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 Type
OBSERVATIONAL
Enrollment
8
Children's Hospital at Montefiore
The Bronx, New York, United States
Clinic for Special Children
Strasburg, Pennsylvania, United States
Shaare Zedek Medical Center
Jerusalem, Israel
King's College Hospital
London, England, United Kingdom
Characterize the disease course and natural history of subjects with Crigler-Najjar syndrome
Time frame: 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
Time frame: Up to 2 years
Assess phototherapy usage over the course of the study
Time frame: Up to 2 years
Assess the humanistic and clinical burden of disease in Crigler-Najjar subjects and caregivers as measured by PedsQL
Time frame: Up to 2 years
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