NCT03078881CompletedNot Applicable
LUSTRO: A Clinical Assessment Study in Crigler-Najjar Syndrome
Audentes Therapeutics4 sites in 3 countries8 target enrollmentStarted: March 10, 2017Last updated:
Conditions
Trial Snapshot
- Phase
- Not Applicable
- Status
- Completed
- Sponsor
- Enrollment
- 8
- Locations
- 4
- Primary Endpoint
- Assess phototherapy usage over the course of the study
Study Overview
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.
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Ages
- 1 Year to — (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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
Outcomes
Primary Outcomes
Assess phototherapy usage over the course of the study
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
Characterize the disease course and natural history of subjects with Crigler-Najjar syndrome
Time Frame: Up to 2 years
Secondary Outcomes
- Assess the humanistic and clinical burden of disease in Crigler-Najjar subjects and caregivers as measured by PedsQL(Up to 2 years)
Investigators
Study Sites (4)
Loading locations...
Similar Trials
Recruiting
Unknown
sCLEC-2 in Stroke StudyAcute ischemic stroke and transient ischemic attackJPRN-UMIN000048954niversity of Yamanashi600
Active, not recruiting
Not Applicable
A prospective study to evaluate the clinical characteristics and disease progression of participants with meibomian gland dysfunction (MGD)meibomian gland dysfunctionJPRN-jRCT1030210166Yamada Hiroyuki50
Recruiting
Not Applicable
Applicability of Positron Emission Tomography using Gallium-68 labelled Fibroblast Activating Protein Inhibitor for the diagnosis, staging and treatment of pancreatic cancer.Cancer - PancreaticPancreatic CancerACTRN12621000935831Royal Brisbane & Women's Hospital62
Completed
Phase 3
Efficacy of Arjuna Ksheerapaka Churna for managing high lipid level in the bodyCTRI/2020/06/026173RGGPG Ayurvedic College30
Completed
Not Applicable
Study For Evaluating The Value Of A Multi Biomarker Approach In Metastatic GEP NETsGastroenteropancreatic Neuroendocrine TumorsNCT02630654Ipsen404