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

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ClinicalTrials.gov Identifier: NCT01903460
Recruitment Status : Completed
First Posted : July 19, 2013
Results First Posted : December 7, 2015
Last Update Posted : December 7, 2015
Sponsor:
Information provided by (Responsible Party):
Shire

Brief Summary:
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 or disease Intervention/treatment Phase
Alagille Syndrome Drug: LUM001 Drug: Placebo Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 20 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, 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
Study Start Date : August 2013
Primary Completion Date : February 2015
Study Completion Date : March 2015

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Liver Diseases
U.S. FDA Resources

Arm Intervention/treatment
Experimental: LUM001
LUM001 administered orally once each day
Drug: LUM001
Placebo Comparator: Placebo
Placebo administered orally once each day
Drug: Placebo



Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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.



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Ages Eligible for Study:   12 Months to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
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

Information from the National Library of Medicine

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


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

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT01903460     History of Changes
Other Study ID Numbers: LUM001-302
2012-005346-38 ( EudraCT Number )
SHP625-302 ( Other Identifier: Shire Development LLC )
First Posted: July 19, 2013    Key Record Dates
Results First Posted: December 7, 2015
Last Update Posted: December 7, 2015
Last Verified: June 2015

Additional relevant MeSH terms:
Syndrome
Liver Diseases
Alagille Syndrome
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