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

Primary Purpose

Alagille Syndrome

Status
Completed
Phase
Phase 2
Locations
United Kingdom
Study Type
Interventional
Intervention
LUM001
Placebo
Sponsored by
Mirum Pharmaceuticals, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Alagille Syndrome

Eligibility Criteria

12 Months - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Birmingham Children's Hospital
  • Leeds Teaching Hospitals
  • Kings College Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

LUM001

Placebo

Arm Description

LUM001 administered orally once each day

Placebo administered orally once each day

Outcomes

Primary Outcome Measures

Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid Level
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
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
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.

Full Information

First Posted
July 16, 2013
Last Updated
March 15, 2019
Sponsor
Mirum Pharmaceuticals, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT01903460
Brief Title
Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome
Acronym
IMAGO
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 Type
Interventional

2. Study Status

Record Verification Date
March 2019
Overall Recruitment Status
Completed
Study Start Date
August 2013 (undefined)
Primary Completion Date
February 2015 (Actual)
Study Completion Date
March 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Mirum Pharmaceuticals, Inc.

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Alagille Syndrome

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
20 (Actual)

8. Arms, Groups, and Interventions

Arm Title
LUM001
Arm Type
Experimental
Arm Description
LUM001 administered orally once each day
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo administered orally once each day
Intervention Type
Drug
Intervention Name(s)
LUM001
Intervention Type
Drug
Intervention Name(s)
Placebo
Primary Outcome Measure Information:
Title
Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid Level
Description
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.
Time Frame
Baseline to 13 weeks or end of treatment
Secondary Outcome Measure Information:
Title
Change From Baseline to Week 13 (End of Treatment) in Liver Enzymes
Description
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.
Time Frame
Baseline to 13 weeks or end of treatment
Title
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
Description
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.
Time Frame
Baseline to 13 weeks or end of treatment

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Months
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Study Director
Organizational Affiliation
Mirum
Official's Role
Study Director
Facility Information:
Facility Name
Birmingham Children's Hospital
City
Birmingham
State/Province
West Midlands
ZIP/Postal Code
B4 6NH
Country
United Kingdom
Facility Name
Leeds Teaching Hospitals
City
Leeds
State/Province
West Yorkshire
ZIP/Postal Code
LS9 7TF
Country
United Kingdom
Facility Name
Kings College Hospital
City
London
ZIP/Postal Code
SE5 9RS
Country
United Kingdom

12. IPD Sharing Statement

Learn more about this trial

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

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