Clinical trials: Inherited Movement Disorders
Tourette Syndrome
Ecopipam Tablets to Study Tourette's Syndrome in Children and Adolescents
This study evaluates the effect of ecopipam tablets in children and adolescents in the treatment of Tourette's Syndrome (TS). Half of the participants will receive ecopipam tablets, while the other half will receive matching…
- Trial name
- D1AMOND (NCT04007991)
- Testing
- Ecopipam
- Where
- 70 sites in United States, Canada and 3 more
- Volunteers
- 153 people
- Sponsor
- Emalex Biosciences Inc.
Acute Liver Failure, Wilson Disease
Daily Versus Alternate Day Plasma Exchange in Wilson Disease With Acute Liver Failure in Children
Wilson disease in children has a varied presentation. Wilson disease with acute liver failure is associated with very high mortality and morbidity.
- Trial name
- NCT06698991
- Testing
- Plasma Exchange, Standard Medical Treatment
- Where
- India
- Volunteers
- 20 people
- Sponsor
- Institute of Liver and Biliary Sciences, India
Angelman Syndrome
Clinical Trial of Levodopa/Carbidopa ( Sinemet) Therapy in Angel Man Syndrome
The purpose of this study is to evaluate the safety and effectiveness (how well a drug works) of Carbidopa/levodopa (Sinemet) in individuals with Angelman syndrome.
- Trial name
- NCT03235037
- Testing
- Levodopa
- Volunteers
- 10 people
- Sponsor
- Bennett Lavenstein
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
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
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
Tic Disorders, Tourette Syndrome
Comparison of Keppra and Clonidine in the Treatment of Tics
The goal of this study is to confirm that levetiracetam has a better tic-suppressing profile than that of the widely used tic-suppressing medication, clonidine.
- Trial name
- NCT00370838
- Testing
- Levetiracetam, Clonidine
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Harvey S. Singer
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
Tourette Syndrome in Children, Tourette Syndrome in Adolescence
Extinction Learning in Youth With Tourette Syndrome
Aversive sensory phenomena such as premonitory urges play a central role in the behavioral treatment model of tics. Extinction learning and extinction recall are learning processes implicated within this model, but have not been…
- Conditions
- Tourette Syndrome in Children, Tourette Syndrome in Adolescence, Habit Reversal Training, Tic
- Trial name
- NCT03765463
- Testing
- Habit Reversal Training
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Johns Hopkins University
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
Pantothenate Kinase-Associated Neurodegeneration
Efficacy and Safety Study of Fosmetpantotenate (RE-024) in PKAN Participants
This study investigated whether fosmetpantotenate (RE-024), a phosphopantothenate replacement therapy, was safe and effective in treating participants with PKAN.
- Trial name
- PKAN (NCT03041116)
- Testing
- Fosmetpantotenate
- Where
- 20 sites in United States, Canada and 7 more
- Volunteers
- 84 people
- Sponsor
- Travere Therapeutics, Inc.
Tourette Syndrome, Tic Disorders
Evaluating and Promoting Resilience in Adolescents With Tourette Syndrome
This study adapts and tests PRISM (Promoting Resilience in Stress Management), a short skills-based program that teaches resilience, for teens ages 12-18 with Tourette syndrome or persistent tic disorder.
- Conditions
- Tourette Syndrome, Tic Disorders, Persistent (Chronic) Motor or Vocal Tic Disorder
- Trial name
- PRISM-TS (NCT07905898)
- Testing
- PRISM Intervention, Usual care intervention
- Where
- United States
- Volunteers
- 95 people
- Sponsor
- Wake Forest University Health Sciences
Meige Syndrome, Meigs Syndrome
A Comparative Study of GPI's DBS and Pallidotomy in the Treatment of Meige Syndrome
In this study, we will compare the degree of postoperative symptom improvement, postoperative complication rate, postoperative quality of life improvement degree of patients with Meige syndrome undergoing pallidotomy (unilateral…
- Conditions
- Meige Syndrome, Meigs Syndrome, Meige Disease, Meig Syndrome
- Trial name
- NCT04618887
- Testing
- pallidotomy, deep brain stimulation
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Peking University People's Hospital
Angelman Syndrome, Nervous System Diseases
Dietary Supplements for the Treatment of Angelman Syndrome
Angelman syndrome (AS) is a complex genetic disorder that affects the nervous system. The purpose of this study is to determine the effectiveness of certain dietary supplements in treating the symptoms of AS.
- Trial name
- NCT00348933
- Testing
- Betaine, Creatine, Metafolin
- Where
- 4 sites in United States
- Volunteers
- 90 people
- Sponsor
- University of California, San Diego
Tic Disorder, Chronic Motor or Vocal, Tourette Syndrome in Children
Evaluation of a Cognitive Psychophysiological Treatment for Tourette Syndrome and Tic Disorders
It is a randomized controlled trial to compare two behavioural treatments, namely CBIT and CoPs. This study would be the first head-to-head randomized trial between two established treatments rather than supportive counseling or…
- Conditions
- Tic Disorder, Chronic Motor or Vocal, Tourette Syndrome in Children, Tourette Syndrome
- Trial name
- NCT03225430
- Testing
- Cognitive behavioural treatment for tics, Cognitive psychophysiological
- Where
- Canada
- Volunteers
- 144 people
- Sponsor
- Centre de Recherche de l'Institut Universitaire en santé Mentale de Montréal
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 Syndrome
Safety of Cultured Allogeneic Adult Umbilical Cord or Placental Derived Mesenchymal Stem Cells for Tourette Syndrome
This trial will study the safety and efficacy of intravenous infusion of cultured allogeneic adult umbilical cord or placental derived mesenchymal stem cells for the treatment of Tourette Syndrome
- Trial name
- NCT05158439
- Testing
- Cultured allogeneic adult umbilical cord or placental derived mesenchymal stem cells
- Sponsor
- The Foundation for Orthopaedics and Regenerative Medicine
Tourette's Syndrome
Ecopipam Treatment of Tourette's Syndrome in Subjects 7-17 Years
Tourette's Syndrome is a neurological disease characterized by motor and vocal tics. It has been hypothesized that abnormal interactions of dopamine with its receptors may cause the tics.
- Trial name
- NCT02102698
- Testing
- Ecopipam
- Where
- 11 sites in United States
- Volunteers
- 40 people
- Sponsor
- Emalex Biosciences Inc.
Lysosomal Storage Disorders, Gaucher Disease
Genetic Studies of Lysosomal Storage Disorders
The purpose of this study is to identify genetic, biochemical, and clinical factors that are associated with disease severity in people with Gaucher disease and other lysosomal storage disorders.
- Conditions
- Lysosomal Storage Disorders, Gaucher Disease, Parkinson Disease
- Trial name
- NCT00001215
- Where
- United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- National Human Genome Research Institute (NHGRI)
Tourette Syndrome, Obsessive Compulsive Disorder
Developmental Phenomenology of Obsessive Compulsive Disorder and Tourette Syndrome in Children and Adolescents
OBJECTIVES: I. Characterize the natural history, associated features, and severity of symptoms of obsessive compulsive disorder and Tourette syndrome in children and adolescents. II.
- Trial name
- NCT00004324
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- National Center for Research Resources (NCRR)