NCT02302690

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
49

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Nov 2014

Typical duration for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

November 1, 2014

Completed
24 days until next milestone

First Submitted

Initial submission to the registry

November 25, 2014

Completed
2 days until next milestone

First Posted

Study publicly available on registry

November 27, 2014

Completed
2.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2017

Completed
Last Updated

December 13, 2017

Status Verified

December 1, 2017

Enrollment Period

2.6 years

First QC Date

November 25, 2014

Last Update Submit

December 12, 2017

Conditions

Crigler Najjar Syndrome

Outcome Measures

Primary Outcomes (1)

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

    1 day visit

Interventions

Blood sample for immunologyBIOLOGICAL

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Patients with Crigler Najjar syndrome

You may qualify if:

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

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Hopital Antoine Béclère

Clamart, 92141, France

Location

MeSH Terms

Conditions

Crigler-Najjar Syndrome

Interventions

Blood Specimen Collection

Condition Hierarchy (Ancestors)

Hyperbilirubinemia, HereditaryMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Specimen HandlingClinical Laboratory TechniquesDiagnostic Techniques and ProceduresDiagnosisPuncturesSurgical Procedures, OperativeInvestigative Techniques

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 25, 2014

First Posted

November 27, 2014

Study Start

November 1, 2014

Primary Completion

June 1, 2017

Study Completion

June 1, 2017

Last Updated

December 13, 2017

Record last verified: 2017-12

Locations

FR(1)