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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT03078881
Other study ID # AT342-01
Secondary ID
Status Completed
Phase
First received
Last updated
Start date March 10, 2017
Est. completion date January 23, 2019

Study information

Verified date February 2019
Source Audentes Therapeutics
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial 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.


Recruitment information / eligibility

Status Completed
Enrollment 8
Est. completion date January 23, 2019
Est. primary completion date January 23, 2019
Accepts healthy volunteers No
Gender All
Age group 1 Year and older
Eligibility 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 Design


Related Conditions & MeSH terms


Locations

Country Name City State
Israel Shaare Zedek Medical Center Jerusalem
United Kingdom King's College Hospital London England
United States Children's Hospital at Montefiore Bronx New York
United States Clinic for Special Children Strasburg Pennsylvania

Sponsors (1)

Lead Sponsor Collaborator
Audentes Therapeutics

Countries where clinical trial is conducted

United States,  Israel,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Characterize the disease course and natural history of subjects with Crigler-Najjar syndrome Up to 2 years
Primary 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
Primary Assess phototherapy usage over the course of the study Up to 2 years
Secondary Assess the humanistic and clinical burden of disease in Crigler-Najjar subjects and caregivers as measured by PedsQL Up to 2 years
See also
  Status Clinical Trial Phase
Terminated NCT03223194 - Gene Transfer Clinical Study in Crigler-Najjar Syndrome Phase 1
Completed NCT00461799 - Orlistat Treatment of Crigler-Najjar Disease N/A
Recruiting NCT05687474 - Baby Detect : Genomic Newborn Screening
Recruiting NCT03466463 - Gene Therapy for Severe Crigler Najjar Syndrome N/A
Terminated NCT03343756 - HepaStem Long-Term Safety Registry