Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome

Phase 2

Completed

• Conditions: Alagille Syndrome
• Interventions: Drug: LUM001; Drug: Placebo

• Registration Number: NCT02057692
• Lead Sponsor: Mirum Pharmaceuticals, Inc.

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2014-02-05
• Study First Submitted QC: 2014-02-05
• Study First Posted: 2014-02-07
• Study Start: 2014-11-24
• Primary Completion: 2016-11-16
• Study Completion: 2016-11-16
• Results First Submitted: 2017-11-07
• Results First Submitted QC: 2018-01-18
• Results First Posted: 2018-01-23
• Status Verified: 2019-03
• Last Update Submitted: 2019-03-15
• Last Update Posted: 2019-03-26

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 37

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
LUM001	LUM001	LUM001 for oral administration
Placebo	Placebo	Placebo administered orally once each day

Primary Outcome Measures

Name	Time	Method
Change From Baseline to Endpoint (Week 13/Early Termination) in Pruritus	Baseline, Week 13/Early Termination	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

Name	Time	Method
Change From Baseline to Endpoint (Week 13/Early Termination) in Fasting Serum Bile Acid (sBA) Level	Baseline, Week 13/Early Termination	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	Baseline, Week 13/Early Termination	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	Baseline, Week 13/Early Termination	Liver enzyme levels of total bilirubin and direct bilirubin were reported here.

Trial Locations

Locations (13)

Children's Hospital Los Angeles; 🇺🇸 Los Angeles, California, United States

University of California at San Francisco; 🇺🇸 San Francisco, California, United States

Children's Hospital Colorado; 🇺🇸 Aurora, Colorado, United States

Children's Healthcare of Atlanta; 🇺🇸 Atlanta, Georgia, United States

Ann & Robert H. Lurie Children's Hospital of Chicago; 🇺🇸 Chicago, Illinois, United States

Riley Hospital for Children; 🇺🇸 Indianapolis, Indiana, United States

Cincinnati Children's Hospital Medical Center; 🇺🇸 Cincinnati, Ohio, United States

The Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States

Children's Hospital of Pittsburgh of UPMC; 🇺🇸 Pittsburgh, Pennsylvania, United States

Baylor College of Medicine/Texas Children's Hospital; 🇺🇸 Houston, Texas, United States

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