Immunity Against AAV in Crigler Najjar Patient

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.

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Key information

Conditions

Crigler Najjar Syndrome Congenital, Hereditary, and Neonatal Diseases and Abnormalities Crigler-Najjar Syndrome Genetic Diseases, Inborn Hyperbilirubinemia, Hereditary Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Sex eligibility

All sexes

Study type

Observational

Primary location

Hopital Antoine Béclère

Clamart, 92141, France

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

The patient has a Crigler-Najjar syndrome
The molecular diagnosis has been confirmed by UGT1A1 gene DNA sequencing

Exclusion criteria

The patient is liver transplanted
The patient is suffering from severe associated pathologies precluding further enrolment in a future interventional clinical study

Treatment and study plan

Blood sample for immunology

Biological

Primary outcomes

To assess the presence of neutralizing AAV antibodies in the serum of CN patients

Time frame: 1 day visit

Sponsors and collaborators

Lead sponsor

Genethon

Other

Registry information

Official study title

Study of the Prevalence of Pre-existing Immunity Against the Adeno-associated Virus (AAV) in Patients With Crigler-Najjar Syndrome

Important dates

Study start: 2014
Primary completion: 2017
Study completion: 2017
First posted: Nov 27, 2014
Registry last updated: Dec 13, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed