The goal of this research study is to test the efficacy of a novel immunosuppressive agent, belumosudil, in allogeneic hematopoietic stem cell transplant (HSCT) recipients who have been newly...
The goal of this observational study is to identify candidate biomarkers in individuals with Rett Syndrome (RTT). The main questions it aims to answer are:
Do these biomarkers change during clinical...
Concurrent electroencephalography (EEG) and new tripolar EEG (tEEG) will be recorded from adult and pediatric patients. In some patients stereo EEG (sEEG) will also be recorded concurrently.
Teleconsultation, or the use of video telecommunications technology to deliver expert recommendations for care remotely, has been used to improve the safety and quality of emergency care for children...
The objective of the Pediatric Gastroparesis Registry 2 is to create a national prospective registry of children, adolescents, and young adults with gastroparesis and gastroparesis-like syndrome ...
The goal of this clinical trial is to determine the impact of a parental support package for new trainee mothers which focuses on mentorship, lactation, and sleep, and access to perinatal care. The...
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...
