Clinical trials: Inherited Movement Disorders
Hepatolenticular Degeneration; Wilson
Clinical Value of DWI-ADC Matching in the Short-term Prognosis of Wilson's Disease
In earlier studies, it was found that patients of Wilson disease with new diagnosed who only has neurological symptoms often had DWI hyper-intensity in brain MRI, which was more common in putamen and midbrain, indicating that the…
- Trial name
- NCT06196931
- Where
- China
- Volunteers
- 200 people
- Sponsor
- Hospital Affiliated to the Institute of Neurology, Anhui University of Chinese Medicine
Huntington Disease
Open-label Extension Study to Evaluate the Safety and Tolerability of WVE-120102 in Patients With Huntington's Disease
WVE-HDSNP2-002 is an open-label extension (OLE) study to evaluate the safety, tolerability, PK, PD, and clinical effects of WVE-120102 in adult patients with early manifest HD who carry a targeted single nucleotide polymorphism…
- Trial name
- NCT04617860
- Testing
- WVE-120102
- Where
- 20 sites in Australia, Canada and 5 more
- Volunteers
- 36 people
- Sponsor
- Wave Life Sciences USA, Inc.
Angelman Syndrome
A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome
The purpose of this study is to evaluate the safety and efficacy of MVX-220 gene therapy in children and adults with Angelman syndrome with UBE3A gene deletion, uniparental disomy, or imprinting center defect genotypes.
- Trial name
- ASCEND-AS (NCT07181837)
- Testing
- MVX-220
- Where
- 3 sites in United States
- Volunteers
- 12 people
- Sponsor
- MavriX Bio, LLC
Tourette Syndrome in Children, Food Intolerance
Peripheral Mechanisms of Humoral Immune Effector-mediated Tourette's Syndrome and the Role of Tuina Intervention
Tourette syndrome is a refractory type of tic disorder. Previous clinical studies have confirmed that Tuina has a good effect on TS, but the mechanism of action is still uncertain.
- Conditions
- Tourette Syndrome in Children, Food Intolerance, Inflammatory Response
- Trial name
- NCT06379373
- Testing
- Spine manipulation; westren medicine
- Where
- China
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- The First Affiliated Hospital of Zhejiang Chinese Medical University
Wilson Disease
Circadian Variation of Urinary Copper Excretion in Wilson Disease Patients
Wilson's disease (WD) is a genetic disorder characterized by an accumulation of copper in the body, mainly in the liver and brain.
- Trial name
- VARCUWIC (NCT06430359)
- Testing
- urine and blood test
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Hospices Civils de Lyon
Tourette Syndrome, Behavioral Problem
Parent Behavioral Training for Disruptive Behaviors in Tourette Syndrome
This is a randomized controlled trial to assess the efficacy of a parent training program to control disruptive behaviors in children with Tourette Syndrome.
- Trial name
- NCT02882490
- Testing
- Parent Training, Supportive Therapy
- Where
- Spain
- Volunteers
- 42 people
- Sponsor
- Hospital Clinic of Barcelona
Genetic/Metabolic Liver Diseases
China Registry for Genetic / Metabolic Liver Diseases
CR-GMLD registry started on June 13, 2015 to collect cases of genetic/metabolic liver diseases from tertiary or secondary hospitals in mainland China.
- Trial name
- NCT03131427
- Testing
- Standard of care
- Where
- 12 sites in China
- Volunteers
- 20,000 people
- Sponsor
- Beijing Friendship Hospital
Huntington Disease
Huntington's Disease Biobank: Advancing Remote Monitoring and Deep Phenotyping
This observational study aims to identify novel biomarkers of disease onset and progression in Huntington's disease by integrating remote monitoring with fluid biomarkers.
- Trial name
- NCT06941662
- Testing
- computer vision, remote monitoring, blood, urine, wrist bands, mobile-app
- Where
- United States
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Stanford University
Huntington's Disease
Child to Adult Neurodevelopment in Gene Expanded Huntington's Disease
Huntington's Disease (HD) is an autosomal dominant disease manifested in a triad of cognitive, psychiatric, and motor signs and symptoms. HD is caused by a triplet repeat (CAG)expansion in the gene Huntingtin (HTT).
