Immunity Against AAV in Crigler Najjar Patient

Genethon

Status: Completed

Conditions: Crigler Najjar Syndrome

Treatments: Biological: Blood sample for immunology

Study type: Observational

Funder types: Other

Identifiers

NCT02302690

CRIG-COL-01

Details and patient eligibility

About

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.

Enrollment: 49 patients

Sex: All

Volunteers: No Healthy Volunteers

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