Clinical Assessment Study in Crigler-Najjar Syndrome

LUSTRO: A Clinical Assessment Study in Crigler-Najjar Syndrome

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT03078881

Acronym

LUSTRO

Enrollment

Registered

2017-03-14

Start date

2017-03-10

Completion date

2019-01-23

Last updated

2019-02-06

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Crigler-Najjar Syndrome

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.

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

1 Years to No maximum

Healthy volunteers

Inclusion criteria

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

Design outcomes

Primary

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

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

Countries

Israel, United Kingdom, United States

Outcome results