An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Subjects With Alagille Syndrome (IMAGINE)

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
Shire Identifier:
First received: January 23, 2014
Last updated: June 10, 2015
Last verified: June 2015
This is a multicentre, extension study of LUM001 in children diagnosed with Alagille Syndrome who have completed participation in a core LUM001 treatment protocol. The primary objective is to evaluate long-term safety and tolerability of LUM001. Efficacy will be assessed by evaluating the effect of LUM001 on the biochemical markers and pruritus associated with Alagille Syndrome.

Condition Intervention Phase
Alagille Syndrome
Drug: LUM001
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Multicentre Extension Study to Evaluate the 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

Resource links provided by NLM:

Further study details as provided by Shire:

Primary Outcome Measures:
  • Safety and tolerability [ Time Frame: 72 weeks ] [ Designated as safety issue: Yes ]
    Adverse events, changes in vital signs, laboratory and other safety parameters from baseline to week 48

Secondary Outcome Measures:
  • Efficacy [ Time Frame: 48 weeks ] [ Designated as safety issue: No ]
    Changes in serum bile acids, pruritus, and other biochemical markers of cholestasis and liver disease from baseline to week 48

Estimated Enrollment: 18
Study Start Date: December 2013
Estimated Study Completion Date: December 2016
Estimated Primary Completion Date: September 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: LUM001
LUM001 administered orally once each day
Drug: LUM001


Ages Eligible for Study:   12 Months to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Completed a core treatment protocol of LUM001 in the treatment of cholestatic liver disease in pediatric subjects with Alagille Syndrome
  2. Ability to understand and willingness to sign informed consent/assent prior to initiation of any study procedures.

Exclusion Criteria:

  1. Experienced an adverse event or serious adverse event related to the study drug during a core LUM001 treatment protocol that led to the discontinuation of the subject from the core LUM001 treatment study
  2. History of non-adherence during the subject's participation in the core LUM001 treatment protocol.
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Please refer to this study by its identifier: NCT02047318

United Kingdom
Birmingham Children's Hospital
Birmingham, West Midlands, United Kingdom, B4 6NH
Leeds Teaching Hospital
Leeds, West Yorkshire, United Kingdom, LS9 7TF
Kings College Hospital
London, United Kingdom, SE5 9RS
Sponsors and Collaborators
  More Information

Responsible Party: Shire Identifier: NCT02047318     History of Changes
Other Study ID Numbers: LUM001-303  2013-003832-54 
Study First Received: January 23, 2014
Last Updated: June 10, 2015
Health Authority: United States: Food and Drug Administration
United Kingdom: Medicines and Healthcare Products Regulatory Agency

Additional relevant MeSH terms:
Alagille Syndrome
Liver Diseases
Abnormalities, Multiple
Bile Duct Diseases
Biliary Tract Diseases
Cardiovascular Abnormalities
Cardiovascular Diseases
Cholestasis, Intrahepatic
Congenital Abnormalities
Digestive System Diseases
Genetic Diseases, Inborn
Heart Defects, Congenital
Pathologic Processes processed this record on May 30, 2016