Clinical trials: Ataxia, open to volunteers
Spinocerebellar Ataxia Genotype Type 1, Spinocerebellar Ataxia Genotype Type 3
Cerebellar DBS in SCA1 and SCA3 Study
The purpose of this study is to evaluate the safety and feasibility of deep brain stimulation (DBS) in the deep cerebellar nuclei (DCN) in patients with Spinocerebellar Ataxia (SCA) types 1 and 3.
- Trial name
- NCT07752095
- Testing
- UG3NS140720 Deep Brain Stimulation System
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Gordon H. Baltuch
Spinocerebellar Ataxia Type 3
Optimizing Parameters of Transcranial Temporal Interference Stimulation for Spinocerebellar Ataxia Type 3
The purpose of this research study is to identify the optimal biological dose for tTIS that targets DN in SCA3 while achieving the best possible balance between therapeutic efficacy and safety.
- Trial name
- NCT07743866
- Testing
- Transcranial Temporal Interference Stimulation (tTIS)
- Where
- China
- Volunteers
- 24 people
- Sponsor
- First Affiliated Hospital of Fujian Medical University
Metastatic Cancer
A Study of Reduced-dose Radiation in People With Metastatic Tumors With a Genetic Change
This study will test whether reduced-dose radiotherapy is an effective treatment for metastatic tumors with an ATM mutation. The researchers want to find the lowest dose of radiation that would still be effective to treat these…
- Trial name
- NCT05010031
- Testing
- Palliative radiotherapy
- Where
- 7 sites in United States
- Volunteers
- 45 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Inclusion Body Myositis, Myotonic Dystrophy
Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders
Currently, there are no standardised fall risk scores or guidelines on when to use appropriate assistive gait devices (AGDs) for people with neuromuscular disorders (NMDs).
- Conditions
- Inclusion Body Myositis, Myotonic Dystrophy, Limb-girdle and Facioscapulohumeral Muscular Dystrophies, Pompe Disease
- Trial name
- FBIndex (NCT06605612)
- Testing
- FBIndex
- Where
- Germany
- Volunteers
- 108 people
- Sponsor
- LMU Klinikum
Friedreich's Ataxia
A Study to Learn More About Friedrich's Ataxia in People Living in the Gulf Cooperation Council (GCC) Region of the World
In this study, researchers will learn more about a disease called Friedrich's Ataxia, also known as FA. This is known as an "observational" study, which collects health information about study participants without changing their…
- Trial name
- NCT07887828
- Volunteers
- 40 people
- Sponsor
- Biogen
Cerebellar Ataxia
A Registered Cohort Study on Cerebellar Ataxia in the Organization in South-East China for Cerebellar Ataxia Research (OSCCAR)
Cerebellar ataxia is a form of ataxia originating in the cerebellum. Cerebellar ataxia can occur as a result of many diseases and may present with symptoms of an inability to coordinate balance, gait, extremity and eye movements.
- Trial name
- NCT04010214
- Testing
- No intervention
- Where
- China
- Volunteers
- 1,500 people, healthy volunteers welcome
- Sponsor
- Ning Wang, MD., PhD.
Pancreas Cancer, Peutz-Jeghers Syndrome (PJS)
The Cancer of the Pancreas Screening-5 CAPS5)Study
Johns Hopkins clinical research office quality assurance group will monitor and audit this study at Johns Hopkins. The Sub Investigator at each site will be responsible for internal monitoring at their site.
- Conditions
- Pancreas Cancer, Peutz-Jeghers Syndrome (PJS), Gene Mutation, Germline Mutation Carrier
- Trial name
- CAPS5 (NCT02000089)
- Testing
- Secretin, MRI, Tumor marker gene test with CA19-9
- Where
- 9 sites in United States
- Volunteers
- 9,000 people
- Sponsor
- Johns Hopkins University
Pelvic Floor Dyssynergia, Phonation Disorder
Dynamics of Pelvic Floor Muscles With Different Phonation Patterns Among Female Students
The purpose of the study is to know if there is relation between the dynamics of pelvic floor muscles and different phonation pattern.
