Clinical trials: Cerebellar Ataxias
Mitochondrial Diseases
EPI-743 for Mitochondrial Respiratory Chain Diseases
This study will evaluate the safety and efficacy of EPI-743 in participants with severe mitochondrial respiratory chain diseases who are considered to be within 90 days of end-of-life care.
- Trial name
- NCT01370447
- Testing
- EPI-743
- Where
- 16 sites in United States
- Sponsor
- PTC Therapeutics
Friedreich's Ataxia
Safety and Pharmacology Study of VP 20629 in Adults With Friedreich's Ataxia
The objectives of the study are: To evaluate the safety and tolerability of single and multiple oral doses of VP 20629 in subjects with Friedreich's ataxia (FA).
- Trial name
- NCT01898884
- Testing
- VP 20629
- Where
- 5 sites in United States
- Volunteers
- 46 people
- Sponsor
- Shire
Friedreich's Ataxia (FA)
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
This is a phase 1b, first in-human, open-label, dose-finding study investigating the safety and tolerability of SGT-212 in participants with Friedreich's ataxia (FA).
- Trial name
- FALCON (NCT07180355)
- Testing
- SGT-212
- Where
- 3 sites in United States
- Volunteers
- 10 people
- Sponsor
- Solid Biosciences Inc.
Friedreich Ataxia
Characterization of the Interruptions of the GAA Expansion and Study of Their Influence on the Severity of Friedreich's Ataxia
Friedreich's ataxia (FA) is the most frequent recessive genetic ataxia with an estimated prevalence of 1/50 000. The first symptoms appear around the age of 10 years with a progressive course and the need for an armchair 10- 15…
- Trial name
- INTREP-AF (NCT04346238)
- Testing
- Reuse of existing data from patients' medical records
- Where
- France
- Volunteers
- 120 people
- Sponsor
- University Hospital, Montpellier
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
Friedreich Ataxia
Idebenone to Treat Friedreich's Ataxia
This study will determine whether a drug called idebenone is safe and effective in reducing the level of oxidants that are believed to damage the nervous system and hearts in patients with Friedreich's ataxia.
- Trial name
- NCT00229632
- Testing
- Idebenone
- Where
- United States
- Volunteers
- 51 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Autoimmune Encephalitis, Imbic Encephalitis
HLA Analysis in Autoimmune Encephalitis and Related Disorders
Autoimmune encephalitis (AE) are characterized by subacute onset of memory deficits, altered mental status or psychiatric symptoms, frequently associated with seizures, inflammatory cerebrospinal fluid and in cases with prominent…
- Conditions
- Autoimmune Encephalitis, Imbic Encephalitis, Autoimmune Cerebellar Ataxia, Stiff-person Syndrome
- Trial name
- ICARE (NCT04106596)
- Where
- France
- Volunteers
- 160 people
- Sponsor
- Hospices Civils de Lyon
Friedreich's Ataxia
A Study Investigating the Safety and Tolerability of Deferiprone in Patients With Friedreich's Ataxia
The primary objective of this study is to demonstrate the safety and tolerability of deferiprone in subjects with Friedreich's ataxia (FRDA).
- Trial name
- NCT00530127
- Testing
- deferiprone
- Where
- 6 sites in Australia, Belgium and 4 more
- Volunteers
- 80 people
- Sponsor
- ApoPharma
Ataxia Telangiectasia
Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With a Novel Form of Anaplerosis
Study design: Parallel group, placebo-controlled, dose-escalation each 2 months for 12 months. Dose based on percent (%) of calculated caloric intake.
- Trial name
- A-TC7 (NCT04513002)
- Testing
- Triheptanoin
- Where
- Australia
- Volunteers
- 30 people
- Sponsor
- The University of Queensland
Hereditary Ataxia
Telerehabilitation for Core Stability and Strength in Hereditary Ataxia
This is a two-arm, controlled clinical trial with a parallel design. Participants will be randomly assigned to an intervention or control group.
- Trial name
- TRCORE-AH2 (NCT07200505)
- Testing
- TR Mixed CORE Exercices, TR Asyncronous CORE Exercices
- Where
- Spain
- Volunteers
- 24 people
- Sponsor
- Universitat de Lleida
Cerebellar Ataxia
Augmenting Balance in Individuals With Cerebellar Ataxias
This study evaluates the effectiveness of a 12-week in home balance training program with and without sensory augmentation for individuals with ataxia.
