Clinical trials: Inherited Movement Disorders
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.
Chorea Associated With Huntington Disease
Alternatives for Reducing Chorea in Huntington Disease
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics of SD-809 extended release (ER) in participants switching from tetrabenazine to SD-809 ER.
- Trial name
- ARC-HD (NCT01897896)
- Testing
- SD-809
- Where
- 38 sites in United States, Australia and 1 more
- Volunteers
- 119 people
- Sponsor
- Auspex Pharmaceuticals, 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.
Huntington Disease
Study of WVE-003 in Patients With Huntington's Disease
This is a Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of WVE-003 in adult patients with early-manifest HD who…
- Trial name
- NCT05032196
- Testing
- SAD: 30mg WVE-003, SAD: 60mg WVE-003, SAD: 90mg WVE-003
- Where
- 23 sites in Australia, Canada and 8 more
- Volunteers
- 47 people
- Sponsor
- Wave Life Sciences USA, Inc.
Huntington Disease
A Study to Evaluate Safety, Tolerability, and Pharmacokinetics of SAGE-718 Oral Solution in Patients With Huntington's Disease - Part B
This study is a phase 1, double-blind, placebo-controlled, multiple ascending dose study to determine the safety, tolerability, and pharmacokinetics of SAGE-718 oral solution in healthy adults (Part A) with an open-label cohort…
- Trial name
- NCT03787758
- Testing
- SAGE-718
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Supernus Pharmaceuticals, Inc.
Wilson's Disease
Trientine Tetrahydrochloride Administered Once a Day for the First Line Treatment of Wilson's Disease Patients.
The goal of this clinical trial is to learn if a new trientine tetrahydrochloride (TETA 4HCl) formulation administered once a day compared to d-Penicillamine (DPA) as a first line treatment for people living with Wilson's disease…
- Trial name
- TRADITiONAL (NCT07465718)
- Testing
- TETA 4HCl formulation, D-Penicillamine
- Where
- 10 sites in United States, China and 2 more
- Volunteers
- 38 people
- Sponsor
- Orphalan
Early Manifest Huntington Disease
A Dose Range Finding Study With Open-Label Extension to Evaluate the Safety of Oral LMI070/Branaplam in Early Manifest Huntington's Disease
This is the first study of branaplam in adults with Huntington's Disease (HD) to determine the correct dose required to lower mutant huntingtin protein (mHTT) levels in the cerebrospinal fluid (CSF) to a degree expected to be…
- Trial name
- VIBRANT-HD (NCT05111249)
- Testing
- Branaplam
- Where
- 12 sites in Canada, France and 3 more
- Volunteers
- 26 people
- Sponsor
- Novartis Pharmaceuticals
Bowel Incontinence
A Study to Evaluate the Safety, Tolerability and Effectiveness of the Renew Insert in Treating Accidental Bowel Leakage
The purpose of this study is to determine whether the use of the Renew Insert is effective, tolerable and safe in the treatment of accidental bowel leakage due to bowel incontinence.
- Trial name
- REST (NCT01475474)
- Testing
- Renew Insert
- Where
- 3 sites in United States
- Volunteers
- 97 people
- Sponsor
- Renew Medical
Tourette Syndrome
Evaluate the Efficacy, Safety and Tolerability of TSUPPORT for Adults With Tourette Syndrome
A 28-week single-arm trial to evaluate the efficacy, safety and tolerability of TSupport (a Traditional Chinese Medicine) for adults with Tourette Syndrome.
- Trial name
- NCT05183594
- Testing
- TSupport (a Traditional Chinese Medicine)
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Tasly Pharmaceuticals, Inc.
Sleep Problems
Assessment of the Pharmacokinetics of Circadin® in Children With Neurodevelopmental Disorders and Sleep Disturbances
There is increasing evidence that chronic sleep disorders in children with autism spectrum disorder (ASD), Angelman Syndrome (AS) and Smith-Magenis syndrome (SMS) are associated with disturbed melatonin secretion and melatonin…
- Trial name
- NCT01903681
- Testing
- Circadin 2 mg, Circadin 10 mg
- Where
- Netherlands
- Volunteers
- 16 people
- Sponsor
- Neurim Pharmaceuticals Ltd.
Angelman Syndrome
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
The primary objective of this study is to evaluate the effect of GTX-102 in cognitive function in participants with deletion-type Angelman Syndrome (AS).
