Clinical trials: Pediatric Neurological Disorder
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.
Acute Liver Failure, Fulminant Hepatic Failure
TReatment for ImmUne Mediated PathopHysiology
TReatment for ImmUne Mediated PathopHysiology (TRIUMPH) is a multi-center, three arm, randomized, controlled trial of immunosuppressive therapy for children with acute liver failure.
- Conditions
- Acute Liver Failure, Fulminant Hepatic Failure, Hepatic Encephalopathy, Acute Liver Injury
- Trial name
- TRIUMPH (NCT04862221)
- Testing
- High-dose methylprednisolone, Equine anti-thymocyte globulin, Prednisolone
- Where
- 17 sites in United States
- Volunteers
- 44 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Concussion, Brain, Concussion, Mild
Mobile Subthreshold Exercise Program for Concussion--R01
Approximately 1.9 million youth sustain a concussion each year, and up to 30% experience persistent post-concussive symptoms (PPCS) such as headache, dizziness, and difficulty focusing that continue for weeks or months.
- Conditions
- Concussion, Brain, Concussion, Mild, Brain Injuries, Traumatic, Persistent Post Traumatic Headache
- Trial name
- MSTEP-R01 (NCT04688255)
- Testing
- MSTEP, Stretching
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Seattle Children's Hospital
Tonsillitis
Suprazygomatic Maxillary Nerve Block in Pediatric Tonsillectomy
Tonsillectomies are among the most common surgical procedures performed in pediatric populations, with more than 14,000 procedures performed annually in Ontario, with more than 80% being performed to treat sleep-disordered…
- Trial name
- NCT07176533
- Testing
- Suprazygomatic maxillary nerve block (SZMN) with bupivacaine
- Where
- Canada
- Volunteers
- 60 people
- Sponsor
- London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's
Cerebral Palsy
Effects of PEDAL Program With and Without EMS On Mobility And Gait In Children With Cerebral Palsy
PEDAL is a program that focuses on juvenile endurance and limb building for kids with cerebral palsy. Studying how PEDAL with and without Electrical Muscle Stimulation (EMS) affects these metrics in an effort to determine whether…
- Trial name
- NCT06460311
- Testing
- PEDAL and EMS, PEDAL
- Where
- Pakistan
- Volunteers
- 22 people
- Sponsor
- Riphah International University
Obsessive-Compulsive Disorder, Tic Disorder
Pathogenesis of Pediatric Obsessive-compulsive Disorder: Based on the Microbiota-gut-brain Axis
This study aims to investigate the characteristics of the microbiota-gut-brain axis in pediatric patients with obsessive-compulsive disorder (OCD) through performing an integrated analysis of gut microbiota, serum metabolomics…
- Trial name
- NCT06754319
- Where
- China
- Volunteers
- 125 people, healthy volunteers welcome
- Sponsor
- Shanghai Mental Health Center
Mechanically Ventilated, Critically Ill Children
Sedation Efficacy of Dexmedetomidine Versus Midazolam in Critically Ill Ventilated Children
There is a significant lack of adequately powered randomized clinical trial (RCT) data to determine the comparative safety and effectiveness of sedative treatments in pediatric patients.
- Trial name
- NCT04082767
- Testing
- Precedex, Midazolam
- Where
- Canada
- Volunteers
- 120 people
- Sponsor
- Douglas Fraser
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
Children's Distress During Flu Vaccination
Reducing Children's Distress Towards Flu Vaccinations
Millions of children in North America receive an annual flu vaccination, many of whom are at risk of experiencing severe distress. Children frequently use technologically advanced devices such as computers and cell phones.
- Trial name
- NCT01529021
- Where
- Canada
- Volunteers
- 57 people, healthy volunteers welcome
- Sponsor
- University of Calgary
Central Nervous System Neoplasms, Pediatric Cancer
Pediatric Prospective Personalized Immune and Target Identification Trial
PPROSPERITIT is a prospective clinical study assessing the use of comprehensive molecular profiling to define the best matching targeted and immune treatment for relapsed, refractory or very high risk pediatric CNS tumors.
- Conditions
- Central Nervous System Neoplasms, Pediatric Cancer, GEN1 Gene Mutation, Tumor, Brain
- Trial name
- PPROSPERITIT (NCT04859543)
- Testing
- FoundationOneHeme
- Where
- 3 sites in Austria, Czechia
- Volunteers
- 80 people
- Sponsor
- Masaryk University
Central Nervous System Tumor, Pediatric
The Use of 5-aminolevulinic Acid (ALA) as an Intraoperative Tumor Marker for Resection of Pediatric Central Nervous System (CNS) Tumors
Surgery is the cornerstone treatment of most pediatric CNS tumors, including astrocytomas, ependymomas, medulloblastomas, and many other pathologies.
