Overview

This trial is active, not recruiting.

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

Summary

This 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 5 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, a 26-week long-term stable dosing period, and an optional 52 week follow-up treatment period for eligible subjects who choose to stay on treatment with LUM001. Subjects' participation in the optional follow-up treatment period will continue until the first of the following occur: (i) completion of 52 weeks of additional treatment (Week 100), or (ii) in the event that a new study of LUM001 opens to enrollment.

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
Arm
(Experimental)
LUM001 administered orally once each day
lum001
LUM001 administered orally once each day
(Placebo Comparator)
Placebo administered orally once each day during randomized withdrawal period
placebo
Placebo administered orally once each day during randomized withdrawal period

Primary Outcomes

Measure
Evaluate effect of LUM001 on serum bile acid levels in children with ALGS using an analysis of covariance (ANCOVA) model treatment
time frame: 48 weeks
Evaluate effect of LUM001 on biochemical markers of cholestasis in children with ALGS
time frame: 48 weeks
Evaluate effect of LUM001 on pruritus in children with ALGS as measured by the average daily Itch Reported Outcome (ItchRO)
time frame: 48 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. Females of childbearing potential must have a negative serum pregnancy test during screening. 5. Sexually active females must be prepared to use an effetive method of contraception during the trial. 6. Subject is expected to have a consistent caregiver(s) for the duration of the study. 7. Caregivers (and age appropriate subjects) must complete at least 10 eDiary reports (morning or evening) during each of two consecutive weeks of the screening period . Exclusion Criteria: 1. Chronic diarrhea requiring ongoing intravenous fluid or nutritional intervention. 2. Surgical interruption of the enterohepatic circulation. 3. Previous liver transplant. 4. Decompensated cirrhosis, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy. 5. History or presence of other concomitant liver disease, gallstones, or kidney stones. 6. Diagnosis of human immunodeficiency virus (HIV). 7. Cancers, except for in situ carcinoman, or cancers treated at least 5 years prior to screening with no evidence of recurrence. 8. Any female who is pregnant or lactating, or planning to become pregnant during the study period.

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 November 2016.
Information provided to ClinicalTrials.gov by Shire.