Patients with severe immune thrombocytopenia (ITP) present with similarly low platelet counts but varying bleeding symptoms, making it difficult to predict the disease course and to decide on an...
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 purpose of this study is to obtain preliminary data in advance of a larger clinical trial aimed to test whether a single session of green light exposure can lead to a clinically significant...
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...
This 2-year study will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic (PD) effects of ocrelizumab in children and adolescents ages ≥ 10 to ≤ 18 years with relapsing-remitting...
The objectives of this multicenter pragmatic clinical trial are to compare the effectiveness and relative safety of balanced fluid resuscitation versus 0.9% "normal" saline in children with septic...
In this research study we want to learn more about the character of neutrophils that are present in the blood of children with sepsis. Sepsis is a severe type of infection, affecting various parts of...
In this research study we want to learn more about the characteristics of neutrophils that are present in the blood and secretions from a breathing tube of patients with sepsis. Sepsis is a severe...
Investigators want to learn the role of indoor environmental exposures on respiratory symptoms, and, separately, on lung function deficits in school-aged children with bronchopulmonary dysplasia (BPD)...
Pain remains a common and frequently debilitating symptom, particularly during adulthood of Fibrous Dysplasia/McCune-Albright Syndrome (FD/MAS). For many FD/MAS patients, the amount of pain perceived...
The primary objective of this study is to evaluate several interventions given to participants with severe asthma. Interventions are administered in a crossover manner with 16-week treatment periods...
