Clinical trials: Ataxia
Friedreich Ataxia
Digital Outcome Assessment Using AI Active Gaming and Motion Capture in Friedreich Ataxia
Friedreich ataxia (FA) is a rare, inherited condition that progressively affects balance, coordination, and walking. Clinical trials of new FA treatments rely largely on standard clinical rating scales, but these can be tiring…
- Trial name
- STEP-OUT FA (NCT07731971)
- Testing
- Laboratory validation session (VSimulator), Home-based active gaming assessment
- Where
- United Kingdom
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- University of Exeter
Friedreich Ataxia
A Study to Find Out How BIIB141 (Omaveloxolone) is Processed in the Body and to Learn More About Its Safety in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old
In this study, researchers will learn more about BIIB141, also known as omaveloxolone or SKYCLARYS®. This drug has been approved, or made available for doctors to prescribe, for people with Friedrich's Ataxia (FA) who are at…
- Trial name
- BOLD (NCT06054893)
- Testing
- Omaveloxolone
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- Biogen
Coordination Impairment, Children
Motor-Cognitive Training , Cognitive Performance and Balance
This RCT design investigates the effects of an 8-week motor-cognitive training program on cognitive performance and balance in preschool-aged children.
- Conditions
- Coordination Impairment, Children, Balance
- Trial name
- NCT07045922
- Testing
- Motor-Cognitive Training Program, Regular Physical education classes
- Where
- Turkey (Türkiye)
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Princess Nourah Bint Abdulrahman University
Ataxia, Gait
Ataxia GAA-FGF14 - Descriptive Genetic and Clinical Study
Cerebellar ataxias of late onset are of undetermined etiology in many cases. A new cause of late-onset cerebellar ataxia was discovered in January 2023 corresponding to an expansion of GAA triplets in intron 1 of the FGF14 gene.
- Trial name
- GAA-FGF14 (NCT05884086)
- Where
- France
- Volunteers
- 20 people
- Sponsor
- Central Hospital, Nancy, France
Motor Neuron Diseases, Spinocerebellar Ataxias
Rare Disease Registry Database for Movement Disorders in Southern Anhui Province
According to the World Health Organization (WHO) definition, a rare disease is a disease affecting fewer than 6.5 people per 10,000; in China, it generally refers to diseases with a prevalence of less than 1/10,000 (i.e., with a…
- Conditions
- Motor Neuron Diseases, Spinocerebellar Ataxias, Hereditary Muscular Dystrophy, Hereditary Spastic Paraplegia
- Trial name
- W-MoSCA (NCT07838883)
- Where
- China
- Volunteers
- 180 people
- Sponsor
- First Affiliated Hospital of Wannan Medical College
Friedreich Ataxia
Efficacy Study of Epoetin Alfa in Friedreich Ataxia
Friedreich's ataxia (FRDA) is a rare genetic disorder characterised by severe neurological disability and cardiomyopathy. Friedreich's ataxia is the consequence of frataxin deficiency.
- Trial name
- FRIEMAX (NCT01493973)
- Testing
- Epoetin alfa
- Where
- 3 sites in Italy
- Volunteers
- 56 people
- Sponsor
- Federico II University
Spastic Ataxia
Phenotypes, Biomarkers and Pathophysiology in Spastic Ataxias
The aim of this study is to determine the clinical spectrum and natural progression of Spastic Ataxias (SPAX) and related disorders in a prospective multicenter natural history study, identify digital, imaging and molecular…
- Trial name
- SPAX-PBP (NCT04297891)
- Testing
- Clinical rating scale to measure Ataxia disease severity and progression, Clinical rating scale to measure spastic paraplegia disease severity and progression, Disease-specific severity index for adults with autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS)
- Where
- 9 sites in Canada, France and 5 more
- Volunteers
- 250 people, healthy volunteers welcome
- Sponsor
- Dr. Rebecca Schule
Spinocerebellar Ataxia
Safety and Efficacy of Intravenous Immune Globulin in Treating Spinocerebellar Ataxia
This is a preliminary study to determine the safety and efficacy of intravenous immune globulin in treating Spinocerebellar Ataxia.
- Trial name
- NCT01350440
- Testing
- IVIG
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- University of South Florida
Episodic Ataxia Syndrome, Cerebellar Diseases
Characteristics of Episodic Ataxia Syndrome
Episodic ataxia (EA) is a rare genetic disease characterized by episodes of imbalance, incoordination, and slurring of speech. The underlying cause of EA is only partly understood, and currently there are no established…
- Trial name
- NCT00266760
- Where
- 6 sites in United States, Canada and 1 more
- Volunteers
- 39 people
- Sponsor
- University of California, Los Angeles
Friedreich Ataxia
A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it by Their Own Doctors
In this study, researchers will learn more about the safety of BIIB141, also known as omaveloxolone or SKYCLARYS®. This is a drug available for doctors to prescribe for people with Friedreich's Ataxia, also known as FA.
