This is a trial in which 350 primary lung transplant recipients will be randomized (1:1) to receive either Tocilizumab (six doses over 20 weeks) plus standard triple maintenance immunosuppression or...
The goal of this study is to find the best way to help participants and families manage the stress of facing a serious illness and be better able to "bounce back" or be resilient after a difficult...
COMPRENDO (ChildhOod Malignancy Peer Research NavigatiOn) is a multi-site randomized clinical trial (RCT) that uses a Hybrid Type 1 design, to test the effects of a clinical intervention on patient...
This is a pediatric kidney transplant study comparing the safety and efficacy of an immunosuppressive regimen of belatacept and sirolimus to tacrolimus and Mycophenolate Mofetil (MMF). Two hundred...
In this research study, the investigators want to learn more about the role of new innovative surgical devices, the Karl Storz Curved and Straight Fetoscopes for in-utero surgery. A fetoscope is like...
Shwachman-Diamond syndrome (SDS) is a genetic condition characterized by bone marrow failure, medical co-morbidities, and leukemia predisposition. SDS-Like patients share clinical features with SDS...
This is a prospective multicenter biomarker study evaluating the prognostic impact of ctDNA detection at diagnosis in patients with Ewing sarcoma or osteosarcoma.
The goal of this study is to examine the safety and treatment effects of sirolimus for targeting social communication deficits in people with genetic disorders associated with PTEN germline mutations,...
Overall, this observational cohort study aims too:
Implement rapid trio WGS for all children presenting to our health systems with epilepsy onset under 12 months of age.
Utilize electronic healthcare...
This study will examine both Human papillomavirus (HPV) vaccine effectiveness and Primary high-risk HPV PHS screening triage strategies in women living with HIV (WLHIV) by partnering with the...
The REVEAL Pediatric Study is a multi-center, Phase 1/2 open-label, dose-escalation and dose-expansion study of TSHA-102, an investigational gene therapy, in pediatric females with Rett Syndrome.
The...
Calcium Release Deficiency Syndrome (CRDS) is a novel inherited arrhythmia syndrome secondary to RyR2 loss-of-function that confers a risk of sudden cardiac death. Diagnosis of CRDS presently requires...
The investigators will be evaluating bone marrow composition via magnetic resonance imaging in adolescents diagnosed with cystic fibrosis (CF) compared to healthy, matched controls. The investigators...
A within-subjects design will be used for this preliminary investigation of four biomarkers across two contexts of use: prediction of treatment response (i.e., stratification) and quantification of...
The goal of this study is to test a web-based psychoeducational resource for adolescents with central disorders of hypersomnolence and their families. The investigators hope to assess the website's...
The goal of this pilot trial is to learn more about the role of Fetal Endotracheal Occlusion (FETO) as an intervention in fetuses with severe congenital diaphragmatic hernia (CDH). The research team...
The goal of this research study is to learn whether investigators can successfully give the PediRISE program to families-in other words, whether most families are interested in participating in a...
In this research study, the investigators want to learn more about the safety and effectiveness of a fetal surgery, known as fetoscopic laser photocoagulation (FLP), for the treatment of a pregnancy...
The goal of this observational prospective study is to determine the health impact of parenthood on United States (US) people with CF in the era of CF transmembrane regulator protein (CFTR) modulators...
The aim of this study is to compare the impact of common (standard of care) language vs positive language used by clinicians during inhalational induction of anesthesia on anxiety and negative...
