Clinical Assessment Study in Crigler-Najjar Syndrome | NCT03078881

Studies
Clinical Assessment Study in Crigler-Najjar Syndrome

NCT03078881

Sponsor

Audentes Therapeutics

Status

Completed

Last Update Posted

Feb 6, 2019Actual

Enrollment

8Actual

Phase

Not provided

Conditions

Crigler-Najjar Syndrome

Interventions

Not provided

Countries

United States
Israel
United Kingdom

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Feb 2019

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Mar 10, 2017Actual

Primary Completion Date

Jan 23, 2019Actual

Completion Date

Jan 23, 2019Actual

First Submitted Date

Mar 8, 2017

First Submission Date that Met QC Criteria

Mar 13, 2017

First Posted Date

Mar 14, 2017Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Feb 4, 2019

Last Update Posted Date

Feb 6, 2019Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

8Actual

Outcomes Module

Primary Outcomes

Measure	Description	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 Outcomes

Measure	Description	Time Frame
Assess the humanistic and clinical burden of disease in Crigler-Najjar subjects and caregivers as measured by PedsQL		Up to 2 years

Other Outcomes

Not provided

Eligibility Module

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

1 Year

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

The study will enroll Crigler-Najjar syndrome subjects aged equal or greater than 1 year of age requiring daily phototherapy.

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Suyash Prasad, MBBS, MRCP,MRCPCH, FFPM	Audentes Therapeutics	Study Director

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Children's Hospital at Montefiore		The Bronx	New York	10467	United States
Clinic for Special Children

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D003414	Crigler-Najjar Syndrome

Ancestor Terms

ID	Term
D006933	Hyperbilirubinemia, Hereditary
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Browse Leaves

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Browse Branches

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