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NCT Number: NCT03078881

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.

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Key information

Conditions

Crigler-Najjar Syndrome Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hyperbilirubinemia, Hereditary Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Age range

1 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Shaare Zedek Medical Center, Jerusalem, Israel

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Primary outcomes

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

    Time frame: Up to 2 years

  2. Assess variation in bilirubin levels over the course of the study and the variation of bilirubin levels over a 24-hr period

    Time frame: Up to 2 years

  3. Assess phototherapy usage over the course of the study

    Time frame: 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

    Time frame: Up to 2 years

Sponsors and collaborators

Lead sponsor

Audentes Therapeutics

Industry

Registry information

Official study title

LUSTRO: A Clinical Assessment Study in Crigler-Najjar Syndrome

Acronym: LUSTRO

Important dates

Study start
2017
Primary completion
2019
Study completion
2019
First posted
Mar 14, 2017
Registry last updated
Feb 6, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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