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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:
ClinicalTrials.gov Identifier:
NCT01903460
First Posted: July 19, 2013
Last Update Posted: December 7, 2015
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.
Information provided by (Responsible Party):
Shire
July 16, 2013
July 19, 2013
November 2, 2015
December 7, 2015
December 7, 2015
August 2013
February 2015   (Final data collection date for primary outcome measure)
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.
Efficacy [ Time Frame: 13 weeks ]
Change from baseline in fasting serum bile acids compared to placebo
Complete list of historical versions of study NCT01903460 on ClinicalTrials.gov Archive Site
  • 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.
Efficacy [ Time Frame: 13 weeks ]
Change from baseline in liver enzymes and pruritus compared to placebo
Not Provided
Safety and Tolerability [ Time Frame: 13 weeks ]
Evaluation of adverse events, changes in vital signs, laboratory and other safety parameters
 
Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome
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
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.
Not Provided
Interventional
Phase 2
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Alagille Syndrome
  • Drug: LUM001
  • Drug: Placebo
  • Experimental: LUM001
    LUM001 administered orally once each day
    Intervention: Drug: LUM001
  • Placebo Comparator: Placebo
    Placebo administered orally once each day
    Intervention: Drug: Placebo
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
20
March 2015
February 2015   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
12 Months to 18 Years   (Child, Adult)
No
Contact information is only displayed when the study is recruiting subjects
United Kingdom
 
 
NCT01903460
LUM001-302
2012-005346-38 ( EudraCT Number )
SHP625-302 ( Other Identifier: Shire Development LLC )
Yes
Not Provided
Not Provided
Shire
Shire
Not Provided
Not Provided
Shire
June 2015

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP