Clinical trials: Inherited Movement Disorders
Angelman Syndrome
A Study in Adults and Adolescents With Angelman Syndrome (STARS)
The purpose of the study is to assess the safety and tolerability of oral OV101 (gaboxadol) in adult and adolescent subjects with Angelman syndrome. In addition, several exploratory efficacy outcome measures will be investigated.
- Trial name
- NCT02996305
- Testing
- OV101 Regimen 1, OV101 regimen 2
- Where
- 13 sites in United States, Israel
- Volunteers
- 88 people
- Sponsor
- Healx AI
Tourette Syndrome, Tic Disorder, Chronic Motor or Vocal
Open-label MNS for Tourette Syndrome
A recent report (Morera Maiquez et al 2020) described reduced tic severity in people with Tourette syndrome during 1-minute epochs of median nerve stimulation (MNS) at 10 Hz.
- Trial name
- NCT05016765
- Testing
- Active, self-directed electrical stimulation of the median nerve
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Washington University School of Medicine
Wilson Disease
Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease
The study will evaluate the efficacy and safety of ALXN1840 (formerly called WTX101) administered for 48 weeks compared to standard of care (SoC) in Wilson Disease (WD) participants aged 12 and older in the Primary Evaluation…
- Trial name
- NCT03403205
- Testing
- ALXN1840, SoC Therapy
- Where
- 56 sites in United States, Australia and 20 more
- Volunteers
- 214 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
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.
Epilepsy; Seizure, Neuromuscular Diseases
North Carolina Genomic Evaluation by Next-generation Exome Sequencing, 2
The "North Carolina Clinical Genomic Evaluation by Next-gen Exome Sequencing, 2 (NCGENES 2)" study is part of a larger consortium project investigating the clinical utility, or net benefit of an intervention on patient and family…
- Conditions
- Epilepsy; Seizure, Neuromuscular Diseases, Brain Malformation, Intellectual Disability
- Trial name
- NCGENES2 (NCT03548779)
- Testing
- Pre-visit prep, usual care + exome seq
- Where
- 3 sites in United States
- Volunteers
- 548 people
- Sponsor
- University of North Carolina, Chapel Hill
Huntington Disease
Open-label Extension Study to Evaluate the Safety and Tolerability of WVE-120101 in Patients With Huntington's Disease
WVE-HDSNP1-002 is an open-label extension (OLE) study to evaluate the safety, tolerability, PK, PD, and clinical effects of WVE-120101 in adult patients with early manifest HD who carry a targeted single nucleotide polymorphism…
- Trial name
- NCT04617847
- Testing
- WVE-120101
- Where
- 18 sites in Australia, Canada and 5 more
- Volunteers
- 27 people
- Sponsor
- Wave Life Sciences USA, Inc.
Tourette Syndrome
Thalamic Deep Brain Stimulation for the Treatment of Refractory Tourette Syndrome
This research is being performed to try to understand if the use of deep brain stimulation or DBS can treat the symptoms of Tourette syndrome that do not respond well to current medications.
- Trial name
- NCT01817517
- Testing
- Medtronic Activa Deep Brain Stimulation System
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Johns Hopkins University
Tourettes Syndrome, Tic
N-acetylcysteine (NAC) for Children With Tourette Syndrome
Tourette syndrome is a childhood-onset neuropsychiatric disorder characterized by multiple motor and vocal tics that last for at least a year in duration.
- Trial name
- NCT01172288
- Testing
- N-Acetylcysteine (NAC)
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Yale University
Tourette's Disorder
Safety and Tolerability Study of Once-weekly Oral Aripiprazole in Children and Adolescents With Tourette's Disorder
The goal of the current trial is to obtain long term efficacy, safety and tolerability data of once weekly aripiprazole in children and adolescents with Tourette's Disorder.
