NCT05898620
A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome
This study will evaluate the efficacy and safety profiles of the investigational gene therapy, NGN-401, in females with typical Rett syndrome.
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This study will evaluate the efficacy and safety profiles of the investigational gene therapy, NGN-401, in females with typical Rett syndrome.
The purpose of this study is to learn more about changes in glucose levels in hospitalized infants with intestinal failure receiving parenteral nutrition or PN (nutrients delivered intravenously), as ...
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 ...
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.
This is a prospective, single-blinded, randomized study to assess the ability of taping methods used to secure intravenous (IV) catheters to resist the IV from being pulled away from the skin. ...
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 ...