Clinical trials: Ataxia
Non-progressive Congenital Ataxia, Ataxic Cerebral Palsy
ARTEMIS - The ARTEMIS Cohort
This multinational European observational clinical study focuses on non-progressive congenital ataxia (NPCA), a very rare early-onset neurological condition also within the cerebral palsy (CP) concept as ataxic CP.
- Trial name
- ARTEMIS (NCT07421219)
- Where
- 7 sites in Belgium, Denmark and 5 more
- Volunteers
- 50 people
- Sponsor
- Vastra Gotaland Region
Ataxia Telangiectasia
Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With a Novel Form of Anaplerosis
Study design: Parallel group, placebo-controlled, dose-escalation each 2 months for 12 months. Dose based on percent (%) of calculated caloric intake.
- Trial name
- A-TC7 (NCT04513002)
- Testing
- Triheptanoin
- Where
- Australia
- Volunteers
- 30 people
- Sponsor
- The University of Queensland
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
Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia Study
The purpose of this phase 3 multi-center, open-label extension study is to evaluate the long-term safety of ACTIMMUNE® (interferon-γ 1b) in participants with Friedreich's Ataxia.
- Trial name
- STEADFAST (NCT02593773)
- Testing
- Interferon γ-1b
- Where
- 4 sites in United States
- Volunteers
- 86 people
- Sponsor
- Amgen
Opsoclonus-myoclonus Syndrome
Study of Cytokines in Children With Opsoclonus-Myoclonus Syndrome
The purpose of this study is to determine if cytokines, inflammatory mediators, are increased in spinal fluid and blood, correlate with disease activity, and could serve as biomarkers or therapeutic targets in children with…
- Trial name
- OMS (NCT00806182)
- Where
- United States
- Volunteers
- 400 people, healthy volunteers welcome
- Sponsor
- National Pediatric Neuroinflammation Organization, Inc.
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
Ataxia Telangiectasia
Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With Nicotinamide Riboside
Study design: Single arm open-label clinical trial in ataxia-telangiectasia to test the effects of nicotinamide riboside on ataxia scales, immune function, and neurofilament light chain.
- Trial name
- ATNAD (NCT06324877)
- Testing
- Nicotinamide riboside
- Where
- Australia
- Volunteers
- 10 people
- Sponsor
- The University of Queensland
SCA7
Riluzole in Patients With Spinocerebellar Ataxia Type 7
Spinocerebellar ataxia type 7 (SCA7) belongs to the dominant forms of inherited cerebellar ataxias (CA), being one of the rarest form.
- Trial name
- NCT03660917
- Testing
- Riluzole
- Where
- Italy
- Volunteers
- 34 people
- Sponsor
- S. Andrea Hospital
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
Friedreich Ataxia
Characterisation of the Cognitive Profile of Patients Suffering From Friedreich's Ataxia
Friedreich's Ataxia (FA) Friedreich's Ataxia is a neurodegenerative disease caused by a homozygous expansion of the GAA triplet repeats of the frataxin gene (FXN). FA usually begins in childhood or adolescence.
- Trial name
- CPCAF (NCT05874388)
- Where
- France
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Encephalopathy, Psychosis
Neuroimmunology Registry and Biobank
A variety of antineuronal antibodies have been detected in the cerebrospinal fluid (CSF) of patients with neurological diseases.
- Conditions
- Encephalopathy, Psychosis, Impaired Consciousness, Epilepsy
- Trial name
- NCT06958341
- Where
- 2 sites in Germany
- Volunteers
- 300 people
- Sponsor
- Charite University, Berlin, Germany
Ataxic Cerebral Palsied Children, Core Stability
Core Stability in Ataxic Cerebral Palsied Children
Forty children with cerebellar ataxia ranged in age from five to nine years old, they were randomly assigned into two matched control and study groups.
- Conditions
- Ataxic Cerebral Palsied Children, Core Stability, Balance and Coordination, Severity of the Ataxia
- Trial name
- NCT04823936
- Testing
- core stability training
- Where
- Egypt
- Volunteers
- 40 people
- Sponsor
- Cairo University
Ataxia Telangiectasia in Children, Ataxia Telangiectasia
Home-based Complex Intervention for Children With Ataxia Telangiectasia
Ataxia telangiectasia is a rare, genetic and progressive condition with no known cure. Therapies present a mainstream management option and have the potential to offer optimisation of fitness and general health.
- Trial name
- NCT05692622
- Testing
- Whole-body exercise and respiratory exercise
- Where
- United Kingdom
- Volunteers
- 40 people
- Sponsor
- University of Plymouth
Teenagers, Athletes
Effect of a Bean Protein-added Supplement on Body Composition in High-performance Adolescent Athletes
Background: Sport is a specialized competitive physical activity that is regulated and requires daily physical training. The workouts allow athletes to increase strength, endurance exercise performance, and muscle mass.
- Trial name
- NCT05307159
- Testing
- bean protein supplement
- Where
- Mexico
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Hospital Regional de Alta Especialidad de la Península de Yucatán
Friedreich Ataxia
Evaluation of the Effects of Calcitriol's in the Neurological Symptoms of Friedreich's Ataxia Patients
Friedreich's Ataxia (FA) is an autosomal recessive disease the mutation of which leads to a deficiency of a protein called frataxin, which is responsible for the symptoms of the disease.
- Trial name
- CalcitriolFA (NCT04801303)
- Testing
- Calcitriol, Blood analysis for frataxin's level measurement, Blood analysis for hypercalcemia's control
- Where
- Spain
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Berta Alemany
Ataxia-Telangiectasia, Ataxia-Telangiectasia (A-T)
A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T)
A pivotal, randomized, double-blind, placebo-controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of Ataxia-Telangiectasia (A-T).
- Trial name
- NCT06673056
- Testing
- N-Acetyl-L-Leucine
- Where
- 11 sites in United States, Germany and 4 more
- Volunteers
- 60 people
- Sponsor
- IntraBio Inc
Spinocerebellar Ataxia
Coordination Training With Complete Body Video Games in Children and Adults With Degenerative Ataxias
Exergame training might offer a novel treatment approach even in largely nonambulatory subjects with multisystemic degenerative spinocerebellar ataxia.
- Trial name
- Move'n Fun (NCT02874911)
- Testing
- General training
- Volunteers
- 10 people
- Sponsor
- University Hospital Tuebingen
Constipation
Biofeedback for Dyssynergic Constipation
Constipation affects 4% of adults in the United States (U.S.). An estimated half of constipated patients are unable to relax pelvic floor muscles during defecation, a type of constipation called pelvic floor dyssynergia (PFD).
- Trial name
- NCT00127257
- Testing
- Biofeedback, Diazepam, Pelvic floor retraining
- Where
- United States
- Volunteers
- 117 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
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