Clinical trials: Inherited Movement Disorders
Tuberous Sclerosis, STXBP1 Encephalopathy With Epilepsy
Gait in Rare Diseases
The aim of this pilot study is to explore whether the knowledge and experience gained during the T-GaiD project (Treatment of Gait Disorders in Dravet Syndrome - NCT03857451) can be transferred to other populations with similar…
- Trial name
- GAGA (NCT05161494)
- Where
- Belgium
- Volunteers
- 41 people
- Sponsor
- Universiteit Antwerpen
Pantothenate Kinase-Associated Neurodegeneration
CoA-Z in Pantothenate Kinase-associated Neurodegeneration (PKAN)
The purpose of this study is to learn more about how people with the condition pantothenate kinase-associated neurodegeneration (PKAN) respond to a specialized study product.
- Trial name
- NCT04182763
- Testing
- CoA-Z
- Where
- United States
- Volunteers
- 77 people
- Sponsor
- Oregon Health and Science University
Tourette Syndrome, Apathy
Apathy in Tourette Syndrome and Changes in Quality of Life
Tourette syndrome is a complex neuropsychological disorder with both motor and vocal tics associated with multiple psychological co-morbidities. Apathy has not been widely studied in patients with Tourette syndrome.
- Trial name
- NCT02765178
- Testing
- Children's Motivation Scale, Yale Global Tic Severity Scale (YGTSS), Studies Depression Scale for Children (CES-DC)
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- University of Florida
Dental Malocclusion, Maxillary Deficiency
Effect on Periodontal Phenotype of Flapless Piezoincission During Orthodontic Treatment
Research Question While the reduction in treatment times and the patient satisfaction after periodontally accelerated osteogenic orthodontics (PAOO) are well sustained in the scientific literature, there is still controversy…
- Trial name
- Piezo_Ortho (NCT06315348)
- Testing
- flapless alveolar decortication with augmentation
- Where
- Spain
- Volunteers
- 40 people
- Sponsor
- Universidad Complutense de Madrid
Cardiac Disease, Respiratory Disease
Clinical Use Cases Assessment of the Gabi System in Young Children With Underlying Medical Conditions (BRIDGE Study)
Subjects will use the Gabi system on a daily basis for 3 months, each time the subject is resting or asleep. The Gabi system will recording the SpO2, pulse rate, respiratory rate and movements of the subject.
- Conditions
- Cardiac Disease, Respiratory Disease, Congenital Cardiac Defect, Cardiovascular Diseases
- Trial name
- BRIDGE (NCT05319236)
- Testing
- Gabi System
- Where
- 4 sites in United States, Belgium
- Volunteers
- 101 people
- Sponsor
- Gabi SmartCare
Pantothenate Kinase-Associated Neurodegeneration
Efficacy and Safety Study of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN)
A multi-center, placebo controlled, double-blind trial comparing the efficacy and safety of 18 months of treatment with deferiprone versus placebo in patients with PKAN.
- Trial name
- NCT01741532
- Testing
- Deferiprone oral solution
- Where
- 4 sites in United States, Germany and 2 more
- Volunteers
- 89 people
- Sponsor
- ApoPharma
Tourette Syndrome
Safety and Efficacy of NBI-98854 in Pediatric Subjects With Tourette Syndrome
This is a Phase 2, double-blind, placebo-controlled, randomized withdrawal study to evaluate the safety and maintenance of efficacy of an optimized once-daily (qd) dose of NBI-98854 in pediatric subjects with TS.
- Trial name
- NCT03530293
- Testing
- Valbenazine
- Where
- 49 sites in United States, Puerto Rico
- Volunteers
- 81 people
- Sponsor
- Neurocrine Biosciences
Wilson Disease
A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients With Wilson Disease
Wilson's disease (WD), also known as Wilson's disease, is a rare autosomal recessive metabolic disorder caused by a mutation of the copper transport ATPase β (ATP7B) gene located on the long arm of chromosome 13 (13q14.3).