- Trial name
- NCT06623890
- Testing
- Omaveloxolone
- Where
- 14 sites in United States, Austria and 5 more
- Volunteers
- 300 people
- Sponsor
- Biogen
Friedreich Ataxia
FRDA Investigator Initiated Study (IIS) With Elamipretide
To evaluate the safety, tolerability, and activity of Elamipretide in treating vision loss in Friedreich Ataxia (FRDA).
- Trial name
- ELViS-FA (NCT05168774)
- Testing
- Elamipretide
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Children's Hospital of Philadelphia
Friedreich Ataxia
Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia
The primary objective is to evaluate the potential effectiveness of an individualized intensive rehabilitation intervention using the "Prokin 252" stabilometric platform in the treatment of adolescent and adult patients with…
- Trial name
- NCT06692296
- Testing
- Balance training over stabilometric platform, Balance training by means of conventional rehabilitation
- Where
- Italy
- Volunteers
- 24 people
- Sponsor
- IRCCS Eugenio Medea
Friedreich's Ataxia
Patient Registry of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS)
This is a multi-centre, multi-national, prospective, observational study of Friedreich's Ataxia (FRDA) with a control group to: obtain natural history data on individuals affected by FRDA relate clinical assessments and results…
- Trial name
- EFACTS (NCT02069509)
- Where
- 16 sites in Austria, Belgium and 8 more
- Volunteers
- 1,200 people, healthy volunteers welcome
- Sponsor
- European Friedreich's Ataxia Consortium for Translational Studies
Friedreich's Ataxia
Biomarkers in Friedreich's Ataxia
The purpose of this project is to characterize measures of cardiac performance and neuromuscular physiology in FA patients using novel techniques, including echocardiography and magnetic resonance imaging (MRI), metabolic…
- Trial name
- NCT02497534
- Where
- United States
- Volunteers
- 203 people, healthy volunteers welcome
- Sponsor
- University of Florida
Angelman Syndrome, Nervous System Diseases
Dietary Supplements for the Treatment of Angelman Syndrome
Angelman syndrome (AS) is a complex genetic disorder that affects the nervous system. The purpose of this study is to determine the effectiveness of certain dietary supplements in treating the symptoms of AS.
- Trial name
- NCT00348933
- Testing
- Betaine, Creatine, Metafolin
- Where
- 4 sites in United States
- Volunteers
- 90 people
- Sponsor
- University of California, San Diego
Friedreich's Ataxia
Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)
The purpose of this study is to confirm the treatment effects of vatiquinone on the key measures of FA disease progression.
- Trial name
- PROVE-FA (NCT07681713)
- Testing
- Vatiquinone
- Where
- 9 sites in United States, Belgium and 4 more
- Volunteers
- 120 people
- Sponsor
- PTC Therapeutics
Ataxia Telangiectasia
Whole Body MRI for Cancer Surveillance in A-T
Ataxia Telangiectasia (A-T) is an inherited disorder characterised by cerebellar neurodegeneration, immunodeficiency and respiratory disease.
- Trial name
- NCT05252819
- Testing
- Magnetic Resonance Imaging and an optional blood test
- Where
- United Kingdom
- Volunteers
- 37 people
- Sponsor
- University of Nottingham
Autosomal Recessive Cerebellar Ataxia
Validation of the RADIAL Algorithm for Diagnosis of Autosomal Recessive Cerebellar Ataxia
RADIAL is an algorithm which has been developed following a review of the literature on 67 autosomal recessive cerebellar ataxias (ARCA) and personal clinical experience.
- Trial name
- RADIAL-VALID (NCT04261127)
- Testing
- Genetic diagnosis (PMDA panel), Use of RADIAL algorithm
- Where
- 8 sites in France
- Volunteers
- 400 people, healthy volunteers welcome
- Sponsor
- University Hospital, Strasbourg, France
Friedreich's Ataxia
Patient Reported Outcomes in Friedreich's Ataxia Patients After Withdrawal From Treatment With Idebenone (PROTI)
This is a Phase IIIb Double-Blind, Randomised, Placebo-Controlled Study. The aim is to further investigate the effects of idebenone in patients with Friedreich's ataxia.
- Trial name
- PROTI (NCT01303406)
- Testing
- Idebenone
- Where
- 6 sites in Austria, Germany and 2 more
- Volunteers
- 29 people
- Sponsor
- Santhera Pharmaceuticals
Localized Resectable Neuroblastoma, Localized Unresectable Neuroblastoma
Cyclophosphamide and Prednisone With or Without Immunoglobulin in Treating Abnormal Muscle Movement in Children With Neuroblastoma
This randomized phase III trial is studying cyclophosphamide, prednisone, and immunoglobulin to see how well they work compared to cyclophosphamide and prednisone alone in treating patients with abnormal trunk muscle movements…
- Conditions
- Localized Resectable Neuroblastoma, Localized Unresectable Neuroblastoma, Regional Neuroblastoma, Stage 4 Neuroblastoma
- Trial name
- NCT00033293
- Testing
- Clinical Observation, Cyclophosphamide, Laboratory Biomarker Analysis
- Where
- 104 sites in United States, Australia and 1 more
- Volunteers
- 53 people
- Sponsor
- Children's Oncology Group