- Trial name
- NCT02887703
- Testing
- Sensory Augmentation
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Kathleen Sienko
Spinocerebellar Ataxia Type 3, Machado-Joseph Disease
Astrocytic Markers and the Pre-ataxic Period of SCA3/MJD - BIGPRO Study Astrocytes
The study will consist of a prospective observation of subjects in a natural history design. The investigators will monitor changes of clinical scales, quality of life, messenger ribonucleic acid (mRNA) of candidate genes (CCL11…
- Conditions
- Spinocerebellar Ataxia Type 3, Machado-Joseph Disease, SCA3, MJD
- Trial name
- NCT04419974
- Testing
- Molecular Diagnosis, Clinical Scales - Baseline, Blood Draw - Baseline
- Where
- Brazil
- Volunteers
- 95 people, healthy volunteers welcome
- Sponsor
- Hospital de Clinicas de Porto Alegre
Cerebellar Ataxia
Dual Task Training for Cerebellar Ataxia
Combining cognitive training with physical training to improve balance is a new approach for reducing the risk of falls in patient populations who are at risk for falls.
- Trial name
- NCT04648501
- Testing
- Dual-task training
- Where
- Hong Kong
- Volunteers
- 36 people
- Sponsor
- The Hong Kong Polytechnic University
Cerebellar Ataxia
Effects of Deep Brain Stimulation of the Dentate Nucleus on Cerebellar Ataxia
Cerebellar ataxias are a group of disorders caused by cerebellar affections, for which currently no specific treatment is available.
- Trial name
- NCT03341416
- Testing
- deep brain stimulation on stimulation - active
- Where
- Brazil
- Volunteers
- 10 people
- Sponsor
- University of Sao Paulo General Hospital
Mild Cognitive Impairment (MCI), Alzheimer Disease (AD)
Biomarkers in Neurodegenerative Diseases
The general purpose of this observational study is to examine biomarkers associated with the pathology of neurodegenerative diseases to potentially develop novel therapeutic approaches.
- Conditions
- Mild Cognitive Impairment (MCI), Alzheimer Disease (AD), Dementia With Lewy Bodies (DLB), Frontotemporal Lobar Degeneration (FTLD)
- Trial name
- NCT04055532
- Testing
- Neuraceq
- Where
- United States
- Sponsor
- University of Minnesota
Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 7
Integrated Functional Evaluation of the Cerebellum
One of the main objectives of this project is to validate potential biological, clinical and/or imaging biomarkers in SCA patients through a multimodal assessment, for future ASOs trials.
- Trial name
- CERMOI (NCT04288128)
- Testing
- Lumbar puncture, Magnetic Resonance Imaging (MRI)
- Where
- France
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Friedreich's Ataxia
Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia
The purpose of this study is to examine the effects of EPI-743 on visual function and neurologic function in patients with Friedreich's ataxia.
- Trial name
- NCT01728064
- Testing
- EPI-743 400 mg, EPI-743 200 mg
- Where
- 3 sites in United States
- Volunteers
- 60 people
- Sponsor
- PTC Therapeutics
Ataxia
Effects of Cerebellar tACS-iTBS in Ataxia
Ataxia refers to a group of neurological disorders characterized by impaired coordination and balance due to dysfunction in the cerebellum or its connections.
- Trial name
- EtABeta (NCT06420271)
- Testing
- Real (real iTBS/tACS + exergaming)
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- I.R.C.C.S. Fondazione Santa Lucia
Spinocerebellar Ataxia Type 3
Efficacy Of Oral Trehalose In Spinocerebellar Ataxia 3
This study evaluates the effectiveness of oral trehalose in alleviating the neuropathological and motor behaviour deficits among patients with SCA3.
- Trial name
- NCT04399265
- Testing
- Trehalose, Maltose
- Where
- Malaysia
- Volunteers
- 20 people
- Sponsor
- National University of Malaysia
Machado-Joseph Disease
Weight in Lower Limbs Improves Gait Ataxia of in Machado-Joseph Disease Patients
Few studies evaluated the efficacy of adding weights on the lower limbs in patients with ataxic disorders. There is no current evidence on which would be the most appropriate weight for use in this context.
- Trial name
- NCT02906046
- Testing
- Weight in Lower Limbs
- Volunteers
- 20 people
- Sponsor
- Federal University of Health Science of Porto Alegre