Clinical trials: Inherited Movement Disorders
Huntington's Disease
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RO7234292 (ISIS 443139) in Huntington's Disease Patients Who Participated in Prior Investigational Studies of RO7234292 (ISIS 443139)
This study will test the safety, tolerability, pharmacokinetics and pharmacodynamics of RO7234292 administered intrathecally to adult patients with Huntington's Disease.
- Trial name
- NCT03342053
- Testing
- RO7234292 (RG6042)
- Where
- 9 sites in Canada, Germany and 1 more
- Volunteers
- 46 people
- Sponsor
- Hoffmann-La Roche
Chronic Motor or Vocal Tic Disorder, Tourette's Disorder
Aripiprazole in Children and Adolescents With Chronic Tic Disorder or Tourette's Disorder
The purpose of this trial is to demonstrate the efficacy and safety of aripiprazole in children and adolescents aged 6~18 years with chronic tic disorders or Tourette's disorder
- Trial name
- NCT00706589
- Testing
- aripiprazole
- Where
- 6 sites in South Korea
- Volunteers
- 61 people
- Sponsor
- Korea Otsuka Pharmaceutical Co., Ltd.
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's Disease
A Study Evaluating if Pridopidine is Safe, Efficacious, and Tolerable in Patients With Huntington's Disease
The purpose of the study is to collect and assess long term data on the safety, tolerability, and efficacy of pridopidine in patients with Huntington's disease (HD).
- Trial name
- Open PRIDE-HD (NCT02494778)
- Testing
- Pridopidine
- Where
- 46 sites in United States, Australia and 9 more
- Volunteers
- 248 people
- Sponsor
- Prilenia
Huntington Disease
An Extension of the HORIZON Protocol Evaluating the Safety of Dimebon (Latrepirdine) in Subjects With Huntington Disease
An open-label extension study of the HORIZON protocol evaluating the safety of dimebon (latrepirdine)in subjects with Huntington disease.
- Trial name
- HORIZON PLUS (NCT01085266)
- Testing
- Dimebon (latrepirdine)
- Volunteers
- 362 people
- Sponsor
- Medivation, Inc.
Huntington Disease
Study to Evaluate the Pharmacodynamics, Safety and Efficacy of SKY-0515 in Participants With Huntington's Disease
The goal of this clinical trial is to test if the drug SKY-0515, an oral medication, can lower harmful proteins linked to Huntington's Disease (HD) and improve the symptoms of participants with HD.
- Trial name
- FALCON-HD (NCT07378644)
- Testing
- SKY-0515
- Where
- 31 sites in United States, Argentina and 8 more
- Volunteers
- 400 people
- Sponsor
- Skyhawk Therapeutics, Inc.
Huntington Disease
Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of MBF-015 in Huntington's Disease Patients
This is a Phase IIa (proof of concept), single center clinical trial to evaluate the safety and efficacy of daily MBF-015 oral treatment during 28 days in Huntington's Disease patients on top of standard of care.
- Trial name
- NCT06469853
- Testing
- MBF-015 16 mg oral capsules
- Where
- Spain
- Volunteers
- 10 people
- Sponsor
- Medibiofarma S.L.
Angelman Syndrome
A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome
The primary objective of the study is to evaluate the safety and tolerability of multiple-ascending doses of GTX-102 administered by intrathecal (IT) injection to participants with Angelman Syndrome (AS).
- Trial name
- NCT04259281
- Testing
- GTX-102
- Where
- 25 sites in United States, Australia and 6 more
- Volunteers
- 74 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Angelman Syndrome
CHAMPION: A Study to Evaluate the Efficacy and Safety of Obudanersen (ION582) in Children and Adults With Angelman Syndrome (AS)
The primary purpose of the study is to evaluate efficacy of obudanersen in participants with AS due to uniparental disomy or imprinting defects (UPD/ID) as measured through expressive communication.
- Trial name
- NCT07782827
- Testing
- obudanersen
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Tourette Syndrome
Open-label Extension Study of Pramipexole in the Treatment of Children and Adolescents With Tourette Syndrome
The primary objective of this open-label, flexible dose study is to assess the safety and efficacy of pramipexole over a 24-week period in children and adolescents (age 6-17 years inclusive) diagnosed with Tourette Syndrome…
- Trial name
- NCT00681863
- Testing
- pramipexole 0.125 mg BID, pramipexole 0.0625 mg QD, pramipexole 0.125 mg TID
- Where
- 14 sites in United States, Germany
- Volunteers
- 45 people
- Sponsor
- Boehringer Ingelheim
Tourette Syndrome
The Safety and Efficacy of Long-term Treatment of PINS Stimulator System for Tourette Syndrome
The purpose of this clinical study is to verify the long term effectiveness and safety of a bilateral deep brain stimulation (DBS) produced by Beijing PINS Medical Co., Ltd.
- Trial name
- NCT02253498
- Testing
- deep brain stimulation (DBS)
- Volunteers
- 20 people
- Sponsor
- Beijing Pins Medical Co., Ltd
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
Friedreich's Ataxia (FA)
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
This is a phase 1b, first in-human, open-label, dose-finding study investigating the safety and tolerability of SGT-212 in participants with Friedreich's ataxia (FA).
- Trial name
- FALCON (NCT07180355)
- Testing
- SGT-212
- Where
- 3 sites in United States
- Volunteers
- 10 people
- Sponsor
- Solid Biosciences Inc.
Huntington's Disease
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 443139 in Participants With Early Manifest Huntington's Disease
This study tested the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending doses of ISIS 443139 administered intrathecally to adult participants with early manifest Huntington's Disease.
- Trial name
- NCT02519036
- Testing
- ISIS 443139 10 mg, ISIS 443139 30 mg, ISIS 443139 60 mg
- Where
- 9 sites in Canada, Germany and 1 more
- Volunteers
- 46 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures
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.
Huntington Disease
An Open Label Study of ANX005 in Participants With, or at Risk for, Manifest Huntington's Disease
This study is a multi-center, open-label study of intravenous (IV) ANX005 in participants with, or at risk for, manifest Huntington's Disease (HD).
- Trial name
- NCT04514367
- Testing
- ANX005
- Where
- 6 sites in United States
- Volunteers
- 28 people
- Sponsor
- Annexon, Inc.
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.
Wilson Disease
Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840
The main objective of the study is to evaluate the change in liver copper (Cu) concentration following 48 weeks of treatment with ALXN1840 in adult participants with Wilson Disease (WD) who have been previously treated for at…
- Trial name
- NCT04422431
- Testing
- Bis-Choline Tetrathiomolybdate
- Where
- 12 sites in United States, Canada and 6 more
- Volunteers
- 31 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Ocular Motility Disorders, Strabismus
Clinical Validation of AI-Based Quantitative Eye Movement Analysis From Smartphone 9-Gaze Videos (Glandy EOM)
This prospective, multicenter, multinational observational study evaluates the clinical validity of an AI-based quantitative eye movement analysis system (Glandy EOM) that analyzes smartphone-recorded 9-gaze videos.
- Conditions
- Ocular Motility Disorders, Strabismus, Extraocular Muscle Dysfunction, Thyroid Eye Disease
- Trial name
- GLANDY-EOM-VAL (NCT07623837)
- Testing
- Glandy EOM (AI-based quantitative eye movement analysis software)
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- THYROSCOPE INC.