- Trial name
- NCT06716268
- Testing
- phonation and respiratory tasks
- Where
- Egypt
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Cairo University
PD - Parkinson's Disease
Transplantation of Human iPS Cell-derived Dopaminergic Progenitors (CT1-DAP001) for Parkinson's Disease (Phase I/II)
To evaluate the safety and efficacy of transplantation of human induced pluripotent stem cell-derived dopaminergic progenitors, CT1-DAP001, into the corpus striatum in patients with Parkinson's disease
- Trial name
- CT1-DAP001 (NCT06482268)
- Testing
- Human induced pluripotent stem cell-derived dopaminergic progenitors (CT1-DAP001)
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- University of California, San Diego
Friedreich Ataxia, Cardiomyopathies
Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia
The purpose of this study is to test the safety and preliminary efficacy of AAVrh.10hFXN to treat the cardiomyopathy associated with Friedreich's ataxia (FA).
- Conditions
- Friedreich Ataxia, Cardiomyopathies, Cardiac Hypertrophy, Myocardial Fibrosis
- Trial name
- NCT05302271
- Testing
- AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN, Prednisone
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Weill Medical College of Cornell University
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'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
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
Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay, Spastic Paraplegia 7
Effects of a Supervised Rehabilitation Program on Disease Severity in Spastic Ataxias
Spastic ataxias are a group of diseases causing symptoms such as walking difficulties and balance impairments that lead to a high risk of falls. No pharmacological treatments exist to treat these diseases.
- Trial name
- NCT06261424
- Testing
- IMPACT - rehabIlitation prograM for sPAstiC aTaxias
- Where
- 5 sites in Canada
- Volunteers
- 84 people
- Sponsor
- Laval University
CANVAS Syndrome
Pathogenic Insights and Search for Biomarkers in RFC1-ataxia/CANVAS
CANVAS (Cerebellar Ataxia, Neuropathy, Vestibular Areflexia Syndrome), also referred to as RFC1-ataxia, is a recently molecularly characterized neurodegenerative disorder caused by a biallelic expansion of an AAGGG…
- Trial name
- INSIDE-CANVAS (NCT07156214)
- Testing
- Assessment of markers of oxidative stress in peripheral blood samples
- Where
- 2 sites in Italy
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Catholic University of the Sacred Heart
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
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
Advanced Malignant Solid Neoplasm, Advanced Microsatellite Stable Colorectal Carcinoma
Testing the Combination of the Anti-Cancer Drugs Temozolomide and M1774 to Evaluate Their Safety and Effectiveness
This phase I/II trial studies the side effects and best dose of temozolomide and M1774 and how well they works in treating patients with cancer that has spread from where it first started (primary site) to other places in the…
- Conditions
- Advanced Malignant Solid Neoplasm, Advanced Microsatellite Stable Colorectal Carcinoma, Hematopoietic and Lymphatic System Neoplasm, Metastatic Malignant Solid Neoplasm
- Trial name
- NCT05691491
- Testing
- Biopsy Procedure, Biospecimen Collection, Computed Tomography
- Where
- 23 sites in United States
- Volunteers
- 42 people
- Sponsor
- National Cancer Institute (NCI)
Friedreich Ataxia
A Single Ascending Dose Study of DT-216P2 in Normal Healthy Participants
This purpose of the study is to evaluate the safety, tolerability and pharmacokinetics of single ascending doses of DT-216P2 administered either subcutaneously (SC) and intravenously (IV) in normal healthy participants.
- Trial name
- NCT06772870
- Testing
- DT-216P2, Saline
- Where
- Australia
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Design Therapeutics, Inc.
Friedreich Ataxia
Friedreich Ataxia Nerve Ultrasund
"Friedreich ataxia is the most common inherited autosomal recessive ataxia. It is caused by a GAA repeat expansion in the frataxin gene on chromosome 9q21.11.
- Trial name
- FAUST (NCT07508631)
- Testing
- Nerve ultrasound - routine exam
- Where
- France
- Volunteers
- 14 people
- Sponsor
- Centre Hospitalier Universitaire de Nice