Alagille Syndrome and LUM001: The IMAGINE Study – Phase II

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

Protocol Description

Through this trial researchers will study the drug LUM001 and its effects on children with cholestatic liver disease with Alagille Syndrome when taken over an extended period. Investigators believe that LUM001 may help control itching and improve liver tests. As a study extension, this trial is only open to participants of another Alagille Syndrome LUM001 trial, known as the ITCH study. The LUM001 studies are being done as a collaboration of the Childhood Liver Disease Research Network centers.

Study Level: Phase II

Eligibility Criteria

Subject to certain exclusion criteria, the study is open to individuals who have been diagnosed with Alagille Syndrome and evidence of cholestasis with itching. All participants must have completed participation in the ITCH study.
Males and Females: 12 months through 18 years

Requirements

All participants will receive a daily oral dose of LUM001, and an assessment of each participant’s itching will be logged via electronic diary. Dose levels will be adjusted for optimum effect in the first part of the approximately 11-month-long study. Medical record information and blood and urine samples will also be collected for each participant.
Visits: 9
Duration: Up to 48 weeks

Status: Open to Enrollment

Source(s) of Support

Shire
National Institute of Diabetes and Digestive and Kidney Diseases

Primary Investigator

Robert H. Squires, MD

Contact Information

For more information about the study or enrollment, please contact:
Kathy Bukauskas, RN, CCRC
412-692-7703