Clinical trials: Neurodegenerative Disorders
Neurofibromatosis Type 1
Frameshift Peptides of Children With NF1
The objective of this study is to determine if children and young adults with Neurofibromatosis Type 1 (NF1) and either Low Grade Gliomas (LGGs) or Plexiform Neurofibromas (PNs) have a specific frameshift peptide protein profile…
- Trial name
- NCT04212351
- Testing
- Frameshift Array blood sample test
- Where
- 3 sites in United States
- Volunteers
- 60 people
- Sponsor
- Children's National Research Institute
Spinal Muscular Atrophy
An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)
This objectives of this study are to evaluate the safety, tolerability, and pharmacokinetics of a single dose of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA).
- Trial name
- SMNRx (NCT01494701)
- Testing
- nusinersen
- Where
- 4 sites in United States
- Volunteers
- 28 people
- Sponsor
- Biogen
Tuberous Sclerosis, Lymphangioleiomyomatosis
TSC Biosample Repository and Natural History Database
The TSC Biosample Repository collects and stores samples of blood, DNA, and tissues that scientists can request to use in their research. The samples we collect are all linked to clinical data in the TSC Natural History Database.
- Trial name
- NCT05676099
- Testing
- Phlebotomy, Buccal (cheek) swab, Genetic Testing
- Where
- 26 sites in United States, Canada
- Volunteers
- 5,000 people
- Sponsor
- National Tuberous Sclerosis Association
Neuro-Ophthalmology, Optic Neuropathies
Optic Nerve Head Evaluation Through Multimodal Blood Flow Analysis: a Prospective Observational, Multi-center Study
OCT-A and eco-colordoppler are technologies which enable optic nerve head (ONH) blood perfusion detection. The aim of the study is to assess ONH vascular and structural components in optic neuropathies with and without ONH edema…
- Conditions
- Neuro-Ophthalmology, Optic Neuropathies, Optic Atrophy, Hereditary, Leber, Optic Neuropathy, Anterior Ischemic
- Trial name
- FlowONH (NCT07075887)
- Volunteers
- 70 people
- Sponsor
- Barboni
Refractory Myasthenia Gravis, Rituximab
Efficacy and Safety of Low-dose Rituximab in the Treatment of Refractory Myasthenia Gravis
This study is designed to evaluate the effectiveness and safety of low-dose rituximab in patients with refractory myasthenia gravis.
- Trial name
- NCT05332587
- Testing
- Rituximab
- Where
- China
- Volunteers
- 50 people
- Sponsor
- First Affiliated Hospital, Sun Yat-Sen University
Parkinson Disease, Dystonia
Osteopathic Manual Treatment Parkinson's Disease and Truncal Dystonia
This study will determine if posture and heart rate variability will significantly improve in Parkinsons disease with camptocormia after osteopathic manual treatments dystonia.
- Conditions
- Parkinson Disease, Dystonia, Autonomic Dysreflexia
- Trial name
- NCT03307161
- Testing
- osteopathic manual therapy
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- New York Institute of Technology
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
Muscular Atrophy, Spinal
An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
This expanded access program (EAP) will provide access to risdiplam for eligible participants with Type 1 or Type 2 spinal muscular atrophy (SMA) before it is commercially available in the United States for the indication of SMA.
- Trial name
- NCT04256265
- Testing
- Risdiplam
- Where
- 29 sites in United States
- Sponsor
- Genentech, Inc.
Leber Congenital Amaurosis 10, Blindness
Extension Study to Study PQ-110-001 (NCT03140969)
Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study for continued dosing if available data support current and/or future…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders
- Trial name
- INSIGHT (NCT03913130)
- Testing
- QR-110
- Where
- 3 sites in United States, Belgium
- Volunteers
- 9 people
- Sponsor
- Laboratoires Thea
Alström Syndrome (ALMS)
Clinical Study of a Single Ciliopathy: Alström Syndrome
The aim of the study is to characterize the clinical manifestations of ALMS within the ciliopathies to prevent complications and determine preventive and therapeutic targets.
