This will be a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of AGTC-401 administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations...
This study uses frequency domain near-infrared spectroscopy coupled with diffuse correlation spectroscopy (FDNIRS-DCS) technology for monitoring cerebral blood flow (CBF) and cerebral oxygen...
Rapid onset Obesity, Hypoventilation, Hypothalamic dysfunction and Autonomic Dysregulation (ROHHAD) is a syndrome named in 2007. The hallmark of the syndrome is the rapid onset obesity and...
This is a Phase 2, open-label, single arm trial of pamrevlumab (FG-3019) to estimate pamrevlumab's safety and efficacy in non-ambulatory participants with DMD.
The primary objective of the study is to assess the long-term safety of dupilumab in pediatric participants with AD.
The secondary objectives of the study are:
To assess the long-term efficacy of...
Few studies are specifically designed to address health concerns relevant during pregnancy. The consequence is a lack of evidence on best clinical practice. This includes mothers and their babies when...
The goal of this trial is to compare the efficacy of the Bridge-Enhanced Anterior Cruciate Ligament Repair (BEAR™) technique with the current method of treatment for anterior cruciate ligament (ACL)...
This is a multi-center, long-term safety and efficacy follow-up study for participants with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product (eli-cel) in a parent clinical...
Laparoscopic cholecystectomy is associated with considerable postoperative pain and surgeon-administered local anesthetic infiltration is the standard practice for achieving post-operative analgesia....
This is a nonrandomized study of ruxolitinib in combination with a standard multi-agent chemotherapy regimen for the treatment of B-cell acute lymphoblastic leukemia. Part 1 of the study will optimize...
The Gene Discovery Core at The Manton Center for Orphan Disease Research based at Boston Children's Hospital studies families with rare, poorly understood or undiagnosed, but suspected genetic...
Pediatric ACL: Understanding Treatment Outcomes (PLUTO) is a multi-center, prospective cohort study. Specific aims of PLUTO are to evaluate the safety and comparative effectiveness of non-operative...
The purpose of this study is to improve the ability of the investigators to monitor brain health in newborn babies at risk of brain injuries. The researchers will be using an investigational system of...
Parents of infants who have been thrombocytopenic for 3-4 days will be approached for consent to enter the study. For the purposes of the study, thrombocytopenia will be defined as a platelet count ...
The goal of Advanced Fetal Imaging - Phase II is to advance fetal MRI imaging by designing MRI coils specifically for pregnant women and testing recently developed MRI image acquisition techniques....
The purpose of this study is to determine the feasibility of comparing outcomes of patients treated de novo with immunosuppressive therapy (IST) versus matched unrelated donor (MUD) hematopoietic stem...
The investigators propose a robust therapeutic intervention to ameliorate myocardial ischemia/ reperfusion injury and significantly decrease morbidity and mortality in patients requiring...
The purpose of this initiative is to improve care and outcomes for infants with HLHS by expanding the NPC-QIC national registry to gather clinical care process, outcome, and developmental data on...
The main objective of this proposed investigation is to evaluate blood ropivacaine concentrations in infants and toddlers following the initiation of, and over the course of, continuous paravertebral...
This proposal sets forth the platform for a Precision Medicine clinical trial through the New Approaches to Neuroblastoma Therapy (NANT) consortium. The plan is to utilize NANT's established multi...
