Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome (IMAGO)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Shire
ClinicalTrials.gov Identifier:
NCT01903460
First received: July 16, 2013
Last updated: November 2, 2015
Last verified: June 2015
  Purpose
The study is a randomized, double-blind, placebo-controlled study to evaluate the safety and tolerability of LUM001. Efficacy will be assessed by evaluating the effect of LUM001 versus placebo on the biochemical markers and pruritus associated with Alagille Syndrome.

Condition Intervention Phase
Alagille Syndrome
Drug: LUM001
Drug: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Shire:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]
    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.


Secondary Outcome Measures:
  • Change From Baseline to Week 13 (End of Treatment) in Liver Enzymes [ Time Frame: Baseline to 13 weeks or end of treatment ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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.


Enrollment: 20
Study Start Date: August 2013
Study Completion Date: March 2015
Primary Completion Date: February 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: LUM001
LUM001 administered orally once each day
Drug: LUM001
Placebo Comparator: Placebo
Placebo administered orally once each day
Drug: Placebo

  Eligibility

Ages Eligible for Study:   12 Months to 18 Years   (Child, Adult)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01903460

Locations
United Kingdom
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
Sponsors and Collaborators
Shire
  More Information

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT01903460     History of Changes
Other Study ID Numbers: LUM001-302  2012-005346-38  SHP625-302 
Study First Received: July 16, 2013
Results First Received: November 2, 2015
Last Updated: November 2, 2015
Health Authority: United Kingdom: Medicines and Healthcare Products Regulatory Agency

Additional relevant MeSH terms:
Alagille Syndrome
Syndrome
Liver Diseases
Cholestasis
Disease
Pathologic Processes
Digestive System Diseases
Cholestasis, Intrahepatic
Bile Duct Diseases
Biliary Tract Diseases
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on July 21, 2016