Immunity Against AAV in Crigler Najjar Patient

Crigler Najjar Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02302690
• Other study ID #: CRIG-COL-01
• Status: Completed
• Phase: N/A
• First received: November 25, 2014
• Last updated: December 12, 2017
• Start date: November 2014
• Est. completion date: June 2017

Study information

• Verified date: December 2017
• Source: Genethon
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 49
• Est. completion date: June 2017
• Est. primary completion date: June 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Cardiomyopathies
• Crigler Najjar Syndrome
• Crigler-Najjar Syndrome
• Syndrome

Intervention

Biological:
• Blood sample for immunology

Locations

Country	Name	City	State
France	Hopital Antoine Béclère	Clamart

Sponsors (1)

Lead Sponsor	Collaborator
Genethon

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To assess the presence of neutralizing AAV antibodies in the serum of CN patients		1 day visit