Crigler-Najjar syndrome – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Sat, 13 Jun 2026 04:03:16 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Crigler-Najjar syndrome – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Study of GNT0003 and imlifidase in adults with Crigler-Najjar syndrome who require daily phototherapy and have pre-existing AAV8 antibodies https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-gnt0003-and-imlifidase-for-adults-with-severe-crigler-najjar-syndrome-and-anti-aav8-antibodies/ Wed, 29 Apr 2026 15:01:44 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-gnt0003-and-imlifidase-for-adults-with-severe-crigler-najjar-syndrome-and-anti-aav8-antibodies/ This study focuses on treating patients with Crigler-Najjar syndrome, a severe condition that requires daily light therapy (phototherapy) to control high levels of bilirubin in the blood. The main treatment being tested combines two medications: GNT0003, which is a gene therapy given through an intravenous infusion, and imlifidase, which is given before the gene therapy. GNT0003 contains a modified virus that carries a working copy of the UGT1A1 gene, which is defective in people with this condition.

The purpose of this study is to determine if this combination treatment can help reduce bilirubin levels in adults with Crigler-Najjar syndrome who have existing antibodies against the viral carrier. During the study, participants will receive imlifidase followed by a single dose of GNT0003. Other medications that may be used during the study include sirolimus, prednisolone, and methylprednisolone to help manage the body’s response to treatment.

The study will monitor participants for 60 months after treatment to check if the therapy is working and safe. Researchers will measure bilirubin levels in the blood and track how long participants can stay off phototherapy. They will also monitor participants’ overall health and quality of life throughout the study period.

]]> Study on Gene Therapy GNT0003 for Patients with Severe Crigler-Najjar Syndrome Requiring Phototherapy https://clinicaltrials.eu/trial/study-on-gene-therapy-gnt0003-for-patients-with-severe-crigler-najjar-syndrome-requiring-phototherapy/ Wed, 29 Apr 2026 14:26:14 +0000 https://clinicaltrials.eu/trial/study-on-gene-therapy-gnt0003-for-patients-with-severe-crigler-najjar-syndrome-requiring-phototherapy/ This clinical trial is focused on studying a rare genetic condition called Crigler-Najjar syndrome. This condition affects the liver’s ability to process a substance called bilirubin, leading to its buildup in the body. Patients with severe forms of this syndrome often require daily light therapy, known as phototherapy, to help manage bilirubin levels. The study is testing a new treatment called GNT0003, which is a type of gene therapy. This treatment involves using a specially designed virus to deliver a healthy version of the gene responsible for processing bilirubin directly into the liver cells. The goal is to help the liver function more normally and reduce the need for phototherapy.

The purpose of the study is to evaluate the safety and effectiveness of a single dose of GNT0003 given through an intravenous infusion, which means it is delivered directly into the bloodstream. The study is divided into two parts. The first part focuses on determining the safest dose of the treatment, while the second part assesses how well the treatment works at the selected dose. Participants will receive the treatment and be monitored over several weeks to see how their bodies respond. This includes checking for any side effects and measuring changes in bilirubin levels.

Throughout the study, participants will have regular check-ups, including blood tests and other assessments, to ensure their safety and to track the treatment’s impact on their health. The study aims to provide valuable information on whether this gene therapy can offer a new way to manage Crigler-Najjar syndrome and improve the quality of life for those affected by this condition. The trial is expected to continue until 2028, allowing researchers to gather comprehensive data on the long-term effects of the treatment.

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