cluster formation in skin cells from people with Friedreich's ataxia.

  NIH research Johns Hopkins University · Baltimore, United States · NIH-11342528

• Therapeutic Gene Editing for Friedreich’s Ataxia

  A gene-editing approach using AAV-based delivery aims to boost frataxin (FXN) protein in Friedreich’s Ataxia patients, potentially slowing disease progression.

  NIH research Jackson Laboratory · Bar Harbor, United States · NIH-11326250

• Blocking the p38 stress protein to help people with Friedreich ataxia

  Looks at whether blocking the p38 stress-response protein can improve cell health and reduce nerve and heart damage in people with Friedreich ataxia.

  NIH research Children's Hosp of Philadelphia · Philadelphia, United States · NIH-11325344

• How PKC gamma mutations cause cerebellar ataxia (SCA14)

  Researchers are working to understand how inherited changes in the brain protein PKC gamma lead to spinocerebellar ataxia type 14 so future treatments can be developed for people with that condition.

  NIH research University of California, San Diego · La Jolla, United States · NIH-11297231

• Understanding why cognitive problems happen in Spinocerebellar Ataxia Type 1

  This project looks at why thinking and memory decline occur in people with SCA1 and how brain regions and cells contribute to those problems.

  NIH research University of Minnesota · Minneapolis, United States · NIH-11296198

• How spinocerebellar ataxia type 12 affects the brain and movement

  Researchers are looking at how a CAG repeat in the PPP2R2B gene changes RNA and protein forms in people with SCA12 to explain tremor, walking problems, and brain loss.

  NIH research Johns Hopkins University · Baltimore, United States · NIH-11263661

• How Ataxia Telangiectasia harms the cerebellum

  Researchers are looking at whether losing two DNA-repair proteins causes the cerebellum damage that leads to ataxia in children and young adults with Ataxia Telangiectasia.

  NIH research Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center · Torrance, United States · NIH-11262941

• Microglia and a stem-cell gene therapy for Friedreich's ataxia

  This project is developing a gene-corrected blood stem-cell treatment to restore frataxin and help people with Friedreich's ataxia.

  NIH research University of California, San Diego · La Jolla, United States · NIH-11239779

• Home wearable movement monitoring for Ataxia-Telangiectasia

  This project uses small wearable sensors to track everyday movements in children and adults with Ataxia-Telangiectasia to spot meaningful changes in motor function.

  NIH research Massachusetts General Hospital · Boston, United States · NIH-11239081

• Tracking changes and causes of Fragile X-associated tremor/ataxia syndrome (FXTAS)

  Researchers will follow people who carry FMR1 premutations—some with FXTAS, some without, and healthy volunteers—over two years to find brain, movement, and molecular markers linked to disease changes.

  NIH research University of Kansas Lawrence · Lawrence, United States · NIH-11239018

• Understanding Spinocerebellar Ataxia Type 5

  This research explores how changes in a specific gene lead to spinocerebellar ataxia type 5 (SCA5) and damage brain cells, aiming to find new ways to help patients.

  NIH research Oakland University · Rochester, United States · NIH-11221203

• Making gene therapy safer and more effective for Friedreich's ataxia

  Looks at using immune-calming strategies to help AAV gene therapy work better and be safer for people with Friedreich's ataxia.

  NIH research University of Florida · Gainesville, United States · NIH-11179356

• Motor neuron causes of weakness and breathing problems in spinocerebellar ataxia type 1

  Seeing if damage to motor neurons explains muscle weakness, trouble breathing and early death in people with spinocerebellar ataxia type 1.

  NIH research Baylor College of Medicine · Houston, United States · NIH-11169736

• How GEMIN5 gene changes cause a newly described cerebellar ataxia

  This project looks at whether GEMIN5 gene changes damage nerve-cell splicing machinery and whether treatments like CoQ10 can help people with a new form of cerebellar ataxia.

  NIH research University of Pittsburgh at Pittsburgh · Pittsburgh, United States · NIH-11167800

• Understanding how a gene called Tox3 affects brain development and movement problems like ataxia

  This project explores how a specific gene, TOX3, helps build an important part of the brain called the cerebellum, which affects balance and movement.

  NIH research Cedars-Sinai Medical Center · Los Angeles, United States · NIH-11134661

• Understanding how astrocytes contribute to Spinocerebellar ataxia type 1

  This study is looking at how certain brain cells called astrocytes are affected by a gene mutation in people with Spinocerebellar ataxia type 1 (SCA1), to better understand how this might worsen the disease and find new ways to help.

  NIH research University of Minnesota · Minneapolis, United States · NIH-11127776

• How alcohol affects balance, walking, and thinking in people with mild cognitive impairment

  This project compares how different levels of alcohol use affect balance, walking speed, and cognitive decline in people with mild cognitive impairment (MCI) and similar people without MCI.

  NIH research Stanford University · Stanford, United States · NIH-11123318

• Understanding Brain Changes in Spinocerebellar Ataxia Type 1

  This project aims to understand why diseases like Spinocerebellar Ataxia Type 1 and Alzheimer's disease first affect specific parts of the brain.

  NIH research Baylor College of Medicine · Houston, United States · NIH-11123147

• Gene editing to treat Friedreich's Ataxia

  This study is exploring a new way to boost a protein called frataxin in people with Friedreich's Ataxia, a condition that affects the nervous system, by using a special delivery method to help improve their health and possibly slow down or even reverse the disease.

  NIH research Jackson Laboratory · Bar Harbor, United States · NIH-11089354

• Understanding how lipid imbalances affect brain cell development in Spinocerebellar ataxia type 3

  This study is looking at how problems with fats in the body affect the growth of important brain cells called oligodendrocytes, which help protect nerve fibers, in people with Spinocerebellar ataxia type 3 (SCA3), to find new ways to help these cells work better and slow down the disease.

  NIH research University of Michigan at Ann Arbor · Ann Arbor, United States · NIH-11063535

• Understanding how cerebellar nuclei dysfunction affects movement in Spinocerebellar Ataxia Type 1

  This study is looking at how certain parts of the brain called cerebellar nuclei affect movement problems in people with Spinocerebellar Ataxia Type 1 (SCA1), hoping to find new ways to help improve balance and walking for those living with this condition.

  NIH research University of Minnesota · Minneapolis, United States · NIH-11056262

• Understanding how R-loops affect Ataxia Telangiectasia

  This study is looking at how certain structures in our cells, called R-loops, and a gene called ATM are connected to Ataxia Telangiectasia (AT), a condition that affects movement and coordination, to find new ways to help people with this disease.

  NIH research Emory University · Atlanta, United States · NIH-11051880

• Targeting p38 MAPK to treat Friedreich ataxia

  This study is looking at how a specific pathway in the body might be making Friedreich ataxia worse, and it hopes to find new treatments that could help improve symptoms and slow down the disease for those living with it.

  NIH research Children's Hosp of Philadelphia · Philadelphia, United States · NIH-11049076

• Targeting the frataxin gene to treat Friedreich's ataxia

  This study is exploring a new way to help people with Friedreich's ataxia by using special techniques to fix the gene that causes the condition, which could lead to improvements in their symptoms.

  NIH research Florida International University · Miami, United States · NIH-11036612

Related topics

Quick answers

How many Ataxia clinical trials are recruiting right now?

10, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 8 countries. The most sites are in United States (5 trials), Germany (3 trials), France (2 trials), Italy (2 trials), Australia (1 trial). Each study page lists its sites and which ones are recruiting.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

Find the studies that fit you

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