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.
Brief Title
Clinical Assessment Study in Crigler-Najjar Syndrome
Categories
Completion Date
Completion Date Type
Actual
Conditions
Crigler-Najjar Syndrome
Eligibility 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
* 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
Inclusion Criteria
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)
* 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)
Gender
All
Gender Based
false
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Minimum Age
1 Year
NCT Id
NCT03078881
Org Class
Industry
Org Full Name
Audentes Therapeutics
Org Study Id
AT342-01
Overall Status
Completed
Primary Completion Date
Primary Completion Date Type
Actual
Official Title
LUSTRO: A Clinical Assessment Study in Crigler-Najjar Syndrome
Primary Outcomes
Outcome Measure
Characterize the disease course and natural history of subjects with Crigler-Najjar syndrome
Outcome Time Frame
Up to 2 years
Outcome Measure
Assess variation in bilirubin levels over the course of the study and the variation of bilirubin levels over a 24-hr period
Outcome Time Frame
Up to 2 years
Outcome Measure
Assess phototherapy usage over the course of the study
Outcome Time Frame
Up to 2 years
Secondary Outcomes
Outcome Time Frame
Up to 2 years
Outcome Measure
Assess the humanistic and clinical burden of disease in Crigler-Najjar subjects and caregivers as measured by PedsQL
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Actual
First Submit Date
First Submit QC Date
Study Population
The study will enroll Crigler-Najjar syndrome subjects aged equal or greater than 1 year of age requiring daily phototherapy.
Std Ages
Child
Adult
Older Adult
Maximum Age Number (converted to Years and rounded down)
999
Minimum Age Number (converted to Years and rounded down)
1
Investigators
Investigator Type
Principal Investigator
Investigator Name
Nadia Ovchinsky
Investigator Email
NOVCHINS@montefiore.org
Investigator Phone