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.
At Boston Children’s Hospital, clinical trials help us discover new and better ways to care for children. These studies give families the chance to explore promising treatments, especially when other options haven’t worked. Participation is always voluntary, and our team is here to guide you, answer questions, and support you throughout the process.
Search all trials by using the search tool or filtering by specific terms below.
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 ...
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 ...