This trial is active, not recruiting.

Condition alagille syndrome
Treatments lum001, placebo
Phase phase 2
Sponsor Shire
Start date September 2014
End date September 2015
Trial size 30 participants
Trial identifier NCT02160782, 2013-005373-43, LUM001-304


The study is a long-term, open-label study with a double-blind, placebo-controlled, randomized drug withdrawal period in children with ALGS designed to evaluate the safety and efficacy of LUM001. The study is divided into 4 parts: a 6-week open-label, dose escalation period, a 12-week stable dosing period, a 4-week randomized, double-blind, placebo-controlled drug withdrawal period, and a 26-week long-term stable dosing period.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model crossover assignment
Masking double blind (subject, caregiver, investigator)
Primary purpose treatment
LUM001 administered orally once each day
(Placebo Comparator)
Placebo administered orally once each day during randomized withdrawal period

Primary Outcomes

time frame: 4 weeks

Secondary Outcomes

time frame: 4 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 Long-Term, Open-Label Study With a Double-Blind, Placebo-Controlled, Randomized Drug Withdrawal Period of LUM001, an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients With Alagille Syndrome
Trial information was received from ClinicalTrials.gov and was last updated in April 2016.
Information provided to ClinicalTrials.gov by Shire.