Clinical trials: Hereditary Ataxia, open to volunteers
Friedreich Ataxia, Cardiomyopathies
A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ATI-105 in Healthy Adults
The goal of this clinical trial is to learn how ATI-105 administered to healthy participants at works in the body at different doses and with or without food.
- Conditions
- Friedreich Ataxia, Cardiomyopathies, Central Nervous System Diseases, Neuromuscular Diseases
- Trial name
- NCT07869654
- Testing
- ATI-105
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Adjuvia Therapeutics, Inc.
Spinocerebellar Ataxia
Ophthalmological Disorders in Dominant Spinal-cerebellar Ataxias
Spinocerebellar ataxias (SCA) are rare genetic neurological disorders. The most common forms are SCA1, SCA2 and SCA3. Another more recently identified cause of ataxia is SCA27B.
- Trial name
- AO-SCA (NCT07019558)
- Testing
- Neurological assessment, Ophthalmological assessment
- Where
- France
- Volunteers
- 60 people
- Sponsor
- University Hospital, Montpellier
Spinocerebellar Ataxia (SCA)
Multi-Modal Digital Monitoring of Disease Symptoms in Spinocerebellar Ataxia
This is an observational study. Subjects with a documented diagnosis of spinocerebellar ataxia (SCA) will be screened and recruited to participate at two clinical sites: Johns Hopkins University (JHU) and Columbia University…
- Trial name
- NCT07851376
- Where
- 2 sites in United States
- Volunteers
- 25 people
- Sponsor
- BioSensics
Friedreich Ataxia
Patient-reported, Health Economic and Psychosocial Outcomes in Friedreich Ataxia
The PROFA study is an international, multi-centric observational and validation study to assess the patient-reported, psychosocial and economic outcomes of patients with Friedreich Ataxia (FA).
- Trial name
- PROFA (NCT05943002)
- Where
- 6 sites in Austria, France and 1 more
- Volunteers
- 200 people
- Sponsor
- German Center for Neurodegenerative Diseases (DZNE)
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
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'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
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
Spinocerebellar Ataxia
Effects of Oral Administration of Antrodia Cinnamomea Products for Clinical Symptoms in Spinocerebellar Ataxia Patients
This study aims to assess the effect of Antrodia cinnamomea on clinical symptoms in spinocerebellar ataxia patients. To investigate the advancements in neurodegenerative diseases.
- Trial name
- NCT06633003
- Testing
- Antrodia cinnamomea Products
- Where
- Taiwan
- Volunteers
- 60 people
- Sponsor
- ALPS Biotech CO. LTD
Friedreich Ataxia
Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study
This project is a global, multicenter, prospective, longitudinal, observational natural history study that can be used to understand the disease progression and support the development of safe and effective drugs and biological…
- Trial name
- UNIFAI (NCT06016946)
- Where
- 34 sites in United States, Australia and 15 more
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Friedreich's Ataxia Research Alliance
Spinocerebellar Ataxia
The EUROSCA Natural History Study
The key goals of EUROSCA-NHS is to determine and compare the rate of disease progression in SCA1, SCA2, SCA3 and SCA6 including determination of the order and occurrence of non-ataxia symptoms, assessment of activities of daily…
- Trial name
- EUROSCA-NHS (NCT02440763)
- Where
- 16 sites in Austria, Belgium and 8 more
- Volunteers
- 400 people
- Sponsor
- Ataxia Study Group
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
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
Solid Cancers, NSCLC (Advanced Non-small Cell Lung Cancer)
An Phase Ib/II Clinical Trial of TCC1727 Combination Therapy in Advanced Solid Tumors
This is a Phase Ib/II clinical study. The Phase Ib dose-escalation study aims to evaluate and determine the recommended Phase II dose (RP2D) of TCC1727 in combination with benmelstobart /olaparib /topotecanfor patients with…
- Conditions
- Solid Cancers, NSCLC (Advanced Non-small Cell Lung Cancer), Gastric (Stomach) Cancer, Endometrial Cancer
- Trial name
- NCT07371663
- Testing
- TCC1727 tablet 90mg, TCC1727 tablet 120mg, TCC1727 tablet 160mg
- Where
- 3 sites in China
- Volunteers
- 266 people
- Sponsor
- Beijing Tide Pharmaceutical Co., Ltd
Spinocerebellar Ataxia (SCA), Spinocerebellar Ataxia Type 6
Interposed Nucleus aDBS for Ataxia
This is a single-center, open-label study designed to evaluate the feasibility, safety, and preliminary efficacy of cerebellar adaptive deep brain stimulation (aDBS) in adults with spinocerebellar ataxia type 6 (SCA6).
- Trial name
- NCT07325487
- Testing
- Deep Brain Stimulation
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- University of Florida
Spinocerebellar Ataxias
Stemchymal® for Polyglutamine Spinocerebellar Ataxia
The purpose of the clinical trial is to study the therapeutic efficacy and safety of Stemchymal® infusions for polyglutamine spinocerebellar ataxia treatment by a randomized, double-blind, placebo-controlled study design.
- Trial name
- NCT06397274
- Testing
- Stemchymal
- Volunteers
- 20 people
- Sponsor
- Steminent US, Inc.
Spinocerebellar Ataxia (SCA), Spinocerebellar Ataxia Type 6
Deep Brain Stimulation for Spinocerebellar Ataxia
The goal of this clinical trial is to test the safety of placing Deep Brain Stimulators (DBS) in the cerebellum and using electrical stimulation of that part of the brain to treat symptoms related to the participants…
- Trial name
- NCT07288437
- Testing
- Deep Brain Stimulation (DBS)
- Where
- 2 sites in United States
- Volunteers
- 5 people
- Sponsor
- University of California, San Francisco
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
Friedreich Ataxia
Digital Outcome Assessment Using AI Active Gaming and Motion Capture in Friedreich Ataxia
Friedreich ataxia (FA) is a rare, inherited condition that progressively affects balance, coordination, and walking. Clinical trials of new FA treatments rely largely on standard clinical rating scales, but these can be tiring…
- Trial name
- STEP-OUT FA (NCT07731971)
- Testing
- Laboratory validation session (VSimulator), Home-based active gaming assessment
- Where
- United Kingdom
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- University of Exeter
Autosomal Dominant Spinocerebellar Ataxia (SCA1, 2,3,6,7,27B)
Autosomal Dominant Spinocerebellar Ataxias and Social Cognition
Spinocerebellar ataxias are a group of rare neurodegenerative diseases, clinically and genetically highly heterogeneous, with an estimated mean prevalence of 2.7 per 100,000 population.
- Trial name
- SoCoSca (NCT07099651)
- Testing
- tests
- Where
- France
- Volunteers
- 160 people, healthy volunteers welcome
- Sponsor
- University Hospital, Angers