- Trial name
- ChANGE HD (NCT01860339)
- Where
- 6 sites in United States
- Volunteers
- 488 people, healthy volunteers welcome
- Sponsor
- Peggy C Nopoulos
Tic Disorders, Tics
Transcranial Magnetic Stimulation to Augment Behavior Therapy for Tics
The study will examine whether combining Comprehensive Behavioral Intervention for Tics (CBIT) with inhibition of the supplementary motor area (SMA) using transcranial magnetic stimulation (TMS) normalizes activity in the…
- Conditions
- Tic Disorders, Tics, Tic, Motor, Tic Disorder, Childhood
- Trial name
- NCT04578912
- Testing
- Comprehensive Behavioral Intervention for Tics (CBIT), Repetitive Transcranial Magnetic Stimulation (rTMS), Continuous Theta Burst Stimulation (cTBS)
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University of Minnesota
Angelman Syndrome
REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome
The purpose of this study is to evaluate the efficacy and safety of obudanersen in children and adults with Angelman syndrome caused by a deletion or mutation of the UBE3A gene.
- Trial name
- NCT06914609
- Testing
- obudanersen
- Where
- 41 sites in United States, Australia and 10 more
- Volunteers
- 156 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Cardiovascular Diseases, Wilson's Disease
Early Detection of Cardiac Affection in Patients of Wilson's Disease
Wilson disease (WD) is a rare autosomal recessive disorder caused by a genetic defect in ATP7B resulting in limited excretion of excess copper into the bile Pathological copper accumulation occurs in the entire body, with the…
- Trial name
- Wilson's (NCT06051734)
- Volunteers
- 50 people
- Sponsor
- Assiut University
Tourette's Disorder
Efficacy & Safety Study of Once-weekly Oral Aripiprazole in Children and Adolescents With Tourette's Disorder
The goal of the current study is to determine efficacy and safety of once-weekly aripiprazole in reducing Total Tic Severity (TTS) score in children and adolescents with Tourette's Disorder.
- Trial name
- NCT01418339
- Testing
- Aripiprazole
- Where
- 39 sites in United States, Canada and 4 more
- Volunteers
- 135 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Tourette's Syndrome, Chronic Motor Tic Disorder
Brain Activation in Vocal and Motor Tics
This study will investigate the brain areas that are activated by vocal and motor tics in patients with Tourette's syndrome and other tic disorders. Tics are involuntary repetitive movements similar to voluntary movements.
- Conditions
- Tourette's Syndrome, Chronic Motor Tic Disorder, Chronic Motor Vocal Disorder
- Trial name
- NCT00026000
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Major Depression, Mania
Reliability and Validity of the MINI International Neuropsychiatric Interview for Children and Adolescents (MINI-KID)
The primary aims of this study are to assess: 1. The inter-rater and test-retest reliability of the MINI-KID 2. The validity of the standard MINI-KID interview in relation to the parent rated pencil/paper version (MINI-KID-P) and…
- Conditions
- Major Depression, Mania, Anxiety Disorders, Psychotic Disorder
- Trial name
- NCT00579267
- Testing
- MINI-KID
- Where
- United States
- Volunteers
- 230 people, healthy volunteers welcome
- Sponsor
- University of South Florida
Tourette Syndrome
The Pathophysiology of Tourette Syndrome: a Multimodal Study
The purpose of this study is to characterize the functional and anatomical connectivity changes in Tourette syndrome and its relation with iron deficiency.
- Trial name
- PTS (NCT02497807)
- Where
- China
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Second Affiliated Hospital, Zhejiang University, School of Medicine
Microcephaly, Hyperkinesis With Developmental Delay
Intensive Therapy for Children With Microcephaly, Hyperkinetic Movements, or Global Developmental Delay
This trial will consist of a clinical series of up to 50 children with Global Developmental Delay and concomitant microcephaly or hyperkinetic movements.
- Conditions
- Microcephaly, Hyperkinesis With Developmental Delay, Global Developmental Delay
- Trial name
- NCT04816175
- Testing
- Acquire Therapy
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Virginia Polytechnic Institute and State University
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
Tourette's Syndrome
Optimising Anterior Pallidal Deep Brain Stimulation for Tourette's Syndrome
The motor tics associated with Tourette's syndrome may be reduced with deep brain stimulation of the anterior globus pallidus. The best area within this brain region and the best stimulation device settings are currently unknown.
- Trial name
- NCT02112253
- Testing
- Deep brain stimulator ventral electrode up to 2 mA, Deep brain stimulator ventral electrode up to 3 mA, Deep brain stimulator dorsal electrode up to 2 mA
- Where
- Australia
- Sponsor
- The University of Western Australia
Tourette's Disorder
Efficacy & Safety Study of Once-weekly Oral Aripiprazole in Children and Adolescents With Tourette's Disorder (TD)
The goal of the current trial is to determine efficacy and safety of once-weekly aripiprazole in reducing Total Tic Severity (TTS) score in children and adolescents with Tourette's Disorder.
- Trial name
- NCT01418352
- Testing
- Aripiprazole
- Where
- 35 sites in United States, Bulgaria and 3 more
- Volunteers
- 83 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.