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MODERN-Dental: Pediatric Obesity, Cardiometabolic Risks, And Periodontal Disease: Effects Of Semaglutide Treatment
The goal of this clinical trial is to assess whether adjunctive dental cleaning can enhance the effects of semaglutide in children with obesity. The main quest…
Interventional, Randomized, Double-blind, Parallel-group, Placebo-controlled Study to Evaluate the Efficacy and Safety of IV Eptinezumab in Adolescents (12-17 Years) for the Preventive Treatment of Chronic Migraine
To find out if eptinezumab is better than placebo (normal saline solution) in lowering the number of days with migraine in young people ages 12 to 17 with chro…
Supraspinal Processing of Sensory Aspects of Pain
The goal of this basic science study is to learn about the brain mechanisms of chronic pain across different chronic pain syndromes in pediatric patients. The …
Study of Tegavivint, a Transducin Beta-like Protein 1 (TBL1) Inhibitor, With Gemcitabine in Patients With Relapsed or Refractory Osteosarcoma
The goal of this clinical trial is to define the maximum tolerated dose (MTD) and/or Recommended phase 2 dose (RP2D) of Tegavivint in combination with Gemcitab…
Electrophysiologic Sleep Phenotyping and Sleep-Dependent Neuro-maturation in Clinical and Healthy Pediatric Populations
Background: During the first few decades of life, the brain changes dramatically in shape and function. Sleep lets researchers measure these changes. Research…
6-Hz Primed Low and High-Frequency Repetitive Transcranial Magnetic Stimulation on Motor Function and Meta-Plasticity in Children With Cerebral Palsy: TMS-EEG Study
This project examines the use of repetitive transcranial magnetic stimulation (rTMS) as a therapeutic approach to improve motor function in children with cereb…
Vasodilators and Exercise as Adjuvant Therapy for Duchenne Muscular Dystrophy (VASO-REx Study)
Examining two strategies as potential adjuvant therapies for Duchenne muscular dystrophy (DMD); aerobic exercise training (to induce adaptations in skeletal mu…
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy, Safety, and Tolerability of DYNE-251 Administered Intravenously in Ambulatory Male Participants 4 to 18 Years of Age With Duchenne Muscular Dystrophy Amenable to Exon-51 Skipping
The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks…
Effect of Gamification on 6-Minute Walk Test Performance and Engagement in Pediatric Neuromuscular Patients
The goal of this study is to learn if adding a game to the 6-minute walk test for children with neuromuscular disorders will increase enjoyment and motivation …
A Randomized Controlled Trial of Ganaxolone for Children With Autism Spectrum Disorder
The goal of this study is to conduct a randomized, placebo-controlled trial (RCT) of ganaxolone, a neuroactive steroid (NAS), in autistic children and adolesce…
Implementing Precision Medicine for Glomerular Diseases in the Nephrotic Syndrome Study Network (NEPTUNE)
NEPTUNE Match is an additional opportunity offered to NEPTUNE study participants to prospectively recruit and communicate patient-specific clinical trial match…
Sinus Disease in Young Children With Cystic Fibrosis
This is a prospective, observational study examining the impact of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators on ch…