- Trial name
- NCT02890550
- Where
- 14 sites in France
- Volunteers
- 75 people, healthy volunteers welcome
- Sponsor
- University Hospital, Strasbourg, France
Anti NMDA Receptor Encephalitis
Clinical-immunological Features of Anti-NMDAR Encephalitis
Using a retrospective cohort of 501 patients with anti-NMDAR encephalitis to assess clinical and immunological prognostic biomarkers
- Trial name
- Bio-NMDAr (NCT05738668)
- Testing
- Description of clinical characteristic
- Where
- France
- Volunteers
- 400 people
- Sponsor
- Hospices Civils de Lyon
Tourette Syndrome, Tourette's Disorder
Assessment of Children With Tic Onset in the Past 6 Months
The purpose of this research is to study why most children who have tics never develop Tourette syndrome but some do. In other words, we aim to find features that may predict whose tics will go away and whose tics will continue…
- Conditions
- Tourette Syndrome, Tourette's Disorder, Chronic Motor or Vocal Tic Disorder, Transient Tic Disorder
- Trial name
- NewTics (NCT01177774)
- Where
- United States
- Volunteers
- 99 people
- Sponsor
- Washington University School of Medicine
Motor Disorders
Combined Artificial Intelligence and Mobile Application for Remote Infant Motor Screening: Development and Validation
The purpose of this study is therefore five-fold: (1) designation of an APP "Baby Go" version 3.0 to include the assessment, follow-up, and education functions for parental use at home, (2) development and validation of the AI…
- Trial name
- NCT06521918
- Where
- Taiwan
- Volunteers
- 242 people, healthy volunteers welcome
- Sponsor
- National Taiwan University Hospital
Charcot-Marie-Tooth Disease
A Registered Cohort Study on Charcot-Marie-Tooth Disease
The aim of the study is to analyze the natural history data data from Charcot-Marie-Tooth disease and related disorders in China, to assess the clinical, genetic, epigenetic features of patients with Charcot-Marie-Tooth disease…
- Trial name
- NCT04010188
- Where
- China
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- Ning Wang, MD., PhD.
Huntington's Disease
HDClarity: a Multi-site Cerebrospinal Fluid Collection Initiative to Facilitate Therapeutic Development for Huntington's Disease
HDClarity will seek at least 2500 research participants at different stages of Huntington's disease (HD). The primary objective is to collect a high quality CSF sample for evaluation of biomarkers and pathways that will enable…
- Trial name
- HDClarity (NCT02855476)
- Where
- 40 sites in United States, Canada and 8 more
- Volunteers
- 2,500 people, healthy volunteers welcome
- Sponsor
- University College, London
Multiple Sclerosis - Relapsing Remitting, Clinically Isolated Syndrome, CNS Demyelinating
DTI in Children With Multiple Sclerosis
This is a prospective, non-randomised, non-blinded, single center study of children and adolescents with multiple sclerosis and clinically isolated syndrome to detect differences or early changes in diffusion-weighted imaging…
- Trial name
- NCT02361697
- Testing
- DTI-MRI
- Where
- Germany
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- University Hospital Muenster
Tourette's Disorder
Omega-3 Fatty Acids in Tourette's Disorder
This study examines the safety and efficacy of omega-3 fatty acids (fish oil) for the treatment of Tourette's Disorder.
- Trial name
- NCT01315327
- Testing
- Omega-3 Fatty Acids, Olive oil
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- NYU Langone Health
Neurofibromatosis 1, Social Skills
Pilot Randomized Control Trial of Telehealth Group for Improving Peer Relationships (PEERS) in NF1
This is a pilot randomized control trial of the UCLA PEERS protocol delivered via Telehealth with teens with neurofibromatosis type 1 whose parents report that they have difficulty making and keeping friends.
- Trial name
- NCT04860362
- Testing
- PEERS
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- University of Wisconsin, Milwaukee
Parkinson Disease
Parkinson's Research: The Organized Genetics Initiative
The purpose of this trial is to study genetic and other risk factors that may be important in the development of Parkinson's disease.
- Trial name
- PROGENI (NCT00086073)
- Where
- 58 sites in United States, Canada and 1 more
- Volunteers
- 1,800 people
- Sponsor
- Indiana University
Neurofibroma, Plexiform, Neurofibromatosis Type I
R115777 to Treat Children With Neurofibromatosis Type 1 and Progressive Plexiform Neurofibromas
This study will examine whether the experimental drug R115777 (Tipifarnib) can shrink or slow the growth of plexiform neurofibromas in children and young adults with neurofibromatosis type 1 (NF1) and determine what side effects…
- Trial name
- NCT00021541
- Testing
- tipifarnib
- Where
- 12 sites in United States, Germany
- Volunteers
- 62 people
- Sponsor
- National Cancer Institute (NCI)
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