Immunity Against AAV in Crigler Najjar Patient | NCT02302690

Studies
Immunity Against AAV in Crigler Najjar Patient

NCT02302690

Sponsor

Genethon

Status

Completed

Last Update Posted

Dec 13, 2017Actual

Enrollment

49Actual

Phase

Not provided

Conditions

Crigler Najjar Syndrome

Interventions

Blood sample for immunology

Countries

France

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Dec 2017

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Nov 2014

Primary Completion Date

Jun 2017Actual

Completion Date

Jun 2017Actual

First Submitted Date

Nov 25, 2014

First Submission Date that Met QC Criteria

Nov 26, 2014

First Posted Date

Nov 27, 2014Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Dec 12, 2017

Last Update Posted Date

Dec 13, 2017Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief 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.

Detailed Description

Not provided

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

49Actual

Name	Type	Description	Arm Group Labels	Other Names
Blood sample for immunology	Biological

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
To assess the presence of neutralizing AAV antibodies in the serum of CN patients		1 day visit

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Patients with Crigler Najjar syndrome

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Hopital Antoine Béclère		Clamart		92141	France

Condition Browse Module

MeSH Terms

ID	Term
D003414	Crigler-Najjar Syndrome

Ancestor Terms

ID	Term
D006933	Hyperbilirubinemia, Hereditary
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Browse Leaves

Not provided

Browse Branches

Not provided

Intervention Browse Module

MeSH Terms

ID	Term
D001800	Blood Specimen Collection

Ancestor Terms

ID	Term
D013048	Specimen Handling
D019411	Clinical Laboratory Techniques
D019937	Diagnostic Techniques and Procedures
D003933	Diagnosis

Browse Leaves

Not provided

Browse Branches

Not provided