Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome

NCT ID: NCT02057692

Last Updated: 2019-03-26

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

37 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-11-24

Study Completion Date

2016-11-16

Brief Summary

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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.

Detailed Description

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Conditions

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Alagille Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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LUM001

LUM001 for oral administration

Group Type EXPERIMENTAL

LUM001

Intervention Type DRUG

LUM001 administered orally

Placebo

Placebo administered orally once each day

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo administered orally

Interventions

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LUM001

LUM001 administered orally

Intervention Type DRUG

Placebo

Placebo administered orally

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

12 Months

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Childhood Liver Disease Research and Education Network

OTHER

Sponsor Role collaborator

Mirum Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Study Director

Role: STUDY_DIRECTOR

Mirum

Locations

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Children's Hospital Los Angeles

Los Angeles, California, United States

Site Status

University of California at San Francisco

San Francisco, California, United States

Site Status

Children's Hospital Colorado

Aurora, Colorado, United States

Site Status

Children's Healthcare of Atlanta

Atlanta, Georgia, United States

Site Status

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Site Status

Riley Hospital for Children

Indianapolis, Indiana, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, United States

Site Status

Baylor College of Medicine/Texas Children's Hospital

Houston, Texas, United States

Site Status

University of Utah

Salt Lake City, Utah, United States

Site Status

Seattle Children's Hospital

Seattle, Washington, United States

Site Status

The Hospital for Sick Children

Toronto, Ontario, Canada

Site Status

Countries

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United States Canada

Other Identifiers

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LUM001-301

Identifier Type: -

Identifier Source: org_study_id

NCT02055768

Identifier Type: -

Identifier Source: nct_alias

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