- Trial name
- WD (NCT06650319)
- Testing
- LY-M003
- Where
- China
- Volunteers
- 18 people
- Sponsor
- Chaohui Yu
Angelman Syndrome
Angelman Syndrome Natural History Study
The goal of this study is to conduct a prospective, longitudinal natural history study of children and adults with Angelman Syndrome using investigator-observed and parent-reported outcome measures to obtain data that will be…
- Trial name
- NCT04507997
- Where
- 11 sites in United States, Canada
- Volunteers
- 300 people
- Sponsor
- Boston Children's Hospital
Angelman Syndrome
Study on the Brain Network of Angelman Syndrome
The aims of study on Angelman syndrome: 1. Establish the Angelman syndrome database 2. Explore the brain Network of Angelman Syndrome Based on Multi-modal Brain Image and Neural-EEG Data
- Trial name
- NCT03358823
- Where
- China
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- Fudan University
Wilson Disease
Role for Biochemical Assays and Kayser-Fleischer Rings in Diagnosis of Wilson Disease
The investigators aimed to identify factors associated with symptoms and features of Wilson disease from a large cohort during long-term follow-up
- Trial name
- NCT04965545
- Testing
- low copper diet
- Where
- China
- Volunteers
- 1,000 people
- Sponsor
- Second Affiliated Hospital, Zhejiang University, School of Medicine
Collagen Degeneration
Type I Collagen Fragments in the GCF and Saliva During OTM
Investigate the levels of type I Collagen fragments ICTP and CTX in GCF and saliva during initial leveling and alignment of the teeth in a sample of adolescents undergoing orthodontic treatment.
- Trial name
- NCT05020431
- Testing
- Biological measuring of collagen fragments biomarker during orthodontic tooth movement
- Where
- Egypt
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Alexandria University
Tourette Syndrome, Tic Disorders
Cognitive Behavior Therapy and Habit Reversal Training for the Treatment of Chronic Tic Disorders in Children
This study will determine the effectiveness of cognitive behavior therapy (CBT) with habit reversal training (HRT) in treating chronic tic disorders (CTDs) in children and adolescents.
- Trial name
- CBIT (NCT00218777)
- Testing
- Comprehensive Behavioral Intervention for Tics
- Where
- 3 sites in United States
- Volunteers
- 120 people
- Sponsor
- Tourette Association of America
Huntington Disease, Juvenile
REGISTRY-JHD - an Observational Study of the European Huntington's Disease Network (EHDN)
The study aims to monitor the progression of symptoms and signs of those affected by JHD using modified UHDRS scales of motor and function (functional assessment, TFC).
- Trial name
- JHD (NCT01590602)
- Where
- 2 sites in Germany, United Kingdom
- Volunteers
- 78 people
- Sponsor
- European Huntington's Disease Network
Tourette Disorder, Tourette Syndrome
Guanfacine in Children With Tic Disorders
The goal of this pilot study is to obtain preliminary information on the tolerability and efficacy of extended release guanfacine (trade name Intuniv) in children with Tourette Disorder (TD, also called Tourette syndrome).
- Trial name
- NCT01547000
- Testing
- extended-release guanfacine (Intuniv)
- Where
- 3 sites in United States
- Volunteers
- 34 people
- Sponsor
- Yale University
Tourette Syndrome
An Open-label Study to Determine the Efficacy and Safety of Topiramate in the Treatment of Tourette Syndrome.
Previous studies using topiramate in Tourette subjects have shown that with the use of this medication subjects report that their tics get better.
- Trial name
- NCT00206336
- Testing
- Topiramate (drug)
- Volunteers
- 20 people
- Sponsor
- Baylor College of Medicine
Angelman Syndrome
A Trial of Levodopa in Angelman Syndrome
This study is designed to determine whether levodopa will lead to an improvement in the development and tremor in children with Angelman syndrome (AS).
- Trial name
- NCT01281475
- Testing
- Levodopa
- Where
- 7 sites in United States
- Volunteers
- 67 people
- Sponsor
- Wen-Hann Tan
Wilson's Disease
International Wilson's Disease Patient Registry (iWilson Registry)
Longitudinal, observational, non-interventional, standard of care Registry. Data will be collected from the routinely scheduled WD clinic visits at approximately 6-12 month intervals.
- Trial name
- NCT05239858
- Where
- 16 sites in Belgium, France and 5 more
- Volunteers
- 500 people
- Sponsor
- Orphalan
Huntington Disease
Wearable Sensors for Quantitative Assessment of Motor Impairment in Huntington's Disease Huntington's Disease
The principal means of measuring motor impairment in Huntington disease (HD) is the Unified Huntington's Disease Rating Scale (UHDRS) total motor score, which is subjective, categorical, requires significant training to…
- Trial name
- NCT03599076
- Testing
- HD Wear
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- BioSensics
Tourette's Syndrome
A Study of the Effectiveness and Safety of Tetrabenazine MR in Pediatric Subjects With Tourette's Syndrome
The purpose of this clinical trial is to study the therapeutic effect of tetrabenazine MR in children with Tourette's Syndrome, as measured by the improvement in total tic score of the Yale Global Tic Severity Scale (YGTSS).
- Trial name
- TBZ-MR (NCT01133353)
- Testing
- Tetrabenazine MR
- Sponsor
- Bausch Health Americas, Inc.