Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome (IMAGO)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01903460|
Recruitment Status : Completed
First Posted : July 19, 2013
Results First Posted : December 7, 2015
Last Update Posted : December 7, 2015
|Condition or disease||Intervention/treatment||Phase|
|Alagille Syndrome||Drug: LUM001 Drug: Placebo||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||20 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Triple (Participant, Care Provider, Investigator)|
|Official Title:||A Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Paediatric Patients With Alagille Syndrome|
|Study Start Date :||August 2013|
|Actual Primary Completion Date :||February 2015|
|Actual Study Completion Date :||March 2015|
LUM001 administered orally once each day
Placebo Comparator: Placebo
Placebo administered orally once each day
- Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid Level [ Time Frame: Baseline to 13 weeks or end of treatment ]Participants were required to fast for at least 4 hours; only water was permitted prior to collection. A negative change from baseline indicates that the level of bile acid decreased.
- Change From Baseline to Week 13 (End of Treatment) in Liver Enzymes [ Time Frame: Baseline to 13 weeks or end of treatment ]Analysis of liver enzymes included alanine aminotransferase (ALT), aspartate aminotransferase (AST), and alkaline phosphatase (ALP). A negative change from baseline indicates that the level of that enzyme decreased.
- Change From Baseline to Week 13 (End of Treatment) in Pruritus as Measured by The Patient And Observer Itch Reported Outcome (ItchRO) Average Daily Scores [ Time Frame: Baseline to 13 weeks or end of treatment ]The ItchRO was administered as a twice daily electronic diary (eDiary). Children ≥9 years of age completed the patient ItchRO; those between the ages of 5 and 8 completed the patient ItchRO with the assistance of their caregiver. There was no patient report for subjects under the age of 5. ItchRO scores range from 0 to 4, with the higher score indicating increasing itch severity. ItchRO average daily scores were calculated as the sum of daily scores (ie, the maximum of morning and evening scores) divided by the number of days. The average daily score was calculated by using the 7 days pre-treatment for baseline, and the last 7 days of treatment for Week 13. A negative change from Baseline indicates that itch severity decreased.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01903460
|Birmingham Children's Hospital|
|Birmingham, West Midlands, United Kingdom, B4 6NH|
|Leeds Teaching Hospitals|
|Leeds, West Yorkshire, United Kingdom, LS9 7TF|
|Kings College Hospital|
|London, United Kingdom, SE5 9RS|