Open Label Study to Evaluate Efficacy and Long Term Safety of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Progressive Familial Intrahepatic Cholestasis

Status:	Active, not recruiting
Conditions:	Gastrointestinal
Therapuetic Areas:	Gastroenterology
Healthy:	No
Age Range:	Any - 18
Updated:	3/10/2019
Start Date:	March 1, 2014
End Date:	December 31, 2019

Open Label Study of the Efficacy and Long Term Safety of LUM001, an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Pediatric Patients With Progressive Familial Intrahepatic Cholestasis

This is an open label study in children with Progressive Familial Intrahepatic Cholestasis
(PFIC) designed to evaluate the safety and efficacy of LUM001. Efficacy will be assessed by
evaluating the effect of LUM001 on pruritus and the biochemical markers of pruritus
associated with PFIC.

The study is divided into 5 parts: a 4-week dose escalation period, a 4-week stable dosing
period, a 5-week stable dosing period, a 59-week long-term exposure period, and an optional
follow-up treatment period for eligible participants who continue treatment with LUM001.
Participants in the optional follow-up treatment period will continue to receive study drug
until they are eligible to enter another LUM001 study or until LUM001 is available
commercially, whichever occurs first.

We found this trial at

sites

Cincinnati Children's Hospital Medical Center

3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229

1-513-636-4200