The Goal of this study is to investigate if individuals ages 12 years and older, carrying the IL-4RαR576 gene variant, will have a greater response to therapy acting directly on the anti-IL-4R. This...
Despite major technological advances, management of type one diabetes mellitus (T1D) remains suboptimal, putting millions of people at risk for immediate and long-term complications. After meals, a...
This research study is evaluating a drug called ribociclib (LEE011) given in combination with everolimus and other standard of care chemotherapy drugs as a possible treatment for relapsed or...
Klippel-Feil syndrome (KFS) was first described in 1912 by Klippel and Feil as a classic triad are comprised of a short neck, a low posterior hairline and restricted motion of the neck. This disease...
Succinic Semialdehyde Dehydrogenase deficiency (SSADHD) is a rare autosomal recessive disease that interferes with the catabolism of the major inhibitory neurotransmitter gamma-amino butyric acid ...
This research study is evaluating the investigational software for magnetic resonance imaging (MRI) systems and techniques to process magnetic resonance (MR) images
Overall Aim: To evaluate the efficacy of continuous erector spinae block (ESB) versus continuous paravertebral block (PVB) for postoperative analgesia in children and adolescents undergoing surgical...
This phase II trial studies how well naive T-cell depletion works in preventing chronic graft-versus-host disease in children and young adults with blood cancers undergoing donor stem cell transplant....
ZYN002 is a pharmaceutically manufactured Cannabidiol that is developed as a clear gel that can be applied to the skin (called transdermal delivery).
The gel will be applied to clean, dry, intact...
ATHN 9 is a natural history study to assess the safety of various Von Willebrand Factor (VWF) regimens for different indications (on-demand, surgery and prophylaxis) in adult and pediatric...
This is a multicenter, open-label, extension study to evaluate the long-term safety, tolerability, and efficacy of treatment with AG-348 in participants who were previously enrolled in Study AG348-C...
The ICONICC Study is a randomized controlled clinical trial designed to compare the proportion of successful treatment outcomes between children assigned to standard concussion care only, standard...
This is a Phase III clinical trial to compare the efficacy of two dosages of a new infant rehabilitation protocol - I-ACQUIRE - to usual and customary forms of infant rehabilitation in infants who...
Effective patient education improves health literacy and engagement thus improving long-term health outcomes. Health literacy is imperative to make informed health decisions and relies on the ability...
CMV infection and disease remain a significant clinical challenge for pediatric solid organ transplant (SOT) recipients. Current prevention strategies are limited to prophylaxis in which antiviral...
Endometriosis is dependent on angiogenesis (the sprouting of new blood vessels) for its growth and maintenance, but the side effects of currently approved angiogenesis inhibitors make these agents...
This is an investigator initiated, multicenter, open label, randomized phase 3 study for subjects with newly diagnosed ITP from ages 1 to less than 18 years old.
The goal of this study is to test the hypothesis that fiberoptic confocal microscopy (FCM) imaging during repair of common congenital heart defects is a useful adjunct for avoidance of conduction...
This is a prospective, multi-center observational study. The study is designed to measure the clinical effectiveness of elexacaftor, tezacaftor and ivacaftor (ETI) triple combination therapy in people...
This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll...
