Clinical trials: Spinocerebellar Ataxia
Hereditary Cerebellar Ataxia.
A New Method to Treat Hereditary Cerebellar Ataxia - Umbilical Cord Mesenchymal Stem Cells Transplantation
Hereditary cerebellar ataxia is a type of autosomal dominant genetic disease, lesions mainly involving the cerebellum, but the spinal cord and cranial nerves may also be some involvement.
- Trial name
- SCA (NCT01489267)
- Testing
- stem cell transplantation
- Where
- China
- Volunteers
- 20 people
- Sponsor
- General Hospital of Chinese Armed Police Forces
Hereditary Ataxia
Hereditary Ataxia Research on Multi-Omics and Neuroclinical Insights in the Yangtze Delta
The goal of this observational study is to explore the clinical and genetic characteristics, multi-omics profiles, disease mechanisms, biomarkers, and potential therapeutic targets of hereditary ataxia (HA) in patients diagnosed…
- Trial name
- HARMONY (NCT07092358)
- Testing
- None of intervention
- Where
- 2 sites in China
- Volunteers
- 5,000 people
- Sponsor
- Second Affiliated Hospital, Zhejiang University, School of Medicine
Machado Joseph Disease (SCA3)
The Influence of Deep TMS on Cerebellar Signs in Patients With Machado Joseph Disease
To test the effects of low frequency deep rTMS using the novel HCERMJD-coil on cerebellar deficits in patients with SCA3 and to establish its safety in this population.
- Trial name
- NCT02039206
- Testing
- Deep TMS
- Where
- Israel
- Volunteers
- 20 people
- Sponsor
- Brainsway
Friedreich Ataxia
Evaluation of the Effect of Artesunate in Friedreich Ataxia (FA)
This dose-escalation study is aimed at investigating a novel application for artesunate in the treatment of Friedreich ataxia. It will evaluate this novel application of oral artesunate using a surrogate biological marker as…
- Trial name
- ARTEMIS (NCT04921930)
- Testing
- Artesunate Oral Product
- Where
- France
- Volunteers
- 20 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Friedreich Ataxia
Coronary Artery Disease in Patients With Friedreich's Ataxia
Friedreich's ataxia is a debilitating, inherited disease cause by mutations in a protein called frataxin (FXN). FXN is one of several proteins that controls the production of iron-sulfur clusters, molecules that are essential for…
- Trial name
- NCT04649866
- Testing
- plethysmography
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- University of Pittsburgh
Transcranial Magnetic Stimulation, Spinocerebellar Ataxias
rTMS Improves Functions in Spinocerebellar Ataxia
Spinocerebellar ataxia (SCA) is a group of inherited brain disorders. SCA often result in poor limb coordination. This study aims to discover the effects of repeated transcranial magnetic stimulation (rTMS) on balance \& gait in…
- Trial name
- NCT05973019
- Testing
- Repetitive Transcranial Magnetic Stimulation (rTMS)
- Where
- 2 sites in Taiwan
- Volunteers
- 10 people
- Sponsor
- Changhua Christian Hospital
Neurologic Disorder, Stroke
Home Based Tele-exercise for People With Chronic Neurological Impairments
To assess the impact of a 12-week virtual seated physical intervention on cardiovascular health and wellness in people with chronic neurological impairments (CNI).
- Conditions
- Neurologic Disorder, Stroke, Spinal Cord Injuries, Cerebral Palsy
- Trial name
- Telex (NCT04564495)
- Testing
- Seated exercise
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Burke Medical Research Institute
Friedreich Ataxia
A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia
In this study, researchers are learning more about RTA 408, also known as omaveloxolone, BIIB141, or SKYCLARYS®. The main goal of this study is to learn more about the safety of RTA 408 and how it affects physical effort…
- Trial name
- NCT02255435
- Testing
- Omaveloxolone Capsules, 2.5 mg, Omaveloxolone Capsules, 5 mg, Omaveloxolone Capsules, 10 mg
- Where
- 11 sites in United States, Australia and 3 more
- Volunteers
- 172 people
- Sponsor
- Biogen
Friedreich Ataxia
Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia
The purpose of this study is to evaluate the efficacy of TAK-831 versus placebo on upper extremity (arm and hands) motor function and manual dexterity.
- Trial name
- NCT03214588
- Testing
- TAK-831
- Where
- 6 sites in United States
- Volunteers
- 67 people
- Sponsor
- Neurocrine Biosciences
Neurodegenerative Disorders
Development of IPS from Donated Somatic Cells of Patients with Neurological Diseases
Human fibroblasts and possibly other human somatic cells may be reprogrammed into induced pluripotent stem (iPS) cells by the forced expression of transcription factors (1-5).
