Clinical Assessment Study in Crigler-Najjar Syndrome

Crigler-Najjar Syndrome Clinical Trial

— LUSTRO  

Official title:

LUSTRO: A Clinical Assessment Study in Crigler-Najjar Syndrome

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03078881
• Other study ID #: AT342-01
• Status: Completed
• Start date: March 10, 2017
• Est. completion date: January 23, 2019

Study information

• Verified date: February 2019
• Source: Audentes Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

This is a Pre-Phase 1 prospective, non-interventional clinical assessment study to evaluate Crigler-Najjar syndrome subjects requiring daily phototherapy, aged 1 year and older.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 8
• Est. completion date: January 23, 2019
• Est. primary completion date: January 23, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year and older

Eligibility

Key Inclusion Criteria:

• Subject has a diagnosis of Crigler-Najjar syndrome resulting from a confirmed mutation in the UGT1A1 gene

• Subject is aged equal or greater than 1 year of age

• Subject receives daily phototherapy for a minimum of 6 hours within a 24-hour period (daily illumination time)

Exclusion Criteria:

• Subject is currently participating in an interventional study or has received gene or cell therapy

• Subject has received a whole liver, partial liver, or hepatocyte transplant; or subject has a liver transplant scheduled within the planned participation period of this study

• Subject has significant cholestatic disease, in the opinion of the investigator

• Subject is receiving phenobarbital or other known inducer of UGT1A1 within 30 days of screening

• Subject has any clinically significant underlying liver disease (other than Crigler-Najjar syndrome), in the opinion of the investigator

• Subject has a history of, or currently has, a clinically important condition other than Crigler-Najjar syndrome, in the opinion of the investigator

Related Conditions & MeSH terms

• Cardiomyopathies
• Crigler-Najjar Syndrome
• Syndrome

Locations

Country	Name	City	State
Israel	Shaare Zedek Medical Center	Jerusalem
United Kingdom	King's College Hospital	London	England
United States	Children's Hospital at Montefiore	Bronx	New York
United States	Clinic for Special Children	Strasburg	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Audentes Therapeutics

Countries where clinical trial is conducted

United States,  Israel,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Characterize the disease course and natural history of subjects with Crigler-Najjar syndrome		Up to 2 years
Primary	Assess variation in bilirubin levels over the course of the study and the variation of bilirubin levels over a 24-hr period		Up to 2 years
Primary	Assess phototherapy usage over the course of the study		Up to 2 years
Secondary	Assess the humanistic and clinical burden of disease in Crigler-Najjar subjects and caregivers as measured by PedsQL		Up to 2 years