Clinical trials: Pediatric Neurological Disorder
Obstructive Sleep Apnea, Respiratory Insufficiency
Study of the Usability and Efficacy of a New Pediatric CPAP Mask
This study will evaluate a newly developed pediatric mask (known as Pixi) on children aged 2-7 using continuous positive airway pressure (CPAP), or Non-invasive ventilation (NIV) treatment.
- Trial name
- NCT01312948
- Testing
- Prototype mask (known as Pixi)
- Where
- Australia
- Volunteers
- 6 people
- Sponsor
- ResMed
Restless Legs Syndrome
Study to Investigate Pharmacokinetics (PK) of Pramipexole in Pediatric Patients Who Are Individually Optimized to Stable Pramipexole Doses for the Treatment of Idiopathic Restless Legs Syndrome (RLS)
Study to determine the pharmacokinetics (PK) of pramipexole (PPX) after administration of a single dose orally (p.o.) in pediatric patients with the diagnosis of RLS
- Trial name
- NCT02231918
- Testing
- MIRAPEX® - low, MIRAPEX® - medium, MIRAPEX® - high
- Volunteers
- 26 people
- Sponsor
- Boehringer Ingelheim
Amblyopia, Strabismus
Amblyopia and Strabismus Detection Using a Pediatric Vision Scanner
The purpose of this project is to compare the performance of the Pediatric Vision Scanner (PVS) with the Gold Standard Examination to determine the effectiveness of the PVS at detecting amblyopia and strabismus.
- Trial name
- NCT01706991
- Testing
- Pediatric Vision Scanner eye scan
- Where
- United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Rebiscan, Inc.
Muscular Dystrophy, Duchenne
An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen)
The purpose of this extension study is to evaluate the safety, tolerability, and pharmacokinetics of repeat administrations of SRP-5051 (vesleteplirsen) in participants with Duchenne muscular dystrophy (DMD) who participated in…
- Trial name
- NCT03675126
- Testing
- SRP-5051
- Where
- 8 sites in United States, Canada
- Volunteers
- 15 people
- Sponsor
- Sarepta Therapeutics, Inc.
Cerebral Palsy
Efficacy of a Rehabilitation Program With the Pediatric Exoskeleton ATLAS 2030 in Pediatric Patients With Cerebral Palsy
Cerebral Palsy (CP) is the first cause of motor disability in children worldwide. ATLAS 2030 is a robotic gait exoskeleton designed to rehabilitate children with motor disability.
- Trial name
- NCT05926635
- Testing
- ATLAS 2030
- Where
- 5 sites in Spain
- Volunteers
- 60 people
- Sponsor
- MarsiBionics
Metachromatic Leukodystrophy (MLD)
A Study to Learn About Metachromatic Leukodystrophy (MLD) in Children in Spain
The main aim of this study is to learn about metachromatic leukodystrophy (MLD) in children and teenagers in Spain. This includes checking the number of new MLD cases and the frequency of children with MLD in 2022 in Spain.
- Trial name
- mECHromatic (NCT05755568)
- Testing
- No Intervention
- Where
- 10 sites in Spain
- Sponsor
- Takeda
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
Generalized Myasthenia Gravis
An Extension Study Evaluating 6-week Treatment Cycles of Rozanolixizumab in Pediatric Study Participants With Generalized Myasthenia Gravis
The purpose of the study is to assess the long term safety and tolerability of additional 6-week treatment cycles with rozanolixizumab in pediatric participants with generalized Myasthenia Gravis (gMG) aged ≥2 at the time of the…
- Trial name
- NCT06540144
- Testing
- rozanolixizumab
- Where
- 9 sites in Italy, Japan and 2 more
- Volunteers
- 13 people
- Sponsor
- UCB Biopharma SRL
Cerebral Palsy (CP)
Evaluating the Bambini Teens Exoskeleton for Pediatric Cerebral Palsy
The goal of this clinical trial is to evaluate the safety and effectiveness of the Bambini Teens exoskeleton as a gait training tool in children aged 5 to 14 years diagnosed with cerebral palsy (CP).
