Clinical trials: Neurological Disorders
- Condition Neurological Disorders
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study.
- Trial name
- EMBARK (NCT05096221)
- Testing
- delandistrogene moxeparvovec
- Where
- 42 sites in United States, Belgium and 7 more
- Volunteers
- 126 people
- Sponsor
- Sarepta Therapeutics, Inc.
Dravet Syndrome
A Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Fenfluramine (Hydrochloride) in Infants 1 Year to Less Than 2 Years of Age With Dravet Syndrome
The primary purpose of this study is evaluate the safety and tolerability of fenfluramine hydrochloride (HCl) 0.2 to 0.8 mg/kg/day in infants 1 year to less than 2 years of age with Dravet syndrome.
- Trial name
- ORCHID (NCT06118255)
- Testing
- fenfluramine
- Where
- 12 sites in United States, Belgium and 3 more
- Volunteers
- 25 people
- Sponsor
- UCB BIOSCIENCES, Inc.
Brain Tumor, Recurrent, Brain Tumor
Registry of Patients With Brain Tumors Treated With STaRT (GammaTiles)
The objectives of this registry study are to evaluate real-world clinical outcomes and patient reported outcomes that measure the effectiveness and safety of STaRT.
- Conditions
- Brain Tumor, Recurrent, Brain Tumor, Brain Tumor, Primary, Brain Tumor - Metastatic
- Trial name
- NCT04427384
- Testing
- GammaTile
- Where
- 54 sites in United States
- Volunteers
- 600 people
- Sponsor
- GT Medical Technologies, Inc.
Hydrocephalus
Assessment of CSF Shunt Flow and Headaches With a Thermal Measurement Device
This study evaluates the performance of a device for non-invasively assessing cerebrospinal fluid (CSF) shunt flow over multiple measurements performed by a study subject during headache and non-headache periods.
- Trial name
- NCT06996600
- Testing
- wireless thermal anisotropy measurement device
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Rhaeos, Inc.
Epilepsy With POS With or Without Secondary Generalization
Study to Look at How Effective Briviact is as add-on Treatment for Patients With Epilepsy With Partial Onset Seizures
Study is the first study after commercialization of brivaracetam. It is designed to collect real world information on the effectiveness of brivaracetam in patients with Partial Onset Seizure epislepsy who are treated in standard…
- Trial name
- BASE (NCT02687711)
- Where
- 44 sites in Denmark, Germany and 7 more
- Volunteers
- 544 people
- Sponsor
- UCB Biopharma S.P.R.L.
Amblyopia
Prospective Evaluation of a Smartphone Application, GoCheckKids™, to Detect Amblyopia Risk Factors in Young Children
The objective is evaluation of GoCheck Kids, a smartphone application ('App'), for use as a photo screening device in a pediatric population to detect amblyopia risk factors (ARFs).
- Trial name
- NCT03447171
- Testing
- App Vision Screening
- Where
- United States
- Volunteers
- 207 people
- Sponsor
- Gobiquity Mobile Health
Fibromyalgia
Pregabalin In Adolescent Patients With Fibromyalgia
The main purpose of this study is to evaluate the long-term safety of pregabalin in adolescent patients who participated in the previous fibromyalgia study (A0081180) and who wish to recieve open-label pregabalin
- Trial name
- NCT01020526
- Testing
- pregabalin
- Where
- 26 sites in United States, Czechia and 1 more
- Volunteers
- 63 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Parkinson's Disease (PD)
Coordinated Reset Deep Brain Stimulation
The study will explore the viability of an alternative/novel programming strategy in Deep Brain Stimulation (DBS) for patients with Parkinson's Disease (PD).
- Trial name
- RESET-DBS (NCT03732898)
- Testing
- Novel Programming (nDBS), Conventional Programming (cDBS)
- Where
- 2 sites in Germany
- Volunteers
- 15 people
- Sponsor
- Boston Scientific Corporation
GM1 Gangliosidosis
Interviews and Video Capture in Patients With GM1 Gangliosidosis
GM1 gangliosidosis is a rare disease for which there is a limited understanding of disease progression and meaningful outcome measures.
- Trial name
- NCT04310163
- Testing
- Natural history
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- LYSOGENE
GM2 Gangliosidosis, Niemann-Pick Type C Disease
A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
This open-label study aims to gather long-term safety, tolerability, PK, biomarker, and clinical efficacy data relating to daily administration of Nizubaglustat in participants previously enrolled in the Phase 2 RAINBOW study…
- Trial name
- PRISMA (NCT07399704)
- Testing
- AZ-3102
- Where
- 3 sites in Brazil
- Volunteers
- 21 people
- Sponsor
- Azafaros B.V.
