Clinical trials: Inherited Movement Disorders
- Condition Inherited Movement Disorders
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
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
Angelman Syndrome
Long-term Extension of GTX-102 in Angelman Syndrome
The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)
- Trial name
- NCT06415344
- Testing
- GTX-102
- Where
- 23 sites in United States, Australia and 6 more
- Volunteers
- 255 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Tourette Syndrome
Alternatives for Reducing Tics in Tourette Syndrome (TS): A Study of TEV-50717 (Deutetrabenazine) for the Treatment of Tourette Syndrome in Children and Adolescents
This is a study to evaluate the efficacy and safety of deutetrabenazine (TEV-50717) tablets for the reduction of motor and phonic tics associated with TS in children and adolescents 6 through 16 years of age.
- Trial name
- ARTISTS1 (NCT03452943)
- Testing
- TEV-50717
- Where
- 42 sites in United States, Canada and 4 more
- Volunteers
- 119 people
- Sponsor
- Teva Branded Pharmaceutical Products R&D, Inc.
Tourettes Disorder, Tourette Disorder
Expanded Access Program for Treatment With Ecopipam for Tourette's Disorder
The goal of this expanded access study is to learn about the long-term effects of Ecopipam in participants with Tourette's Disorder (TD).
- Trial name
- EBS-101-TD-392 (NCT07093541)
- Testing
- ecopipam HCl ~2mg/kg/day
- Where
- 26 sites in United States
- Sponsor
- Emalex Biosciences Inc.
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.
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'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.
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.
HSP, Hereditary Spastic Paraplegia
Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)
Safety and Efficacy of AAV9/AP4B1 For Patients with AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47): A Phase 1/2 Single-Center, Open-Label Study of Stereotactic Intra-cisterna Magna Administration.
- Conditions
- HSP, Hereditary Spastic Paraplegia, Hereditary Spastic Paraparesis, Hereditary Spastic Paraplegia Type 50
- Trial name
- NCT06948019
- Testing
- BFB-101 (AAV9-CBh-AP4B1)
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- BlackfinBio Ltd
Tic Disorder, Tourette Syndrome
Phase III Study of 5LGr to Treat Tic Disorder
The purpose of this study is to evaluate the effective and safety of 5LGr in pediatric patients with tic disorders.
- Conditions
- Tic Disorder, Tourette Syndrome, Chronic Tic Disorder
- Trial name
- 5LGr (NCT01501695)
- Testing
- 5LGr, tiapride
- Volunteers
- 603 people
- Sponsor
- Tasly Pharmaceuticals, Inc.
Tourette Syndrome
Aripiprazole Oral Solution in the Treatment of Children and Adolescents With Tourette's Syndrome
This study is a multicenter, randomized, double-blind, placebo-controlled trial designed to assess the safety, efficacy, tolerability and steady-state plasma trough concentration of flexible-dosed aripiprazole once-daily…
- Trial name
- NCT03487783
- Testing
- Aripiprazole Oral Solution
- Where
- China
- Volunteers
- 121 people
- Sponsor
- Otsuka Beijing Research Institute
Angelman Syndrome, Fragile X Syndrome
Single Dose Pharmacokinetic (PK) Study
The trial is a Phase 1 Single Dose PK Study in Adolescent Subjects with Fragile X syndrome (FXS) or Angelman syndrome (AS). The primary objective of the study is to evaluate the pharmacokinetics (PK) of OV101 following a single 5…
- Trial name
- NCT03109756
- Testing
- OV101
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Ovid Therapeutics Inc.
TOURETTE SYNDROME
A Pilot Study Of SD-809 (Deutetrabenazine) In Moderate To Severe Tourette Syndrome (TS)
The purpose of this study is to evaluate safety, tolerability and preliminary efficacy of SD-809 in the treatment of motor and phonic tics of Tourette Syndrome and to evaluate the pharmacokinetic of SD-809 and its metabolites.
- Trial name
- NCT02674321
- Testing
- SD-809
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- Auspex Pharmaceuticals, Inc.
Achondroplasia, Hypochondroplasia
Prospective Longitudinal Multicentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal Dysplasia
The study aims to identify which Syde®-derived digital outcomes are reliable in FGFR3-related Skeletal Dysplasia. This requires to set-up a natural history study to measure limb movements in patients with ACH or HCH.
- Trial name
- SKY-MOT3 (NCT07388966)
- Where
- 4 sites in Belgium, France and 1 more
- Volunteers
- 140 people
- Sponsor
- SYSNAV
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 Syndrome
Safety and Efficacy Study of NBI-98854 in Children and Adolescents With Tourette Syndrome
Phase 2, double-blind, placebo-controlled study to assess the safety and efficacy of NBI-98854 administered once daily (qd) for a total of 6 weeks of treatment.
- Trial name
- NCT02679079
- Testing
- NBI-98854
- Where
- 44 sites in United States
- Volunteers
- 98 people
- Sponsor
- Neurocrine Biosciences