- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01903460
Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome (IMAGO)
March 15, 2019 updated by: Mirum Pharmaceuticals, Inc.
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.
Study Overview
Study Type
Interventional
Enrollment (Actual)
20
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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London, United Kingdom, SE5 9RS
- Kings College Hospital
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West Midlands
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Birmingham, West Midlands, United Kingdom, B4 6NH
- Birmingham Children's Hospital
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West Yorkshire
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Leeds, West Yorkshire, United Kingdom, LS9 7TF
- Leeds Teaching Hospitals
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
1 year to 18 years (ADULT, CHILD)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- Diagnosis of Alagille Syndrome
- Evidence of cholestasis
- Moderate to severe pruritus
- Ability to understand and willingness to sign informed consent/assent prior to initiation of any study procedures
Exclusion Criteria:
- Surgical disruption of the enterohepatic circulation
- Liver transplant
- History or presence of other concomitant liver disease
- Females who are pregnant or lactating
- Known HIV infection
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: RANDOMIZED
- Interventional Model: PARALLEL
- Masking: TRIPLE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
EXPERIMENTAL: LUM001
LUM001 administered orally once each day
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PLACEBO_COMPARATOR: Placebo
Placebo administered orally once each day
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
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Baseline to 13 weeks or end of treatment
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
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Baseline to 13 weeks or end of treatment
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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
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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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Baseline to 13 weeks or end of treatment
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
August 1, 2013
Primary Completion (ACTUAL)
February 1, 2015
Study Completion (ACTUAL)
March 1, 2015
Study Registration Dates
First Submitted
July 16, 2013
First Submitted That Met QC Criteria
July 16, 2013
First Posted (ESTIMATE)
July 19, 2013
Study Record Updates
Last Update Posted (ACTUAL)
March 28, 2019
Last Update Submitted That Met QC Criteria
March 15, 2019
Last Verified
March 1, 2019
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Digestive System Diseases
- Pathologic Processes
- Cardiovascular Diseases
- Disease
- Congenital Abnormalities
- Genetic Diseases, Inborn
- Biliary Tract Diseases
- Heart Defects, Congenital
- Cardiovascular Abnormalities
- Abnormalities, Multiple
- Bile Duct Diseases
- Cholestasis, Intrahepatic
- Cholestasis
- Liver Diseases
- Syndrome
- Alagille Syndrome
Other Study ID Numbers
- LUM001-302
- 2012-005346-38 (EUDRACT_NUMBER)
- SHP625-302 (OTHER: Shire Development LLC)
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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