- Trial name
- NCT00874783
- Where
- Israel
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Hadassah Medical Organization
Spinocerebellar Ataxia 3
Machado-Joseph Disease in Israel
Machado-Joseph disease (MJD) or spinocerebellar ataxia type 3 (SCA-3) is the most common dominant ataxia. The genetic cause of this late-onset degenerative disorder is the expansion of a (CAG)n tract located in the exonic region…
- Trial name
- NCT02175290
- Where
- Israel
- Volunteers
- 250 people
- Sponsor
- Meir Medical Center
Friedreich Ataxia
A Study to Learn How BIIB141 (Omaveloxolone) Affects the Health of Participants With Friedrich's Ataxia Who Took it During Pregnancy and/or During Breastfeeding and About the Health of Their Babies
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 Friedrich's Ataxia, also known as FA.
- Trial name
- NCT06628687
- Testing
- Omaveloxolone
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Biogen
Friedreich's Ataxia, Motor Disorders
Electroencephalogram in Patients With Friedreich's Ataxia for the Study of the Structural and Functional Connectome.
Prospective, exploratory, multicenter pilot study investigating the structural and functional connectome in patients with Friedreich's Ataxia (FRDA) using high-density electroencephalogram (HD-EEG).
- Trial name
- CONNETTOMA (NCT07095062)
- Testing
- HD-EEG recordings, combined with cognitive and motor assessment
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- IRCCS Eugenio Medea
Ataxia Telangiectasia, Leukemia
Treatment of Leukemia and Lymphoma in Children With Ataxia Telangiectasia
Ataxia telangiectasia (A-T) is a multisystem disease with diverse manifestations, including progressive neurodegeneration, immunodeficiency, respiratory disease, and genomic instability.
- Conditions
- Ataxia Telangiectasia, Leukemia, Lymphoma
- Trial name
- NCT04037189
- Where
- Israel
- Volunteers
- 250 people
- Sponsor
- Rabin Medical Center
Movement Disorder
Phenotype/Genotype Correlations in Movement Disorders
The goal of this protocol is to identify families with inherited movement disorders and evaluate disease manifestations to establish an accurate clinical diagnosis by using newest technological advances and investigate the…
- Trial name
- NCT00018889
- Where
- United States
- Volunteers
- 2,500 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 3
A Safety and Pharmacokinetics Trial of VO659 in SCA1, SCA3 and HD
The goal of this first-in-human clinical trial is to assess the safety and tolerability of four doses of a new study drug called VO659 in people with genetic disorders called spinocerebellar ataxia type 1, type 3 or Huntington's…
- Conditions
- Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 3, Huntington Disease
- Trial name
- NCT05822908
- Testing
- VO659
- Where
- 14 sites in Denmark, France and 4 more
- Volunteers
- 68 people
- Sponsor
- Vico Therapeutics B. V.
Friedreich Ataxia
An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia
An open label study designed to evaluate the safety, PK, PD, and clinical effects of long-term daily administration of CTI-1601 enrolling adolescent and adult patients with FRDA who have participated in a prior clinical study of…
- Trial name
- NCT06447025
- Testing
- CTI-1601
- Where
- 10 sites in United States
- Volunteers
- 85 people
- Sponsor
- Larimar Therapeutics, Inc.
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
Inclusion Body Myositis, Myotonic Dystrophy 1
The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.
The planned project is an intervention study to assess the risk of falling after adaptation of an assistive gait devices in patients with the following neuromuscular diseases: Inclusion body myositis, myotonic dystrophy, limb…
- Conditions
- Inclusion Body Myositis, Myotonic Dystrophy 1, Myotonic Dystrophy 2, Facio-Scapulo-Humeral Dystrophy
- Trial name
- Risk of fall (NCT07072676)
- Testing
- Assistive gait devices combined with physiotherapy
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- LMU Klinikum
Hereditary Spastic Paraplegia, Spastic Ataxia
TreatHSP Platform: Adaptive Natural History Platform for Ataxias, HSPs, and Spastic Ataxias
Ataxias, hereditary spastic paraplegias (HSP), and spastic ataxias (collectively referred to as SPAX diseases) are rare neurological conditions that cause progressive problems with walking, balance, coordination, and daily…
- Trial name
- TreatHSP (NCT07798674)
- Where
- 30 sites in Austria, Czechia and 7 more
- Volunteers
- 4,000 people, healthy volunteers welcome
- Sponsor
- Heidelberg University