Clinical trials

Cerebral palsy (CP) is the most common physical disability in early childhood causing serious motor and sensory impairments. Effective interventions for the recovery of motor functions are of profound significance to children with CP, their families, caregivers, and health professionals. Robot-assisted rehabilitation represents a frontier with potential to improve motor functions and induce brain reorganization in children with CP.

This project examines the use of repetitive transcranial magnetic stimulation (rTMS) as a therapeutic approach to improve motor function in children with cerebral palsy (CP). By applying 6-Hz primed low and high-frequency rTMS and measuring brain responses through TMS-EEG, the study aims to enhance neural plasticity and motor recovery. The goal is to promote faster rehabilitation and reduce long-term healthcare needs.

The investigators propose a study that compares breathlessness and airway obstruction during intense exercise in 34 children and adolescents with Exercise-Induced Laryngeal Obstruction after breathing in ipratropium bromide or placebo. It is hypothesized that breathlessness and airway obstruction will be lower following breathing in ipratropium bromide compared with placebo.

The purpose of this Phase II clinical trial is to establish the safety and effectiveness of trametinib, a targeted therapy, for the treatment of newly or recently diagnosed Langerhans Cell Histiocytosis (LCH) among pediatric patients.

This study aims to test the overall hypothesis that the membrane tissue binding capacity of cytokines in the biopsied tissue of patients with Inflammatory Bowel Disease (IBD) is predictive of/strongly correlated to clinical response/outcomes observed. The key questions under investigation are: Aim 1: To assess the fluorescent signal intensity at baseline (control antibody with control biopsy and control antibody with IBD biopsy). Aim 2: To characterize the cellular landscape by surveying surface markers using bar-coded antibodies and performing gene expression profiling on every cell within inflamed tissue of patients with IBD. Aim 3: Develop algorithm using artificial intelligence to predict responders versus non-responders and to further subclassify IBD patients using phenotype data.

The overarching goal of this study is to establish a first ever registry in the U.S. to collect outcomes data to evaluate changes in the gastrointestinal (GI) mucosa following direct food application utilizing Confocal Laser Endomicroscopy (CLE). This will be assessed in pediatric and adult patients who present to the outpatient clinic with persistent irritable bowel syndrome-like symptoms while testing negative for celiac disease and have either negative or very low/low levels of Immunoglobulin E (IgE) serological tests.

Low blood sugars are known to cause brain damage in newborn babies. One of the most common causes of low blood sugars persisting beyond the new born period is a condition called congenital hyperinsulinism (HI). This is a disease whereby the pancreas secretes too much insulin and causes low blood sugars. Twenty to forty percent of these babies will have brain damage. There are two forms of this disease. In one form only a small part of the pancreas makes too much insulin (focal HI) and in the other, the whole pancreas make too much insulin (diffuse HI). Another very similar disease is insulinoma which occurs after birth, but also causes hyperinsulinism. If a surgeon could know which part of the pancreas has the focal lesion he could remove it and cure the patient. The purpose of this study is to investigate whether a new investigational drug called Fluorodopa F 18, when used with a PET scan, can find the focal lesion and guide the surgeon to remove it, thus curing the patient and preventing further brain damage.

The purpose of this research is to look at the effect of massage therapy on the pain, anxiety, and quality of life that pediatric patients have after undergoing spinal fusion surgery. This is a single-site, prospective, randomized, interventional study design that will involve post-thoracic and post-lumbar spinal fusion surgeries of pediatric patients from 7 to 19 years of age that present to Cook Children's Medical Center in Fort Worth, Texas. These patients will be identified prior to their scheduled spinal fusion surgery and recruited to enroll in the study. The planned spinal fusion surgeries are not considered part of this research project, but rather considered standard of care and would occur whether the patient is enrolled in this project or not. Enrolled participants will be followed during their inpatient stay and through their subsequent follow-up visits at weeks 2, 6, and 12. Participants will be randomly assigned to either a massage therapy group or a group that receives the standard (normal) care for recovery after surgery. The final study involvement will occur at week 16 (post-hospital discharge) where a study team member will administer a quality of life (PedsQL) questionnaire via phone or mail with the subject. Data will be collected after study related procedures are completed.

This study will non-invasively obtain levels of GABA in the brain of children with SCN1A+DS and neurodeveloping children through evoked and induced cortical responses, correlate them with the BOLD responses, and with the levels of GABA in their blood.