- Trial name
- 5-ALA (NCT02050243)
- Testing
- 5ALA
- Where
- Israel
- Volunteers
- 100 people
- Sponsor
- Tel-Aviv Sourasky Medical Center
Gaucher Disease
Efficacy and Safety of Eliglustat in Chinese Pediatric Patients With Gaucher Disease Type 1 and Type 3
Primary Objective: Evaluate the efficacy and safety of eliglustat in Chinese pediatric patients (≥12 to <18 years old) with Gaucher disease type 1 and type 3.
- Trial name
- NCT06523517
- Testing
- Eliglustat Tartrate Capsules
- Where
- China
- Volunteers
- 5 people
- Sponsor
- Peking Union Medical College Hospital
Ehler's Danlos Syndrome, Obstructive Sleep Apnea
Obstructive Sleep Apnoea in Children and Adolescents With Ehlers-Danlos Syndrome
Ehlers-Danlos Syndrome (EDS) is a clinically and genetically heterogeneous group of inherited connective tissue disorders characterized by joint hypermobility, skin hyperextensibility, and tissue fragility.
- Trial name
- NCT02712060
- Testing
- No intervention
- Where
- Switzerland
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- University of Zurich
Diabetes Mellitus, Type 1, Behavior, Health
Game-based Pediatric Diabetes Education
The goal of this study is to evaluate the impact of a mobile app video game called Digital Vaccine for Type 1 Diabetes (DVx-T1D)™, proposed as a low-risk, non-invasive, digital therapeutic candidate for behavior change in…
- Trial name
- DVx-T1D (NCT06264258)
- Testing
- DVx-T1D™ app
- Where
- United States
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Carnegie Mellon University
Neurological Impairment
Music Therapy for Pain Modification in Pediatric Palliative Care
The purpose of this within-subjects randomized controlled trial is to determine whether two music therapy interventions are efficacious in reducing pain symptoms, in order to improve the quality of care for children in pediatric…
- Trial name
- NCT01456884
- Testing
- Live guitar and vocal music therapy, Vibroacoustic Music Therapy
- Where
- Canada
- Volunteers
- 13 people
- Sponsor
- University of British Columbia
Pain, Injection Site Reaction
Safety of Pediatric COVID-19 Vaccination
This is a prospective, observational study. During the study, children and adolescents (ages ≥ 5 to < 16) will be followed post administration of mRNA COVID-19 vaccines.
- Conditions
- Pain, Injection Site Reaction, Adverse Drug Event
- Trial name
- NCT05157191
- Testing
- Observational
- Where
- 5 sites in United States
- Volunteers
- 299 people, healthy volunteers welcome
- Sponsor
- Duke University
Healthy Volunteers (Meningococcal Infection)
Immunogenicity and Safety of a Quadrivalent Meningococcal Conjugate Vaccine When Administered Concomitantly With Routine Pediatric Vaccines in Healthy Infants and Toddlers in the US
The purpose of this study was to compare the immunogenicity and describe the safety of MenACYW conjugate vaccine and MENVEO® when both are administered concomitantly with routine pediatric vaccines to healthy infants and toddlers…
- Trial name
- NCT03537508
- Testing
- MenACYW conjugate vaccine, MenACYW-135 conjugate vaccine, DTaP-IPV//Hib vaccine
- Where
- 70 sites in United States, Puerto Rico
- Volunteers
- 2,627 people, healthy volunteers welcome
- Sponsor
- Sanofi Pasteur, a Sanofi Company
Duchenne Muscular Dystrophy (DMD)
A Study to Evaluate the Safety and Tolerability of RAG-18 in Pediatric Patients With Duchenne Muscular Dystrophy
This is an open-label, single-arm, dose-escalation trial to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary efficacy of RAG-18 in pediatric patients with Duchenne Muscular…
- Trial name
- NCT07282652
- Testing
- RAG-18
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Peking Union Medical College Hospital
Bone Marrow Disease, Pain
Study of Fentanyl-Propofol-EMLA of L.M.X4 Technique for Bone Marrow Aspiration
St. Jude Children's Research Hospital is studying the best ways to prevent pain during and after procedures such as bone marrow aspiration and lumbar puncture with intrathecal (in the spinal fluid) chemotherapy.
- Trial name
- NCT00187135
- Testing
- Fentanyl, EMLA, L.M.X4
- Where
- United States
- Volunteers
- 168 people
- Sponsor
- St. Jude Children's Research Hospital
Tourette Syndrome
Safety, Tolerability, and Efficacy of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome
This is a Phase 2b, randomized, double-blind, placebo-controlled, dose-optimization study to evaluate the efficacy, safety, and tolerability of NBI-98854 titrated to the subject's optimal dose administered once daily (qd) for a…
- Trial name
- NCT03325010
- Testing
- Valbenazine
- Where
- 35 sites in United States, Puerto Rico
- Volunteers
- 127 people
- Sponsor
- Neurocrine Biosciences
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