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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
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