This trial is active, not recruiting.

Condition progressive familial intrahepatic cholestasis (pfic)
Treatment lum001
Phase phase 2
Sponsor Shire
Start date February 2014
End date February 2017
Trial size 24 participants
Trial identifier NCT02057718, 2013-003833-14, LUM001-501


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 the biochemical markers and pruritus associated with PFIC.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
LUM001 administered orally once each day

Primary Outcomes

time frame: 13 weeks

Secondary Outcomes

time frame: 13 weeks

Eligibility Criteria

Male or female participants from 12 months up to 18 years old.

Inclusion Criteria: 1. Diagnosis of Progressive Familial Intrahepatic Cholestasis 2. Ability to understand and willingness to sign informed consent/assent prior to initiation Exclusion Criteria: 1. Surgical disruption of the enterohepatic circulation 2. Liver transplant 3. History or presence of other concomitant liver disease 4. Females who are pregnant or lactating

Additional Information

Official title 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
Trial information was received from ClinicalTrials.gov and was last updated in October 2015.
Information provided to ClinicalTrials.gov by Shire.