Clinical trials: Hereditary Ataxia
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
Spinocerebellar Ataxias
An Open-label Trial of Intravenous Immune Globulin (IVIG)in Treating Spinocerebellar Ataxias
The purpose of this study is to learn how Intravenous Immune Globulin (IVIG) will affect Spinocerebellar Ataxia (SCA) symptoms and how it will affect motor and nervous system function in participants Subtypes of SCA to be…
- Trial name
- NCT02287064
- Testing
- Intravenous Immune Globulin (IVIG)
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- University of South Florida
Friedreich Ataxia
A Natural History Study to TRACK Brain and Spinal Cord Changes in Individuals with Friedreich Ataxia (TRACK-FA)
This is a natural history study prospectively investigating neuroimaging markers of disease progression in children and adults with Friedreich ataxia (FA). There will be three assessment periods (baseline, 12 and 24 months).
- Trial name
- (TRACK-FA) (NCT04349514)
- Testing
- Natural history
- Where
- 7 sites in United States, Australia and 3 more
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- Monash University
Spinocerebellar Ataxia
The Study of Transcranial Magnetic Stimulation in the Regulation of Spinocerebellar Ataxia
Spinocerebellar ataxia (SCA) is a group of hereditary neurological diseases caused by gene mutations leading to degenerative changes in the cerebellum, brainstem, and spinal cord.
- Trial name
- NCT06904716
- Testing
- Transcranial magnetic stimulation
- Where
- China
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- First Affiliated Hospital of Chongqing Medical University
Spinocerebellar Ataxias, Spastic Paraplegias
Natural History, Genetic Bases and Phenotype-genotype Correlations in Autosomal Dominant Spinocerebellar Degenerations
The autosomal dominant spinocerebellar degenerations are a highly heterogeneous, clinically and genetically, group of rare diseases and of severe evolution.
- Trial name
- NCT00136630
- Where
- 8 sites in France
- Volunteers
- 175 people, healthy volunteers welcome
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Cancer
Caspofungin Acetate in Treating Aspergillosis in Patients With Hematologic Cancer or in Patients Who Have Undergone a Stem Cell Transplant
RATIONALE: Antifungals, such as caspofungin acetate, may be effective in treating fungal infections caused by chemotherapy or stem cell transplant.
- Trial name
- NCT00110045
- Testing
- caspofungin acetate
- Where
- 13 sites in Belgium, France and 5 more
- Volunteers
- 171 people
- Sponsor
- European Organisation for Research and Treatment of Cancer - EORTC
Spinocerebellar Ataxias
Efficacy of Cerebellar Transcranial Magnetic Stimulation to Treat Hereditary Spinocerebellar Ataxias
Spinocerebellar ataxia (SCA) is a type of autosomal dominant ataxia and there is currently no effective treatment. The goal of this clinical trial is to learn the efficacy of navigated iTBS (Intermittent theta-burst stimulation)…
- Trial name
- NCT06898645
- Testing
- navigated iTBS (Intermittent theta-burst stimulation) targeting the cerebellum
- Volunteers
- 80 people
- Sponsor
- Xijing Hospital
Spinocerebellar Ataxia Type 3
A Pharmacokinetics and Safety Study of BIIB132 in Adults With Spinocerebellar Ataxia 3
The primary objective of this study is to evaluate the safety and tolerability of multiple ascending doses of BIIB132 administered via intrathecal (IT) injection to participants with spinocerebellar ataxia type 3 (SCA3).
- Trial name
- NCT05160558
- Testing
- BIIB132
- Where
- 20 sites in United States, Germany and 4 more
- Volunteers
- 8 people
- Sponsor
- Biogen
Ataxia Telangiectasia
Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T)
This project aims to evaluate the safety and efficacy of precision genetic therapy for patients with Ataxia-telangiectasia (A-T), a rare neurodegenerative disease caused by mutations in the ATM gene.
