Clinical trials: Cerebellar Ataxia
Friedreich Ataxia, Cardiomyopathy, Secondary
Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy
The purpose of Study LX2006-03, a multicenter, Phase 2, open-label, randomized, controlled study, is to evaluate the efficacy and safety of LX2006 gene therapy in participants with Friedreich ataxia (FA) cardiomyopathy (CM).
- Trial name
- SUNRISE-FA 2 (NCT07721025)
- Testing
- LX2006, Usual Care
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- Lexeo Therapeutics
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
Phosphomannomutase 2 Deficiency, PMM2-CDG
A Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 in Participants With PMM2-CDG
This study is evaluating the safety, effectiveness, and how the body absorbs, distributes, and eliminates GLM101, for participants with PMM2-CDG, including children, adolescents, and adults.
- Trial name
- NCT06892288
- Testing
- GLM101 (Part A, Double-blind), GLM101 (Part B, Open-label)
- Where
- 15 sites in United States, Belgium and 8 more
- Volunteers
- 50 people
- Sponsor
- Glycomine, Inc.
Spinocerebellar Ataxias
Cerebellar Transcranial Direct Current Stimulation in Spinocerebellar Ataxia 38
Spinocerebellar ataxia 38 (SCA 38) is a very rare autosomal dominant inherited disorder caused by a mutation in ELOV5 gene, specifically expressed in cerebellar Purkinje cells, encoding an enzyme involved in the synthesis of…
- Trial name
- NCT05951010
- Testing
- Transcranial Direct Current Stimulation
- Where
- Italy
- Volunteers
- 7 people
- Sponsor
- University of Cagliari
Multiple Sclerosis
Bilateral Anodal Cerebellar tDCS for Multidomain Dysfunctions in Patients With Multiple Sclerosis
The goal of this clinical trial is to learn if brain stimulation can improve movement and daily function in people with multiple sclerosis (MS).
- Trial name
- CerebellertDCS (NCT07555379)
- Testing
- Bilateral Cerebellar Transcranial Direct Current Stimulation (ctDCS)
- Where
- United Arab Emirates
- Volunteers
- 40 people
- Sponsor
- University of Sharjah
Cerebellar Ataxia
Exercise Training Program for Cerebellar Ataxia
The purpose of this study is to determine whether a person's ability to adapt (i.e. short term motor learning) predicts their ability to benefit from physical therapy exercises.
- Trial name
- NCT01307176
- Testing
- Home exercise program
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Friedreich Ataxia
Single Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia
To evaluate the safety and tolerability of single ascending doses of CTI-1601 in participants with Friedreich's ataxia
- Trial name
- NCT04176991
- Testing
- CTI-1601
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Larimar Therapeutics, Inc.
CLIFAHDD, Congenital Contractures of the Limbs and Face, Hypotonia, and Developmental Delay
Aprepitant for the Treatment of CLIFAHDD
There are no drug treatments for individuals with Congenital Contractures of the Limb and Face, Hypotonia, and Developmental Delay (CLIFAHDD), an ultra-rare and severe neurodevelopmental disease.
- Conditions
- CLIFAHDD, Congenital Contractures of the Limbs and Face, Hypotonia, and Developmental Delay, NALCN Channelopathy, Ataxia - Other
- Trial name
- NCT07845825
- Testing
- Aprepitant Powder for Oral Suspension
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- The University of Texas Health Science Center at San Antonio
Mitochondrial Diseases, Kearns-Sayre Syndrome
Global Registry and Natural History Study for Mitochondrial Disorders
The main goal of the project is provision of a global registry for mitochondrial disorders to harmonize previous national registries, enable world-wide participation and facilitate natural history studies, definition of outcome…
- Conditions
- Mitochondrial Diseases, Kearns-Sayre Syndrome, MIDD, SANDO
- Trial name
- GENOMIT (NCT05554835)
- Where
- 34 sites in Austria, Germany and 1 more
- Volunteers
- 6,000 people
- Sponsor
- LMU Klinikum
Ataxia
Special Drug Use Investigation (Retrospective) for Arixtra® (Fondaparinux) Venous Thromboembolism Treatment (Over 100kg)
The purpose of this post-marketing surveillance study is to collect and assess information retrospectively on safety and effectiveness of fondaparinux injection in patients with venous thromboembolism (VTE) whose body weight is…
- Trial name
- NCT01428544
- Testing
- Fondaparinux sodium
- Volunteers
- 5 people
- Sponsor
- GlaxoSmithKline
Pancreatic Ductal Adenocarcinoma, Pancreatic Cyst
The Pancreas Interception Center (PIC) for Early Detection, Prevention, and Novel Therapeutics
The long-term goal of our PIC is to develop effective strategies that can be applied clinically at the point-of-care to prevent, intercept, or detect PDAC at an early stage, thereby reducing PDAC burden and saving lives.