- Trial name
- NCT01416441
- Testing
- Aripiprazole
- Where
- 80 sites in United States, Bulgaria and 8 more
- Volunteers
- 170 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
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
Minocycline in the Treatment of Angelman Syndrome
There is mounting evidence to suggest that a treatment for Angelman syndrome is not just possible, but probable. The lack of known molecular targets associated with AS has hampered the development of specific therapeutics.
- Trial name
- NCT01531582
- Testing
- minocycline
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- University of South Florida
Tourette Syndrome, Chronic Tic Disorder
Proof of Concept Study of an Oral Orthotic to Reduce Tic Severity in Chronic Tic Disorder and Tourette Syndrome
The purpose of this study is to assess the feasibility of a trial on an oral orthotic for reducing tic severity in children ages 7-25 years with Tourette syndrome (TS) or Chronic Tic Disorder (CTD).
- Trial name
- NCT02067819
- Testing
- Active Oral Orthotic Treatment
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Weill Medical College of Cornell University
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 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
Huntington's Disease
Coenzyme Q10 in Huntington's Disease (HD)
The goals of this trial are to determine if coenzyme Q10 is effective in slowing the worsening symptoms of Huntington's disease and to learn about the safety and acceptability of long-term coenzyme Q10 use by determining its…
- Trial name
- 2CARE (NCT00608881)
- Testing
- coenzyme Q10
- Where
- 49 sites in United States, Australia and 1 more
- Volunteers
- 609 people
- Sponsor
- Massachusetts General Hospital
Angelman Syndrome
An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome
This open-label study (OV101-18-002) will evaluate the long-term (52 weeks) safety of OV101 in subjects with AS and provide additional OV101 treatment to those subjects who completed Study OV101-15-001 (NCT02996305).
- Trial name
- ELARA (NCT03882918)
- Testing
- OV101
- Where
- 11 sites in United States, Israel
- Volunteers
- 141 people
- Sponsor
- Healx AI
Tourette's Disorder (TD)
Trial to Evaluate the Long-term Efficacy of Oral Aripiprazole in the Treatment of Pediatric Participants With Tourette's Disorder
To evaluate the long-term efficacy of oral aripiprazole in pediatric participants for the treatment of Tourette's Disorder (TD).
- Trial name
- NCT03661983
- Testing
- Aripiprazole
- Where
- 42 sites in United States, Canada and 1 more
- Volunteers
- 36 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Tourette's Disorder
ABF Tourette's Disorder Post Marketing Surveillance Study
This is a Post-Marketing Surveillance study of Abilify® tablets in accordance with Korean regulations on New Drug Re-examination (i.e. New Drug Re-examination Standards: KFDA Notification No. 2010-94 dated 27 December 2010).
- Trial name
- NCT01795105
- Where
- 4 sites in South Korea
- Volunteers
- 692 people
- Sponsor
- Korea Otsuka Pharmaceutical Co., Ltd.
Tourette Disorder
Ecopipam Tablets to Study Tourette's Disorder in Children, Adolescents and Adults
This Phase 3 multicenter study evaluates the maintenance of efficacy, safety and tolerability of ecopipam tablets in children, adolescents and adults in the treatment of Tourette's Disorder (TD).
- Trial name
- D1AMOND (NCT05615220)
- Testing
- Ecopipam Hydrochloride
- Where
- 95 sites in United States, Bulgaria and 10 more
- Volunteers
- 216 people
- Sponsor
- Emalex Biosciences Inc.
Tourette Syndrome
Safety, Tolerability, and Efficacy of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome
This is a Phase 2b, randomized, double-blind, placebo-controlled, dose-optimization study to evaluate the efficacy, safety, and tolerability of NBI-98854 titrated to the subject's optimal dose administered once daily (qd) for a…
- Trial name
- NCT03325010
- Testing
- Valbenazine
- Where
- 35 sites in United States, Puerto Rico
- Volunteers
- 127 people
- Sponsor
- Neurocrine Biosciences