- Trial name
- NCT07215416
- Testing
- Antisense oligonucleotide targeting the ATM gene
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Timothy Yu
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
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Friedreich's Ataxia
GV101 for Friedreich's Ataxia (FA) Treatment
This is a dose-ranging, randomized, double-blind, placebo-controlled 12-week Phase 2a trial with an up to two week loading dose period.
- Trial name
- NCT07870590
- Testing
- GV101
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Graviton Bioscience Corporation
Friedreich Ataxia
Interferon Gamma-1b in Friedreich Ataxia (FRDA)
Friedreich ataxia (FRDA) is a progressive neurodegenerative disease of children and adults for which there is presently no therapy.
- Trial name
- NCT01965327
- Testing
- Interferon Gamma-1b
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Children's Hospital of Philadelphia
Ataxia Telangiectasia
International Ataxia Rating Scale in Younger Patients
The project will collect information on the mapping of clinical ratings on a number of scales that are used in the assessment of patients with ataxias.
- Trial name
- NCT01942850
- Where
- 5 sites in United States, India and 2 more
- Volunteers
- 63 people
- Sponsor
- Quince Therapeutics S.p.A.
Ataxia-Telangiectasia
Study for Treatment of Cancer in Children With Ataxia-telangiectasia
This is a pilot/feasibility study designed to investigate the feasibility of treating children with Ataxia-Telangiectasia (A-T) and cancer with regimens nearly as intense as non-A-T patients with cancer would receive.
- Trial name
- NCT00187057
- Testing
- vinblastine, vincristine, prednisone, daunorubicin, doxorubicin, methotrexate, cyclophosphamide, L-asparaginase, etoposide, cytarabine, mercaptopurine
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- St. Jude Children's Research Hospital
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
Wheelchair Mobility, Manual Wheelchair Skills Training
Wheelchair Skills Training for People with ARSACS and DM1
Wheelchairs (WC) are often provided to people with ARSACS and MD1 when they are not able to walk anymore. However, giving someone a MWC alone does not guarantee they will use it safely or properly.
- Conditions
- Wheelchair Mobility, Manual Wheelchair Skills Training, ARSACS, Myotonic Dystrophy Type 1
- Trial name
- NCT06596850
- Testing
- Wheelchair Skills Training Program
- Where
- Canada
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Laval University
Acute Myocardial Infarction (AMI), STEMI - ST Elevation Myocardial Infarction (MI)
Effect of Dapagliflozin Administration on the Apoptosis Levels of Patients With Acute Myocardial Infarction
The currently known Sodium-Glucose Transporter 2 (SGLT-2) inhibitors are recognized not only for their effects on improving intravascular glucose levels but also for their cardioprotective effects, including improvements in…
- Conditions
- Acute Myocardial Infarction (AMI), STEMI - ST Elevation Myocardial Infarction (MI), NSTEMI - Non-ST Segment Elevation Myocardial Infarction (MI)
- Trial name
- DAPOPTOSIS-MI (NCT06615674)
- Testing
- Forxiga® 10 mg Film-Coated Tablet, manufactured by AstraZeneca Pharmaceuticals LP, USA for AstraZeneca Pharmaceuticals Co. Ltd., China imported by PT AstraZeneca Indonesia, Indonesia
- Where
- Indonesia
- Volunteers
- 40 people
- Sponsor
- Yoga Yudhistira
Ataxia Telangiectasia
Evaluate the Neurological Effects of EryDex on Subjects With A-T
This is an international, multi-center, randomized, prospective, double-blind, placebo-controlled, Phase 3 study, designed to assess the effect of EryDex (dexamethasone sodium phosphate [DSP] in autologous erythrocytes)…
- Trial name
- NEAT (NCT06193200)
- Testing
- Dexamethasone sodium phosphate
- Where
- 19 sites in United States, Denmark and 7 more
- Volunteers
- 105 people
- Sponsor
- Quince Therapeutics S.p.A.
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