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Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome (ITCH)

Primary Purpose

Alagille Syndrome

Status
Completed
Phase
Phase 2
Locations
International
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

  • Children's Hospital Los Angeles
  • University of California at San Francisco
  • Children's Hospital Colorado
  • Children's Healthcare of Atlanta
  • Ann & Robert H. Lurie Children's Hospital of Chicago
  • Riley Hospital for Children
  • Cincinnati Children's Hospital Medical Center
  • The Children's Hospital of Philadelphia
  • Children's Hospital of Pittsburgh of UPMC
  • Baylor College of Medicine/Texas Children's Hospital
  • University of Utah
  • Seattle Children's Hospital
  • The Hospital for Sick Children

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

LUM001

Placebo

Arm Description

LUM001 for oral administration

Placebo administered orally once each day

Outcomes

Primary Outcome Measures

Change From Baseline to Endpoint (Week 13/Early Termination) in Pruritus
Pruritus was assessed using Itch report outcome measure (ItchRO[Obs]), administered as an electronic diary (eDiary) which was completed by the participants twice daily (morning and evening). ItchRO(Obs) score ranged from 0 to 4, with the higher score indicating increasing itch severity. The highest score between the morning and evening ItchRO(Obs) reports represented the daily score: a measure of the worst itching over the previous 24-hour period.

Secondary Outcome Measures

Change From Baseline to Endpoint (Week 13/Early Termination) in Fasting Serum Bile Acid (sBA) Level
Fasting sBA level was measured by using a liquid chromatography mass spectrometry method.
Change From Baseline to Endpoint (Week 13/Early Termination) in Liver Enzyme Levels
Liver enzyme levels of alanine aminotransferase, alkaline phosphatase, aspartate aminotransferase and gamma glutamyl transferase were reported here.
Change From Baseline to Endpoint (Week 13/Early Termination) in Total and Direct Bilirubin Concentrations
Liver enzyme levels of total bilirubin and direct bilirubin were reported here.

Full Information

First Posted
February 5, 2014
Last Updated
March 15, 2019
Sponsor
Mirum Pharmaceuticals, Inc.
Collaborators
Childhood Liver Disease Research and Education Network
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1. Study Identification

Unique Protocol Identification Number
NCT02057692
Brief Title
Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome
Acronym
ITCH
Official Title
The Evaluation of the Intestinal Bile Acid Transport (IBAT) Inhibitor LUM001 in the Reduction of Pruritus in Alagille Syndrome, a Cholestatic Liver Disease
Study Type
Interventional

2. Study Status

Record Verification Date
March 2019
Overall Recruitment Status
Completed
Study Start Date
November 24, 2014 (Actual)
Primary Completion Date
November 16, 2016 (Actual)
Study Completion Date
November 16, 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Mirum Pharmaceuticals, Inc.
Collaborators
Childhood Liver Disease Research and Education Network

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The study is a randomized, double-blind, placebo-controlled study in children with Alagille Syndrome (ALGS). The study will investigate the effects of LUM001, compared to placebo, on pruritus, serum bile acids, liver enzymes, and other biochemical markers in patients with ALGS.

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
37 (Actual)

8. Arms, Groups, and Interventions

Arm Title
LUM001
Arm Type
Experimental
Arm Description
LUM001 for oral administration
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo administered orally once each day
Intervention Type
Drug
Intervention Name(s)
LUM001
Intervention Description
LUM001 administered orally
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo administered orally
Primary Outcome Measure Information:
Title
Change From Baseline to Endpoint (Week 13/Early Termination) in Pruritus
Description
Pruritus was assessed using Itch report outcome measure (ItchRO[Obs]), administered as an electronic diary (eDiary) which was completed by the participants twice daily (morning and evening). ItchRO(Obs) score ranged from 0 to 4, with the higher score indicating increasing itch severity. The highest score between the morning and evening ItchRO(Obs) reports represented the daily score: a measure of the worst itching over the previous 24-hour period.
Time Frame
Baseline, Week 13/Early Termination
Secondary Outcome Measure Information:
Title
Change From Baseline to Endpoint (Week 13/Early Termination) in Fasting Serum Bile Acid (sBA) Level
Description
Fasting sBA level was measured by using a liquid chromatography mass spectrometry method.
Time Frame
Baseline, Week 13/Early Termination
Title
Change From Baseline to Endpoint (Week 13/Early Termination) in Liver Enzyme Levels
Description
Liver enzyme levels of alanine aminotransferase, alkaline phosphatase, aspartate aminotransferase and gamma glutamyl transferase were reported here.
Time Frame
Baseline, Week 13/Early Termination
Title
Change From Baseline to Endpoint (Week 13/Early Termination) in Total and Direct Bilirubin Concentrations
Description
Liver enzyme levels of total bilirubin and direct bilirubin were reported here.
Time Frame
Baseline, Week 13/Early Termination
Other Pre-specified Outcome Measures:
Title
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
Description
An AE was any unfavorable and unintended sign (including a clinically significant abnormal laboratory finding, for example), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE was considered related to the investigational drug product. A serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in-patient hospitalization; life -threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.TEAEs were defined as AEs/SAEs that started or worsened after the study drug treatment.
Time Frame
From the start of study drug administration up to Week 17

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
Children's Hospital Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
University of California at San Francisco
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
Children's Hospital Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Children's Healthcare of Atlanta
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Ann & Robert H. Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Riley Hospital for Children
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
The Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Children's Hospital of Pittsburgh of UPMC
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
Baylor College of Medicine/Texas Children's Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84113
Country
United States
Facility Name
Seattle Children's Hospital
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
Facility Name
The Hospital for Sick Children
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 1X8
Country
Canada

12. IPD Sharing Statement

Learn more about this trial

Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome

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