Clinical trials: Ataxia, Cerebellar
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
Friedreich Ataxia
A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
The primary objective of the study is to evaluate the efficacy (using the modified Friedreich Ataxia Rating Scale [mFARS]) and safety of vatiquinone in participants with Friedreich ataxia (FA).
- Trial name
- MOVE-FA (NCT04577352)
- Testing
- Vatiquinone
- Where
- 14 sites in United States, Australia and 7 more
- Volunteers
- 146 people
- Sponsor
- PTC Therapeutics
Friedreich's Ataxia
A First in Human Study of RT001 in Patients With Friedreich's Ataxia
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of RT001 in patients with Friedreich's ataxia.
- Trial name
- NCT02445794
- Testing
- Low dose cohort, High dose cohort
- Where
- 2 sites in United States
- Volunteers
- 19 people
- Sponsor
- Biojiva LLC
Cerebellar Ataxia
Training in Ataxia - Individuals With Degenerative Cerebellar Diseases
Balance and aerobic training show promise as treatments for degenerative cerebellar diseases, but the neural effects of both training methods are unknown.
- Trial name
- NCT05002218
- Testing
- Aerobic Training, Balance Training
- Where
- United States
- Volunteers
- 64 people
- Sponsor
- Columbia University
Friedreich Ataxia
Neurology Measures in FA Children
The purpose of this study is to identify ways to follow progression of Friedreich's Ataxia (FA) and be able to measure changes over time in children with FA.
- Trial name
- NCT03418740
- Where
- 3 sites in United States
- Volunteers
- 108 people
- Sponsor
- Children's Hospital of Philadelphia
Spinocerebellar Ataxias, Spinocerebellar Ataxia Type 1
Troriluzole in Adult Participants With Spinocerebellar Ataxia
The purpose of this study is to compare the efficacy of Troriluzole (200 mg once daily) versus placebo after 48 weeks of treatment in subjects with spinocerebellar ataxia (SCA).
- Conditions
- Spinocerebellar Ataxias, Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3
- Trial name
- NCT03701399
- Testing
- troriluzole
- Where
- 23 sites in United States, China
- Volunteers
- 299 people
- Sponsor
- Biohaven Pharmaceuticals, Inc.
Wolfram Syndrome, Diabetes Mellitus
A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome
Wolfram syndrome is a rare genetic disorder characterized by juvenile-onset diabetes mellitus, diabetes insipidus, optic nerve atrophy, hearing loss, and neurodegeneration.
- Conditions
- Wolfram Syndrome, Diabetes Mellitus, Optic Nerve Atrophy, Ataxia
- Trial name
- NCT02829268
- Testing
- dantrolene sodium
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Washington University School of Medicine
Siderosis, Neurological Disorder
Audiovestibular Function in Infratentorial Superficial Siderosis
One in six people in the United Kingdom and over 400 million people worldwide have disabling hearing loss. This figure will double by 2050 as predicted by the World Health Organisation.
- Conditions
- Siderosis, Neurological Disorder, Rare Diseases, Age Related Hearing Loss
- Trial name
- AViSS (NCT04200664)
- Testing
- Hearing assessment, Vestibular/balance assessment, Quality of life assessment
- Where
- 2 sites in United Kingdom
- Volunteers
- 11 people, healthy volunteers welcome
- Sponsor
- University College, London
Fragile X-associated Tremor/Ataxia Syndrome
Treatment of Fragile-X Associated Tremor/Ataxia Syndrome (FXTAS) With Allopregnanolone
The purpose of this study is to examine the safety and efficacy of Allopregnanolone as a possible treatment for symptoms of Fragile X-associated Tremor/Ataxia Syndrome (FXTAS).
- Trial name
- NCT02603926
- Testing
- Allopregnanolone
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Randi J. Hagerman, MD
Friedreich Ataxia
NAD+ Precursor Supplementation in Friedreich's Ataxia
The primary objective is to test the safety and tolerability of short-term therapy with a nicotinamide adenine dinucleotide (NAD+) precursor (MIB-626) in adults with Friedreich's Ataxia (FA) without overt heart failure and with a…
- Trial name
- NCT04817111
- Testing
- MIB-626
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Metro International Biotech, LLC
Advanced Stage Solid Tumors
ATRi Transition Rollover Study
The main purpose of the study was to monitor the safety of participant receiving long-term treatment of Berzosertib as monotherapy.
