View clinical trials related to Crigler-Najjar Syndrome.
Filter by:Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions. With a simple blood test, rare genetic conditions can be easily detected, and the early start of transformative treatment will help avoid severe disabilities and increase the quality of life. Baby Detect Project is an innovative NBS program using a panel of target sequencing that aims to identify 126 treatable severe early onset genetic diseases at birth caused by 361 genes. The list of diseases has been established in close collaboration with the Paediatricians of the University Hospital in Liege. The investigators use dedicated dried blood spots collected between the first day and 28 days of life of babies, after a consent sign by parents.
This research involves collecting data about levator ani syndrome (LAS) associated rectal pain and a comparison of diazepam treatment administration routes. The goal of this research is to see if an alternative route of diazepam administration provides sufficient control of LAS discomfort and low sleep quality while minimizing systemic effects of diazepam (drowsiness).
This is a Phase 1/2, multinational, open-label, study to evaluate the safety and efficacy of an intravenous infusion of GNT0003 in patients with Crigler-Najjar aged ≥10 years and requiring phototherapy. Patients will received a single administration of GNT0003 and will be followed for safety and efficacy of approximately 60 months (5 years): - a follow-up of approximately 12 months (48 weeks) - a long term follow-up of approximately 48 months (4 years), in order to be in line with the latest EMEA Guideline on follow-up of patients administered with gene therapy medicinal products, released on 22 Oct.2009 by the Committee for medicinal products for human use.
All patients having received at least one infusion of the Investigational Medicinal Product (IMP) HepaStem HHALPC during a previous interventional clinical study conducted by Promethera Biosciences
This is a Phase 1/2, multinational, open-label, ascending-dose, delayed-treatment concurrent control clinical study to evaluate the safety and preliminary efficacy of AT342 in subjects with Crigler-Najjar aged ≥1 year. Subjects will receive a single dose of AT342 and will be followed for safety and efficacy for 5 years.
This is a Pre-Phase 1 prospective, non-interventional clinical assessment study to evaluate Crigler-Najjar syndrome subjects requiring daily phototherapy, aged 1 year and older.
Evaluating the efficacity of a new device phototherapy by comparing it with conventional phototherapy. Jaundice occurs in many newborns, and is, in most cases benign, However, owing to the potential neurotoxicity of unconjugated bilirubin, newborns must be monitored to identify those who might develop severe hyperbilirubinemia an, in rare cases, acute bilirubin encephalopathy or kernicterus. Treatment of jaundice in newborn relies on phototherapy, exposing their skin to light of a specific wavelength . Fluorescent tubes or halogen lamps have been used as light sources for phototherapy for many years. Light-emitting diodes (LEDs) are more recent sources which are power efficient, have a longer life and are portable with low heat production. Several technologies and devices are developed using LEDs and specially a compact system.
The proposed research protocol aims at addressing these points by pre-screening CN patients for their AAV serology in link with their medical history and current medical status. A first objective is to assess the presence of neutralizing AAV antibodies in the serum of CN patients.
The purpose of this study is to assess the long-term safety follow-up of patients having been treated with HepaStem.
The purpose of this study is to assess the safety and to appraise the efficacy of one cycle of Hepastem (Heterologous Human Adult Liver-derived Progenitor Cells, HHALPC) infusions in paediatric patients suffering from CN or UCD. The study duration: 12 months starting from the day of treatment: 6 months active surveillance and 6 months observation post-infusion.