Spinal Muscular Atrophy (SMA)
Virtual Reality for the Mitigation of Anxiety During Intrathecal Administration in Participants With Spinal Muscular Atrophy
The primary objective of the study is to evaluate anxiety level during intrathecal administration (IT) under standard of care (SOC) and virtual reality (VR) conditions using a reliable self-rating scale.
- Trial name
- REALITY (NCT05354414)
- Testing
- Virtual reality, Standard of Care
- Where
- 12 sites in France
- Volunteers
- 63 people
- Sponsor
- Biogen
Meningitis, Meningococcal
Study Evaluating Safety, Tolerability, and Immunogenicity of Meningococcal B Vaccine in Healthy Infants
The primary purpose of this study is to evaluate the safety and immunogenicity of an investigational meningococcal B vaccine in healthy infants.
- Trial name
- NCT00798304
- Testing
- meningococcal B rLP2086 vaccine, Routine age appropriate childhood vaccines
- Where
- 7 sites in Spain
- Volunteers
- 46 people
- Sponsor
- Pfizer
Fragile x Syndrome
Ganaxolone Treatment in Children With Fragile X Syndrome
This Phase 2 proof-of-concept study is a double-blind, randomized, placebo-controlled, crossover study to investigate ganaxolone treatment in children with fragile x syndrome (FXS).
- Trial name
- NCT01725152
- Testing
- Ganaxolone
- Where
- 2 sites in United States, Belgium
- Volunteers
- 59 people
- Sponsor
- Marinus Pharmaceuticals
Multiple Sclerosis, Progressive Multifocal Leukoencephalopathy
Risk of Progressive Multifocal Leukoencephalopathy Among Patients With Multiple Sclerosis Exposed to Zeposia
This study will evaluate the risk of progressive multifocal leukoencephalopathy (PML), a rare brain infection caused by John Cunningham virus (JCV), among people with multiple sclerosis who have been treated with ozanimod.
- Trial name
- NCT07732907
- Testing
- Ozanimod
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Bristol-Myers Squibb
Friedreich Ataxia
A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
The primary objective of the study is to evaluate the efficacy (using the modified Friedreich Ataxia Rating Scale [mFARS]) and safety of vatiquinone in participants with Friedreich ataxia (FA).
- Trial name
- MOVE-FA (NCT04577352)
- Testing
- Vatiquinone
- Where
- 14 sites in United States, Australia and 7 more
- Volunteers
- 146 people
- Sponsor
- PTC Therapeutics
Fabry Disease
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.
- Trial name
- FLY (NCT06328608)
- Testing
- PRX-102 1 mg/kg every two weeks
- Where
- 12 sites in United States, Austria and 4 more
- Volunteers
- 22 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Diphtheria, Tetanus
To Evaluate the Immunogenicity and Safety of DTaP-IPV Vaccine Administered as a Boosting Dose to Healthy Children of 4-6 Years
The study objective is to assess the immunogenicity and safety of DTaP-IPV combination vaccine administered as a boosting dose to healthy 4 to 6-year-old children who received three doses of primary immunization against…
- Conditions
- Diphtheria, Tetanus, Pertussis, Poliomyelitis
- Trial name
- Aladdin (NCT04618640)
- Testing
- DTaP-IPV combination vaccine
- Where
- 26 sites in South Korea
- Volunteers
- 249 people, healthy volunteers welcome
- Sponsor
- Boryung Biopharma Co., Ltd.
Epilepsy, Epilepsy in Children
OASIS: RetrOspective Analysis on EEGs for Identifying Seizure Susceptibility in paediatrIcs Using biomarkerS
The goal of this retrospective study is to validate a set of computational biomarkers (BioEP) for seizure susceptibility on retrospective routinely collected non-contributory EEGs in paediatric participants with epilepsy.
- Trial name
- OASIS (NCT06546410)
- Testing
- BioEP
- Where
- 7 sites in United Kingdom
- Volunteers
- 530 people
- Sponsor
- Neuronostics Ltd
Cluster Headache
Data Collection on the ATI Neurostimulation System: SPG Stimulation for Cluster Headache
The primary objectives of the Registry are to: 1. Monitor the transfer of the ATI Neurostimulation System and its safety/clinical performance to a larger number of centers in the post market phase and 2.
- Trial name
- NCT01677026
- Where
- 11 sites in Denmark, Germany
- Volunteers
- 150 people
- Sponsor
- Autonomic Technologies, Inc.
Hearing Loss
CLEAR Clinical Study
Acute in-booth assessment of a new noise reduction algorithm with cochlear implant recipients.
- Trial name
- NCT03126825
- Testing
- CP910/CP920 with noise reduction on, CP910/CP920
- Where
- 2 sites in Australia
- Volunteers
- 48 people
- Sponsor
- Cochlear
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