- Conditions
- Pancreatic Ductal Adenocarcinoma, Pancreatic Cyst, Chronic Pancreatitis, Fatty Pancreas
- Trial name
- NCT05692596
- Testing
- Data collection
- Where
- United States
- Volunteers
- 317 people, healthy volunteers welcome
- Sponsor
- H. Lee Moffitt Cancer Center and Research Institute
Critical Illness, Ventilator Lung
Early and Intensive Occupational Therapy in Mechanical Ventilated Patients
This study evaluates the efficacy of an early and intensive occupational therapy (OT) protocol in critical adult patients requiring mechanical ventilation. Evaluating the functional independence at hospital discharge.
- Conditions
- Critical Illness, Ventilator Lung, Impairment, Cognitive, Impairment, Coordination
- Trial name
- NCT04819529
- Testing
- Early and Intensive Occupational Therapy
- Where
- Chile
- Volunteers
- 226 people
- Sponsor
- University of Chile
Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay
A Rehabilitation Program to Increase Balance and Mobility in Ataxia of Charlevoix-Saguenay
This exploratory study used a pre-post test design. The supervised rehabilitation program was performed three times a week for 8 weeks (two sessions at a rehabilitation gym and one pool session).
- Trial name
- NCT05479656
- Testing
- Training program
- Where
- Canada
- Volunteers
- 10 people
- Sponsor
- Élise Duchesne
GLUT1DS1, Dystonia
Harmonic Ratio in Patients With GLUT1 Deficiency Syndrome
Glucose transporter deficiency syndrome type 1 (GLUT1DS) is a rare, genetically determined, neurometabolic disorder . It is estimated that about 90% of affected patients present various pathological gait patterns.
- Conditions
- GLUT1DS1, Dystonia, Gait Disorders, Neurologic, Gait Ataxia
- Trial name
- NCT05887739
- Testing
- Inertial gait analysis
- Where
- 2 sites in Italy
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- IRCCS National Neurological Institute "C. Mondino" Foundation
Malignancy
rTMS as a Treatment of Visceral Pain Secondary to Malignancy
The purpose of this study is to assess the efficacy of rTMS over primary motor cortex in patients suffering from malignant visceral pain. Thirty four patients were included in the study.
- Trial name
- NCT02092805
- Testing
- Real rTMS(20 Hz, 10 trains, inter train interval 30 sc with total pulses 2000, intensity 80% of motor threshold) daily for 10 consecutive days (5 days/week)
- Volunteers
- 34 people
- Sponsor
- Assiut University
Progressive Myoclonus Epilepsy Type 1, EPM1
Registry and Natural History Study for Progressive Myoclonus Epilepsy Type 1 (EPM1)
The Registry and Natural History Study for Progressive Myoclonus Epilepsy Type 1 (EPM1) is focused on gathering longitudinal clinical data as well as biological samples (blood and/or urine) from male and female patients, of all…
- Conditions
- Progressive Myoclonus Epilepsy Type 1, EPM1, CSTB-related Disease, Myoclonus Epilepsies, Progressive
- Trial name
- EPM1 (NCT06593951)
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Boston Children's Hospital
Parkinson Disease, Hereditary Spastic Paraparesis
Enhancement of Quality of Work And Life
Work participation is essential for quality of life, providing purpose, social interaction, financial security, and shaping social status.
- Conditions
- Parkinson Disease, Hereditary Spastic Paraparesis, Cerebellar Ataxia, Mitochondrial Diseases
- Trial name
- EQuAL (NCT06573866)
- Testing
- Preventive Participatory workplace intervention
- Where
- Netherlands
- Volunteers
- 124 people
- Sponsor
- Radboud University Medical Center
Advanced Cancer, Metastatic Cancer
A Study of ART0380 for the Treatment of Advanced or Metastatic Solid Tumors
This clinical trial is evaluating a drug called ART0380 in participants with advanced or metastatic solid tumors. The main goals of this study are to: Find the recommended dose of ART0380 that can be given safely to participants…
- Conditions
- Advanced Cancer, Metastatic Cancer, Ovarian Cancer, Primary Peritoneal Cancer
- Trial name
- NCT04657068
- Testing
- ART0380, Gemcitabine, Irinotecan
- Where
- 88 sites in United States, France and 2 more
- Volunteers
- 542 people
- Sponsor
- Artios Pharma Ltd
Ataxia Telangiectasia
Comparative Bioavailability of Betamethasone Oral Solution Metered Spray (GTX-102) in Healthy Subjects
A Randomized, Open-label, Crossover Study to Evaluate the Comparative Bioavailability, Pharmacokinetics, and Safety of GTX-102 Administered as an Oral Spray Compared to Intramuscular Injection - betamethasone and an Oral Solution…
- Trial name
- NCT05531890
- Testing
- GTX-102 medium dose fast Period 1 and Period 2, GTX-102 medium dose slow Period 1 and Period 2, GTX-102 high dose fast Period 1 and Period 2
- Where
- Canada
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Grace Therapeutics Inc.
Friedreich Ataxia
Methylprednisolone Treatment of Friedreich Ataxia
This study will explore whether methylprednisolone treatment is safe, well-tolerated, and beneficial in patients that are diagnosed with Friedreich Ataxia (FRDA).
- Trial name
- NCT02424435
- Testing
- Methylprednisolone
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Children's Hospital of Philadelphia