- Trial name
- NCT03309150
- Testing
- Berzosertib
- Where
- United Kingdom
- Volunteers
- 1 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Ataxia, Cerebellar, Cerebellar Ataxia
Rehabilitative Trial With Cerebello-Spinal tDCS in Neurodegenerative Ataxia
Neurodegenerative cerebellar ataxias represent a group of disabling disorders for which we currently lack effective therapies. Cerebellar transcranial direct current stimulation (tDCS) is a non-invasive technique, which has been…
- Conditions
- Ataxia, Cerebellar, Cerebellar Ataxia, Spinocerebellar Ataxias, Ataxia, Spinocerebellar
- Trial name
- CStDCSAtaxia (NCT03120013)
- Testing
- Anodal cerebellar and cathodal spinal tDCS
- Where
- Italy
- Volunteers
- 21 people
- Sponsor
- Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia
Cerebellar Ataxia, Spinocerebellar Ataxias
Using Real-time fMRI Neurofeedback and Motor Imagery to Enhance Motor Timing and Precision in Cerebellar Ataxia
The aim of the research is to improve motor function in people with cerebellar ataxia by using neuroimaging methods and mental imagery to "exercise" motor networks in the brain.
- Conditions
- Cerebellar Ataxia, Spinocerebellar Ataxias, Cerebellar Degeneration
- Trial name
- NCT05436262
- Testing
- Real-time fMRI with neurofeedback of motor imagery
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Johns Hopkins University
Fragile X Tremor/Ataxia Syndrome
Citocoline for Treatment in Fragile X-associated Tremor/Ataxia Syndrome
The objective of this study is to determine if citocoline is effective for balance abnormalities and to stabilize cognitive decline in patients with fragile X-associated tremor ataxia syndrome.
- Trial name
- NCT02197104
- Testing
- citocoline
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Rush University Medical Center
Fragile X-Associated Tremor/Ataxia Syndrome, Fragile X Premutation Carriers
Memantine Treatment in Fragile X-Associated Tremor/Ataxia Syndrome
The purpose of this study is to determine if memantine is effective in treating symptoms of Fragile X-associated Tremor Ataxia Syndrome.
- Trial name
- NCT00584948
- Testing
- Memantine
- Where
- 2 sites in United States
- Volunteers
- 94 people, healthy volunteers welcome
- Sponsor
- University of California, Davis
Spinocerebellar Ataxias, Tai Chi
Could Tai-chi Help Maintain Balance of Spinocerebellar Ataxia Patients
Spinocerebellar atrophy is the most common autosomal dominant inherited ataxia. There are over thirty subtypes, which characterize neurologic features differently.
- Trial name
- NCT03687190
- Testing
- Tai chi, conventional medicine
- Volunteers
- 21 people
- Sponsor
- Changhua Christian Hospital
Nervous System Disease, Genetic Syndrome
Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Telangiectasia Patients
Objectives: The objective of study was to evaluate the safety and the efficacy of EryDex (Dexamethasone sodium phosphate encapsulated in autologous erythrocytes, using the EryDex System - EDS) at two dose levels (low dose and…
- Trial name
- ATTeST (NCT02770807)
- Testing
- EryDex Low dose DSP, EryDex High dose DSP
- Where
- 22 sites in United States, Australia and 10 more
- Volunteers
- 176 people
- Sponsor
- Quince Therapeutics S.p.A.
Stiff-Person Syndrome
Stem Cell Transplantation for Stiff Person Syndrome (SPS)
Non-myeloablative regimens (as the investigators use herein) are designed to maximally suppress the immune system without destruction of the bone marrow stem cell compartment.
- Trial name
- SPS (NCT02282514)
- Testing
- Autologous Hematopoietic Stem Cells, Cyclophosphamide, Mesna
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- Northwestern University
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
Ataxia Telangiectasia
Open-Label Extension of EryDex Study IEDAT-04-2022
This is an international, multi-center, prospective, open-label, non-comparative study aiming to provide access to treatment with EryDex to ataxia telangiectasia (A-T) patients who completed the IEDAT-04-2022 trial which studied…
- Trial name
- OLE_NEAT (NCT06664853)
- Testing
- Dexamethasone sodium phosphate
- Where
- 21 sites in United States, Denmark and 7 more
- Volunteers
- 101 people
- Sponsor
- Quince Therapeutics S.p.A.