Clinical Assessment Study in Crigler-Najjar Syndrome (LUSTRO)

LUSTRO: A Clinical Assessment Study in Crigler-Najjar Syndrome

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.

Study Overview

• Status: Completed
• Conditions: Crigler-Najjar Syndrome

• Study Type: Observational
• Enrollment (Actual): 8

Contacts and Locations

Study Locations

• Israel
  • Jerusalem, Israel, 9103102
    • Shaare Zedek Medical Center
• United Kingdom
  • England
    • London, England, United Kingdom, SE5 9RS
      • King's College Hospital
• United States
  • New York
    • Bronx, New York, United States, 10467
      • Children's Hospital at Montefiore
  • Pennsylvania
    • Strasburg, Pennsylvania, United States, 17579
      • Clinic for Special Children

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• 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.

Description

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

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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 Outcome Measures

Outcome Measure	Time Frame
Assess the humanistic and clinical burden of disease in Crigler-Najjar subjects and caregivers as measured by PedsQL	Up to 2 years

Collaborators and Investigators

• Sponsor: Audentes Therapeutics
• Investigators: Study Director: Suyash Prasad, MBBS, MRCP,MRCPCH, FFPM, Audentes Therapeutics

Study record dates

Study Major Dates

• Study Start (Actual): March 10, 2017
• Primary Completion (Actual): January 23, 2019
• Study Completion (Actual): January 23, 2019

Study Registration Dates

• First Submitted: March 8, 2017
• First Submitted That Met QC Criteria: March 13, 2017
• First Posted (Actual): March 14, 2017

Study Record Updates

• Last Update Posted (Actual): February 6, 2019
• Last Update Submitted That Met QC Criteria: February 4, 2019
• Last Verified: February 1, 2019

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Heart Diseases; Cardiovascular Diseases; Metabolic Diseases; Disease; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Hyperbilirubinemia, Hereditary; Syndrome; Cardiomyopathies; Crigler-Najjar Syndrome
• Other Study ID Numbers: AT342-01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No