Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome
- 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
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 37
- 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
- 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 & 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)
The Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
The Hospital for Sick Children
🇨🇦Toronto, Ontario, Canada
Ann & Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
Baylor College of Medicine/Texas Children's Hospital
🇺🇸Houston, Texas, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States
University of California at San Francisco
🇺🇸San Francisco, California, United States
Riley Hospital for Children
🇺🇸Indianapolis, Indiana, United States
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
Children's Healthcare of Atlanta
🇺🇸Atlanta, Georgia, United States
Children's Hospital Los Angeles
🇺🇸Los Angeles, California, United States
Children's Hospital of Pittsburgh of UPMC
🇺🇸Pittsburgh, Pennsylvania, United States
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States