This trial is active, not recruiting.

Condition alagille syndrome
Treatments lum001, placebo
Phase phase 2
Sponsor Shire
Collaborator Childhood Liver Disease Research and Education Network
Start date November 2014
End date November 2016
Trial size 36 participants
Trial identifier NCT02057692, LUM001-301, NCT02055768


The study is a randomized, double-blind, placebo-controlled study in children with Alagille Syndrome (ALGS). The study will investigate the effects of LUM001, compared to placebo, on pruritus, serum bile acids, liver enzymes, and other biochemical markers in patients with ALGS.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking double blind (subject, caregiver, investigator)
Primary purpose treatment
LUM001 for oral administration
LUM001 administered orally
(Placebo Comparator)
Placebo administered orally once each day
Placebo administered orally

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 Alagille Syndrome 2. Evidence of cholestasis 3. Moderate to severe pruritus 4. Ability to understand and willingness to sign informed consent/assent prior to initiation of any study procedures 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 5. Known HIV infection

Additional Information

Official title The Evaluation of the Intestinal Bile Acid Transport (IBAT) Inhibitor LUM001 in the Reduction of Pruritus in Alagille Syndrome, a Cholestatic Liver Disease
Trial information was received from ClinicalTrials.gov and was last updated in August 2016.
Information provided to ClinicalTrials.gov by Shire.