- Trial name
- Aspire (NCT06617429)
- Testing
- GTX-102
- Where
- 28 sites in United States, Canada and 4 more
- Volunteers
- 129 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Huntington's Disease
Safety and Tolerability of WVE-120102 in Patients With Huntington's Disease
PRECISION-HD2 is a Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple doses of WVE-120102 in…
- Trial name
- PRECISION-HD2 (NCT03225846)
- Testing
- WVE-120102
- Where
- 26 sites in United States, Australia and 6 more
- Volunteers
- 88 people
- Sponsor
- Wave Life Sciences USA, Inc.
Huntington Disease
PRidopidine's Outcome On Function in Huntington Disease, PROOF- HD
This study will evaluate the efficacy and safety of pridopidine 45mg twice daily (BID) in patients with early stage manifest Huntington Disease (HD).
- Trial name
- NCT04556656
- Testing
- Pridopidine
- Where
- 58 sites in United States, Austria and 9 more
- Volunteers
- 499 people
- Sponsor
- Prilenia
Alzheimer Disease, Alzheimer Dementia
Alzheimer's Autism and Cognitive Impairment Stem Cell Treatment Study
The purpose of the study is to evaluate the use of autologous Bone Marrow Derived Stem Cells (BMSC) as a means to improve cognitive impairment as occurs in Alzheimer's Disease and other dementias and to improve behavior and…
- Conditions
- Alzheimer Disease, Alzheimer Dementia, Vascular Dementia, Lewy Body Disease
- Trial name
- ACIST (NCT03724136)
- Testing
- Intravenous Bone Marrow Stem Cell (BMSC) Fraction, Intranasal Topical Bone Marrow Stem Cell (BMSC) Fraction, Near Infrared Light
- Where
- 3 sites in United States, United Arab Emirates
- Volunteers
- 100 people
- Sponsor
- MD Stem Cells
Chorea
First Time Use of SD-809 in Huntington Disease
The purpose of this study is to determine whether SD-809 tablets are effective in the treatment of chorea associated with Huntington's Disease.
- Trial name
- First-HD (NCT01795859)
- Testing
- SD-809
- Where
- 43 sites in United States, Australia and 1 more
- Volunteers
- 90 people
- Sponsor
- Teva Branded Pharmaceutical Products R&D, Inc.
Wilson Disease
Pharmacokinetic Study of Oral ALXN1840 in Japanese and Non-Japanese Adult Healthy Participants
The main purpose of the study is to confirm how long ALXN1840 stays in the body of Japanese and non-Japanese healthy participants (that is, pharmacokinetic [PK] profile).
- Trial name
- NCT05641311
- Testing
- ALXN1840
- Where
- United Kingdom
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Alexion Pharmaceuticals, Inc.
Chorea, Huntington
Open-Label Rollover Study for Continuing Valbenazine Administration for the Treatment of Chorea Associated With Huntington Disease
This is a Phase 3, open-label study to evaluate the long-term safety and tolerability of valbenazine, and to provide participants continued access to valbenazine for the treatment of chorea associated with Huntington disease.
- Trial name
- NCT04400331
- Testing
- Valbenazine
- Where
- 34 sites in United States, Canada
- Volunteers
- 154 people
- Sponsor
- Neurocrine Biosciences
Sleep Disorders
Efficacy and Safety of Circadin® in the Treatment of Sleep Disturbances in Children With Neurodevelopment Disabilities
The purpose of this study is to establish the efficacy and safety of Circadin in children with neurodevelopmental disorders and to determine the dose, this randomized, placebo-controlled study is planned to evaluate the efficacy…
- Trial name
- NCT01906866
- Testing
- Circadin 2/5/10 mg
- Where
- 28 sites in United States, Finland and 3 more
- Volunteers
- 125 people
- Sponsor
- Neurim Pharmaceuticals Ltd.
Huntington Disease
A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease
The purpose is to assess safety and tolerability of votoplam and to determine whether votoplam slows disease progression in patients with early symptomatic Huntington's disease (HD) compared to the control arm.
- Trial name
- INVEST-HD (NCT07326709)
- Testing
- Votoplam (blinded)
- Where
- 117 sites in United States, Argentina and 25 more
- Volunteers
- 770 people
- Sponsor
- Novartis Pharmaceuticals
Huntington Disease, Alzheimer Disease
A Phase 1 Study To Evaluate The Effect Of Dimebon On The Pharmacokinetics Of Dextromethorphan
This drug-drug interaction study is being conducted to evaluate the potential effect of Dimebon on the pharmacokinetics on dextromethorphan, a probe substrate of the cytochrome P450 2D6 (CYP2D6) enzyme, after multiple dose…
- Trial name
- NCT00788047
- Testing
- Dextromethorphan, Dimebon + Dextromethorphan
- Where
- United States
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Pfizer