Expanded Access Protocol for an Intermediate Size Population - RAVICTI for Byler Disease

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ClinicalTrials.gov Identifier: NCT02094222

Expanded Access Status : No longer available

First Posted : March 21, 2014

Last Update Posted : February 18, 2019

Sponsor:

Information provided by (Responsible Party):

Robert Squires, Jr., University of Pittsburgh

Tracking Information

First Submitted Date

March 19, 2014

First Posted Date

March 21, 2014

Last Update Posted Date

February 18, 2019

Descriptive Information

Brief Title

Expanded Access Protocol for an Intermediate Size Population - RAVICTI for Byler Disease

Brief Summary

Byler Disease is the result of a homozygous missense (G308V) mutation in the ATP8B1 gene. The disease is typically manifest in the first year of life on the basis of complications of cholestasis; common presentations include jaundice, poor growth, bleeding related to vitamin K deficiency, and/or weak bones related to vitamin D deficiency. Early management of Byler Disease is directed at nutritional issues which tend to be responsive to medical intervention, unlike the pruritus/scratching which remains a devastating problem. Progressive liver disease develops in Byler Disease and can lead to cirrhosis and end-stage liver disease. This is an open label expanded access protocol of RAVICTI in children with Byler Disease. The primary hypothesis is that the administration of RAVICTI in these children is feasible, well tolerated and safe. It is also hypothesized that RAVICTI treatment leads to an improvement in biochemical markers of liver disease and it may ameliorates or prevents the development of scratching behavior as a manifestation of pruritus attributed to the liver disease.

Detailed Description

Not Provided

Study Type

Expanded Access

Intervention

Drug: RAVICTI

open label expanded access protocol of titrated dosing regimen of RAVICTI for up to 60 weeks

Other Name: glycerol phenylbutyrate

Publications *

Not Provided

* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information

Expanded Access Status

No longer available

Contacts

Contact information is only displayed when the study is recruiting subjects

Listed Location Countries

Not Provided

Removed Location Countries

Administrative Information

NCT Number

NCT02094222

Responsible Party

Robert Squires, Jr., University of Pittsburgh

Study Sponsor

Robert Squires, Jr.

Collaborators

Not Provided

Investigators

Principal Investigator:

Robert H Squires, MD

University of Pittsburgh

PRS Account

University of Pittsburgh

Verification Date

February 2019

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