Overview

This trial is active, not recruiting.

Condition alagille syndrome
Treatment lum001
Phase phase 2
Sponsor Shire
Collaborator Lumena Pharmaceuticals, Inc.
Start date December 2013
End date August 2017
Trial size 19 participants
Trial identifier NCT02047318, 2013-003832-54, LUM001-303

Summary

The purpose of this extension study is to determine the long-term safety and tolerability of an investigational treatment (LUM001) in children with ALGS who have completed participation in the LUM001-302 study.

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
Arm
(Experimental)
LUM001 administered orally once each day
lum001
Dosing of LUM001 with the objective of achieving optimal control of pruritus at a dose level that is tolerated by the subject and up to a maximum daily dose of 280 μg/kg LUM001 or 20 mg total dose.

Primary Outcomes

Measure
Safety and tolerability
time frame: 72 weeks

Secondary Outcomes

Measure
Efficacy
time frame: 72 weeks

Eligibility Criteria

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

Inclusion Criteria: - Male or female, 12 months to 18 years of age. - Competent to provide informed consent and assent (per IRB/EC), as appropriate. - Completed participation in study LUM001-302. - Females of childbearing potential must have a negative urine pregnancy test. - Sexually active females must be prepared to use an effective method of contraception during the trial. - Subjects above the age of assent and caregivers and children must be able to read and understand English. - Caregivers (and age appropriate subjects) must have access to phone for scheduled calls from study site. - Caregivers (and age appropriate subjects) must be willing and able to complete a daily electronic diary (ItchRO) during the first consecutive 12 weeks of the study and then for 4 consecutive weeks following the Week 24 and Week 44 visits. - Caregivers (and age appropriate subjects) must digitally accept the licensing agreement in the ItchRO electronic diary software at the outset of the study. Exclusion Criteria: - Experienced an adverse event or serious adverse event (SAE) related to the study drug during the LUM001-302 protocol that led to the discontinuation of the subject from the core study. - Any conditions or abnormalities (including laboratory abnormalities) which, in the opinion of the Investigator or the Sponsor Medical Monitor, may compromise the safety of the subject, or interfere with the subject participating in or completing the study. - History or presence of gallstones or kidney stones. - History of non-adherence during the subject's participation in the LUM001-302 protocol. Non-adherence is defined by dosing compliance of less than 80% in the LUM001-302 protocol. - Unlikely to comply with the study protocol, or unsuitable for any other reason, as judged by the investigator.

Additional Information

Official title A Multicentre Extension Study to Evaluate the Long-Term Safety and Durability of the Therapeutic Effect of LUM001, an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome
Trial information was received from ClinicalTrials.gov and was last updated in July 2016.
Information provided to ClinicalTrials.gov by Shire.