- Trial name
- NCT07146932
- Testing
- Pediatric Lower Limb Exoskeleton (Bambini Teens)
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- COSMO ROBOTICS CO., Ltd
Autism, Autistic Disorder
Safety and Efficacy Study in the Treatment of Intestinal Problems Associated With Autism
The purpose of this study is to determine if human immunoglobulin given by mouth twice a day is effective in treating the persistent gastrointestinal (GI) problems such as diarrhea, constipation, abdominal pain, and bloating, in…
- Conditions
- Autism, Autistic Disorder, Child Development Disorders, Pervasive, Gastrointestinal Diseases
- Trial name
- NCT00110708
- Testing
- Oralgam (human immunoglobulin)
- Where
- 22 sites in United States
- Volunteers
- 120 people
- Sponsor
- PediaMed Pharmaceuticals
Neurogenic Detrusor Overactivity
Open-label, Long-term Safety, Efficacy, and Pharmacokinetics Study of Vibegron in Pediatric Subjects 2 Years to < 18 Years of Age With NDO and on CIC
The purpose of this study is to evaluate the safety, efficacy, and PK of Vibegron in pediatric participants with NDO who are regularly using CIC
- Trial name
- KANGUROO (NCT05491525)
- Testing
- Vibegron
- Where
- 38 sites in United States, Belgium and 14 more
- Volunteers
- 71 people
- Sponsor
- Urovant Sciences GmbH
Epilepsy
Safety and Tolerability Study of Diazepam Buccal Film (DBF) in Subjects With Epilepsy
This Phase 3, multicenter, open-label study of chronic, intermittent use of study drug (DBF) is designed to evaluate the safety and tolerability of the buccal formulation of diazepam in children, adolescents and adults with…
- Trial name
- DBF (NCT03428360)
- Testing
- Diazepam Buccal Soluble Film 5, 7.5,10, 12.5,15 or 17.5 mg with individual dose determined according to age and weight category
- Where
- 6 sites in United States
- Volunteers
- 149 people
- Sponsor
- Aquestive Therapeutics
Limb-girdle Muscular Dystrophy
A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4
This is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4).
- Trial name
- EMERGENE (NCT06246513)
- Testing
- SRP-9003, Glucocorticoid
- Where
- 10 sites in United States, Belgium and 4 more
- Volunteers
- 17 people
- Sponsor
- Sarepta Therapeutics, Inc.
Pediatric Growth Hormone Deficiency
A Study to Compare the Experiences of Taking Daily Growth Hormone Injections to Weekly Ngenla in Children With Low Levels of Growth Hormone
The purpose of this study is to learn about: how children stick to taking their injections their experience with the study medicines (Ngenla and daily growth hormone) prescribed to children with low levels of growth hormone.
- Trial name
- NCT06113952
- Testing
- Sharps bin to collect used needles/injections
- Sponsor
- Pfizer
Cerebral Palsy, Muscle Spasticity
Dysport® Pediatric Lower Limb Spasticity Study
The purpose of this research study is to determine whether Dysport® is effective in the treatment of increased stiffness of the calf muscles and to evaluate the safety of this treatment in children with Cerebral Palsy.
- Conditions
- Cerebral Palsy, Muscle Spasticity, Children
- Trial name
- NCT01249417
- Testing
- Botulinum toxin type A
- Where
- 29 sites in United States, Chile and 4 more
- Volunteers
- 241 people
- Sponsor
- Ipsen
Solid Tumor, Unspecified, Child, Relapsed Solid Neoplasm
A Study of Avapritinib in Pediatric Patients With Solid Tumors Dependent on KIT or PDGFRA Signaling
This is a Phase 1/2, multicenter, open-label trial of avapritinib in participants 2 to < 18 years of age with advanced relapsed/refractory (R/R) solid tumors, including central nervous system (CNS) tumors, that harbor a PDGFRA…
- Conditions
- Solid Tumor, Unspecified, Child, Relapsed Solid Neoplasm, CNS Tumor
- Trial name
- NCT04773782
- Testing
- avapritinib
- Where
- 26 sites in United States, Australia and 7 more
- Volunteers
- 29 people
- Sponsor
- Blueprint Medicines Corporation
Migraine
Fremanezumab Managed Access Program for Pediatric Patients
This is a Managed Access Program (MAP) based on a licensed physician's unsolicited request on a compassionate basis. Specifically, this MAP is for male and female pediatric patients 6 to 17 years with a diagnosis of either…
- Trial name
- NCT03539393
- Testing
- Fremanezumab
- Sponsor
- Teva Branded Pharmaceutical Products R&D LLC
Hunter Syndrome
Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome
The purpose of the study is to collect data on CSF biomarkers in patients with Hunter Syndrome that would serve as reference data for comparison with cognitively impaired patients with Hunter syndrome, patients with other…
- Trial name
- NCT01449240
- Testing
- No treatment
- Where
- 7 sites in United States, United Kingdom
- Volunteers
- 10 people
- Sponsor
- Shire
Fibromyalgia
Adolescent Fibromyalgia Study
This study will compare pregabalin with placebo over a 15 week period in adolescents with fibromyalgia aged 12-17 years to evaluate the safety and efficacy of pregabalin.
- Trial name
- NCT01020474
- Testing
- pregabalin (Lyrica)
- Where
- 36 sites in United States, Czechia and 2 more
- Volunteers
- 107 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Amblyopia, Strabismus
Amblyopia and Strabismus Detection Using a Pediatric Vision Scanner
The purpose of this project is to compare the performance of the Pediatric Vision Scanner (PVS) with the Gold Standard Examination to determine the effectiveness of the PVS at detecting amblyopia and strabismus.
- Trial name
- NCT01832883
- Testing
- Pediatric Vision Scanner
- Where
- United States
- Volunteers
- 78 people, healthy volunteers welcome
- Sponsor
- Rebiscan, Inc.