Clinical Assessment Study in Crigler-Najjar Syndrome

NCT03078881 | Completed

• 1 other identifier: AT342-01
• Study Type: observational
• Target: 8
• Locations: 3 countries
• Sites: 4

Brief 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.

Conditions

Crigler-Najjar Syndrome

Outcome Measures

Primary Outcomes (3)

• Characterize the disease course and natural history of subjects with Crigler-Najjar syndrome

  Up to 2 years

• 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

• Assess phototherapy usage over the course of the study

  Up to 2 years

Secondary Outcomes (1)

• Assess the humanistic and clinical burden of disease in Crigler-Najjar subjects and caregivers as measured by PedsQL

  Up to 2 years

Eligibility Criteria

• Age: 1 Year+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The study will enroll Crigler-Najjar syndrome subjects aged equal or greater than 1 year of age requiring daily phototherapy.

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Audentes Therapeutics: lead

Study Sites (4)

Children's Hospital at Montefiore

The Bronx, New York, 10467, United States

Location

Clinic for Special Children

Strasburg, Pennsylvania, 17579, United States

Location

Shaare Zedek Medical Center

Jerusalem, 9103102, Israel

Location

King's College Hospital

London, England, SE5 9RS, United Kingdom

Location

MeSH Terms

Conditions

Crigler-Najjar Syndrome

Condition Hierarchy (Ancestors)

Hyperbilirubinemia, Hereditary; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Suyash Prasad, MBBS, MRCP,MRCPCH, FFPM

  Audentes Therapeutics

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 8, 2017
• First Posted: March 14, 2017
• Study Start: March 10, 2017
• Primary Completion: January 23, 2019
• Study Completion: January 23, 2019
• Last Updated: February 6, 2019

Record last verified: 2019-02

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1); IL (1); US (2)